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Lineage Cell Therapeutics Reports Third Quarter 2019 Financial Results and Provides Business Update – Business Wire

CARLSBAD, Calif.--(BUSINESS WIRE)--Lineage Cell Therapeutics, Inc. (NYSE American and TASE: LCTX), a clinical-stage biotechnology company developing novel cellular therapies for unmet medical needs, reported financial and operating results for the third quarter ended September 30, 2019. Lineage management will host a conference call and webcast today at 4:30 p.m. Eastern Time/1:30 p.m. Pacific Time to discuss its third quarter 2019 financial results and to provide a business update.

We are excited about our cell therapy programs and how they may benefit patients with serious medical conditions such as dry AMD, spinal cord injury, and cancer, stated Brian M. Culley, CEO of Lineage. We believe that Lineage has one of the largest and most comprehensive patent estates in cell therapy and that our clinical-stage programs are making important advances. We also recently implemented additional cost-cutting measures that will reduce our planned 2020 net operational spend to $16 million, $8 million to $12 million less than our previous estimate of $24 million to $28 million. Under this plan, our primary goal will be to complete enrollment in our Phase I/IIa clinical study of OpRegen early next year and collect the follow-up data to guide our late-stage study design and partnership discussions. We also have completed the transfer of OPC1 to our manufacturing facility and will continue our efforts to introduce manufacturing enhancements to OPC1 in preparation for the initiation of a randomized clinical study in 2021. We believe reducing our cash burn and focusing on OpRegen, our nearest-term high value asset, as well as on finding a strong marketing partner for Renevia, is the best way to create near-term shareholder value. In August 2020, we also are entitled to receive our final payment of $24.6 million in principal and interest for the 2018 sale of AgeX Therapeutics shares to Juvenescence, an amount which exceeds our anticipated cash needs from now through the end of next year.

Additionally, we are looking forward to hosting two therapeutic area experts in ophthalmology and spinal cord injury at Solebury Trouts KOL Event for analysts and investors in New York City on November 15, 2019, added Mr. Culley. Our executive team will be joined by renowned experts Allen C. Ho, M.D. FACS, Wills Eye Hospital Attending Surgeon and Director of Retina Research, and John Steeves, B.Sc., Ph.D., Emeritus Principal Investigator at ICORD and Professor in the Department of Neuroscience at the University of British Columbia. We will be providing an update on our OpRegen and OPC1 clinical programs, as well as an update on the SCiStar Clinical Study for the treatment of spinal cord injury.

Recent Significant Highlights

Near Term Milestones for 2019 and 2020

Balance Sheet Highlights

Cash, cash equivalents and marketable securities totaled $35.7 million as of September 30, 2019. Marketable securities include our remaining ownership stakes in OncoCyte, AgeX and Hadasit Bio-Holdings Ltd (Hadasit), which are now all under 20% of their respective total outstanding shares. Lineage sold 6,250,000 shares of OncoCytes common stock in the third quarter of 2019 for net proceeds of $10.7 million. Lineage also sold 651,839 shares of AgeX common stock in the third quarter of 2019 for net proceeds of $1.6 million and 647,397 shares of Hadasit common stock in July 2019 for net proceeds of $1.2 million.

Lineages promissory note due from Juvenescence Limited had an outstanding balance (principal plus accrued interest) of $23.2 million as of September 30, 2019. Unless earlier converted into Juvenescence ordinary shares, the promissory note is payable in cash, plus accrued interest at 7% per year, at maturity in August 2020. If Juvenescence completes an initial public offering (IPO) resulting in gross proceeds of not less than $50.0 million, the promissory note automatically converts into the Juvenescence securities issued in the IPO based on the per-share price to the public in the IPO, subject to an upward adjustment in the number of shares that would be issued to Lineage upon such conversion if the 20-day volume-weighted average trading price of one share of common stock of AgeX before the IPO is priced above $3.00. If the promissory note is converted, the Juvenescence ordinary shares will be a marketable security that Lineage may use to supplement its liquidity, as needed and as market conditions allow.

In summary, as of September 30, 2019, the value of the Companys cash, marketable securities, and the balance of a promissory note due to it in August 2020 were in excess of $58.9 million.

Lineage expects to spend approximately $6 million in the fourth quarter of 2019. The Company has implemented significant cost savings initiatives and now anticipates that net operational spend for 2020 will be $16 million. This planned spending level represents a significant reduction from 2019 forecasted spending levels of $34 million and 2018 spending levels of $43 million for Lineage and Asterias Biotherapeutics, Inc. (Asterias) combined. Lineage acquired Asterias on March 8, 2019.

Third Quarter Operating Results

Note regarding AgeX: On August 30, 2018, Lineage deconsolidated AgeX from its consolidated financial statements due to the sale by Lineage of 14,400,000 shares of AgeX common stock to Juvenescence and the related decrease of Lineages ownership position in AgeX from 80.4% to 40.2%. Accordingly, Lineage ceased recognizing revenue and expenses related to AgeX and its programs on such date.

Revenues: Lineages revenue is generated primarily from research grants, licensing fees and royalties. Total revenues for the three months ended September 30, 2019 were $0.6 million, a decrease of $0.4 million as compared to the same period in 2018. The decrease was primarily related to a $0.4 million decrease in grant revenues, which is primarily based on the timing of grant-related activities.

Operating Expenses: Operating expenses are comprised of research and development (R&D) expenses and general and administrative (G&A) expenses. Total operating expenses for the three months ended September 30, 2019 were $8.9 million, a decrease of $2.4 million as compared to the same period in 2018.

R&D Expenses: R&D expenses for the three months ended September 30, 2019 were $4.3 million, a decrease of $0.6 million as compared to the same period in 2018. The decrease was primarily related to a $0.8 million decrease from the AgeX deconsolidation and the absence of AgeX R&D expenses incurred after August 30, 2018, offset by a net increase of $0.2 million in Lineage programs primarily related to: (1) an increase of $1.4 million in OPC1 and VAC2 expenses (these programs were acquired in the Asterias merger) offset by (2) decreases of $1.2 million in Renevia, OpRegen and other research-related expenses.

G&A Expenses: G&A expenses for the three months ended September 30, 2019 were $4.6 million, a decrease of $1.8 million as compared to the same period in 2018. The decrease was primarily attributable to a $0.8 million decrease in AgeX related general and administrative expenses, a $0.5 million reduction in legal and patent expenses, a $0.4 million decrease in salaries, benefits and severance costs primarily related to terminated personnel and a $0.3 million reduction in consulting expenses, offset by a $0.2 million increase in rent expense, which is primarily related to the implementation of ASC 842 Leases in 2019.

Loss from Operations: Loss from operations for the three months ended September 30, 2019 was $8.4 million, a decrease of $2.0 million as compared to the same period in 2018.

Other Income/(Expenses), Net: Other income/(expenses), net for the three months ended September 30, 2019 reflected other expense, net of ($9.1) million, compared to other income, net of $76.9 million for the same period in 2018. The variance was primarily related to the gain on the deconsolidation of AgeX in 2018 and the changes in the value of investments in marketable equity securities for the applicable periods.

Conference Call and Webcast

Lineage will host a conference call and webcast today, at 1:30pm PT/4:30pm ET to discuss its third quarter 2019 financial results and to provide a business update. Interested parties may access the conference call by dialing (866) 888-8633 from the U.S. and Canada and (636) 812-6629 from elsewhere outside the U.S. and Canada and should request the Lineage Cell Therapeutics Call. A live webcast of the conference call will be available online in the Investors section of Lineages website. A replay of the webcast will be available on Lineages website for 30 days and a telephone replay will be available through November 19, 2019, by dialing (855) 859-2056 from the U.S. and Canada and (404) 537-3406 from elsewhere outside the U.S. and Canada and entering conference ID number 1473397.

About Lineage Cell Therapeutics, Inc.

Lineage Cell Therapeutics is a clinical-stage biotechnology company developing novel cell therapies for unmet medical needs. Lineages programs are based on its proprietary cell-based therapy platform and associated development and manufacturing capabilities. With this platform Lineage develops and manufactures specialized, terminally-differentiated human cells from its pluripotent and progenitor cell starting materials. These differentiated cells are developed either to replace or support cells that are dysfunctional or absent due to degenerative disease or traumatic injury or administered as a means of helping the body mount an effective immune response to cancer. Lineages clinical assets include (i) OpRegen, a retinal pigment epithelium transplant therapy in Phase I/IIa development for the treatment of dry age-related macular degeneration, a leading cause of blindness in the developed world; (ii) OPC1, an oligodendrocyte progenitor cell therapy in Phase I/IIa development for the treatment of acute spinal cord injuries; and (iii) VAC2, an allogeneic cancer immunotherapy of antigen-presenting dendritic cells currently in Phase I development for the treatment of non-small cell lung cancer. Lineage is also evaluating potential partnership opportunities for Renevia, a facial aesthetics product that was recently granted a Conformit Europenne (CE) Mark. For more information, please visit http://www.lineagecell.com or follow the Company on Twitter @LineageCell.

Forward-Looking Statements

Lineage cautions you that all statements, other than statements of historical facts, contained in this press release, are forward-looking statements. Forward-looking statements, in some cases, can be identified by terms such as believe, may, will, estimate, continue, anticipate, design, intend, expect, could, plan, potential, predict, seek, should, would, contemplate, project, target, tend to, or the negative version of these words and similar expressions. Such statements include, but are not limited to, statements relating to Lineages cost-savings efforts, manufacturing plans, enrollment activities, data presentations, clinical study advancement, drug evaluation, and anticipated net operational spend for the fourth quarter of 2019 and full year 2020. Forward-looking statements involve known and unknown risks, uncertainties and other factors that may cause Lineages actual results, performance or achievements to be materially different from future results, performance or achievements expressed or implied by the forward-looking statements in this press release, including risks and uncertainties inherent in Lineages business and other risks in Lineages filings with the Securities and Exchange Commission (the SEC). Lineages forward-looking statements are based upon its current expectations and involve assumptions that may never materialize or may prove to be incorrect. All forward-looking statements are expressly qualified in their entirety by these cautionary statements. Further information regarding these and other risks is included under the heading Risk Factors in Lineages periodic reports with the SEC, including Lineages Annual Report on Form 10-K filed with the SEC on March 14, 2019 and its other reports, which are available from the SECs website. You are cautioned not to place undue reliance on forward-looking statements, which speak only as of the date on which they were made. Lineage undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law.

Tables to follow

LINEAGE CELL THERAPEUTICS, INC. AND SUBSIDIARIES

CONDENSED CONSOLIDATED BALANCE SHEETS

(IN THOUSANDS)

September 30,2019 (Unaudited)(Notes 1 and 3)

December 31,2018(Notes 1 and 6)

ASSETS

CURRENT ASSETS

Cash and cash equivalents

$

14,366

$

23,587

Marketable equity securities

21,318

7,154

Promissory note from Juvenescence (Note 5)

23,238

-

Trade accounts and grants receivable, net

157

767

Receivables from affiliates, net (Note 10)

164

2,112

Prepaid expenses and other current assets

2,342

2,738

Total current assets

61,585

36,358

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Lineage Cell Therapeutics Reports Third Quarter 2019 Financial Results and Provides Business Update - Business Wire

Marker Therapeutics Reports Third Quarter 2019 Operating and Financial Results – P&T Community

HOUSTON, Nov. 12, 2019 /PRNewswire/ -- Marker Therapeutics, Inc.(Nasdaq:MRKR), a clinical-stage immuno-oncology company specializing in the development of next-generation T cell-based immunotherapies for the treatment of hematological malignancies and solid tumor indications, today provided a corporate update and reported financial results for the third quarter ended September 30, 2019.

"We continue to make progress in advancing our next-generation T-cell based immunotherapies for the treatment of hematological malignancies and solid tumors," said Peter L. Hoang, President and CEO of Marker Therapeutics. "Our partner-sponsored MultiTAA T-cell therapy trials at the Baylor College of Medicine continue to show promising results. In addition, we continue to expand our team and build out our infrastructure to support future Marker-sponsored clinical trials. We expect the next 12 to 18 months to be an exciting and productive time for our Company."

Continued Mr. Hoang: "We recently filed an Investigational New Drug (IND) application with the U.S. Food and Drug Administration (FDA) for our MultiTAA T-cell therapy as part of a planned Marker Phase 2 study in post-allogeneic hematopoietic stem cell transplant patients with acute myeloid leukemia in both the adjuvant and active disease setting. The FDA reviewed our submission and requested additional information regarding certain quality and technical specifications for two reagents supplied by third party vendors that are used in our manufacturing process. Because the FDA requires these data in order to clear the IND, the Marker AML trial has been placed on clinical hold until our complete response to the technical questions is satisfactory to the FDA. While these reagents are not present in the final product, we worked with respective manufacturers of these reagents to satisfy the FDA's questions and subsequently submitted a complete response to the FDA in late October. We currently project to initiate our Phase 2 trial in 2020 and look forward to providing an update on our clinical path forward upon receiving the FDA's feedback."

PROGRAM UPDATES

Multi-Antigen Targeted (MultiTAA) T-Cell Therapies

Marker Submits Response to FDA Clinical Hold on AML Trial The Company worked with regulatory and quality groups at the respective manufacturers to address the FDA's request and submitted a complete response to the issues raised by the FDA on October 28, 2019. The FDA will respond within 30 daysafter receiving Marker's complete response, indicating whether the hold is lifted and, if not, specifying the reasons the clinical trial remains on hold.Marker expects to initiate its Phase 2 clinical trial of MultiTAA therapy for the treatment of post-transplant AML in 2020.

T Cell-Based Vaccines

Phase 2 Triple Negative Breast Cancer Trial ProgressingMarker continues to advance its T cell-based vaccine program in triple negative breast cancer. To date, results have shown:

Phase 2 Platinum-Sensitive Advanced Ovarian Cancer Trial Update Marker will be discontinuing the development of TPIV200 in patients with platinum-sensitive advanced ovarian cancer based on an unblinded review of interim results from its Phase 2 study conducted by an independent Data and Safety Monitoring Board (DSMB). Although the DSMB did not express any safety concerns with respect to TPIV200, Marker has elected to suspend the trial because it did not meet the threshold for probability of success based upon the Company's pre-specified criteria. Pending full review of the data, Marker anticipates closing the trial in the first quarter of 2020.

CORPORATE UPDATES

THIRD QUARTER 2019 FINANCIAL RESULTS

Net loss for the quarter ended September 30, 2019 was $5.5 million, compared to a net loss of $4.4 million for the quarter ended September 30, 2018.

Research and development expenses during the three months ended September 30, 2019 were $3.1 million, compared to $1.9 million during the three months ended September 30, 2018. The increase of $1.2 million was primarily attributable to increases in personnel-related expenses, relating to the build-up of Marker's internal infrastructure.

General and administrative expenses were $2.5 million during the three months ended September 30, 2019 as compared to $2.6 million during the three months ended September 30, 2018. The decrease was primarily attributable to $0.6 million of merger-related expenses incurred during the three months ended September 30, 2018, offset by increased expenses in headcount-related and legal and other professional expenses.

CASH POSITION AND GUIDANCE

At September 30, 2019, Marker had cash and cash equivalents of $48.5 million. The Company believes that its existing cash and cash equivalents will fund its current operations through at least the fourth quarter of 2020.

Conference Call and Webcast

The Company will host a webcast and conference call to discuss its third quarter 2019 financial results and provide an update on recent corporate activities today at 5:00 p.m. EST.

The webcast will be accessible in the Investors section of the Company's website at http://www.markertherapeutics.com. Individuals can participate in the conference call by dialing 877-407-8913 (domestic) or 201-689-8201 (international) and referring to the "Marker Therapeutics Third Quarter 2019 Earnings Call."

The archived webcast will be available for replay on the Marker website following the event.

About Marker Therapeutics, Inc.Marker Therapeutics, Inc. is a clinical-stage immuno-oncology company specializing in the development of next-generation T cell-based immunotherapies for the treatment of hematological malignancies and solid tumor indications. Marker's cell therapy technology is based on the selective expansion of non-engineered, tumor-specific T cells that recognize tumor associated antigens (i.e. tumor targets) and kill tumor cells expressing those targets. This population of T cells is designed to attack multiple tumor targets following infusion into patients and to activate the patient's immune system to produce broad spectrum anti-tumor activity. Because Marker does not genetically engineer its T cell therapies, we believe that our product candidates will be easier and less expensive to manufacture, with reduced toxicities, compared to current engineered CAR-T and TCR-based approaches, and may provide patients with meaningful clinical benefit. As a result, Marker believes its portfolio of T cell therapies has a compelling product profile, as compared to current gene-modified CAR-T and TCR-based therapies.

Marker is also advancing a number of innovative peptide and gene-based immuno-therapeutics for the treatment of metastatic solid tumors, including the Folate Receptor Alpha program (TPIV200) for breast cancer and the HER2/neu program (TPIV100/110) for breast cancer, currently in Phase 2 clinical trials.

To receive future press releases via email, please visit:https://www.markertherapeutics.com/email-alerts/

Forward-Looking Statement DisclaimerThis release contains forward-looking statements for purposes of the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Statements in this news release concerning the Company's expectations, plans, business outlook or future performance, and any other statements concerning assumptions made or expectations as to any future events, conditions, performance or other matters, are "forward-looking statements." Forward-looking statements include statements regarding our intentions, beliefs, projections, outlook, analyses or current expectations concerning, among other things: our research, development and regulatory activities and expectations relating to our non-engineered multi-tumor antigen specific T cell therapies; our TPIV200 and TPIV100/110 programs; the effectiveness of these programs or the possible range of application and potential curative effects and safety in the treatment of diseases; and, the timing and success of our clinical trials, as well as clinical trials conducted by our collaborators. Forward-looking statements are by their nature subject to risks, uncertainties and other factors which could cause actual results to differ materially from those stated in such statements. Such risks, uncertainties and factors include, but are not limited to the risks set forth in the Company's most recent Form 10-K, 10-Q and other SEC filings which are available through EDGAR at http://www.sec.gov. The Company assumes no obligation to update our forward-looking statements whether as a result of new information, future events or otherwise, after the date of this press release.

Marker Therapeutics, Inc.

Condensed Consolidated Balance Sheets

(Unaudited)

September 30,

December 31,

2019

2018

ASSETS

Current assets:

Cash and cash equivalents

$ 48,477,670

$ 61,746,748

Prepaid expenses and deposits

1,906,062

141,717

Interest receivable

78,145

108,177

Total current assets

50,461,877

61,996,642

Non-current assets:

Property, plant and equipment, net

438,881

147,668

Right-of-use assets, net

501,714

-

Total non-current assets

940,595

147,668

Total assets

$ 51,402,472

$ 62,144,310

LIABILITIES AND STOCKHOLDERS' EQUITY

Current liabilities:

Accounts payable and accrued liabilities

$ 2,858,808

$ 2,754,572

Lease liability

199,266

-

Warrant liability

129,000

49,000

Total current liabilities

3,187,074

2,803,572

Non-current liabilities:

Lease liability, net of current portion

333,480

-

Total non-current liabilities

333,480

-

Total liabilities

3,520,554

2,803,572

Commitments and contingencies

-

-

Stockholders' equity:

Preferred stock - $0.001 par value, 5 million shares authorized and 0 shares issued and outstanding at September 30, 2019 and December 31, 2018, respectively

-

-

Common stock, $0.001 par value, 150 million shares authorized, 45.7 million and 45.4 million shares issued and outstanding as of September 30, 2019 and December 31, 2018, respectively

45,723

45,440

Additional paid-in capital

370,290,447

365,400,748

Accumulated deficit

(322,454,252)

(306,105,450)

Total stockholders' equity

47,881,918

Originally posted here:
Marker Therapeutics Reports Third Quarter 2019 Operating and Financial Results - P&T Community

Neon Therapeutics Reports Third Quarter 2019 Financial Results and Recent Highlights – Associated Press

CAMBRIDGE, Mass., Nov. 12, 2019 (GLOBE NEWSWIRE) -- Neon Therapeutics, Inc. (Nasdaq: NTGN), a clinical-stage immuno-oncology company developing neoantigen-based therapeutics, today reported financial results for the third quarter ended September 30, 2019 and provided a business update.

We continue to make important progress in advancing our leadership in the field of neoantigen-targeted therapies and are pleased to have recently presented updates across our personalized cancer vaccine and adoptive T cell therapy programs at the SITC Annual Meeting. In the remaining months of 2019, we look to continue building on our leadership position by completing our process development work that will enable our planned Clinical Trial Application in Europe for Phase 1 development of our neoantigen-targeted T cell therapy candidate, NEO-PTC-01, said Hugh ODowd, Neons Chief Executive Officer.

Recent Highlights

-- RECON Bioinformatics Platform: Neon scientists published in the scientific journal Immunity a breakthrough process for predicting which neoantigens will be presented by MHC class II molecules in the tumor microenvironment. o Predicting the relevant cancer-specific antigens is a crucial precursor to developing immunotherapies that effectively train T cells to traffic to the tumor and destroy malignant cells. In the paper, titled Defining HLA-II ligand processing and binding rules with mass spectrometry enhances cancer epitope prediction, Neons proprietary mono-allelic profiling technology called MAPTAC facilitated the development of convolutional neural network-based predictors. o These algorithms achieved up to a 61-fold improvement in predicting MHC class II peptides compared to publicly available tools. o This MHC class II technology will be integrated into Neons RECON bioinformatics platform and is expected to improve the efficacy of immunotherapies developed by Neon by predicting recruitment of CD4+ T cells, which are believed to be important in controlling tumor growth. -- NEO-PV-01: Neon presented updated results at the SITC Annual Meeting from the ongoing, multicenter Phase 1b NT-001 clinical trial evaluating NEO-PV-01, Neons personal neoantigen vaccine candidate, in combination with OPDIVO (nivolumab) in patients with advanced or metastatic melanoma, smoking-associated non-small cell lung cancer (NSCLC) and bladder cancer. o Across all three distinct tumor types, results demonstrated prolonged and consistent improvements in progression-free survival (PFS) and overall survival (OS) that compare favorably to that observed with checkpoint inhibitor monotherapy, based on historical benchmark data. o Further, neoantigen-specific immune responses and epitope spread to RECON-predicted targets were associated with longer PFS, and major pathological responses post-administration of NEO-PV-01 in melanoma patients were also associated with longer PFS. o The safety data for NT-001 were consistent with the safety profile for OPDIVO monotherapy. These updated results come from 82 patients who received at least one dose of OPDIVO in the Phase 1b NT-001 trial. -- NEO-PTC-01:Neon presented at the SITC Annual Meeting an update on preclinical and process development work for NEO-PTC-01, its personal neoantigen-targeted T cell therapy candidate consisting of multiple T cell populations targeting the most therapeutically relevant neoantigens from each patients tumor. o NEO-PTC-01 leverages Neons RECON bioinformatics platform to individually select a set of neoantigen targets for each patient, and NEO-STIM, its proprietary process to directly prime, activate and expand neoantigen-targeting T cells ex vivo.Neon believes that this approach will allow NEO-PTC-01, a non-engineered product that leverages peripheral blood mononuclear cells (PBMCs) as starting material, to specifically target each patients individual tumor with T cells that can drive a robust and persistent anti-tumor response. o In the SITC update, Neon demonstrated that it can reproducibly generate a potent T cell product from PBMCs of melanoma patients, as well as at therapeutic scale using a healthy donor sample. This process development work showed that NEO-PTC-01 induced multiple CD8+ and CD4+ T cell responses from both the memory and the nave T cell compartments. o Neon is focusing the initial clinical development of NEO-PTC-01 in patients with solid tumors that are refractory to checkpoint inhibitors. Neon expects to file a clinical trial application, or CTA, in Europe by the end of 2019 to evaluate NEO-PTC-01 in the solid tumor setting.

Expected Near-Term Milestones

-- NEO-PTC-01: Planned European CTA filing to evaluate NEO-PTC-01 in a refractory solid tumor setting (2H 2019). -- NEO-PTC-01: Planned Phase 1 initiation in a refractory solid tumor setting (1H 2020). -- NEO-PV-01: Planned clinical results and correlative immune data, including 12-month follow-up, from NT-002 Phase 1b trial in first-line metastatic NSCLC (Q3 2020).

Third Quarter 2019 Financial Results and Financial Guidance:

-- R&D Expenses:Research and development expenses were $14.1 million for the third quarter of 2019, compared to $14.4 million for the same period last year. The decrease was primarily due to a reduction in clinical development and manufacturing costs, partially offset by an increase in personnel-related costs and costs related to the preparation for the planned CTA filing in Europe for Phase 1 development of NEO-PTC-01. -- G&A Expenses:General and administrative expenses were $5.1 million for the third quarter of 2019, compared to $4.6 million for the same period last year. The increase was primarily due to personnel-related costs and costs associated with being a public company. -- Net Loss: Net loss was $19.0 million for the third quarter of 2019, compared to $18.4 million for the same period last year. -- Cash Position:As of September 30, 2019, cash, cash equivalents and marketable securities were $44.3 million, as compared to cash, cash equivalents and marketable securities of $103.3 million as of December 31, 2018. -- Financial Guidance: Based on its current operating plan, Neon expects that its existing cash and cash equivalents will enable the Company to fund its operating expenses and capital expenditure requirements into June 2020.

OPDIVO is a registered trademark of Bristol-Myers Squibb Company.

About Neon Therapeutics

Neon Therapeutics is a clinical-stage immuno-oncology company and a leader in the field of neoantigen-targeted therapies, dedicated to transforming the treatment of cancer by directing the immune system towards neoantigens. Neon is using its neoantigen platform to develop both vaccine and T cell therapies, including NEO-PV-01, a clinical-stage neoantigen vaccine for the treatment of metastatic melanoma, non-small cell lung cancer, and bladder cancer; NEO-PTC-01, a neoantigen T cell therapy for the treatment of solid tumors; and NEO-SV-01, a neoantigen vaccine for the treatment of a subset of hormone receptor-positive (HR+) breast cancer.

For more information, please visit neontherapeutics.com.

Forward-Looking Statements

This press release contains forward-looking statements of Neon Therapeutics, Inc. within the meaning of the Private Securities Litigation Reform Act of 1995. These forward-looking statements may include, but may not be limited to, express or implied statements regarding our ability to obtain and maintain regulatory approval of our product candidates; the potential timing and advancement of our clinical trials; the potential timing and manner of data readouts from our ongoing and planned clinical trials; the design and potential efficacy of our therapeutic approaches; financial plans and projections; and our ability to replicate results achieved in our preclinical studies or clinical trials in any future studies or trials. Any forward-looking statements in this press release are based on managements current expectations and beliefs 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: uncertainties related to the initiation, timing and conduct of studies and other development requirements for our product candidates; the risk that any one or more of our product candidates will not be successfully developed and commercialized; the risk that the results of preclinical studies and clinical trials may not be predictive of future results in connection with future studies or trials; the risk that Neons collaborations will not continue or will not be successful; risks related to our ability to protect and maintain our intellectual property position; risks related to our capital requirements and use of capital; and risks related to the ability of our licensors to protect and maintain their intellectual property position. For a discussion of these and other risks and uncertainties, and other important factors, any of which could cause Neons actual results to differ from those contained in the forward-looking statements, see the section entitled Risk Factors in Neons most recent Quarterly Report on Form 10-Q, as filed with the Securities and Exchange Commission, as well as discussions of potential risks, uncertainties, and other important factors in Neons other filings with the Securities and Exchange Commission. All information in this press release is as of the date of the release, and Neon undertakes no duty to update this information unless required by law.

Selected Consolidated Balance Sheet Data (Unaudited)(amounts in thousands)

September 30, 2019 December 31, 2018 ------------------ ----------------- Cash, cash equivalents and marketable securities $ 44,278 $ 103,311 Working capital (1) $ 36,215 $ 92,737 Total assets $ 62,317 $ 114,088 Total stockholders equity $ 45,334 $ 101,249

______________________________________________

(1) Working capital is defined as current assets less current liabilities.

Consolidated Statements of Operations (Unaudited)(amounts in thousands, except per share data)

Three Months Ended Nine Months Ended September 30, September 30, 2019 2018 2019 2018 ----------- ----------- ----------- ----------- Operating expenses: Research and development $ 14,120 $ 14,441 $ 47,027 $ 42,403 General and administrative 5,134 4,612 16,122 12,524 --------- - --------- - --------- - --------- - Total operating expenses 19,254 19,053 63,149 54,927 --------- - --------- - --------- - --------- - Loss from operations (19,254 ) (19,053 ) (63,149 ) (54,927 ) Other income (expense), net Interest income 278 672 1,252 1,136 Other expense (4 ) (10 ) (39 ) (20 ) --------- - --------- - --------- - --------- - Total other income, net 274 662 1,213 1,116 --------- - --------- - --------- - --------- - Net loss (18,980 ) (18,391 ) (61,936 ) (53,811 ) Accretion of redeemable convertible preferred stock to (6,371 ) redemption value --------- - --------- - --------- - --------- - Net loss attributable to common stockholders $ (18,980 ) $ (18,391 ) $ (61,936 ) $ (60,182 ) - ------- - - ------- - - ------- - - ------- - Net loss per share attributable to common stockholders, $ (0.68 ) $ (0.67 ) $ (2.23 ) $ (5.55 ) basic and diluted - ------- - - ------- - - ------- - - ------- - Weighted average common shares outstanding, basic and 27,935 27,358 27,792 10,834 diluted --------- - --------- - --------- - --------- -

Investor Contact: Paul Cox, Corporate Affairs and Strategy pcox@neontherapeutics.com 617-337-4762

Media Contact: Stephanie Simon, Ten Bridge Communications stephanie@tenbridgecommunications.com 617-581-9333

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Neon Therapeutics Reports Third Quarter 2019 Financial Results and Recent Highlights - Associated Press

Kiadis Pharma changes strategy to focus solely on development of Natural Killer (NK) Cell therapeutics and terminates development of ATIR101 -…

Amsterdam, The Netherlands, 12 November 2019 Kiadis Pharma N.V. (Kiadis Pharma or the Company) (Euronext Amsterdam and Brussels: KDS), a clinical-stage biopharmaceutical company, today announced that it has completed a strategic portfolio review and has decided to change its strategy and focus all resources and investments on the companys NK-cell therapy platform and product candidates. The company will discontinue development of ATIR101 and stop its ongoing phase 3 trial.

Kiadis NK-cell program consists of off-the-shelf and haplo donor cell therapy products for the treatment of liquid and solid tumors. Kiadis proprietary off-the-shelf NK-cell platform is based on NK-cells from unique universal donors, expanded and activated ex vivo using our PM21 particle technology. The Kiadis off-the-shelf platform has the potential to make NK-cell therapy products rapidly and economically available for a broad patient population across a potentially wide range of indications.

The companys pipeline includes:

Arthur Lahr, CEO of Kiadis Pharma commented, We believe that our proprietary NK-cell therapy platform has broad potential as stand-alone or adjunctive treatments for patients with both liquid and solid tumors. Our off-the-shelf NK-cell platform is based on NK-cells from unique universal donors, expanded and activated with our PM21 particle technology, to make our NK-cell therapy products rapidly and economically available for patients across a potentially broad range of indications. The proof-of-concept trials for our NK pipeline programs, in which 38 patients have been treated, is very promising and was the basis for our acquisition of Cytosen Therapeutics, Inc. earlier this year. To confirm findings from these trials, we will start two Phase 1/2 clinical trials in 2020. We believe that investing in our NK platform and rapidly advancing development of our off-the-shelf and haplo donor derived NK-cell therapies in solid and liquid tumors will bring value to patients and our investors.

Lahr continued, As part of our strategic portfolio review, we reviewed progress of our phase 3 study, which was designed to show superiority of ATIR101 over the PTCy protocol. We identified that in the phase 3 a higher percentage of patients than expected dropped out of the study before receiving ATIR101. We subsequently collected additional recent external data, which show that outcomes with PTCy have better survival and lower severe GVHD than literature showed when we designed and started the phase 3 study. Based on these data, we no longer believe that the phase 3 ATIR study as currently designed with 250 patients can demonstrate superiority over PTCy and at a minimum would require a much larger trial. In the best interest of patients, we have therefore taken the decision to discontinue the ATIR101 study with immediate effect and are proceeding with close down activities.

RestructuringKiadis is implementing a restructuring program to refocus the organization on its NK-cell therapy platform, which will result in a reduction of approximately half of its workforce, a reduction in external clinical trial costs associated with the phase 3 study, and a reduced company cash burn. The company ended the third quarter of 2019 with approximately 47 million of cash.

About Kiadis K-NK-Cell Therapies Kiadis NK-cell programs consist of off-the-shelf and haplo donor cell therapy products for the treatment of liquid and solid tumors as adjunctive and stand-alone therapies.

Our NK-cell PM21 particle technology enables improved ex vivo expansion and activation of anti-cancer cytotoxic NK-cells supporting multiple high-dose infusions. Kiadis proprietary off-the-shelf NK-cell platform is based on NK-cells from unique universal donors. The Kiadis off-the-shelf K-NK platform can make NK-cell therapy product rapidly and economically available for a broad patient population across a potentially wide range of indications.

Administered as an adjunctive immunotherapeutic on top of HSCT, K-NK002 provides functional, mature and potent NK-cells from a haploidentical family member. In addition, Kiadis is developing K-NK003 for the treatment of relapse/refractory acute myeloid leukemia and has pre-clinical programs evaluating NK-cell therapy for the treatment of solid tumors.

Kiadis Contacts:

About KiadisFounded in 1997, Kiadis Pharma, is a fully integrated biopharmaceutical company committed to developing innovative cell-based therapies for patients with life-threatening diseases. With headquarters in Amsterdam, the Netherlands, and offices and activities in the US and across Europe, Kiadis Pharma is leveraging the natural strengths of humanity and our collective immune system to source the best cells for life.

Kiadis Pharma is listed on the regulated market of Euronext Amsterdam and Euronext Brussels since July 2, 2015, under the symbol KDS. Learn more at http://www.kiadis.com.

Forward Looking Statements Certain statements, beliefs and opinions in this press release are forward-looking, which reflect Kiadis Pharmas or, as appropriate, Kiadis Pharmas directors current expectations and projections about future events. By their nature, forward-looking statements involve a number of risks, uncertainties and assumptions that could cause actual results or events to differ materially from those expressed or implied by the forward-looking statements. These risks, uncertainties and assumptions could adversely affect the outcome and financial impact of the plans and events described herein. A multitude of factors including, but not limited to, changes in demand, regulation, competition and technology, can cause actual events, performance or results to differ significantly from any anticipated development. Forward-looking statements contained in this press release regarding past trends or activities should not be taken as a representation that such trends or activities will continue in the future. As a result, Kiadis Pharma expressly disclaims any obligation or undertaking to release any update or revisions to any forward-looking statements in this press release as a result of any change in expectations or any change in events, conditions, assumptions or circumstances on which these forward-looking statements are based. Neither Kiadis Pharma nor its advisers or representatives nor any of its subsidiary undertakings or any such persons officers or employees guarantees that the assumptions underlying such forward-looking statements are free from errors nor does either accept any responsibility for the future accuracy of the forward-looking statements contained in this press release or the actual occurrence of the forecasted developments. You should not place undue reliance on forward-looking statements, which speak only as of the date of this press release.

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Kiadis Pharma changes strategy to focus solely on development of Natural Killer (NK) Cell therapeutics and terminates development of ATIR101 -...

bluebird bio and Forty Seven Announce a Research Collaboration to Study an All Antibody Conditioning Regimen for Use in Combination with Autologous…

CAMBRIDGE, Mass. & MENLO PARK, Calif.--(BUSINESS WIRE)--bluebird bio, Inc. (Nasdaq: BLUE) and Forty Seven, Inc. (Nasdaq:FTSV) announced today that they have entered into a research collaboration to pursue clinical proof-of-concept for Forty Sevens novel antibody-based conditioning regimen, FSI-174 (anti-cKIT antibody) plus magrolimab (anti-CD47 antibody), with bluebirds ex vivo lentiviral vector hematopoietic stem cell (LVV HSC) gene therapy platform. This collaboration will focus on a conditioning approach aimed to deliver reduced toxicity and will initially target diseases that have the potential to be corrected with transplantation of autologous gene-modified blood-forming stem cells. If successful, the new conditioning regimen could allow for more patients to undergo gene therapy.

Autologous hematopoietic stem cell transplantation (HSCT) and most ex vivo LVV HSC gene therapies require that a patients own stem cells first be depleted from the bone marrow to facilitate the engraftment of the new (or gene-modified) HSCs through a process called conditioning. Conditioning is performed using chemotherapy or radiation, which can place patients at risk for infection and require hospitalization until bone marrow cells have recovered. In addition, conventional conditioning can place patients at risk for secondary malignancy and infertility. As a result, the overall toxicity profile of current conditioning regimens limits the types of patients who are eligible for gene therapy. It is hoped that novel antibody based conditioning regimens could avoid these toxicities.

We are excited about this collaboration, combining our industry-leading LVV HSC gene therapy platform with Forty Sevens novel antibody-based conditioning regimen, said Philip Gregory, chief scientific officer, bluebird bio. We believe that, if successful, this novel conditioning modality could not only increase the number of patients and physicians who may consider gene therapy but also improve the overall risk benefit profile for stem cell-based gene therapy, as well as potentially reduce time and costs associated with hospital visits.

Forty Seven is advancing the pioneering work on CD47 and cKIT from our scientific founder, Irv Weissmans lab. We have shown that antibody blockade of CD47 can synergize with other antibodies targeting cancer to promote tumor engulfment. Based on this experience, coupled with the results of preclinical studies, we are eager to explore this dual-antibody approach for the potential treatment of non-malignant diseases, says Jens Peter Volkmer, M.D., Founder and Vice President of Research and Development at Forty Seven.

Forty Sevens President and Chief Executive Officer, Mark McCamish, M.D., Ph.D., commented, bluebird is a leading gene therapy company and we are excited to collaborate with them. Stem cell transplantation is potentially curative for a variety of blood diseases, including genetic blood disorders like sickle cell disease and beta-thalassemia. If successful, we believe our chemo- and radiation-free, all-antibody approach could expand transplantation beyond genetic blood disorders to a range of indications for which current transplantation approaches are suboptimal. In 2020, we plan to evaluate FSI-174 in healthy volunteers, before initiating a combination study of Forty Sevens novel all-antibody conditioning regimen and bluebirds gene therapy product.

Under the terms of the agreement, bluebird bio will provide its ex vivo LVV HSC gene therapy platform and Forty Seven will contribute its innovative antibody-based conditioning regimen for the collaboration.

About FSI-174 and MagrolimabFSI-174 is a humanized monoclonal antibody targeting cKIT, which is a receptor that is highly expressed on hematopoietic stem cells. Magrolimab is a humanized monoclonal antibody targeting CD47, which is a dont eat me signal to macrophages and is expressed on all cells. Magrolimab is currently being investigated in Phase 2 clinical trials to treat cancer and has established clinical efficacy in four indications, including myelodysplastic syndrome, acute myeloid leukemia, diffuse large B cell lymphoma and follicular lymphoma, with a favorable safety profile in over 350 patients treated, including some patients treated continuously for over two years. When combined, FSI-174 sends a positive signal to macrophages to target blood forming stem cells for removal and magrolimab disengages inhibitory signals that block phagocytosis. Combination of these antibodies has shown efficient removal of blood forming stem cells, allowing for transplantation in pre-clinical models.

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 by researching cerebral adrenoleukodystrophy, sickle cell disease, transfusion-dependent -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.

bluebird bio is a trademark of bluebird bio, Inc.

About Forty Seven Inc.Forty Seven, Inc. is a clinical-stage immuno-oncology company that is developing therapies targeting cancer immune evasion pathways based on technology licensed from Stanford University. Forty Sevens lead program, magrolimab, is a monoclonal antibody against the CD47 receptor, a dont eat me signal that cancer cells commandeer to avoid being ingested by macrophages. This antibody is currently being evaluated in multiple clinical studies in patients with myelodysplastic syndrome, acute myeloid leukemia, non-Hodgkins lymphoma, ovarian cancer and colorectal carcinoma.

For more information, please visit http://www.fortyseveninc.com or contact info@fortyseveninc.com.

Follow Forty Seven on social media: @FortySevenInc, LinkedIn

Forward-Looking StatementsThis release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as "may," "will," potentially, and similar expressions (as well as other words or expressions referencing future events, conditions, or circumstances) are intended to identify forward-looking statements. These statements include those related to the research and development plans for bluebird bios and Forty Sevens respective platforms and product candidates, the timing and success of Forty Sevens collaboration with bluebird bio, Forty Sevens plans to pursue clinical proof-of-concept for FSI-174 plus magrolimab with the LVV HSC gene therapy platform, the focus on diseases that have the potential to be corrected with transplantation of autologous gene-modified blood-forming stem cells, the tolerability and efficacy of FSI-174 and magrolimab, Forty Sevens plans to continue development of FSI-174 plus magrolimab, as well as related timing for clinical trials of the same.

Any forward-looking statements are based on the companies 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 exploratory antibody-based conditioning platform will not be successful or will not be safe or effective in clinical trials, the risks that the collaboration between bluebird bio and Forty Seven will not continue or be successful, and the risk that the parties will not be successful in advancing the collaboration in development, the risk that potential product candidates that bluebird bio and Forty Seven develop may not progress through clinical development or receive required regulatory approvals within expected timelines or at all, the risk that clinical trials may not confirm any safety, potency or other product characteristics described or assumed in this press release and the risk that such product candidates may not be beneficial to patients or successfully commercialized. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the companies actual results to differ from those contained in the forward-looking statements, see the section entitled Risk Factors in each companys most recent Form 10-K as well as discussions of potential risks, uncertainties and other important factors in subsequent filings with the Securities and Exchange Commission at http://www.sec.gov. All information contained in this press release are not guarantees of future performance and speak only as of the date hereof, and each of bluebird bio and Forty Seven disclaims any obligation to update this information to reflect future events or circumstances unless required by law.

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bluebird bio and Forty Seven Announce a Research Collaboration to Study an All Antibody Conditioning Regimen for Use in Combination with Autologous...

CAR-T Cell Therapy Market Impressive Growth| Bluebird, Autolus, Cellectis, Celyad, Eureka Therapeutics – Markets Gazette 24

The recent research report on CAR-T Cell Therapy Market provided by DataBridge Market Research offers worldwide industry study, size, share, development, current trends, and forecast till 2026. CAR-T Cell Therapy global market analysis answers various questions like present market position, upcoming market opportunities, worldwide and regional distribution for shareholders, mainly which CAR-T Cell Therapy market segments they should target upon, during the next five years to set up their efforts and investments. The study focus on both qualitative as well as quantitative side and follows Industry benchmark and NAICS standards to built coverage of players for final compilation of study.

Global CAR-T cell therapy market is set to witness a healthy CAGR of 46.35% in the forecast period of 2019- 2026.

For Better Understanding of CAR-T Cell Therapy Market Request Sample Report Enabled with Respective Tables and Figures: https://www.databridgemarketresearch.com/request-a-sample/?dbmr=global-car-t-cell-therapy-market

As per qualitative study key players of CAR-T Cell Therapy market are: Autolus., Bellicum Pharmaceuticals, Inc., Bluebird Inc., CELGENE CORPORATION, Cellectis, Celyad, Eureka Therapeutics, Fortress Biotech., Immune Therapeutics, Juno Therapeutics, Kite Pharma, Novartis AG, Sorrento Therapeutics, Inc., TILT Biotherapeutics, Ziopharm Oncology.

Staying on top of market trends & drivers is essential for decision makers to leverage this emerging opportunity. The Global CAR-T Cell Therapy Market research publication released by DBMR addresses all this aspects and provides the latest scoop and detailed insights on all major & emerging business segments.

Global CAR-T Cell Therapy Market Dynamics:

Market Drivers

Rising demand for chemotherapy among consumer is driving the market growth

Increasing number of cancer patient worldwide is also acting as a driver for this market

Increasing awareness among population about the effectiveness of CAR T- cell therapy will also propel the growth of this market

Market Restraints

High cost of the therapy will restrain the growth of this market

Complexity related with manufacturing process will also hamper the growth of this market

High toxicity associated with the therapy will also act as a restrain for the market growth

Data Collection Matrix

We looked for primary and secondary sources from both the supply and demand sides of the global CAR-T Cell Therapy market for collecting data and information to prepare this encyclopedic research study. From the supply side, our primary sources were technology distributors and wholesalers and manufacturers, whereas our secondary sources were economic and demographic data reports, independent investigations, government publications, and company publications and reports. From the demand side, we relied on mystery shopping, consumer surveys, and end-user surveys for primary research and reference customers and case studies for secondary research.

Global CAR-T Cell Therapy Market Segmentation:

By Target Antigen: CD 19, CD 20, GD2, CD22, CD30, CD33, HER1, HER2, Meso, Egfrvlll, Others

By Application: Acute Lymphocytic Leukemia, Chronic Lymphocytic Leukemia, Non Hodgkin Leukemia, Multiple Myeloma, Pancreatic Cancer

Competitive Analysis:

Each manufacturer or CAR-T Cell Therapy market players growth rate, revenue figures, and gross profit margin is provided in a tabular, simple format for few years and an individual section on CAR-T Cell Therapy market recent development such as collaboration, acquisition, mergers, and any new service or new product launching in the market is offered.

This market research report is a careful investigation of current scenario of the market and future estimations which spans several market dynamics. Competitor strategies such as new product launches, expansions, agreements, joint ventures, partnerships, and acquisitions can be utilized well by the industry to take better steps for selling goods and services.

Currently profiled leading players in the CAR-T Cell Therapy market report: Autolus., Bellicum Pharmaceuticals, Inc., Bluebird Inc., CELGENE CORPORATION, Cellectis, Celyad, Eureka Therapeutics, Fortress Biotech., Immune Therapeutics, Juno Therapeutics, Kite Pharma, Novartis AG, Sorrento Therapeutics, Inc., TILT Biotherapeutics, Ziopharm Oncology.

To know more about the table of contents, you can click @ https://www.databridgemarketresearch.com/toc/?dbmr=global-car-t-cell-therapy-market

Table of Contents in CAR-T Cell Therapy Market Report:

1 Introduction

1.1 Objectives of the Study

1.2 Definitions & Exclusions

1.3 Market Covered

1.3.1 CAR-T Cell Therapy Market, By Type of Service

1.3.2 CAR-T Cell Therapy Market, By End User

1.3.3 CAR-T Cell Therapy Market, By Region

2 Research Methodology

2.1 Market Breakdown & Triangulation

2.1.1 Secondary Data

2.1.1.1 Associations/Organizations

2.1.1.2 Paid Databases

2.1.2 Primary Data

2.1.2.1 Demand Side and Supply Side

2.2 Market Size Estimation

2.2.1 Bottom-Up Approach

2.2.2 Top-Down Approach

3 CAR-T Cell Therapy Market, By Region

3.1 North America

3.2 Europe

3.3 Asia-Pacific

3.4 MEA

3.5 LATAM

4 Competitive Landscape

4.1 Service Launches

4.2 Agreements/Collaborations/Partnerships/Alliances

4.3 Acquisitions

4.4 Investments

Buy Full Copy Global CAR-T Cell Therapy Report @ https://www.databridgemarketresearch.com/checkout/buy/enterprise/global-car-t-cell-therapy-market

Key questions answered in this report Global CAR-T Cell Therapy Market

What will the market size be in 2023 and what will the growth rate be?

What are the key market trends?

What is driving Global CAR-T Cell Therapy Market?

What are the challenges to market growth?

Who are the key vendors in Market space?

What are the key market trends impacting the growth of the Global CAR-T Cell Therapy Market ?

What are the key outcomes of the five forces analysis of the Global CAR-T Cell Therapy Market?

Thanks for reading this article, you can also get individual chapter wise section or region wise report version like North America, Europe or Asia.

About Data Bridge Market Research:

An absolute way to forecast what future holds is to comprehend the trend today!Data Bridge set forth itself as an unconventional and neoteric Market research and consulting firm with unparalleled level of resilience and integrated approaches. We are determined to unearth the best market opportunities and foster efficient information for your business to thrive in the market.

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CAR-T Cell Therapy Market Impressive Growth| Bluebird, Autolus, Cellectis, Celyad, Eureka Therapeutics - Markets Gazette 24

Gene Editing Tool in Ongoing Sickle Cell Trial of BIVV003 Supported by Early Study – Sickle Cell Anemia News

A technique called zinc finger nuclease (ZFN) gene editing technology can be used to modify immature red blood cells called precursor cells to boost the production of fetal hemoglobin and help ensure red blood cells maintain a normal shape in people with sickle cell disease (SCD), a study shows.

Sanofi has launched a Phase 1/2 trial (NCT03653247) evaluating the safety, tolerability and efficacy of a BIVV003, a gene editing therapy using the ZFN technology (by Sangamo Therapeutics) in adults with severe SCD. This trial, taking place at four U.S. sites, is currentlyrecruiting eligible patients.

ZFN technology findings will be presented by Samuel Lessard, PhD, a researcher at Sanofi, in the poster, Zinc Finger Nuclease-Mediated Disruption of the BCL11A Erythroid Enhancer Results in Enriched Biallelic Editing, Increased Fetal Hemoglobin, and Reduced Sickling in Erythroid Cells Derived from Sickle Cell Disease Patients, (abstract No. 974) at the 61st Annual Meeting of the American Society of Hematology (ASH), December 710 in Orlando.

Sickle cell is caused by mutations in the HBB gene, which provides instructions for making part of hemoglobin, a protein responsible for transporting oxygen in the blood.

These mutations change the structure of the protein, resulting in the production of abnormal hemoglobin fibers. These fibers tend to stiffen red blood cells, changing their shape from normal disc-like cells to those with a sickle-like shape.

BIVV003 is an investigational gene edited cell therapy being developed under an agreement between Bioverativ, a Sanofi company, and Sangamo.

It uses Sangamos proprietary ZFN gene editing technology to modify a short sequence of the BCL11Agene in red blood precursor cells acquired from the patients own hematopoietic stem cells (stem cells that give rise to other blood cells) to raise production of fetal hemoglobin, the main form of hemoglobin found in fetuses.

Fetal hemoglobin production is normally switched off in adults and largely disappears at ages 6 months to 1 year, but artificial ways of introducing fetal hemoglobin show increasing promise in treating SCD by preventing red blood cells from taking on a damaging sickle shape.

Sangamoannounced the findings from a proof-of-concept ex-vivo(lab) study aiming to validate the ZFN gene editing technology using immature red blood cell isolated from four healthy donors and one sickle cell patient.

Study findings demonstrated that more than 90% of edited donor cells incorporated genetic modifications in both copies of the BCL11A gene, leading to an increase of 27% to 38% in fetal hemoglobin levels compared to baseline (studys start).

Edited cells from the SCD patient also tended to incorporate these modifications in both gene copies. As a result, patient edited cells produced up to 28% more fetal hemoglobin compared to unedited cells.

Patient red blood cells obtained from already edited precursor cells were also less likely to change to a sickle-like shape, supporting BIVV003 as a potential cell therapy for SCD, the study reports. Further experiments in red blood precursor cells from additional SCD patients are underway.

Sangamo is also enrolling patients with transfusion-dependent beta-thalassemia (TDT) in thePhase 1/2 THALES trial (NCT03432364) to evaluate the safety, tolerability, and efficacy of ST-400, another experimental gene-edited cell therapy that uses the same gene-editing approach as BIVV003.

The companyannounced it will present data from the first three THALESpatients at the ASH meeting.

Joana is currently completing her PhD in Biomedicine and Clinical Research at Universidade de Lisboa. She also holds a BSc in Biology and an MSc in Evolutionary and Developmental Biology from Universidade de Lisboa. Her work has been focused on the impact of non-canonical Wnt signaling in the collective behavior of endothelial cells cells that make up the lining of blood vessels found in the umbilical cord of newborns.

Total Posts: 94

Margarida graduated with a BS in Health Sciences from the University of Lisbon and a MSc in Biotechnology from Instituto Superior Tcnico (IST-UL). She worked as a molecular biologist research associate at a Cambridge UK-based biotech company that discovers and develops therapeutic, fully human monoclonal antibodies.

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Gene Editing Tool in Ongoing Sickle Cell Trial of BIVV003 Supported by Early Study - Sickle Cell Anemia News