Takeda Drives Immuno-Oncology With Three Cell Therapy Collaborations

Takeda Pharmaceutical has started 2019 with a spate of cell therapy collaborationsdesigned to advance the company’s novel immuno-oncology portfolio. The three collaborations will accelerate the discovery of next-generation cancer immunotherapies.

This morning, Osaka, Japan-based Takeda struck deals with Memorial Sloan Kettering Cancer Center, Noile-Immune Biotech Inc. and Crescendo Biologics. Takeda said the diversification into next-generation cell therapy builds directly on its three strategic pillars in oncology: hematologic malignancies, lung cancer and immuno-oncology.

Phil Rowlands, head of Takeda’s Oncology Therapeutic Area Unit, said the company has been excited by momentum in oncology research and development, specifically around the “curative potential of cell-based therapies.” Rowland said the company looks forward to collaborating with pioneers in the field of cell therapy to “fuel research and discovery with the aim of targeting novel mechanisms of action in the cancer-immunity cycle to help us fulfill our aspiration to cure cancer.”

In its partnership with Memorial Sloan Kettering, Takeda will work to develop novel chimeric antigen receptor T-cell (CAR-T) products for the treatment of multiple myeloma, acute myeloid leukemia and additional solid tumor indications. The collaboration with the famed cancer center will be co-led by former Juno Therapeutics scientific founder Michel Sadelain, who is currently head of the Center for Cell Engineering at Memorial Sloan Kettering.

In September of 2017, Takeda and biotech startup Noile-Immune Biotech forged a deal to develop CAR-T therapies, shortly after the U.S. Food and Drug Administration approved the first CAR-T therapy, Novartis’ Kymriah. This morning, Takeda and Noile-Immune have strengthened their relationship. The company noted that the successes seen so far in the program have spurred the move. Takeda exclusively licensed two assets, NIB-102 and NIB-103 for the treatment of various solid tumor indications. The company will co-develop these CAR-T programs with Noile, using that company’s proprietary “Prime” (proliferation inducing and migration-enhancing) CAR-T platform. The company plans to gain regulatory approval for human testing of NIB-102 by the end of this year.

In its third deal with Crescendo Biologics, Takeda will exercise its option for an exclusive oncology-targeted Humabody license to allow the company to evaluate the Humabody VHs for the development of novel CAR-T therapeutics. Takeda said the development will “leverage the unique properties of single-domain tumor-targeted binders as an alternative to conventional single-chain variable fragment (scFv)-based approaches.”

In addition to the collaborations, Takeda also established a new internal translational cell therapy engine with bioengineering, chemistry, manufacturing and control (CMC), clinical and translational expertise. This new division will be led by Stefan Wildt. The group will aim to “rapidly translate innovative and differentiated cell therapy concepts into the clinic,” Takeda said.

Wildt said there’s an incredible opportunity to combine promising innovation through external partners with “the power of a fit-for-purpose translational cell therapy engine” that will accelerate the development of novel therapies.

https://www.biospace.com/article/takeda-drives-immuno-oncology-portfolio-with-three-cell-therapy-collaborations/

Unity Biotechnology selects UBX1967 as lead ophthalmology candidate

Unity Biotechnology has selected UBX1967 as the lead development candidate in the ophthalmology pipeline for advancement into studies to enable an Investigational New Drug application and executed the compound license agreement for the compound with Ascentage Pharma under a previously agreed compound library agreement. UBX1967 is being evaluated in a range of age-related diseases of the eye, including neovascular age-related macular degeneration, proliferative diabetic retinopathy and diabetic macular edema. Unity plans to file an IND application for UBX1967 in 2H19.
https://thefly.com/landingPageNews.php?id=2843517

Catalyst Biosciences initiated at Oppenheimer

Catalyst Biosciences initiated with Outperform at Oppenheimer. Oppenheimer analyst Kevin DeGeeter initiated Catalyst Biosciences with an Outperform rating and a price target of $24. The analyst notes that the company offers a “unique approach” in hemophilia treatment, asking key questions about the “best method to transition inhibitor patients from products designed to treat bleeding to prophylaxis for preventing bleeds” and the optimal treatment of “pediatric hemophilia B patients that are not candidates for subcutaneous non-factor replacement therapies or gene therapy”. DeGeeter contends that investors are under-appreciating Catalyst Biosciences’ Factor VIIa program and anticipates the investment story for the stock to solidify this year.
https://thefly.com/landingPageNews.php?id=2843503

Novavax spikes as high as 12% after announcing positive results for NanoFlu

Shares of Novavax are spiking in the after-hours after announcing top-line results of its Phase 2 clinical trial of NanoFlu. The trial compared the safety and immune responses of various quadrivalent formulations of NanoFlu, with or without Novavax' Matrix-M adjuvant, with two U.S.-licensed influenza vaccines in 1,375 healthy adults 65 years of age and older. Shares are currentlu up 9.5% in after-hours trading to $2.23 per share.
https://thefly.com/landingPageNews.php?id=2843511

Verastem aims to continue expanding commercial traction of copiktra

Verastem highlighted the company’s recent progress and outlined strategic priorities for 2019. “2018 was a pivotal year for Verastem Oncology, as the U.S. Food and Drug Administration’s approval of COPIKTRA and other key accomplishments strongly positioned us to execute on our 2019 corporate priorities that are focused on increasing revenues, initiating additional clinical studies of COPIKTRA and advancing our pipeline,” said Robert Forrester, President and Chief Executive Officer of Verastem Oncology. “We are pleased with the strong vote of confidence we have received in duvelisib, including validating licensing agreements in key Asian markets, recognition of our pivotal Phase 3 data in the medical journal Blood, and more. We are also entering 2019 with a strong balance sheet derived from the successful completion of multiple financing transactions, which we believe provides us with important financial strength to achieve our planned corporate objectives. We look forward to keeping the momentum going, and to sharing ongoing updates on our progress.” Verastem Oncology’s 2019 focus is to execute on business priorities aimed at increasing the company’s sales and revenues: Continuing to expand on the commercial traction of COPIKTRA in CLL/SLL and FL for appropriate patients; Expansion of the open-label, multicenter, Phase 2 clinical trial evaluating the efficacy and safety of duvelisib monotherapy in adult patients with histologically confirmed relapsed or refractory PTCL. This study is expected to enroll approximately 120 patients; Initiating a confirmatory Phase 3 study evaluating duvelisib for the treatment of patients with relapsed or refractory FL after at least two prior systemic therapies. The confirmatory study is expected to start in the second half of 2019; Initiating additional investigational studies of duvelisib as a monotherapy and in combination with other anti-cancer agents, such as checkpoint inhibitors, in both hematological and solid tumor malignancies; Working with the LLS to advance the PTCL program including the expansion of the Phase 2 combination study of duvelisib and romidepsin for patients with relapsed or refractory PTCL; Additional ex-U.S. partnerships for duvelisib; Presenting and publishing additional duvelisib data; and advancing the company’s focal adhesion kinase inhibitor defactinib, which is designed to treat cancer through modulation of the tumor microenvironment and enhancement of anti-tumor immunity. Defactinib is currently being evaluated in three separate clinical collaborations in combination with immunotherapeutic agents for the treatment of several different cancer types including pancreatic cancer, non-small cell lung cancer, and mesothelioma.
https://thefly.com/landingPageNews.php?id=2843453

Flexion CEO comments first full year launch of Zilretta

“2018 was a foundational year for ZILRETTA, and in the first full year of the launch, we saw strong product uptake as we established a broad and growing base of prescribers. Furthermore, the clinical feedback on ZILRETTA from physicians and patients alike continues to be overwhelmingly positive and gratifying,” said Michael Clayman, M.D., President and Chief Executive Officer. “We now enter 2019 with a product-specific J code, which we believe will be a key driver of continued ramp, as it provides prescribers with a well-known and clearly defined reimbursement mechanism that is utilized by both Medicare and private payers. Each year, roughly five million people in the U.S. receive intra-articular injections for osteoarthritis knee pain, and we believe ZILRETTA will play an increasingly significant role in the treatment paradigm for this large and growing patient population.”
https://thefly.com/landingPageNews.php?id=2843455

Regenxbio provides corporate update on candidate development programs

Regenxbio (RGNX) provided a year-end 2018 corporate update. Highlights include: further positive interim update from RGX-314 Phase I trial for wet AMD, in which 50% of subjects treated in Cohort 3 continue to remain free of anti-VEGF injections at nine months and mean RGX-314 intraocular protein expression in recently dosed Cohort 4 was higher than in previously reported Cohort 3 at one month; received FDA clearance to expand RGX-314 Phase I protocol immediately into a Phase IIa clinical trial, and on track to initiate Phase IIb trial in late 2019; plans to expand RGX-314 into additional retinal conditions, with the first such IND submission anticipated in 2H19; RGX-121 was well-tolerated in first patient dosed at initial eight-week safety assessment; additional recruitment and site activation continues; interim trial updates for RGX-121, RGX-111 and RGX-501 anticipated in 2H19; first anticipated FDA regulatory action for a proprietary NAV Technology-based treatment, Novartis’ (NVS) Zolgensma for the treatment of SMA Type I, expected in May; the company had over $470M in cash, cash equivalents and marketable securities as of December 31, 2018, and expects to end 2019 with over $330M in cash, cash equivalents and marketable securities, excluding any projected commercial revenue from Zolgensma.
https://thefly.com/landingPageNews.php?id=2843459