The emerging roles of the gut microbiome in allogeneic hematopoietic stem cell transplantation – DocWire News

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Gut Microbes. 2021 Jan-Dec;13(1):1966262. doi: 10.1080/19490976.2021.1966262.

ABSTRACT

Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is used for the treatment of hematologic cancers and disorders. However, graft-versus-host disease (GVHD) in which the donor immune cells attack the genetically-disparate recipient is a significant cause of morbidity. Acute GVHD is an inflammatory condition and the gastrointestinal system is a major organ affected but is also tied to beneficial graft-versus-tumor (GVT) effects. There is increasing interest on the role of the microbiome on immune function as well as on cancer progression and immunotherapy outcomes. However, there are still significant unanswered questions on the role the microbiome plays in GVHD progression or how to exploit the microbiome in GVHD prevention or treatment. In this review, concepts of HSCT with the focus on GVHD pathogenesis as well as issues in preclinical models used to study GVHD will be discussed with an emphasis on the impact of the microbiome. Factors affecting the microbiome and GVHD outcome such as obesity are also examined. The bridging of preclinical models and clinical outcomes in relation to the role of the microbiome will also be discussed along with possibilities for therapeutic exploitation.

PMID:34455917 | DOI:10.1080/19490976.2021.1966262

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The emerging roles of the gut microbiome in allogeneic hematopoietic stem cell transplantation - DocWire News

Event to benefit 2 families with kids fighting cancer – Youngstown Vindicator

News

Aug 30, 2021

AUSTINTOWN Local Relay for Life Team M&M The Flamingo Mafia and the Ice House Inn are joining to present a family friendly even Sept. 19 to raise money for the families of two area children battling cancer. Dozens of businesses, entertainers and volunteers have signed on to help.

The day-long Family Fun Day will feature live bands, comedians, food vendors, an auction, childrens activities (dunk tank, bounce house, etc.) and more.

Hours are noon to 9 p.m. Tickets are $10 for general admission and $5 for children 12 and younger. There is no admission charge for children under 3.

Advance tickets are available at the Ice House Inn, 5516 W. Webb Road, Austintown, or by calling Rosa at 330-559-0465. Tickets also will be available for purchase at the door the day of the event.

Nora Sabella, 7, and Joey Gaskell, 4, each went through rigorous treatments after their initial diagnoses only for their families to later learn they had both relapsed.

So many of us have personally experienced the devastating impact cancer has on families. For these families to go through everything theyve already been through only to face this all over again is unimaginable, said Tracy Greene, member and spokesperson for Team Flamingo Mafia.

Our team wanted to do something to help anyway we can. One way is to try to alleviate some of the financial burden, if we can, to allow them to focus on taking care of their children and each other.

ABOUT NORA

Nora, a student St. Charles School, lives in Boardman with her dad and mom, Jeff and Amy Sabella, her big sister, Nina Sabella, and Pickles the dog. She is battling relapsed neuroblastoma.

She was first diagnosed with Stage 4, High Risk Neuroblastoma on Jan. 29, 2018. She went through six cycles of chemotherapy, a stem cell transplant, 12 radiation treatments, six courses of immunotherapy, and many surgeries at Akron Childrens Hospital. She finished with frontline treatment March 24, 2019.

Nora immediately started a vaccine trial at Nationwide Childrens Hospital. After nearly two years on the trial, a lesion was found in Noras abdomen during routine scans.

On Nov. 23, 2020, Nora had a second tumor resection surgery, and the family was devastated to learn that she had relapsed. While small, the lesion was attached to her renal artery and renal vein, making it impossible to remove 100 percent. Nora started chemotherapy and immunotherapy the following week.

As of April this year, she had completed five cycles and there is still a tiny spot visible with subtle growth. So Nora started an intense chemotherapy regimen on April 28. She had a reaction to one of the chemotherapy drugs the second day and coded after becoming unresponsive. But the toxicity was quickly reversed with medication.

Another cycle of intense chemotherapy followed. Scans showed no change, and it was decided to do surgery. This surgery was originally to remove her kidney, but the surgeon was able to remove the tumor and save the organ.

After a two-week recovery, Nora started her third cycle of intense chemotherapy in combination with radiation treatments. Scans are planned for four weeks after her last radiation treatment.

Nora said she hopes the bad guy is gone and that shell be considered in remission. With remission comes another year of treatment at Memorial Sloan Ketteringin New York City for a vaccine trial process that will take approximately one year to complete. The family will have to travel to NYC up to 10 times in that year. The vaccine trial is to help her body learn to fight the neuroblastoma on its own.

ABOUT JOEY

Joey lives in Austintown and is the son of Todd and Jessica Gaskell. He wasnt attending school but hopes to start this year.

He was diagnosed with pre-B cell acute lymphoblastic leukemia in October 2018 at just 18 months old. His initial treatment was supposed to be about two years but two weeks before the end of his treatment, it was discovered he had relapsed in his central nervous system.

Joey is currently in remission again after an intensive first month of relapse treatment followed by fout months of frequent hospital stays, clinic visits for chemo and transfusions.

His current relapse treatment plan will consist of intensive chemo and radiation for about one-and-a-half years with a lot of hospital stays.

We just hope to see the community give these children, these families, the support they desperately need, said team co-captain Rosa Sheets. Any and all proceeds from this event are going directly to these two families. We appreciate any help and support people in our community and beyond can and are willing to give. It will be a fun-packed, family- and kid-friendly day.

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Event to benefit 2 families with kids fighting cancer - Youngstown Vindicator

Operational Highlights and Financial Results for the Year Ended June 30, 2021 – GlobeNewswire

NEW YORK, Aug. 30, 2021 (GLOBE NEWSWIRE) -- Mesoblast Limited (Nasdaq:MESO; ASX:MSB), global leader in allogeneic cellular medicines for inflammatory diseases, today reported operational highlights and financial results for the fourth quarter and full-year ended June 30, 2021 (FY2021).

During this calendar year we made significant progress in both regulatory and clinical outcomes for our lead product candidate, remestemcel-L, after experiencing a disappointing set-back last year said Silviu Itescu, Chief Executive of Mesoblast. We are pleased with recent recommendations by FDAs CBER to meet with the review team and address remaining CMC items for remestemcel-L in the treatment of steroid-refractory acute graft versus host disease in children. Additionally, our most recent meeting with the FDA has provided clarity on the pathway towards an emergency use authorization for remestemcel-L in the treatment of COVID ARDS.

Operational Highlights

Remestemcel-L Outcome of recent meeting with FDA on regulatory pathway for emergency use authorization in the treatment of COVID-19 ARDS:

Remestemcel-L in the treatment of steroid-refractory acute graft versus host disease (SR-aGVHD) in children:

Rexlemestrocel-L in the treatment of chronic heart failure and chronic low back pain:

Manufacturing

Financial Highlights

DETAILED CLINICAL ACTIVITIES FOR THE FISCAL YEAR FY2021

Remestemcel-L

Acute Respiratory Distress Syndrome due to COVID-19

Mesoblast recently presented results from the randomized controlled trial of remestemcel-L in 222 ventilator-dependent COVID-19 patients with moderate/severe acute respiratory distress syndrome (ARDS) at the biennial Stem Cells, Cell Therapies, and Bioengineering in Lung Biology and Diseases conference hosted by the University of Vermont, Burlington, VT, and at the International Society for Cell & Gene Therapy (ISCT) Scientific Signatures Series event on Cell and Gene-Based Therapies in Lung Diseases and Critical Illnesses.

The presented data included improved respiratory function in patients treated with remestemcel-L, as well as 90-day survival outcomes showing remestemcel-L significantly reduced mortality by 48% at 90 days compared to controls in a pre-specified exploratory analysis of 123 treated patients under 65 years old. The trial had been halted after the third interim analysis since the 30-day primary endpoint would not be attained.

Key presentation findings were:

Mesoblast plans to move forward with an additional Phase 3 trial in COVID-19 ARDS with the next step being to agree with the FDA the final protocol and potency assay.

Inflammatory Bowel Disease Crohns Disease and Ulcerative Colitis

A randomized, controlled study of remestemcel-L delivered by an endoscope directly to areas of inflammation and tissue injury in up to 48 patients with medically refractory Crohns disease and ulcerative colitis commenced at Cleveland Clinic in October 2020. The investigator-initiated study is the first in humans using local cell delivery in the gut and will enable Mesoblast to compare clinical outcomes using this delivery method with results from an ongoing randomized, placebo-controlled trial in patients with biologic-refractory Crohns disease where remestemcel-L was administered intravenously.

Rexlemestrocel-L

Chronic Heart Failure

The results from the landmark DREAM-HF randomized controlled trial in 537 treated patients with chronic heart failure with reduced left ventricular ejection fraction (HFrEF) who received rexlemestrocel-L (REVASCOR) or control sham, demonstrated that a single dose of rexlemestrocel-L resulted in substantial and durable reductions in heart attacks, strokes, and cardiac deaths. The trials primary endpoint of reduction in volume overload related hospitalizations was not achieved. The results of this trial identify New York Heart Association (NYHA) class II HFrEF patients as the optimal target population for greatest rexlemestrocel-L treatment effect, and therefore a focus for developing rexlemestrocel-L in the largest market in heart failure.

The incidence of heart attacks and strokes were reduced by 60% over a median follow-up period of 30 months following a single dose of rexlemestrocel-L in the entire population of 537 treated patients. The incidence of death from cardiovascular causes was reduced by 60% in the 206 patients with NYHA class II disease, a significant reduction which was evident in both ischemic and non-ischemic subgroups as well as diabetic and nondiabetic patients.

The results also show that the NYHA class II patients in the control group, following an initial period of approximately 20 months of disease stability, progressed to cardiac death rates in-line with NYHA class III patients. NYHA class II patients treated with a single dose of rexlemestrocel-L did not show such cardiac death progression.

The combination of the three pre-specified outcomes of cardiac death, heart attack or stroke into a single composite outcome - called the three-point major adverse cardiovascular events (MACE) is a well-established endpoint used by the FDA to determine cardiovascular risk. Rexlemestrocel-L reduced this three-point MACE by 30% compared to controls across the entire population of 537 treated patients. In the NYHA class II subgroup of 206 patients, rexlemestrocel-L reduced the three-point MACE by 55% compared to controls.

Mesoblast expects feedback from the FDA in the next quarter on the potential pathway to US regulatory approval for rexlemestrocel-L in patients with chronic heart failure.

Chronic Low Back Pain due to Degenerative Disc Disease

The results from the randomized controlled trial of its allogeneic mesenchymal precursor cell (MPC) therapy rexlemestrocel-L in 404 enrolled patients with chronic low back pain (CLBP) due to degenerative disc disease (DDD) refractory to conventional treatments indicate that a single injection of rexlemestrocel-L+hyaluronic acid (HA) carrier may provide a safe, durable, and effective opioid-sparing therapy for patients with chronic inflammatory back pain due to degenerative disc disease, and that greatest benefits are seen when administered earlier in the disease process before irreversible fibrosis of the intervertebral disc has occurred. The trial's composite outcomes of pain reduction together with functional responses to treatment were not met by either MPC group.

The rexlemestrocel-L+HA treatment group achieved substantial and durable reductions in CLBP compared to control through 24 months across the entire evaluable study population (n=391) compared with saline controls. Greatest pain reduction was observed in the pre-specified population with CLBP of shorter duration than the study median of 68 months (n=194) and subjects using opioids at baseline (n=168) with the rexlemestrocel-L+HA group having substantially greater reduction at all time points (1, 3, 6, 12, 18 and 24 months) compared with saline controls. There was no appreciable difference in the safety of MPC groups compared to saline control over the 24-month period of follow-up in the entire study population. In subjects using opioids at baseline, the MPC+HA demonstrated a reduction in the average opioid dose over 24 months, while saline control subjects had essentially no change.

There is a significant need for a safe, efficacious, and durable opioid-sparing treatment in patients with chronic low back pain due to severely inflamed degenerative disc disease. Mesoblast has filed a request and expects to receive feedback from the FDA on the pathway to US regulatory approval in patients with chronic low back pain due to degenerative disc disease.

Intellectual Property

Mesoblast has an extensive patent portfolio with over 1,000 patents and patent applications across 77 patent families, and patent terms extending through 2041. These patents cover composition of matter, manufacturing, and therapeutic applications of mesenchymal lineage cells, and provide strong commercial protection for our products in all major markets, including the United States, Europe, Japan and China. During the fiscal year Mesoblast has significantly expanded its patent portfolio, focusing on areas of its strategic commercial interests.

Licensing agreements with JCR, Grnenthal, Tasly and Takeda highlight the strength of Mesoblast's extensive intellectual property portfolio covering mesenchymal lineage cells. Mesoblast will continue to use its patents to prosecute its commercial rights as they relate to its core strategic product portfolio. When consistent with the Companys strategic objectives, it may consider providing third parties with commercial access to its patent portfolio.

DETAILED FINANCIAL RESULTS

Financial Results for the Year Ended June 30, 2021 (FY2021)

In August we entered into a contractual amendment to extend the interest-only period of its current senior debt facility to at least January 2022 and as a result no loan repayments will be required prior to January 2022. Mesoblast is in active discussions to refinance the facility.

We expect to recognize the existing US$21.9 million of remestemcel-L pre-launch inventory on the balance sheet if we receive FDA approval.

As a result of the above and other remeasurements on revaluation of assets and liabilities, the loss after tax for FY2021 was US$98.8 million compared to US$77.9 million for FY2020. The net loss attributable to ordinary shareholders was 16.33 US cents per share for FY2021, compared with 14.74 US cents per share for FY2020.

Conference Call

There will be a webcast today, beginning at 7.00pm EDT (Monday, August 30, 2021); 9.00am AEST (Tuesday, August 31). It can be accessed via: https://webcast.boardroom.media/mesoblast-limited/20210826/NaN61036c41df5665001c97fc67

The archived webcast will be available on the Investor page of the Companys website: http://www.mesoblast.com

About Mesoblast

Mesoblast is a world leader in developing allogeneic (off-the-shelf) cellular medicines for the treatment of severe and life-threatening inflammatory conditions. The Company has leveraged its proprietary mesenchymal lineage cell therapy technology platform to establish a broad portfolio of late-stage product candidates which respond to severe inflammation by releasing anti-inflammatory factors that counter and modulate multiple effector arms of the immune system, resulting in significant reduction of the damaging inflammatory process.

Mesoblast has a strong and extensive global intellectual property portfolio with protection extending through to at least 2041 in all major markets. The Companys proprietary manufacturing processes yield industrial-scale, cryopreserved, off-the-shelf, cellular medicines. These cell therapies, with defined pharmaceutical release criteria, are planned to be readily available to patients worldwide.

Mesoblast has completed Phase 3 trials of rexlemestrocel-L for advanced chronic heart failure and chronic low back pain. Remestemcel-L is being developed for inflammatory diseases in children and adults including steroid refractory acute graft versus host disease and moderate to severe acute respiratory distress syndrome. Two products have been commercialized in Japan and Europe by Mesoblasts licensees, and the Company has established commercial partnerships in Europe and China for certain Phase 3 assets.

Mesoblast has locations in Australia, the United States and Singapore and is listed on the Australian Securities Exchange (MSB) and on the Nasdaq (MESO). For more information, please see http://www.mesoblast.com, LinkedIn: Mesoblast Limited and Twitter: @Mesoblast

References / Footnotes

Forward-Looking Statements

This announcement includes forward-looking statements that relate to future events or our future financial performance and involve known and unknown risks, uncertainties and other factors that may cause our actual results, levels of activity, performance or achievements to differ materially from any future results, levels of activity, performance or achievements expressed or implied by these forward-looking statements. We make such forward-looking statements pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995 and other federal securities laws. Forward-looking statements should not be read as a guarantee of future performance or results, and actual results may differ from the results anticipated in these forward-looking statements, and the differences may be material and adverse. Forward-looking statements include, but are not limited to, statements about the initiation, timing, progress and results of Mesoblasts preclinical and clinical studies, and Mesoblasts research and development programs; Mesoblasts ability to advance product candidates into, enroll and successfully complete, clinical studies, including multi-national clinical trials; Mesoblasts ability to advance its manufacturing capabilities; the timing or likelihood of regulatory filings and approvals, manufacturing activities and product marketing activities, if any; the commercialization of Mesoblasts product candidates, if approved; regulatory or public perceptions and market acceptance surrounding the use of stem-cell based therapies; the potential for Mesoblasts product candidates, if any are approved, to be withdrawn from the market due to patient adverse events or deaths; the potential benefits of strategic collaboration agreements and Mesoblasts ability to enter into and maintain established strategic collaborations; Mesoblasts ability to establish and maintain intellectual property on its product candidates and Mesoblasts ability to successfully defend these in cases of alleged infringement; the scope of protection Mesoblast is able to establish and maintain for intellectual property rights covering its product candidates and technology; estimates of Mesoblasts expenses, future revenues, capital requirements and its needs for additional financing; Mesoblasts financial performance; developments relating to Mesoblasts competitors and industry; and the pricing and reimbursement of Mesoblasts product candidates, if approved. You should read this press release together with our risk factors, in our most recently filed reports with the SEC or on our website. Uncertainties and risks that may cause Mesoblasts actual results, performance or achievements to be materially different from those which may be expressed or implied by such statements, and accordingly, you should not place undue reliance on these forward-looking statements. We do not undertake any obligations to publicly update or revise any forward-looking statements, whether as a result of new information, future developments or otherwise.

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Operational Highlights and Financial Results for the Year Ended June 30, 2021 - GlobeNewswire

Incyte and MorphoSys Announce the European Commission Approval of Minjuvi (tafasitamab) in Combination With Lenalidomide for the Treatment of Adults…

WILMINGTON, Del. & PLANEGG, Germany & MUNICH--(BUSINESS WIRE)--Incyte (Nasdaq:INCY) and MorphoSys AG (FSE:MOR; NASDAQ:MOR) today announced that the European Commission (EC) has granted conditional marketing authorization for Minjuvi (tafasitamab) in combination with lenalidomide, followed by Minjuvi monotherapy, for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) who are not eligible for autologous stem cell transplant (ASCT). The EC decision follows the positive opinion received from the European Medicines Agencys Committee for Medicinal Products for Human Use (CHMP) in June 2021 recommending the conditional marketing authorization of Minjuvi.

People living with relapsed or refractory DLBCL in the EU, have historically had limited treatment options and a poor prognosis. However, with the ECs approval of Minjuvi, eligible patients now have a new and much needed treatment option, said Herv Hoppenot, Chief Executive Officer, Incyte. We will now focus our efforts on working with individual countries in Europe to provide people access to this new treatment.

The approval of Minjuvi is a crucial milestone for patients with relapsed or refractory DLBCL in Europe, said Jean-Paul Kress, M.D., Chief Executive Officer, MorphoSys. DLBCL is the most common type of non-Hodgkin lymphoma in adults and Minjuvi addresses an urgent unmet medical need for the 30-40% of people who do not respond to or relapse, after initial therapy.

The conditional approval is based on the results from the L-MIND study evaluating the safety and efficacy of Minjuvi in combination with lenalidomide as a treatment for patients with relapsed or refractory DLBCL who are not eligible for autologous stem cell transplant (ASCT). The results showed best objective response rate (ORR) of 56.8% (primary endpoint), including a complete response (CR) rate of 39.5% and a partial response rate (PR) of 17.3%, as assessed by an independent review committee. The median duration of response (mDOR) was 43.9 months after a minimum follow up of 35 months (secondary endpoint). Minjuvi together with lenalidomide was shown to provide a clinically meaningful response and the side effects were manageable. Warnings and precautions for Minjuvi include infusion-related reactions, myelosuppression, including neutropenia and thrombocytopenia, infections and tumour lysis syndrome.

The data from the L-MIND study demonstrate the potential benefits, including long duration of response, that tafasitamab may have for eligible DLBCL patients, said Professor Pier Luigi Zinzani M.D., Ph.D., Head of the Lymphoma Group at University of Bologna. It is encouraging to see new treatments become available for these patients, especially given the historical lack of treatment options in this area.

Incyte and MorphoSys share global development rights to tafasitamab; Incyte has exclusive commercialization rights to tafasitamab outside the United States. Tafasitamab is co-marketed by Incyte and MorphoSys under the brand name Monjuvi in the U.S., and is marketed by Incyte under the brand name Minjuvi in the EU.

About Diffuse Large B-Cell Lymphoma DLBCL is the most common type of non-Hodgkin lymphoma in adults worldwide, comprising 40% of all cases4, and is characterized by rapidly growing masses of malignant B-cells in the lymph nodes, spleen, liver, bone marrow or other organs5. It is an aggressive disease with about one in three patients not responding to initial therapy or relapsing thereafter6. In Europe, each year approximately 16,000 patients are diagnosed with relapsed or refractory DLBCL7,8,9.

About L-MIND The L-MIND trial is a single arm, open-label Phase 2 study (NCT02399085) investigating the combination of tafasitamab and lenalidomide in patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) who have had at least one, but no more than three prior lines of therapy, including an anti-CD20 targeting therapy (e.g., rituximab), who are not eligible for high-dose chemotherapy (HDC) or autologous stem cell transplant (ASCT). The studys primary endpoint is overall response rate (ORR). Secondary outcome measures include duration of response (DoR), progression-free survival (PFS) and overall survival (OS). The study reached its primary completion in May 2019.

For more information about L-MIND, visit https://clinicaltrials.gov/ct2/show/NCT02399085.

About Minjuvi (tafasitamab) Tafasitamab is a humanized Fc-modified cytolytic CD19 targeting monoclonal antibody. In 2010, MorphoSys licensed exclusive worldwide rights to develop and commercialize tafasitamab from Xencor, Inc. Tafasitamab incorporates an XmAb engineered Fc domain, which mediates B-cell lysis through apoptosis and immune effector mechanism including Antibody-Dependent Cell-Mediated Cytotoxicity (ADCC) and Antibody-Dependent Cellular Phagocytosis (ADCP).

In the United States, Monjuvi (tafasitamab-cxix) is approved by the U.S. Food and Drug Administration in combination with lenalidomide for the treatment of adult patients with relapsed or refractory DLBCL not otherwise specified, including DLBCL arising from low grade lymphoma, and who are not eligible for autologous stem cell transplant (ASCT). This indication is approved under accelerated approval based on overall response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).

In Europe, Minjuvi (tafasitamab) received conditional approval, in combination with lenalidomide, followed by Minjuvi monotherapy, for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) who are not eligible for autologous stem cell transplant (ASCT).

Tafasitamab is being clinically investigated as a therapeutic option in B-cell malignancies in several ongoing combination trials.

Minjuvi and Monjuvi are registered trademarks of MorphoSys AG. Tafasitamab is co-marketed by Incyte and MorphoSys under the brand name Monjuvi in the U.S., and marketed by Incyte under the brand name Minjuvi in the EU.

XmAb is a registered trademark of Xencor, Inc.

Safety Information from the EU Summary of Product Characteristics (SmPC) Infusion-related reactions may occur and have been reported more frequently during the first infusion. Patients should be monitored closely throughout the infusion and should be advised to contact their healthcare professionals if they experience signs and symptoms of infusion related reactions including fever, chills, rash or breathing problems within 24 hours of infusion. A premedication should be administered to patients prior to starting tafasitamab infusion. Based on the severity of the infusion-related reaction, tafasitamab infusion should be interrupted or discontinued and appropriate medical management should be instituted.

Fatal and serious infections, including opportunistic infections, occurred in patients during treatment with Minjuvi.

Minjuvi should be administered to patients with an active infection only if the infection is treated appropriately and well controlled. Patients with a history of recurring or chronic infections may be at increased risk of infection and should be monitored appropriately. Patients should be advised to contact their healthcare professionals if fever or other evidence of potential infection, such as chills, cough or pain on urination, develops.

Treatment with Minjuvi in combination with lenalidomide should not be initiated in female patients unless pregnancy has been excluded.

The most common adverse reactions were infections, neutropenia, asthenia, anemia, diarrhea, thrombocytopenia, cough, oedema peripheral, pyrexia and decreased appetite.

Minjuvi may cause serious adverse reactions. The most common serious adverse reactions were infection, including pneumonia and febrile neutropenia.

Treatment with tafasitamab can cause serious or severe myelosuppression including neutropenia, thrombocytopenia and anemia. Complete blood counts should be monitored throughout treatment and prior to administration of each treatment cycle.

About Incyte Incyte is a Wilmington, Delaware-based, global biopharmaceutical company focused on finding solutions for serious unmet medical needs through the discovery, development and commercialization of proprietary therapeutics. For additional information on Incyte, please visit Incyte.com and follow @Incyte.

About MorphoSys MorphoSys (FSE & NASDAQ: MOR) is a commercial-stage biopharmaceutical company dedicated to the discovery, development and commercialization of innovative therapies for people living with cancer and autoimmune diseases. Based on its leading expertise in antibody and protein technologies, MorphoSys is advancing its own pipeline of new drug candidates and has created antibodies which are developed by partners in different areas of unmet medical need. In 2017, Tremfya (guselkumab) developed by Janssen Research & Development, LLC and marketed by Janssen Biotech, Inc., for the treatment of plaque psoriasis became the first drug based on MorphoSys antibody technology to receive regulatory approval. In July 2020, the U.S. Food and Drug Administration (FDA) granted accelerated approval of the companys proprietary product Monjuvi (tafasitamab-cxix) in combination with lenalidomide in patients with a certain type of lymphoma. Headquartered near Munich, Germany, the MorphoSys Group, including the fully owned U.S. subsidiaries MorphoSys US Inc. and Constellation Pharmaceuticals, Inc., has more than 750 employees. For more information visit http://www.morphosys.com or http://www.morphosys-us.com.

Tremfya is a registered trademark of Janssen Biotech, Inc.

Incyte Forward-looking Statements Except for the historical information set forth herein, the matters set forth in this press release, including statements regarding the Companys expectations relating to the use of tafasitamab for treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL), the Companys ongoing clinical development program for tafasitamab, and its DLBCL program generally, contain predictions, estimates, and other forward-looking statements.

These forward-looking statements are based on the Companys current expectations and subject to risks and uncertainties that may cause actual results to differ materially, including unanticipated developments in and risks related to: unanticipated delays; further research and development and the results of clinical trials possibly being unsuccessful or insufficient to meet applicable regulatory standards or warrant continued development; the ability to enroll sufficient numbers of subjects in clinical trials and the ability to enroll subjects in accordance with planned schedules; the effects of the COVID-19 pandemic and measures to address the pandemic on the Companys clinical trials, supply chain, and other third-party providers and development and discovery operations; determinations made by the European Commission and other regulatory authorities; the Companys dependence on its relationships with its collaboration partners; the efficacy or safety of the Companys products and the products of the Companys collaboration partners; the acceptance of the Companys products and the products of the Companys collaboration partners in the marketplace; market competition; sales, marketing, manufacturing, and distribution requirements; and other risks detailed from time to time in the Companys reports filed with the Securities and Exchange Commission, including its annual report and its quarterly report on Form 10-Q for the quarter ended June 30, 2021. The Company disclaims any intent or obligation to update these forward-looking statements.

MorphoSys Forward-looking Statements This communication contains certain forward-looking statements concerning the MorphoSys group of companies. The forward-looking statements contained herein represent the judgment of MorphoSys as of the date of this release and involve known and unknown risks and uncertainties, which might cause the actual results, financial condition and liquidity, performance or achievements of MorphoSys, or industry results, to be materially different from any historic or future results, financial conditions and liquidity, performance or achievements expressed or implied by such forward-looking statements. In addition, even if MorphoSys' results, performance, financial condition and liquidity, and the development of the industry in which it operates are consistent with such forward-looking statements, they may not be predictive of results or developments in future periods. Among the factors that may result in differences are that MorphoSys' expectations may be incorrect, the inherent uncertainties associated with competitive developments, clinical trial and product development activities and regulatory approval requirements, MorphoSys' reliance on collaborations with third parties, estimating the commercial potential of its development programs and other risks indicated in the risk factors included in MorphoSys' Annual Report on Form 20-F and other filings with the U.S. Securities and Exchange Commission. Given these uncertainties, the reader is advised not to place any undue reliance on such forward-looking statements. These forward-looking statements speak only as of the date of publication of this document. MorphoSys expressly disclaims any obligation to update any such forward-looking statements in this document to reflect any change in its expectations with regard thereto or any change in events, conditions or circumstances on which any such statement is based or that may affect the likelihood that actual results will differ from those set forth in the forward-looking statements, unless specifically required by law or regulation.

_____________________ 1 DRG Epidemiology data. 2 Kantar Market Research (TPP testing 2018). 3 Friedberg, Jonathan W. Relapsed/Refractory Diffuse Large B-Cell Lymphoma. Hematology Am Soc Hematol Educ Program 2011; 2011:498-505. doi: 10.1182/asheducation-2011.1.498. 4 Cancer Research UK. Diffuse large B cell lymphoma. Available at https://www.cancerresearchuk.org/about-cancer/non-hodgkin-lymphoma/types/diffuse-large-B-cell-lymphoma. Accessed: May 2021. 5 Sarkozy C, et al. Management of relapsed/refractory DLBCL. Best Practice Research & Clinical Haematology. 2018 31:20916. doi.org/10.1016/j.beha.2018.07.014. 6 Skrabek P, et al. Emerging therapies for the treatment of relapsed or refractory diffuse large B cell lymphoma. Current Oncology. 2019 26(4): 253265. doi.org/10.3747/co.26.5421. 7 DRG Epidemiology data. 8 Kantar Market Research (TPP testing 2018). 9 Friedberg, Jonathan W. Relapsed/Refractory Diffuse Large B-Cell Lymphoma. Hematology Am Soc Hematol Educ Program 2011; 2011:498-505. doi: 10.1182/asheducation-2011.1.498.

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Incyte and MorphoSys Announce the European Commission Approval of Minjuvi (tafasitamab) in Combination With Lenalidomide for the Treatment of Adults...

Thomas Smeenk with an update on the FDA Phase II clinical trial program of the Hemostemix blood-derived, stem cell therapeutics product (ACP-01) -…

In a recent InvestorIntel interview, Peter Clausi speaks with Thomas Smeenk, Co-Founder, President and CEO ofHemostemix Inc. (TSXV: HEM | OTC: HMTXF) about the Phase II Clinical Trial on Hemostemixs ACP-01 which has the potential to treat patients with severe critical limb ischemia.

In this InvestorIntel interview, which may also be viewed on YouTube (click here to subscribe to the InvestorIntel Channel), Thomas went on to say that Hemostemix is in the process of completing the source document verification. With about 60% of source documents verified, Thomas said that they expect the source document verification to be complete within the year.

To watch the full interview,click here

About Hemostemix Inc.

Hemostemix is a publicly traded autologous stem cell therapy company. A winner of the World Economic Forum Technology Pioneer Award, the Company developed and is commercializing its lead product ACP-01 for the treatment of CLI, PAD, Angina, Ischemic Cardiomyopathy, Dilated Cardiomyopathy and other conditions of ischemia. ACP-01 has been used to treat over 500 patients, and it is the subject of a randomized, placebo-controlled, double blind trial of its safety and efficacy in patients with advanced critical limb ischemia who have exhausted all other options to save their limb from amputation.

On October 21, 2019, the Company announced the results from its Phase II CLI trial abstract entitled Autologous Stem Cell Treatment for CLI Patients with No Revascularization Options: An Update of the Hemostemix ACP-01 Trial With 4.5 Year Followup which noted healing of ulcers and resolution of ischemic rest pain occurred in 83% of patients, with outcomes maintained for up to 4.5 years.

The Company owns 91 patents across five patent families titled: Regulating Stem Cells, In Vitro Techniques for use with Stem Cells, Production from Blood of Cells of Neural Lineage, and Automated Cell Therapy.

To know more about Hemostemix Inc.,click here

Disclaimer:Hemostemix Inc. is an advertorial member of InvestorIntel Corp.

Thisinterview, which was produced by InvestorIntel Corp. (IIC)does not contain, nor does it purport to contain, a summary of all the material information concerning theCompany being interviewed. IIC offers no representations or warranties that any of the information contained in this interview is accurate or complete.

This presentationmay containforward-looking statements within the meaning ofapplicable Canadian securities legislation.Forward-looking statements are based on the opinions and assumptions of managementof the Companyas of the date made. Theyare inherently susceptible to uncertainty and other factors that could cause actual events/results to differ materially from these forward-looking statements.Additional risks and uncertainties, including those that the Company does not know about now or that it currently deems immaterial, may also adversely affect the Companys business or any investment therein.

Anyprojectionsgivenare principally intended for use as objectives and are not intended, and should not be taken, as assurances that the projected results will be obtained by the Company.The assumptions used may not prove to be accurateanda potential decline in the Companys financial condition or results of operations may negatively impact the value of its securities. Prospectiveinvestors are urged to review the Companys profile onSedar.comand to carry out independent investigations in order to determine their interest in investing in the Company.

If you have any questions surrounding the content of this interview, please emailinfo@investorintel.com.

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Thomas Smeenk with an update on the FDA Phase II clinical trial program of the Hemostemix blood-derived, stem cell therapeutics product (ACP-01) -...

Cryo-Cell International Announces Uplisting to The Nasdaq Capital Market – GlobeNewswire

Cryo-Cell International, Inc. (Symbol CCEL), The Leader In Cord Blood And Cord Tissue Cryopreservation Services, Today Announced That The Companys Common Stock Has Been Approved For Listing On The Nasdaq Capital Market (Nasdaq).

Trading to Begin August 31, 2021 with Current Ticker CCEL

MIAMI, Aug. 30, 2021 (GLOBE NEWSWIRE) -- Cryo-Cell International has met Nasdaqs stringent financial, liquidity and corporate governance listing requirements. As a result, Cryo-Cell will be listed on Nasdaq as of Tuesday, August 31st 2021. This is expected to improve the liquidity of Cryo-Cell common stock, broaden its institutional shareholder base and ultimately enhance long-term shareholder value.

Cryo-Cells Chairman of the Board and Co-Chief Executive Officer, David Portnoy, stated: I am pleased to announce Cryo-Cells uplisting to Nasdaq, a major financial milestone that the Company had been considering, as previously stated several months ago. I believe that the significant gap between Cryo-Cells current market valuation and our intrinsic value should narrow as our visibility in the investment community grows, and this is a step in that direction. Cryo-Cell International has transformed in 2021 from being a profitable cord blood bank to a fully integrated, biopharmaceutical company that is expected to provide much needed access to experimental treatments for patients at the upcoming Cryo-Cell Institute for Cellular Therapies.

About Cryo-Cell International, Inc.

Founded in 1989, Cryo-Cell International, Inc. is the worlds first private cord blood bank. More than 500,000 parents from 87 countries have entrusted Cryo-Cell International with their babys cord blood and cord tissue stem cells. In addition to its private bank, Cryo-Cell International has a public banking program in partnership with Duke University. Cryo-Cells public bank has provided cord blood for more than 600 transplantations and operates cord blood donation sites across the U.S in prominent hospitals such as CedarsSinai Hospital in Los Angeles and Baptist Hospital in Miami. Cryo-Cells facility is FDA registered, cGMP-/cGTP-compliant and licensed in all states requiring licensure. Besides being AABB accredited as a cord blood facility, Cryo-Cell was also the first U.S. (for private use only) cord blood bank to receive FACT accreditation for adhering to the most stringent cord blood quality standards set by any internationally recognized, independent accrediting organization. Cryo-Cell owns the exclusive rights to PrepaCyte-CB, the industrys most advanced cord blood processing technology.

Cryo-Cells mission has been to provide clients with state-of-the-art cord blood and cord tissue cryopreservation services, raise awareness of the opportunity for expectant parents to bank or donate their babys cord blood and support the advancement of regenerative medicine. In February 2021, Cryo-Cell entered into a license agreement with Duke University that transformed Cryo-Cell into an autonomous, vertically integrated cellular therapy company that will be able to treat patients.

For more information, please visitIR.cryo-cell.com

Forward-Looking Statement

Statements herein the terms believes, intends, projects, anticipates, expects, and similar expressions as used are intended to reflect forward-looking statements of the Company. The information contained herein is subject to various risks, uncertainties and other factors that could cause actual results to differ materially from the results anticipated in such forward-looking statements or paragraphs, many of which are outside the control of the Company. These uncertainties and other factors include the impact of the COVID-19 pandemic on our sales, operations and supply chain, the success of the Companys global expansion initiatives and product diversification, the Companys actual future ownership stake in future therapies emerging from its collaborative research partnerships, the success related to its IP portfolio, the Companys future competitive position in stem cell innovation, future success of its core business and the competitive impact of public cord blood banking on the Companys business, the success of the Companys initiative to expand its core business units to include biopharmaceutical manufacturing and operating clinics, the uncertainty of profitability from its biopharmaceutical manufacturing and operating clinics, the Companys ability to minimize future costs to the Company related to R&D initiatives and collaborations and the success of such initiatives and collaborations, the success and enforceability of the Companys umbilical cord blood and cord tissue license agreements, together with the associated intellectual property and their ability to provide the Company with royalty fees, and those risks and uncertainties contained in risk factors described in documents the Company files from time to time with the Securities and Exchange Commission, including the most recent Annual Report on Form 10-K, Quarterly Reports on Form 10-Q and any Current Reports on Form 8-K filed by the Company. The Company disclaims any obligations to subsequently revise any forward-looking statements to reflect events or circumstances after the date of such statements.

For more information, please contact: Corporate Communications / Investor Relations Diane Glanz, Rph, PharmD. Telephone: +1 (813) 749-2195 Email: Investors@Cryo-Cell.com

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Cryo-Cell International Announces Uplisting to The Nasdaq Capital Market - GlobeNewswire

2021 Pipeline Insight Report on Myocardial Infarction – Featuring AstraZeneca, Mesoblast and Boehringer Ingelheim Among Others -…

DUBLIN--(BUSINESS WIRE)--The "Myocardial Infarction - Pipeline Insight, 2021" clinical trials have been added to ResearchAndMarkets.com's offering.

This "Myocardial infarction - Pipeline Insight, 2021," report provides comprehensive insights about 50+ companies and 50+ pipeline drugs in Myocardial infarction pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

Report Highlights

The companies and academics are working to assess challenges and seek opportunities that could influence Myocardial infarction R&D. The therapies under development are focused on novel approaches to treat/improve Myocardial infarction.

Myocardial infarction Emerging Drugs Chapters

This segment of the Myocardial infarction report encloses its detailed analysis of various drugs in different stages of clinical development, including phase II, I, preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.

Myocardial infarction Emerging Drugs

Dapagliflozin: AstraZeneca

Dapagliflozin is a selective sodium-glucose co-transporter subtype 2 (SGLT2) inhibitor with antihyperglycemic activity. Dapagliflozin selectively and potently inhibits SGLT2 compared to SGLT1, which is the cotransporter of glucose in the gut. The drug is in phase 3 of clinical trials for the treatment of Myocardial infarction.

Remestemcel-L: Mesoblast

AAV-AQP1 is an Adeno-associated virus vector containing gene therapy that contains the human aquaporin-1 gene (AAV2hAQP1) for the treatment of Myocardial infarction. It works on Aquaporin 1 replacement mechanism. It is currently under Phase I/II stage of development. Remestemcel-L is an investigational therapy comprising culture-expanded mesenchymal stromal cells derived from the bone marrow of an unrelated donor. Remestemcel-L is thought to have immunomodulatory properties to counteract the cytokine storms that are implicated in various inflammatory conditions by downregulating the production of pro-inflammatory cytokines, increasing production of anti-inflammatory cytokines, and enabling recruitment of naturally occurring anti-inflammatory cells to involved tissues. The drug is in phase 2 of clinical trials for the treatment of Myocardial infarction.

Myocardial infarction: Therapeutic Assessment

This segment of the report provides insights about the different Myocardial infarction drugs segregated based on following parameters that define the scope of the report, such as:

Major Players in Myocardial infarction

There are approx. 50+ key companies which are developing the therapies for Myocardial infarction. The companies which have their Myocardial infarction drug candidates in the most advanced stage, i.e. phase III include, AstraZeneca.

Phases

This report covers around 50+ products under different phases of clinical development like

Myocardial infarction pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as

Products have been categorized under various Molecule types such as

The drugs have been categorized under various product types like Mono, Combination and Mono/Combination.

Myocardial infarction: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Myocardial infarction therapeutic drugs key players involved in developing key drugs.

Pipeline Development Activities

The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Myocardial infarction drugs.

Myocardial infarction Report Insights

Myocardial infarction Report Assessment

Key Questions Answered

Current Treatment Scenario and Emerging Therapies:

Key Players

For more information about this clinical trials report visit https://www.researchandmarkets.com/r/i2f9gu

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2021 Pipeline Insight Report on Myocardial Infarction - Featuring AstraZeneca, Mesoblast and Boehringer Ingelheim Among Others -...

Mini brains grown in the lab sprout primitive eyes – Freethink

A German-led research team has grown mini brains with light-sensitive eye structures, and they believe it could lead to breakthrough treatments for vision disorders potentially even retinas that could be transplanted into human patients.

Mini brains and more: The mini brains are technically organoids, lab-grown clumps of cells that mimic the function and structure of organs.

Organoids arent nearly as complex as their full-sized counterparts, but theyre useful for research scientists can study organ development, monitor disease progression, and even test new treatments on them.

The eye structures usually developed in pairs at the front of the brain right where youd expect them.

Whats new: When human embryos are about five weeks old, they develop structures called optic cups that will eventually become retinas.

Researchers have grown optic cups in the lab before, and theyve also grown mini brains. Now, researchers at University Hospital Dsseldorf have grown brain organoids with optic cups.

These organoids can help to study brain-eye interactions during embryo development, model congenital retinal disorders, and generate patient-specific retinal cell types for personalized drug testing and transplantation therapies, senior study author Jay Gopalakrishnan said in a press release.

How it works: The researchers had coaxed stem cells into becoming mini brains before, so for the new study, they just added a chemical linked to eye development to the organoids about 20 days into their growth.

Optic cups started developing about 10 days later most often in pairs of two, side-by-side in the front part of the brain, where youd expect eyes to be.

By day 50, the optic cups were fully visible structures that contained lens and corneal tissue and even responded to light mirroring human eye development.

Looking ahead: Of the 314 mini brains grown during the study, 72% had the primitive eyes, suggesting that the method can be reproduced.

The researchers are now trying to get the optic cups to live longer, as that would make the brain organoids even more useful for studies into eye disease treatments, transplantable lab-grown retinas, and more.

Wed love to hear from you! If you have a comment about this article or if you have a tip for a future Freethink story, please email us at tips@freethink.com.

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Mini brains grown in the lab sprout primitive eyes - Freethink

BeiGene and EUSA Pharma Announce China NMPA Approval of QARZIBA (Dinutuximab Beta) for Patients With High-Risk Neuroblastoma – Business Wire

CAMBRIDGE, Mass. & BEIJING & HEMEL HEMPSTEAD, England--(BUSINESS WIRE)--BeiGene, Ltd. (NASDAQ:BGNE; HKEX:06160) and EUSA Pharma (UK), Ltd. today announced that the China National Medical Products Administration (NMPA) has granted QARZIBA (dinutuximab beta) conditional approval for the treatment of high-risk neuroblastoma in patients aged 12 months and above who have previously received induction chemotherapy and achieved at least a partial response, followed by myeloablative therapy and stem cell transplantation, as well as patients with a history of relapsed or refractory (R/R) neuroblastoma with or without residual disease. Dinutuximab beta is a targeted immunotherapy approved by the European Medicines Agency (EMA).i

Dinutuximab beta represents an important biologic therapy for pediatric patients in China, having been listed in the first batch of New Drugs in Urgent Clinical Need Marketed Overseas by the NMPA, commented Xiaobin Wu, Ph.D., President, Chief Operating Officer, and General Manager of China at BeiGene. For these young patients fighting neuroblastoma in China, we are proud to bring the first approved treatment.

We are delighted that the benefit of dinutuximab beta has been recognized in China. This approval represents an important milestone in our mission and collaboration with BeiGene of bringing innovative cancer and rare disease therapies to patients, said Carsten Thiel, Ph.D., Chief Executive Officer of EUSA Pharma.

The approval of dinutuximab beta in China for the treatment of patients with high-risk neuroblastoma was supported by clinical results available from key trials conducted by SIOPEN (The International Society of Paediatric Oncology Europe Neuroblastoma Group) in collaboration with APEIRON Biologics and EUSA Pharma. These randomized controlled trials evaluated the efficacy of dinutuximab beta by comparing the administration of dinutuximab beta with and without interleukin-2 (IL-2) in the first-line treatment of patients with high-risk neuroblastoma and in two single-arm studies in the R/R setting. In the SIOPEN trial (HR-NBL1), the five-year event-free survival (EFS) rate in patients treated with dinutuximab beta was 57% vs. 42% of historical controls (p<0.01) and five-year overall survival (OS) rate was 64% vs. 50% (p0.0001).ii The safety of dinutuximab beta has been evaluated in 514 patients. The most common adverse reactions were pyrexia and pain that occurred despite analgesic treatment. Other frequent adverse reactions were hypersensitivity, vomiting, diarrhea, capillary leak syndrome, and hypotension.

About QARZIBA (dinutuximab beta)

QARZIBA is a monoclonal antibody that is specifically directed against the carbohydrate moiety of disialoganglioside 2 (GD2), which is overexpressed on neuroblastoma cells. Dinutuximab beta was approved by the European Commission in 2017 (See EMA Summary of Product Characteristics (SmPC)) and is indicated for the treatment of high-risk neuroblastoma in patients aged 12 months and above, who have previously received induction chemotherapy and achieved at least a partial response, followed by myeloablative therapy and stem cell transplantation, as well as patients with a history of relapsed or refractory neuroblastoma, with or without residual disease. Prior to the treatment of relapsed neuroblastoma, any actively progressing disease should be stabilized by other suitable measures.

About EUSA Pharma

Founded in March 2015, EUSA Pharma is a world-class biopharmaceutical company focused on oncology and rare disease. The company has extensive commercial operations in the United States and Europe, alongside a direct presence in select other markets across the globe. EUSA Pharma is led by an experienced management team with a strong record of building successful pharmaceutical companies and is supported by significant funding raised from leading life science investor EW Healthcare Partners. For more information please visit http://www.eusapharma.com.

BeiGene Oncology

BeiGene is committed to advancing best and first-in-class clinical candidates internally or with like-minded partners to develop impactful and affordable medicines to patients across the globe. We have a growing R&D team of approximately 2,300 colleagues dedicated to advancing more than 90 clinical trials involving more than 13,000 patients and healthy volunteers. Our expansive portfolio is directed by a predominantly internalized clinical development team supporting trials in more than 40 countries. Hematology-oncology and solid tumor targeted therapies and immuno-oncology are key focus areas for the Company, with both mono- and combination therapies prioritized in our research and development. The Company currently markets three medicines discovered and developed in our labs: BTK inhibitor BRUKINSA in the United States, China, Canada, and additional international markets; and non-FC-gamma receptor binding anti-PD-1 antibody tislelizumab and PARP inhibitor pamiparib in China.

BeiGene also partners with innovative companies who share our goal of developing therapies to address global health needs. We commercialize a range of oncology medicines in China licensed from Amgen and Bristol Myers Squibb. We also plan to address greater areas of unmet need globally through our collaborations including with Amgen, Bio-Thera, EUSA Pharma, Mirati Therapeutics, Seagen, and Zymeworks. BeiGene has also entered into a collaboration with Novartis granting Novartis rights to develop, manufacture, and commercialize tislelizumab in North America, Europe, and Japan.

About BeiGene

BeiGene is a global, science-driven biotechnology company focused on developing innovative and affordable medicines to improve treatment outcomes and access for patients worldwide. With a broad portfolio of more than 40 clinical candidates, we are expediting development of our diverse pipeline of novel therapeutics through our own capabilities and collaborations. We are committed to radically improving access to medicines for two billion more people by 2030. BeiGene has a growing global team of approximately 7,000 colleagues across five continents. To learn more about BeiGene, please visit http://www.beigene.com and follow us on Twitter at @BeiGeneGlobal

BeiGene Forward Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 and other federal securities laws, including statements regarding the planned launch, potential benefits to patients, and opportunity of QARZIBA in China, and other information that is not historical information. Actual results may differ materially from those indicated in the forward-looking statements as a result of various important factors, including BeiGene's ability to demonstrate the efficacy and safety of its drug candidates; the clinical results for its drug candidates, which may not support further development or marketing approval; actions of regulatory agencies, which may affect the initiation, timing and progress of clinical trials and marketing approval; BeiGene's ability to achieve commercial success for its marketed products and drug candidates, if approved; BeiGene's ability to obtain and maintain protection of intellectual property for its technology and drugs; BeiGene's reliance on third parties to conduct drug development, manufacturing and other services; BeiGenes limited operating history and BeiGene's ability to obtain additional funding for operations and to complete the development and commercialization of its drug candidates, as well as those risks more fully discussed in the section entitled Risk Factors in BeiGenes most recent quarterly report on Form 10-Q, as well as discussions of potential risks, uncertainties, and other important factors in BeiGene's subsequent filings with the U.S. Securities and Exchange Commission. All information in this press release is as of the date of this press release, and BeiGene undertakes no duty to update such information unless required by law.

QARZIBA is a registered trademark of EUSA Pharma (UK), Ltd.

GL-DNB-2100014. August 2021.

__________________

References

i European Medicines Agency, Qarziba (previously Dinutuximab beta EUSA and Dinutuximab beta APEIRON Biologics). Accessed: August 2021 via https://www.ema.europa.eu/en/medicines/human/EPAR/qarziba#authorisation-details-section.

ii Ladenstein, R et al. Cancers 2020, 12, 309; doi:10.3390/cancers12020309.

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BeiGene and EUSA Pharma Announce China NMPA Approval of QARZIBA (Dinutuximab Beta) for Patients With High-Risk Neuroblastoma - Business Wire

Study Calls for COVID-19 Vaccination in Patients With Cancer to Enable Optimal Treatment Delivery During Pandemic – OncLive

When utilizing a validated antibody assay against the SARS-CoV-2 spike protein, investigators revealed a high seroconversion rate of 94% among 200 patients with cancer in New York City who had received a full dose of 1 of the FDA-authorized COVID-19 vaccines.

Patients with solid tumors experienced an impressive seroconversion rate of 98% compared with a rate of 85% in those with hematologic malignancies, 70% in those who had received highly immunosuppressive therapies like anti-CD20 agents, and 73% in those who had previously undergone stem cell transplantation. Notably, patients who received treatment with immune checkpoint inhibitors or hormonal therapies experienced seroconversion rates of 97% and 100%, respectively, following vaccination.

We saw very encouraging [data] showing that most patients with a cancer diagnosis have a really high chance of responding to vaccinationsas long as the vaccinations are done in an appropriate manner, [with] both doses administered, Balazs Halmos, MD, MS, study author, director of Thoracic Oncology, and director of Clinical Cancer Genomics at Montefiore Medical Center, told OncLive in an exclusive interview on the research. This was [true] even for [patients who were receiving] active treatment with chemotherapy, targeted therapy, or immunotherapy.

Halmos and colleagues launched this study to develop a better understanding with regard to the immunogenicity of vaccines in a group of patients with a cancer diagnosis in New York City by examining the rates of anti-spike immunoglobulin G (IgG) antibody positivity after receiving 1 of the 3 authorized COVID-19 vaccines.

A total of 213 patients were enrolled to the study through an informed-consent process. Twenty-nine additional patients with cancer who received the SARS-CoV-2 spike IgG testing were identified through retrospective chart review.

A total of 18 patients did not have this test conducted following consent, and thus, they were excluded from the analysis. Twenty additional patients were excluded because they had their test done before having received full vaccination in accordance with FDA guidance. Four additional patients were excluded for other reasons.

As such, 233 patients with cancer were noted to have received all required doses of their COVID-19 vaccine; all these patients were included in the safety analysis. A subset of 200 patients received the IgG test and were included in the immunogenicity analysis. Serological information from these patients were utilized in association studies between cancer subtypes and therapies.

Investigators also examined the link between the quantitative titer of SARS-CoV-2 spike IgG and cancer subtypes and therapies. If the 200 patients, 185 had available IgG titers that were at least 2 days following the last vaccine dose. A total of 15 patients were excluded from the vaccination cohort with titers; these patients had received the vaccine, but titers were checked less than 1 week from their last dose.

Among those included in the efficacy analysis (n = 200), the median age was 67 years (range, 27-90), 58% were female, and 42% were male. The study population was noted to be representative of the diverse population that resides in the Bronx, New York, with 32% of patients identifying as African American, 39% as Hispanic, 22% as Caucasian, 5% as Asian, and 3% as other ethnicities.

Additionally, 67% of patients had a solid tumor diagnosis and 33% had a hematologic malignancy. Among those with solid tumors, 26% had breast cancer, 14% had gastrointestinal cancer, 9% had genitourinary cancer, 5% had gynecologic cancer, 13% had thoracic or head and neck cancer, 1% had skin or musculoskeletal cancer, and 1% had carcinoma of an unknown primary. Among those with hematologic malignancies, 13% had lymphoid disease, 9% had myeloid disease, and 11% had plasma cell disease.

Seventy-five percent of patients had an active malignancy and 67% were receiving active treatment at the time that they received the COVID-19 vaccine. Fifty-six percent of patients were on active chemotherapy. Moreover, 19% of patients were on active chemotherapy within 48 hours of receiving at least 1 of their COVID-19 vaccine doses.

Fifty-four percent of patients completed vaccination with the Pfizer vaccine, 31% with the Moderna vaccine, and 10% with the Johnson & Johnson vaccine. A total of 3 patients had received a complete mRNA vaccination series but the information regarding the type of vaccine (Pfizer vs Moderna) are not yet available.

Additional findings from the study showed that significantly higher titer values were observed in solid tumors vs hematologic malignancies among a subgroup of 185 patients with available IgG titers longer than 7 days post vaccination, at a median of 7858 AU/mL vs a median of 2528 AU/mL, respectively (P = .013).

When comparing patients who were receiving active cancer treatment vs those who were not, no significant differences in seroconversion were reported, at 96% and 93%, respectively. However, investigators did report lower seropositivity rates in those who were on active cytotoxic chemotherapy versus other treatments, at 92% vs 99%, respectively (P = .04). Moreover, significantly lower seroconversion rates were also noted in those who received immunosuppressive therapies like stem cell transplant (73%; P = .0002), CD20 antibody therapy (70%; P = .0001), or CAR T-cell therapy (all seronegative; P = .0002).

Significantly lower titer levels were observed in patients who received CD20 antibody therapy vs the overall patient population, which underscored the susceptibility of patients receiving these treatments during the pandemic.

No statistically significant associations between age, ethnicity, time since immunosuppressive therapy, steroid use, or treatment within 48 hours of a vaccine dose, and seropositivity were reported.

Although all patients who were receiving CDK4/6 inhibitor treatment demonstrated positive anti-spike IgG test results, notably antibody titers were noted to be very low in this subset (n = 5), at a median of 1242 AU/mL vs a median of 6887 AU/mL in the overall cohort. Given the known involvement of the CDK4/6 pathway in immune activation, this might be biologically plausible and warrants further studies into the impact of CDK4/6 inhibitors on vaccine efficacy, the study authors noted.

A trend to lower titers were also reported among subsets of patients who received BCL-2 or BTK inhibitors.

Among a subset of 22 patients with cancer who had previously been infected with COVID-19, the seroconversion rate was 95%. Notably, antibody titers in those who had prior infection with the virus were found to be significantly higher than those who did not have a known prior infection, at a median of 46,737 AU/mL and a median of 5296 AU/mL, respectively (P < .001).

Our study, along with other emerging data, strongly highlights the continued need to vaccinate patients with a cancer diagnosis urgently and broadly, as vaccinations are likely to be highly effective, the study authors concluded. On the other hand, our study highlights at-risk cohorts of patients, in particular patients with hematologic malignancies following receipt of immunosuppressive therapies such as stem cell transplantation, anti-CD20 therapies, and CAR T-cell treatments. These cohorts of patients could potentially benefit from passive immunization with anti-COVID antibodies in the face of the ongoing pandemic.

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Study Calls for COVID-19 Vaccination in Patients With Cancer to Enable Optimal Treatment Delivery During Pandemic - OncLive