bluebird bio Reports Fourth Quarter and Full Year 2019 Financial Results and Highlights Operational Progress – Yahoo Finance

- First conditional approval of ZYNTEGLOTM (autologous CD34+ cells encoding A-T87Q-globin gene) gene therapy for patients 12 years and older with transfusion-dependent -thalassemia who do not have 0/0 genotype in Europe achieved in 2019; Germany launch underway

- Announced positive top-line data from pivotal Phase 2 KarMMa study of ide-cel in relapsed and refractory multiple myeloma

- Presented clinical data across studies of LentiGlobin gene therapy for -thalassemia (betibeglogene autotemcel) and LentiGlobin gene therapy for sickle cell disease (SCD) and bb21217 in multiple myeloma at American Society of Hematology (ASH) Annual Meeting

- Ended quarter with $1.24 billion in cash, cash equivalents and marketable securities

bluebird bio, Inc. (NASDAQ: BLUE) today reported financial results and business highlights for the fourth quarter and full year ended December 31, 2019.

"2019 was truly a transformative year for bluebird, with our first commercial product now launched in Europe and exciting progress across our first four clinical programs and pipeline," said Nick Leschly, chief bluebird. "Notably, our data in SCD continues to build, and at the ASH annual meeting in December we presented data that showed a 99% reduction in the annualized rate of vaso-occlusive crises (VOC) and acute chest syndrome (ACS) in HGB-206 Group C patients with history of VOCs and ACS who had at least six months follow-up. In -thalassemia, the consistency with which patients who do not have a 0/0 genotype in our Northstar-2 (HGB-207) study are achieving transfusion independence is very encouraging and were starting to see indications that we may be able to see similar outcomes with many patients with 0/0 genotypes as well in our Northstar-3 (HGB-212 study). These data put us in a strong position as we progress our European launch, currently underway in Germany. At the end of 2019, we also announced positive top-line data from the pivotal KarMMa study of ide-cel. We and our partners at BMS look forward to submitting these data to the FDA in the first half of this year. Amidst all of our progress in 2019, our birds demonstrated time and again their dedication to patients and ability to meet and learn from the many challenges we have faced along the way. I look forward to facing the challenges of 2020 with this amazing flock."

Recent Highlights:

TRANSFUSION-DEPENDENT -THALASSEMIA

SICKLE CELL DISEASE (SCD)

MULTIPLE MYELOMA

COMPANY

Upcoming Anticipated Milestones:

Fourth Quarter and Full Year 2019 Financial Results

LentiGlobin for -thalassemia Safety

Non-serious adverse events (AEs) observed during the HGB-204, HGB-207 and HGB-212 clinical studies that were attributed to LentiGlobin for -thalassemia were hot flush, dyspnoea, abdominal pain, pain in extremities, thrombocytopenia, leukopenia, neutropenia and non-cardiac chest pain. One serious adverse event (SAE) of thrombocytopenia was considered possibly related to LentiGlobin for -thalassemia for TDT.

Additional AEs observed in clinical studies were consistent with the known side effects of HSC collection and bone marrow ablation with busulfan, including SAEs of veno-occlusive disease.

With more than five years of follow-up to date, there have been no new unexpected safety events, no deaths, no graft failure and no cases of vector-mediated replication competent lentivirus or clonal dominance. In addition, there have been no new reports of veno-occlusive liver disease (VOD) as of the data cutoff presented at ASH.

About LentiGlobin for -Thalassemia (betibeglogene autotemcel)

The European Commission granted conditional marketing authorization for LentiGlobin for -thalassemia, to be marketed as ZYNTEGLO (autologous CD34+ cells encoding A-T87Q-globin gene) gene therapy, for patients 12 years and older with TDT who do not have a 0/0 genotype, for whom hematopoietic stem cell (HSC) transplantation is appropriate, but a human leukocyte antigen (HLA)-matched related HSC donor is not available.

TDT is a severe genetic disease caused by mutations in the -globin gene that result in reduced or significantly reduced hemoglobin (Hb). In order to survive, people with TDT maintain Hb levels through lifelong chronic blood transfusions. These transfusions carry the risk of progressive multi-organ damage due to unavoidable iron overload.

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LentiGlobin for -thalassemia adds functional copies of a modified form of the -globin gene (A-T87Q-globin gene) into a patients own hematopoietic (blood) stem cells (HSCs). Once a patient has the A-T87Q-globin gene, they have the potential to produce HbAT87Q, which is gene therapy-derived hemoglobin, at levels that may eliminate or significantly reduce the need for transfusions.

The conditional marketing authorization for ZYNTEGLO is only valid in the 28 member states of the EU as well as Iceland, Liechtenstein and Norway. For details, please see the Summary of Product Characteristics (SmPC).

The U.S. Food and Drug Administration granted LentiGlobin for -thalassemia Orphan Drug status and Breakthrough Therapy designation for the treatment of TDT.

bluebird bio has initiated its rolling BLA submission of LentiGlobin for -thalassemia for approval in the U.S. and is engaged with the FDA in discussions regarding the requirements and timing of certain information to be provided in the BLA, including information regarding various release assays for LentiGlobin for -thalassemia. Subject to these ongoing discussions, the company is currently planning to complete the BLA submission in the second half of 2020.

LentiGlobin for -thalassemia continues to be evaluated in the ongoing Phase 3 Northstar-2 and Northstar-3 studies. For more information about the ongoing clinical studies, visit http://www.northstarclinicalstudies.com or clinicaltrials.gov and use identifier NCT02906202 for Northstar-2 (HGB-207), NCT03207009 for Northstar-3 (HGB-212).

bluebird bio is conducting a long-term safety and efficacy follow-up study (LTF-303) for people who have participated in bluebird bio-sponsored clinical studies of LentiGlobin for -thalassemia. For more information visit: https://www.bluebirdbio.com/our-science/clinical-trials or clinicaltrials.gov and use identifier NCT02633943 for LTF-303.

About bluebird bio, Inc.

bluebird bio is pioneering gene therapy with purpose. From our Cambridge, Mass., headquarters, were developing gene therapies for severe genetic diseases and cancer, with the goal that people facing potentially fatal conditions with limited treatment options can live their lives fully. Beyond our labs, were working to positively disrupt the healthcare system to create access, transparency and education so that gene therapy can become available to all those who can benefit.

bluebird bio is a human company powered by human stories. Were putting our care and expertise to work across a spectrum of disorders including cerebral adrenoleukodystrophy, sickle cell disease, -thalassemia and multiple myeloma, using three gene therapy technologies: gene addition, cell therapy and (megaTAL-enabled) gene editing.

bluebird bio has additional nests in Seattle, Wash.; Durham, N.C.; and Zug, Switzerland. For more information, visit bluebirdbio.com.

Follow bluebird bio on social media: @bluebirdbio, LinkedIn, Instagram and YouTube.

ZYNTEGLO, LentiGlobin, and bluebird bio are trademarks of bluebird bio, Inc.

The full common name for ZYNTEGLO: A genetically modified autologous CD34+ cell enriched population that contains hematopoietic stem cells transduced with lentiviral vector encoding the A-T87Q-globin gene.

Forward-Looking Statements

This release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including statements regarding the companys financial condition, results of operations, as well as statements regarding the plans for regulatory submissions and commercialization for ZYNTEGLO and the companys product candidates, including anticipated regulatory milestones, the execution of the companys commercial launch plans, planned clinical studies, as well as the companys intentions regarding the timing for providing further updates on the development and commercialization of ZYNTEGLO and the companys product candidates. Any forward-looking statements are based on managements current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to, the risks that the preliminary positive efficacy and safety results from our prior and ongoing clinical trials will not continue or be repeated in our ongoing or future clinical trials; the risk of cessation or delay of any of the ongoing or planned clinical studies and/or our development of our product candidates; the risk that the current or planned clinical trials of our product candidates will be insufficient to support regulatory submissions or marketing approval in the United States and European Union; the risk that regulatory authorities will require additional information regarding our product candidates, resulting in delay to our anticipated timelines for regulatory submissions, including our applications for marketing approval; the risk that we will encounter challenges in the commercial launch of ZYNTEGLO in the European Union, including in managing our complex supply chain for the delivery of drug product, in the adoption of value-based payment models, or in obtaining sufficient coverage or reimbursement for our products; the risk that our collaborations, including the collaborations with Bristol-Myers Squibb and Forty Seven, will not continue or will not be successful; and the risk that any one or more of our product candidates, will not be successfully developed, approved or commercialized. For a discussion of other risks and uncertainties, and other important factors, any of which could cause our actual results to differ from those contained in the forward-looking statements, see the section entitled "Risk Factors" in our most recent Form 10-K, as well as discussions of potential risks, uncertainties, and other important factors in our subsequent filings with the Securities and Exchange Commission. All information in this press release is as of the date of the release, and bluebird bio undertakes no duty to update this information unless required by law.

bluebird bio, Inc.Condensed Consolidated Statements of Operations and Comprehensive Loss(in thousands, except per share data)(unaudited)

For the three months endedDecember 31,

For the year endedDecember 31,

2019

2018

2019

2018

Revenue:

Collaboration revenue

$ 7,159

$ 18,382

$ 36,469

$ 52,353

License and royalty revenue

2,838

861

8,205

2,226

Total revenues

9,997

19,243

44,674

54,579

Operating expenses:

Research and development

161,821

119,722

582,413

448,589

Selling, general and administrative

76,202

53,508

271,362

174,129

Cost of license and royalty revenue

1,073

818

2,978

885

Change in fair value of contingent consideration

1,435

2,156

2,747

2,999

Total operating expenses

240,531

176,204

859,500

626,602

Loss from operations

(230,534)

(156,961)

(814,826)

(572,023)

Interest income, net

6,855

6,209

34,761

14,624

Other (expense) income, net

535

1,916

(10,088)

1,961

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bluebird bio Reports Fourth Quarter and Full Year 2019 Financial Results and Highlights Operational Progress - Yahoo Finance

Could this patch help mend a broken heart? – Medical Design & Outsourcing

(Image from Trinity College Dublin)

Researchers in Ireland have developed a prototype patch that they say does the same job as crucial aspects of heart tissue.

The patch was designed to withstand the mechanical demands of heart tissue and mimic the electrical signaling properties that allow the heart to pump blood throughout the body. The researchers believe it brings medtech one step closer to a functional design that could mend a broken heart.

Cardiac patches lined with heart cells can be applied surgically to restore heart tissue in patients who have had damaged tissue removed after a heart attack and to repair congenital heart defects in infants and children. Ultimately, though, the goal is to create cell-free patches that can restore the synchronous beating of the heart cells, without impairing the heart muscle movement. The bioengineers report their work in the journal Advanced Functional Materials.

Researchers are continuously looking to develop new treatments which can include stem cell treatments, biomaterial gel injections and assistive devices, said senior author Michael Monaghan, an assistant professor at Trinity College Dublin, in a news release. Ours is one of few studies that looks at a traditional material, and through effective design allows us to mimic the direction-dependent mechanical movement of the heart, which can be sustained repeatably. This was achieved through a novel method called melt electrowriting and through close collaboration with the suppliers located nationally we were able to customize the process to fit our design needs.

This work was performed in the Trinity Centre for Biomedical Engineering, based in the Trinity Biomedical Sciences Institute in collaboration with Spraybase, a subsidiary of Avectas Ltd.

The mechanical demands of heart muscle cannot be met using polyester-based thermoplastic polymers, which are predominantly the approved options for biomedical applications, according to the researchers. However, the functionality of thermoplastic polymers could be leveraged by its structural geometry. They made a patch that could control the expansion of a material in multiple directions and tune this using an engineering design approach.

The patches were manufactured via melt electrowriting, a core technology of Spraybase, which the company says is reproducible, accurate and scalable. The patches were also coated with the polymer polypyrrole to provide electrical conductivity while maintaining cell compatibility. The patch withstood repeated stretching, which is a dominant concern for cardiac biomaterials, and showed good elasticity, to accurately mimic that key property of heart muscle.

Essentially, our material addresses a lot of requirements, Monaghan said. The bulk material is currently approved for medical device use, the design accommodates the movement of the pumping heart, and has been functionalized to accommodate signaling between isolated contractile tissues. This study currently reports the development of our method and design, but we are now looking forward to furthering the next generation of designs and materials with the eventual aim of applying this patch as a therapy for a heart attack.

Read more:
Could this patch help mend a broken heart? - Medical Design & Outsourcing

UCLA researchers discover new compound that promotes lung health – UCLA Newsroom

Sarah C.P. Williams | February 18, 2020

A molecule identified by UCLA researchers helps maintain a healthy balance of cells in airway and lung tissue. If the compound, so far only studied in isolated human and mouse cells, has the same effect in people, it may lead to new drugs to treat or prevent lung cancer.

We think this could help us develop a new therapy that promotes airway health, said Dr. Brigitte Gomperts, a UCLA professor of pediatrics and of pulmonary medicine, a member of theEli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA, and lead author of the study. This could not only inform the treatment of lung cancer, but help prevent its progression in the first place.

Humans respiratory systems are constantly being injured by pollution and germs in the air we breathe and must be replenished with healthy cells. That process is driven by airway basal stem cells, which divide to produce both more stem cells and the mucociliary cells that line the airways and lungs.

There are two types of mucociliary cells: mucus cells, which produce the mucus that trap toxic and infectious particles, and ciliated cells, which have finger-like projections that sweep the mucus away to keep the respiratory system healthy and clear. In healthy lungs, airway basal stem cells stay balanced between producing mucociliary cells and self-renewing to maintain a population of stem cells.

In precancerous cells in the lungs, basal stem cells divide more often than usual, generating a large number of stem cells but too few mucociliary cells. The resulting imbalance of cells in the airway leaves the airways unable to properly clear debris, and it creates a greater risk that the precancerous cells will give rise to a tumor.

In the new study, published today in Cell Reports, Gomperts and her colleagues analyzed airway cells from equal numbers of biopsies of healthy people, people with premalignant lung cancer lesions and people with squamous lung cancer. They discovered that one group of molecules collectively called the Wnt/beta-catenin signaling pathway was present at different levels in the basal stem cells of the patient samples versus the cells from healthy people.

And when the researchers altered the levels of these molecules in healthy airway cells from mice, the balance between stem cells and mucociliary cells shifted, mimicking the imbalance seen in lung pre-cancers.

When you activate the Wnt/beta-catenin signaling pathway, these stem cells just divide and divide, said Gomperts, who is also a member of the UCLA Jonsson Comprehensive Cancer Center.

Finally, the team screened more than 20,000 chemical compounds for their ability to reverse this effect in human cells, lowering levels of Wnt and restoring the balance of stem cells and mature airway cells.

One compound stood out for its ability to limit the proliferation of basal stem cells and restore the balance of the stem cells and mucociliary cells to normal. The compound was also less toxic to airway cells than other, previously discovered, molecules that block Wnt/beta-catenin signaling. The team named the compound Wnt Inhibitor Compound 1, or WIC1.

The identification of this new drug is a nice tool to tease apart the biology of the Wnt/beta-catenin signaling pathway and its effects on lung health, said Cody Aros, the first author of the new paper and a UCLA graduate student. Its also very exciting that it may act in a new way than other existing Wnt/beta-catenin signaling pathway inhibitors and has such low toxicity.

Since WIC1 was identified through a random drug screen, the researchers dont yet know exactly how it works, but theyre planning future studies on its mechanism and safety.

The compound tested by the researchers was used in preclinical tests only and has not been tested in humans or approved by the Food and Drug Administration as safe and effective for use in humans.

The newly identified compound is covered by a patent application filed by the UCLA Technology Development Group on behalf of the Regents of the University of California, with Gomperts and Aros as co-inventors.

Funding for the study was provided in part by the National Institutes of Health, the National Cancer Institute, the Tobacco Related Disease Research Program and the Broad Stem Cell Research Center Training Program, including support from the Rose Hills Foundation Graduate Scholarship.

See original here:
UCLA researchers discover new compound that promotes lung health - UCLA Newsroom

Exosome-Based Treatment Targeting KRAS G12D-Mutated Pancreatic Cancer to Enter Human Studies Next Month – Precision Oncology News

NEW YORK Researchers have been hunting for ways to target alterations in KRAS ever since the mutated gene was first discovered in cancer in the early 1980s. Now, as drugmakers race to bring the first drugs targeting mutated KRAS protein to cancer patients, one scientist at MD Anderson Cancer Center is trying a different approach to get at this elusive target: exosomes.

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Exosome-Based Treatment Targeting KRAS G12D-Mutated Pancreatic Cancer to Enter Human Studies Next Month - Precision Oncology News

BEGEV Salvage Regimen Linked to High Complete Remission Rates – Cancer Therapy Advisor

Previous research has suggested that patients with classical Hodgkin lymphoma who are refractory to or relapse after first-line therapy may be more likely to be cured after receiving second-line salvage chemotherapy followed by autologous stem cell transplantation (ASCT) than after receiving standard-dose chemotherapy.1,2 High complete remission rates prior to ASCT have been shown to predict better long-term outcomes for patients with the condition.3 Researchers have investigated a number of combination therapy regimens as induction regimens prior to ASCT for refractory to or relapsed lymphoma, yielding pre-ASCT CR rates ranging from 20% to 54% and overall response rates (ORRs) of 60% to 80%.4

A prospective multicenter single-arm phase 2 study also indicated that the combination of bendamustine, gemcitabine, and vinorelbine (BEGEV) as second-line chemotherapy before ASCT, in 59 patients with relapsed/refractory (R/R) classical Hodgkin lymphoma, resulted in a CR of 73% and ORR of 83%.5

Now, updated 5-year results from that same study have confirmed the benefits, showing a CR rate of 75% and ORR of 83%.6 But experts cautioned that a randomized trial would be needed to determine whether the regimen is superior to other treatments.

The update[d] 5-year results ofBEGEV (bendamustine, gemcitabine, vinorelbine) followed by autologous stem celltransplantation in patients with Hodgkin disease refractory or relapsing tofirst-line therapy confirm the significant data in terms of CR rate (75%) aswell as PFS (59%) and overall survival (78%), lead study author ArmandoSantoro, MD, director of the Cancer Center at Humanitas Research Hospital,Milan, said in an email.

The trial involved 27 patientswith classical Hodgkin lymphoma who were refractory to and 32 patients whorelapsed after receiving 1 line of chemotherapy. The patients received 800 mg/mgemcitabine on days 1 and 4, 20 mg/ m vinorelbine on day 1, and 90 mg/mbendamustine on days 2 and 3. The patients received 100 mg prednisolone on days1 to 4. They were given 4 cycles of BEGEV every 21 days.

Those patients who experiencedcomplete response or partial response after completing the 4 cycles receivedmyeloablative therapy with carmustine, etoposide, cytarabine and melphalan orfotemustine, etoposide, cytarabine and melphalan, which preceded the reinfusionof CD341 cells.

The researchers evaluated diseaseresponses before and after the fourth BEGEV cycle using computed tomography and18F-fluorodeoxyglucosepositron emission tomography scans accordingto the International Working Group response criteria.

The treatment prompted CR in 44patients (75%) and partial response (PR) in 5 (8%), yielding an OR of 83%. Theauthors noted that the CR rate was higher than the 54% CR rate previouslyreported for the ifosfamide, gemcitabine, vinorelbine, and prednisolone (IGEV)regimen as well as other second-line chemotherapy-based salvage regimens.

Of 49 patients who responded toBEGEV and were eligible for ASCT, 43 ended up receiving transplants. After 5years following BEGEV treatment, the PFS for all series was 59% and OS was 78%.More than 90% of patients were at high risk for relapse post-ASCT. At 5 years,33 patients who received transplants experienced continuous CR, 7 relapsed, and3 died due to unrelated factors that included pneumonia, infection, andmultiorgan failure.

Excerpt from:
BEGEV Salvage Regimen Linked to High Complete Remission Rates - Cancer Therapy Advisor

US FDA Grants Orphan Drug Designation for Retrotopes RT001 in the Treatment of Progressive SupraNuclear Palsy (PSP) – Yahoo Finance

LOS ALTOS, Calif., Feb. 18, 2020 (GLOBE NEWSWIRE) -- Retrotope announced today that the U.S. Food and Drug Administration (FDA) Office of Orphan Products Development granted orphan drug designation for its chemically-modified polyunsaturated fatty acid drug (RT001) for the treatment of Progressive SupraNuclear Palsy (PSP). Physicians collaborating with Retrotope have previously received approval from the FDAs Division of Neurology Products to test RT001 in Expanded Access trials of three patients having PSP, an orphan neurodegenerative disorder that causes progressive impairment of balance and walking; impaired eye movement, abnormal muscle rigidity; dysarthria; and dysphagia and eventual death.

PSP is a serious neurodegenerative disease that profoundly affects the quality and length of life in adults. Patients are typically severely disabled within 3-5 years of disease onset. It affects an estimated 17,500 adults in the US. In addition to the motor deficits noted above, affected individuals frequently experience personality changes and cognitive impairment. Symptoms typically begin after age 60 but can begin earlier. The exact cause of PSP is unknown, and is often misdiagnosed as Parkinson disease due to the similarity of symptoms in the early stage of disease. The cause of PSP is not known, but it is a form of tauopathy, in which abnormal phosphorylation and accumulation of the protein tau in the mid brain leads to destruction of vital protein filaments in nerve cells, causing their death. Most cases appear to be sporadic as an acquired tauopathy and there is no approved drug therapy. A regionally specific increase in lipid peroxidation has been observed in PSP and mitochondrial structures and functions are compromised, leading to profound oxidation damage.

RT001 is a chemically stabilized fatty acid that confers resistance to lipid peroxidation in mitochondrial and cellular membranes via a novel mechanism. RT001 has been shown to decrease levels of lipid peroxidation in PSP patient mesenchymal stem cells, and restore mitochondrial structure and function to damaged cells. As RT001 is distributed, as an essential fat, throughout tissues in human, it is expected to lower the amount of lipid peroxidation, restore normal mitochondrial function and prevent mitochondrial cell death.

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Robert Molinari, Ph.D. CEO of Retrotope commented: We want to sincerely thank the FDAs OOPD for this designation which allows us accelerated review and more flexibility in pursuing this indication. We are also grateful to the researchers, patients and clinicians whose work contributed to the results supporting our filing an investigational new drug (IND) application to FDA in this terrible disease.

Peter Milner, MD, Chief Medical Officer of Retrotope, added, PSP is a disease involving modification and dysfunction of tau protein. RT001s mechanism of action both lowers lipid peroxidation and prevents mitochondrial cell death of neurons which is associated with disease onset and progression. Also RT001 has a synergistic downstream benefit in the pathophysiology of PSP. This orphan designation encourages clinical trials and rapid review of data from trials to treat this intractable disease.

About the Orphan Drug ActThe US FDA Orphan Drug Act (ODA) provides orphan designation for drugs and biological products to treat a rare disease or condition upon request of a sponsor. Orphan drugs are usually defined as those products intended for the safe and effective treatment, diagnosis or prevention of rare diseases/disorders that affect fewer than 200,000 people in the U.S., or that affect more than 200,000 persons but are not expected to recover the costs of developing and marketing a treatment drug. Orphan designation qualifies the sponsor of the drug for various development incentives of the ODA, including tax credits for qualified clinical testing. A marketing application for a prescription drug product that has received orphan designation is not subject to a prescription drug user fee unless the application includes an indication for other than the rare disease or condition for which the drug was designated.

About RT001RT001 is a patented, first-in-class, orally available D-PUFA, a deuterated polyunsaturated fatty acid, that incorporates into mitochondrial and cellular membranes and stabilizes them. Retrotope and others have discovered that lipid peroxidation, the free-radical damage of polyunsaturated fats (PUFAs) in mitochondrial and cellular membranes, may be the primary source of cell death in several degenerative diseases. The presence of D-PUFAs (RT001) can help protect (fireproof) against this attack and potentially restore cellular health.

About RetrotopeRetrotope, a privately held, clinical-stage pharmaceutical company, is creating a new category of drugs to treat degenerative diseases. Composed of proprietary compounds that are chemically stabilized forms of essential nutrients, these compounds are being studied as disease-modifying therapies for many intractable diseases, such as Parkinsons, Alzheimers, mitochondrial myopathies, and retinopathies. RT001, Retrotopes first lead candidate, is being tested in clinical trials for the treatment of Friedreich's ataxia, a fatal orphan disease; and in a fatal, childhood neurodegenerative disease called Infantile Neuroaxonal Dystrophy, and now in PSP which is also fatal. Expanded Access trials calibrating endpoint effects of RT001 in ALS, PSP, Huntingtons disease, and others are also underway. For more information about Retrotope, please visit http://www.retrotope.com.

https://www.ninds.nih.gov/Disorders/Patient-Caregiver-Education/Fact-Sheets/Progressive-Supranuclear-Palsy-Fact-Sheet

SOURCE: Retrotope, Inc. 4300 El Camino Real, Suite 201 Los Altos, CA 94022 650-575-7551

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US FDA Grants Orphan Drug Designation for Retrotopes RT001 in the Treatment of Progressive SupraNuclear Palsy (PSP) - Yahoo Finance

Mustang Bio Announces First Subject Treated in Phase 1/2 Trial with the Optimized CD20-targeted CAR T Cell Therapy MB-106The subject achieved a…

NEW YORK, Feb. 18, 2020 (GLOBE NEWSWIRE) -- Mustang Bio, Inc. (Mustang) (NASDAQ: MBIO), a clinical-stage biopharmaceutical company focused on translating todays medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors and rare genetic diseases, today announced that the first subject treated with the optimized MB-106 (CD20-targeted, autologous CAR T cell therapy) manufacturing process, developed in collaboration between Mustang and Fred Hutchinson Cancer Research Center (Fred Hutch), has achieved a complete response (CR) at the lowest starting dose in an ongoing Phase 1/2 clinical trial. The trial is evaluating the safety and efficacy of MB-106 in subjects with relapsed or refractory B-cell non-Hodgkin lymphomas.

Manuel Litchman, M.D., President and Chief Executive Officer of Mustang, said, We are thrilled to announce that we have achieved a complete response in the first subject dosed with MB-106 following Mustang and Fred Hutchs enhancement to the cell process. MB-106 targets CD20, a commercially validated target on the surface of cancer cells that has lacked a strong CAR T-based clinical focus in the U.S. The complete response was seen on Day 28 in a subject with relapsed follicular lymphoma, and no cytokine release syndrome or neurologic toxicity were observed. The response was especially encouraging, since the subject received a dose of only 3.3 x 105 CAR T cells/kg. While this initial success of the optimized MB-106 is important, additional clinical testing is necessary. We are looking forward to follow-up data, as well as continuing to establish the safety of the therapy, which appears to be well tolerated to date. We are excited to work further with Fred Hutch to develop MB-106 and anticipate providing additional clinical results by year end.

The Phase 1/2, open-label, dose-escalation trial is evaluating the maximum tolerated dose of MB-106. Secondary endpoints include safety and toxicity, preliminary antitumor activity as measured by overall response rate and complete remission rate, progression-free survival, and overall survival. Fred Hutch intends to enroll approximately 30 subjects on the trial, which is being led by principal investigator Mazyar Shadman, M.D., M.P.H., Assistant Member of Fred Hutchs Clinical Research Division.

About B-cell Non-Hodgkin Lymphoma (NHL)There are several forms of NHL, including follicular lymphoma, mantle cell lymphoma, marginal zone lymphoma, lymphoplasmacytic lymphoma and small lymphocytic lymphoma, which account collectively for about 45% of all cases of NHL. Most types of NHL are incurable with available therapies, except for allogenic hematopoietic stem cell transplant (allo-SCT). More than 70,000 new cases of B-cell NHL are diagnosed each year in the United States, and more than 19,000 patients die annually due to this group of diseases.

About MB-106 (CD20-targeted CAR T Cell Therapy)CD20 is a membrane-embedded surface molecule which plays a role in the differentiation of B-cells into plasma cells. The CAR T was developed by Mustangs research partner, Fred Hutchinson Cancer Research Center, in the laboratory of Oliver Press, M.D., Ph.D., and Brian Till, M.D., in the Clinical Research Division and exclusively licensed to Mustang Bio in 2017. MB-106 has been optimized as a third-generation CAR derived from a fully human antibody and is currently in a Phase 1/2 open-label, dose-escalation trial at Fred Hutch in B-cell non-Hodgkin lymphoma patients. Additional information on the trial can be found at http://www.clinicaltrials.gov using the identifier NCT03277729.

About Mustang BioMustang Bio, Inc. (Mustang) is a clinical-stage biopharmaceutical company focused on translating todays medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors and rare genetic diseases. Mustang aims to acquire rights to these technologies by licensing or otherwise acquiring an ownership interest, to fund research and development, and to outlicense or bring the technologies to market. Mustang has partnered with top medical institutions to advance the development of CAR T and CRISPR/Cas9-enhanced CAR T therapies across multiple cancers, as well as a lentiviral gene therapy for XSCID. Mustang is registered under the Securities Exchange Act of 1934, as amended, and files periodic reports with the U.S. Securities and Exchange Commission. Mustang was founded by Fortress Biotech, Inc. (NASDAQ: FBIO). For more information, visit http://www.mustangbio.com.

ForwardLooking StatementsThis press release may contain forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934, each as amended. Such statements include, but are not limited to, any statements relating to our growth strategy and product development programs and any other statements that are not historical facts. Forward-looking statements are based on managements current expectations and are subject to risks and uncertainties that could negatively affect our business, operating results, financial condition and stock value. Factors that could cause actual results to differ materially from those currently anticipated include: the risk that the response in the first patient dosed with MB-106 following the enhancement to the cell process may not be repeated, or may not be achieved within the time table we anticipate, due to the results of research and development activities, the timing of starting and completing clinical trials, and uncertainties relating to preclinical and clinical testing generally; risks relating to our growth strategy; our ability to obtain, perform under and maintain financing and strategic agreements and relationships; risks relating to the results of research and development activities; risks relating to the timing of starting and completing clinical trials; uncertainties relating to preclinical and clinical testing; our dependence on third-party suppliers; our ability to attract, integrate and retain key personnel; the early stage of products under development; our need for substantial additional funds; government regulation; patent and intellectual property matters; competition; as well as other risks described in our SEC filings. We expressly disclaim any obligation or undertaking to release publicly any updates or revisions to any forward-looking statements contained herein to reflect any change in our expectations or any changes in events, conditions or circumstances on which any such statement is based, except as required by law.

Company Contacts:Jaclyn Jaffe and William BegienMustang Bio, Inc.(781) 652-4500ir@mustangbio.com

Investor Relations Contact:Daniel FerryLifeSci Advisors, LLC(617) 430-7576daniel@lifesciadvisors.com

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34 Years with a New Heart and Counting | 90.1 FM WABE – WABE 90.1 FM

Whenever Harry Wuest has a doctors appointment in northern Atlantas hospital cluster dubbed Pill Hill, he makes sure to stop by the office of Dr. Douglas Doug Murphy for a quick chat.

And Murphy, unless hes tied up in the operating room, always takes a few minutes to say hello to his former patient. Remember when . . . ? is how the conversation typically starts, and its always tinged with laughter, often joyful, sometimes bittersweet.

Its a reunion of two men who shaped a piece of Georgias medical history.

Almost 35 years ago, Murphy opened the chest of Wuest and sewed in a new heart, giving him a second shot at life. Wuest was the third heart transplant patient at Emory University Hospital.

Tall, lanky, with short curly hair and a quiet demeanor, Wuest is the longest-surviving heart transplant recipient in Georgia and one of the longest-surviving in the world. The 75-year-old accountant still plays golf twice a week and only recently went from working full-time to part-time.

My heart is doing just fine, he says.

Murphy is now the chief of cardiothoracic surgery at Emory Saint Josephs Hospital and still in the operating room almost every day. He has moved on to become the worlds leading expert in robotically assisted heart surgery.

***

Harry Wuest is originally from Long Island, N.Y. After a stint in the U.S. Air Force, he moved to Florida to work and go to school. He wanted to become a physical education teacher. Then, in 1973, he fell ill. It started with some pain on his left side. He didnt think much of it, but when he got increasingly winded and fatigued, he went to see a doctor.

Several months and numerous specialists later, he received the diagnosis: Cardiomyopathy, a disease of the heart muscle that can make the heart become enlarged, thick and rigid, preventing it from pumping enough blood through the body.

They didnt know how I got it, says Wuest, sitting back in a brown leather armchair in the dark, wood-paneled living room of his Stone Mountain home. Maybe it was a virus. And back then, there wasnt much they could do to treat it, except bed rest.

For the next 12 years, Wuest lived life as best as he could. He got a degree in accounting from the University of Central Florida and worked for a real estate developer. There were good days, but there were more bad days. He was often too weak to do anything, and his heart was getting bigger and bigger.

***

The first successful human-to-human heart transplant was performed in Cape Town, South Africa, in 1967 a medical breakthrough that catapulted the surgeon, Dr. Christiaan Barnard, onto the cover of Life magazine and to overnight celebrity status.

This highly publicized event was followed by a brief surge in the procedure around the world, but overall, heart transplants had a rocky start. Most patients died shortly after the surgery, mainly due to organ rejection. Back then, immunosuppressive drugs, which can counteract rejection, were still in their infancy. Many hospitals stopped doing heart transplants in the 1970s.

That changed with the discovery of a highly effective immunosuppressive agent. Cyclosporine got FDA approval in 1983 and altered the world of organ transplants.

It was shortly thereafter when Emory University Hospital decided to launch a heart transplant program, but none of the senior surgeons wanted to do it. Even with the new drug, it was a risky surgery, and mortality was still high.

Its an all-or-nothing operation, Murphy says, as he sits down in his small office overlooking the greyish hospital compound. Hes wearing light blue scrubs from an early morning surgery. At 70, he still has boyish looks, with a lean build and an air of laid-back confidence. If you have a number of bad outcomes initially, it can be detrimental to your career as a surgeon, he says.

But Murphy didnt really have a choice. He remembers that during a meeting of Emorys cardiac surgeons in 1984, he was paged to check on a patient. When he returned, the physicians congratulated him on being appointed the head of the new heart transplant program. He was the youngest in the group and had been recruited from Harvards Massachusetts General Hospital just three years before.

Yeah, thats how I became Emorys first transplant surgeon, says Murphy.

He flew to California to shadow his colleagues at Stanford University Hospital, where most heart transplants were performed at the time. Back home at Emory, he put together a team and rigorously rehearsed the operation. The first transplant patient arrived in April 1985. The surgery was successful, as was the second operation less than a month later.

Around the same time, Harry Wuest wound up in a hospital in Orlando. He needed a transplant, but none of the medical centers in Florida offered the procedure. One of his doctors recommended Emory, and Wuest agreed. I knew I was dying. I could feel it. He was flown to Atlanta by air ambulance and spent several weeks in Emorys cardiac care unit until the evening of May 23, when Murphy walked into his room and said, Weve got a heart.

***

The heart, as the patient later learned, came from a 19-year-old sophomore at Georgia Tech who had been killed in a car crash.

Organ transplants are a meticulously choreographed endeavor, where timing, coordination and logistics are key. While Murphy and his eight-member team were preparing for the surgery, Wuest was getting ready to say farewell to his family his wife and three teenage sons and to thank the staff in the cardiac ward.

I was afraid, he recalls, especially of the anesthesia. It scared the heck out of me. He pauses during the reminiscence, choking briefly. I didnt know if I was going to wake up again.

The surgery took six hours. Transplants usually happen at night because the procurement team, the surgeons who retrieve different organs from the donor, only start working when regularly scheduled patients are out of the operating room.

Despite the cultural mystique surrounding the heart as the seat of life, Murphy says that during a transplant surgery, its not like the big spirit comes down to the operating room. Its very technical. As the team follows a precise routine, emotions are kept outside the door. We dont have time for that. Emotions come later.

After waking up from the anesthesia, Wuests first coherent memory was of Murphy entering the room and saying to a nurse, Lets turn on the TV, so Harry can watch some sports.

Wuest spent the next nine days in the ICU and three more weeks in the hospital ward. In the beginning, he could barely stand up or walk, because he had been bedridden weeks before the surgery and had lost a lot of muscle. But his strength came back quickly. I could finally breathe again, he says. Before the surgery, he felt like he was sucking in air through a tiny straw. I cannot tell you what an amazing feeling that was to suddenly breathe so easily.

Joane Goodroe was the head nurse at Emorys cardiovascular post-op floor back then. When she first met Wuest before the surgery, she recalls him lying in bed and being very, very sick. When she and the other nurses finally saw him stand up and move around, he was a whole different person.

In the early days of Emorys heart transplant program, physicians, nurses and patients were a particularly close-knit group, remembers Goodroe, whos been a nurse for 42 years and now runs a health care consulting firm. There were a lot of firsts for all of us, and we all learned from each other, she said.

Wuest developed friendships with four other early transplant patients at Emory, and he has outlived them all.

When he left the hospital, equipped with a new heart and a fresh hunger for life, Wuest made some radical changes. He decided not to return to Florida but stay in Atlanta. Thats where he felt he got the best care, and where he had found a personal support network. And he got a divorce. Four months after the operation, he went back to working full-time: first in temporary jobs and eventually for a property management company.

After having been sick for 12 years, I was just so excited to be able to work for eight hours a day, he recalls. That was a big, big deal for me.

At 50, he went back to school to get his CPA license. He also found new love.

Martha was a head nurse in the open-heart unit and later ran the cardiac registry at Saint Josephs Hospital. Thats where Wuest received his follow-up care and where they met in 1987. Wuest says for him it was love at first sight, but it took another five years until she finally agreed to go out with him. Six months later, they were married.

Having worked in the transplant office, I saw the good and the bad, Martha Wuest says. A petite woman with short, perfectly groomed silver hair, she sits up very straight on the couch, her small hands folded in her lap.Not every transplant patient did as well as Harry. And I had a lot of fear in the beginning. Now he may well outlive her, she says with a smile and a wink.

Wuests surgeon, meanwhile, went on to fight his own battles. Two and a half years into the program, Murphy was still the only transplant surgeon at Emory and on call to operate whenever a heart became available. Frustrated and exhausted, he quit his position at Emory and signed up with Saint Josephs (which at the time was not part of the Emory system) and started a heart transplant program there.

At St. Joes, Murphy continued transplanting hearts until 2005. In total, he did more than 200 such surgeries.

Being a heart transplant surgeon is a grueling profession, he says, and very much a younger surgeons subspecialty.

He then shifted his focus and became a pioneer in robotically assisted heart surgery.He has done more than 3,000 operations with the robot, mostly mitral valve repairs and replacements more than any other cardiac surgeon in the world.

***

Since Murphy sewed a new heart into Wuest, 35 years ago, there has been major progress in the field of heart transplants,but it has been uneven.

Medications to suppress the immune system have improved, says Dr. Jeffrey Miller, a transplant surgeon and heart failure specialist at Emory. As a result, we are seeing fewer cases of rejections of the donor heart.

Also, there are new methods of preserving and transporting donor hearts.

Yet patients requiring late-stage heart failure therapy, including transplantation, still exceed the number of donor hearts available. In 2019, 3,551 hearts were transplanted in the United States, according to the national Organ Procurement and Transplantation Network. But 700,000 people suffer from advanced heart failure, says the American Heart Association.

New technologies and continued research are providing hope to many of these patients. There has been significant progress in the development of partial artificial hearts, known as Left Ventricular Assist Devices, or LVADs, says Miller.

These are implantable mechanical pumps that assist the failing heart. Patients are back out in society living normal lives while theyre waiting for their donor hearts, he explains.

LVADs are used not only as bridge devices but as destination therapy as well, maintaining certain patients for the remainder of their lives.

Also, total artificial hearts have come a long way since the first artificial pump was implanted in a patient in 1969.

Long-term research continues into xenotransplantation, which involves transplanting animal cells, tissues and organs into human recipients.

Regenerative stem cell therapy is an experimental concept where stem cell injections stimulate the heart to replace the rigid scar tissue with tissue that resumes contraction, allowing for the damaged heart to heal itself after a heart attack or other cardiac disease.

Certain stem cell therapies have shown toreverse the damage to the heart by 30 to 50 percent, says Dr. Joshua Hare, a heart transplant surgeon and the director of the Interdisciplinary Stem Cell Institute at the University of Miamis Miller School of Medicine.

All of these ideas have potential, says Miller. But they have a lot of work before were ready to use them as alternatives to heart transplantation. I dont think were talking about the next few years.

Besides Emory, other health care systems in Georgia that currently have a heart transplant program are Piedmont Healthcare, Childrens Healthcare of Atlanta and Augusta University Health.

Organ rejection remains a major issue, and long-term survival rates have not improved dramatically over the past 35 years. The 10-year survival is currently around 55 percent of patients, which makes long-term-survivors like Harry Wuest rare in the world of heart transplants.

The United Network of Organ Sharing, or UNOS, which allocates donor hearts in the United States, doesnt have comprehensive data prior to 1987. An informal survey of the 20 highest-volume hospitals for heart transplants in the 1980s found only a scattering of long-term survivors.

***

Being one of the longest-living heart transplant recipients is something that Wuest sees as a responsibility to other transplant patients, but also to the donors family, which hes never met. If you as a transplant recipient reject that heart, thats like a second loss for that family.

Part of this responsibility is living a full and active life. Both he and Martha have three children from their previous marriages, and combined they have 15 grandchildren. Most of their families live in Florida, so they travel back and forth frequently. Wuest still works as a CPA during tax season, and he does advocacy for the Georgia Transplant Foundation. In addition to golf, he enjoys lifting weights and riding his bike.

Hes had some health scares over the years. In 2013, he was diagnosed with stage 1 kidney cancer, which is in remission. Also, he crossed paths with his former surgeon, and not just socially. In 2014, Murphy replaced a damaged tricuspid valve in Wuests new heart. That operation went well, too.

Murphy says there are several reasons why Wuest has survived so long. Obviously, his new heart was a very good match. But a patient can have the best heart and the best care and the best medicines and still die a few months or years after the transplantation, the surgeon says. Attitude plays a key role.

Wuest was psychologically stable and never suffered from depression or anxiety, Murphy says. Hes a numbers guy. He knew the transplant was his only chance, and he was set to pursue it.

Wuest attributes his longevity to a good strong heart from his donor; good genetics; great doctors and nurses; and a life that he loves. Im just happy to be here, he says.

Quoting his former surgeon and friend, he adds: Doug always said, Having a transplant is like running a marathon. And Im in for the long haul.

Katja Ridderbusch is an Atlanta-based journalist who reports for news organizations in the U.S. and her native Germany. Her stories have appeared in Kaiser Health News, U.S. News & World Report and several NPR affiliates.

This is a slightly modified version of the article 34 Years with a New Heart, published by Georgia Health News on February 18, 2020.

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34 Years with a New Heart and Counting | 90.1 FM WABE - WABE 90.1 FM

FREE Brunch with a Doc! Regenerative Options for Orthopedic Conditions – Englewood Herald

Join us for brunch and to hear how the unique stem cell and blood platelet procedures offered at the Centeno-Schultz Clinic are helping patients with joint pain avoid surgery and get back to the activities they love!

Stem Cell and Blood Platelet Procedures offer a viable alternative for individuals who are suffering from joint pain, or who may be considering elective surgery or joint replacement due to injury or arthritis. Patients avoid the lengthy periods of downtime, and painful rehabilitation that typically follow invasive surgeries.

Commonly Treated Conditions:

- Knee Pain, Osteoarthritis

- Knee ACL & Meniscal Tears

- Hip Arthritis, Pain & Labral Tears

- Shoulder Pain, Rotator Cuff Tears & Tendinitis

- Low Back, Neck & Nerve Pain, Sciatica

- Tennis/Golfers Elbow

- Achilles Tendinitis

- Foot & Ankle Pain, Plantar Fasciitis

- Sacroiliac Joint Instability

- AND MUCH MORE!

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FREE Brunch with a Doc! Regenerative Options for Orthopedic Conditions - Englewood Herald

Stem Cell Therapy seminar – The Wilton Bulletin

Bridges by EPOCH at Trumbull, a memory care assisted living community, located at 2415 Reservoir Ave., will host the following complimentary event.

Dine and Discover Stem Cell Therapy for Stroke and Traumatic Brain Injury (TBI) Patients: Thursday, Feb. 20, at 5 p.m.; With Guest Speaker Dr. Peter McAllister, co-founder and Chief Medical Officer, New England Institute for Clinical Research.

Stroke and traumatic brain injury strike millions of people each year. Dr. McAllister will discuss the various treatment options for the long-term effects of these conditions, including weakness, spasticity, language and cognitive difficulties and pain. He'll also discuss new research on stem cell therapy that could help restore function for stroke and TBI patients.

Caregivers may bring their loved ones with memory loss to enjoy a meal and an activity with Bridges by EPOCH residents during this seminar.

Dinner will be served.

Call 203-935-8530 to RSVP.

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Stem Cell Therapy seminar - The Wilton Bulletin