News
Key Takeaways:
- Sangamo Therapeutics, a genomic medicine company, reported on Pfizer Inc.’s announcement of positive topline results from the Phase 3 AFFINE trial (NCT04370054) evaluating giroctocogene fitelparvovec, an investigational gene therapy that Sangamo is co-developing with and licensing to Pfizer for the treatment of adults with moderately severe to severe hemophilia A.
- Sangamo is eligible to earn from Pfizer up to $220 million in milestone payments upon the achievement of certain regulatory and commercial milestones for giroctocogene fitelparvovec and product sales royalties of 14% - 20% if giroctocogene fitelparvovec is approved and commercialized, subject to certain reductions.
Key Takeaways:
- Pinetree Therapeutics, a biotechnology company pioneering next generation targeted protein degraders (TPD) to combat drug resistance in oncology and beyond, announced it has entered into an exclusive option and global license agreement with AstraZeneca for a preclinical EGFR degrader candidate.
- Under the terms of the agreement, AstraZeneca will be granted an exclusive option to license Pinetree’s preclinical EGFR degrader for global development and commercialization.
- In exchange, Pinetree will receive upfront and near-term payments of up to $45 million and is eligible to receive additional development and commercial milestone payments for a total deal value of over $500 million, as well as tiered royalties on net sales worldwide.
Key Takeaways:
- Kyverna Therapeutics, a patient-centered, clinical-stage biopharmaceutical company focused on developing cell therapies for patients suffering from autoimmune diseases, announced the designation as Regenerative Medicine Advanced Therapy (RMAT) by the U.S. Food and Drug Administration (FDA) for its autologous, fully human CD19 chimeric antigen receptor (CAR) T-cell product candidate, KYV-101, to be used for the treatment of patients suffering from refractory stiff-person syndrome.
Key Takeaways:
- Kazia Therapeutics Limited , an oncology-focused drug development company, is pleased to announce results from GBM-AGILE, a phase II/III study that included an evaluation of paxalisib versus standard of care (SOC) for patients with glioblastoma (NCT03522298), a life-threatening brain cancer, where there is an urgent unmet need for new therapeutics.
- GBM AGILE trial data shows clinically meaningful improvement in a prespecified secondary analysis for overall survival in paxalisib-treated, newly diagnosed unmethylated patients with glioblastoma
Key Takeaways:
- Palvella Therapeutics, a clinical-stage biopharmaceutical company focused on developing and commercializing novel therapies to treat patients suffering from serious, rare genetic skin diseases for which there are no U.S. Food and Drug Administration (FDA)-approved therapies, and Pieris Pharmaceuticals, Inc. announced they have entered into a definitive merger agreement to combine the companies in an all-stock transaction.
- The combined company will focus on developing and commercializing Palvella’s lead clinical product candidate, QTORIN™ 3.9% rapamycin anhydrous gel (QTORIN™ rapamycin), for the treatment of microcystic lymphatic malformations (microcystic LMs), cutaneous venous malformations, and other serious, functionally debilitating skin diseases driven by the overactivation of the mammalian target of rapamycin (mTOR) pathway.
- Upon completion of the proposed merger, the combined company will operate under the name Palvella Therapeutics, Inc., will be headquartered in Wayne, PA, and is expected to trade on The Nasdaq Capital Market.
Key Takeaways:
- Abeona Therapeutics Inc., a cell and gene therapy company with proprietary adeno-associated virus (AAV)-based capsids, and Beacon Therapeutics, an ophthalmic gene therapy company and Syncona portfolio company, announced an agreement by which Beacon will evaluate Abeona’s patented AAV204 capsid for the development and commercialization of potential gene therapies for select ophthalmology indications.
- AAV204, a novel AAV capsid from the AIM™ capsid library licensed by Abeona from the University of North Carolina at Chapel Hill, has been shown to achieve high macular and optic nerve transduction levels after para-retinal administration and has also been shown to facilitate transduction of both the inner and outer retina after intravitreal administration in mice and non-human primates.
Key Takeaways:
- Merz Therapeutics announces the successful acquisition of INBRIJA® (levodopa inhalation powder) and (F)AMPYRA® (fampridine), and related assets from Acorda Therapeutics, Inc. AMPYRA® (dalfampridine) is Food and Drug Administration (FDA) approved in the U.S. and commercialized as FAMPYRA in the European Union and other territories throughout the world.
- The completion of this deal, valued at USD 185 million in cash, bolsters Merz Therapeutics’ market position by enhancing offerings for people living with Parkinson’s disease and expanding into the multiple sclerosis (MS) space.
