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The US FDA grants Breakthrough Therapy Designation to bemarituzumab for FGFR2b+ gastric cancer
On 19th April, Amgen announced that the US FDA has granted breakthrough therapy designation to bemarituzumab (anti-FGFR2b) as a frontline treatment option in combination with mFOLFOX6 for HER2neg, fibroblast growth factor receptor 2b (FGFR2b) overexpressing metastatic and locally advanced gastric and gastroesophageal (GEJ) adenocarcinoma. The designation is based on results from the Phase 2 FIGHT study.
Background: Gastric cancer (GC) is an aggressive cancer, with particularly high incidence rates in East Asian countries and a 5-year survival rate of only 5.5% for metastatic GC. It is the third most common cause of deaths by cancer worldwide.
As many as 30% patients with HER2neg GC express FGFR2b which has been shown to play a prominent in driving gastric tumorigenesis. It is a poor pathological and prognostic marker for GC associated with worse survival outcomes and no approved treatment options.
Details: BTD was granted for FGFR2b overexpression in at least 10% of tumor cells as demonstrated by an FDA approved companion diagnostic assay.
The FIGHT study investigated bemarituzumab + mFOLFOX6 vs. mFOLFOX6 in 155 patients with previously untreated advanced, HER2neg GC/GEJ cancer with FGFR2b overexpression. The trial showed clinically meaningful improvement in primary endpoint of PFS (9.5 vs. 7.4 months; HR: 0.68, p=0.0727) and key secondary endpoints of OS (NR vs. 12.9 months; HR: 0.58, p=0.0268) and ORR (47% vs. 33%) for bemarituzumab + mFOLFOX6 vs. mFOLFOX6, respectively. Median duration of response was 12.2 vs. 7.1 months. Patients with higher expression showed better survival outcomes with mPFS in patients with IHC 2+/3+ at ≥10% of sample being 14.1 vs. 7.3 months (HR: 0.44).
Implications: Amgen acquired bemarituzumab from Five Prime Therapeutics in a $1.9B acquisition, completed on 16th April. Results from the FIGHT study will support a pivotal Phase III study in GC/GEJ cancer patients. Bemarituzumab has the potential to become first-in-class therapy for the treatment of GC patients with FGFR2b overexpression, thereby addressing the high unmet need for this patient segment.

FDA approves JEMPERLI (dostarlimab-gxly) for dMMR endometrial cancer
“The approval of JEMPERLI is a transformative milestone for AnaptysBio. This event provides important validation for our proprietary SHM antibody discovery platform and provides significant potential future milestone and royalty revenue to support AnaptysBio’s growth,” said Hamza Suria, president and chief executive officer of AnaptysBio. “While AnaptysBio has partnered certain pipeline assets, our primary value-creation strategy remains focused on advancing wholly-owned, first-in-class therapeutic antibodies from discovery through development, and we look forward to multiple upcoming clinical data readouts from our product pipeline through 2021 and 2022.”

Pre-BLA Meeting Requested with the FDA to Discuss Path to Eryaspase’s Approval in ALL

• Results from the NOPHO-sponsored Ph 2 clinical trial demonstrated that eryaspase + chemotherapy combination, administered every two weeks, provides a sustained asparaginase enzyme activity level, and is generally well tolerated with few hypersentivity reactions. 
• The trial results were presented at the 62nd ASH Annual Meeting held in December 2020. 
• The investigators positioned eryaspase as a potential attractive treatment option for patients who developed hypersensitivities to PEG-asparaginase. 
• ERYTECH initiated a dialogue with the US FDA in 2020 to evaluate the potential of eryaspase to be approved for the treatment of hypersensitive ALL patients based on the NOPHO-sponsored Phase 2 clinical trial.
• The FDA confirmed the unmet medical need and, following the review of a comprehensive data package submitted by ERYTECH, invited the Company to request a pre-BLA meeting to discuss the potential for the NOPHO-sponsored Phase 2 clinical trial to support marketing approval in the United States.  
• ERYTECH has requested a pre-BLA meeting and subject to the feedback received in that meeting, the Company plans to submit a Biologics License Application (BLA) in the second half of 2021 for its lead product candidate eryaspase for the treatment of hypersensitive ALL patients.

• IDMC of the JUPITER-06 clinical trial has determined that toripalimab + paclitaxel/cisplatin as first-line treatment for patients with advanced ESCC has achieved the pre-specified primary endpoints of PFS and OS at the interim analysis.
• JUPITER-06 is a randomized, double-blind, placebo-controlled, multi-center, Ph 3 clinical trial initiated in 2019 with 514 patients enrolled.
• The interim analysis showed that toripalimab + paclitaxel/cisplatin combination significantly prolonged the PFS and OS of patients with advanced ESCC, compared with paclitaxel/cisplatin chemotherapy alone.
• Data from the study are expected later this year.

ULTRA-V Ph 3 Trial Evaluating the Triple Combination of UKONIQ™ (umbralisib), Ublituximab, and Venetoclax launched
Richard R. Furman, MD, Director of CLL Research Center at Weill Cornell Medicine and Study Chair for the ULTRA-V Phase 2 and Phase 3 trials stated, “We are excited to launch this pivotal Phase 3 study based on the promising Phase 1 clinical results reported to date on the triplet combination of UKONIQ, ublituximab and venetoclax in patients with CLL. While recent approvals provide excellent treatment options for patients, disease progression and treatment tolerability still remain problematic for many patients. Our belief is that time-limited treatment regimens, such as U2 plus venetoclax, have the potential to produce meaningful responses without the need to expose patients to continuous therapy and related toxicities. We look forward to presenting the results of the Phase 2 portion of this study at a future medical meeting. I want to thank my colleagues for their strong support of the Phase 2 ULTRA-V trial and look forward to continuing and expanding our efforts now in Phase 3.”

Vertex and Obsidian Therapeutics enter collaboration to discover novel regulatory gene editing therapies for serious diseases
On 22nd April 2021, Vertex Pharmaceuticals and Obsidian Therapeutics entered into a multi-year strategic research collaboration and licensing agreement focused on the discovery of novel therapies that regulate gene editing for the treatment of serious diseases. This leverages Obsidian’s proprietary cytoDRiVE® platform to discover gene-editing medicines whose therapeutic activity can be precisely controlled using small molecules and Vertex’s scientific and clinical capabilities in small molecule, cell and genetic therapies.