Biomarin: Participant Diagnosed With Leukemia In Late-Stage Hemophilia Gene Therapy Trial

 

• In an SEC filing, BioMarin Pharmaceutical Inc 
  BMRN-2.71%+ Free Alerts
   said that, in August, a participant in its valoctocogene roxaparvovec (BMN 270) phase 3 study was diagnosed with B-cell acute lymphoblastic leukemia (B-ALL). 
• Based on BioMarin's assessment of the case to date, including initial genetic testing of the leukemic cells, BioMarin believes at this time that this cancer is unrelated to BMN 270.
• In August, the hemophilia A gene therapy received conditional European approval.
• The overall rate of all cancers observed in all BMN 270 trial patients (2 in approximately 400 patient-years of observation) appears consistent with expected cancer rates in persons with Hemophilia.
• BioMarin said it started a comprehensive assessment of the case. 
• Genomic analyses are underway, and BioMarin expects to confirm the absence of BMN 270 vector integration events contributing to leukemic growth.
• BioMarin submitted an IND safety report to the FDA consistent with the company's commitments to regulatory authorities.
• No authority has requested a hold on any trial at this time. 
• The company says it remains on track to resubmit the marketing application for BMN 270 to the FDA by the end of this month.

https://www.benzinga.com/general/biotech/22/09/28833630/biomarin-says-one-participant-diagnosed-with-leukemia-in-late-stage-hemophilia-gene-therapy-trial

Terns: Wainwright Has Doubts On NASH-Focused Stock, builds Neutral Case

 

• HC Wainwright initiated Terns Pharmaceutical Inc 
  TERN+14.4%+ Free Alerts
   with a Neutral rating and a price target of $6.00. The biopharmaceutical company has a portfolio of small molecule programs for NASH and obesity. 
• The analyst says the Phase 2a DUET study of oral TERN-501 in Noncirrhotic Adults with NASH "lacks sufficient supporting evidence," while the rest of the company's pipeline "is too early." 
• Terns' recent clinical track record has been disappointing, Arce tells investors.
• The analyst notes some recent data as underwhelming, including the Phase 2a LIFT study of TERN-101 reported last year and Part 1 of the Phase 1b AVIATION study of TERN-201 10mg reported in March. 
• Although top-line results from AVIATION Part 2 (20mg) are expected in 2H22, we do not expect the company to pursue further development with TERN-201, says the analyst.
• DUET study dosing started in July, and top-line data is expected in 2H23. While acknowledging the pipeline potential, more data is needed to be more constructive. The DUET readout is unlikely to represent a significant stock catalyst.

https://www.benzinga.com/general/biotech/22/09/28834690/with-some-doubts-hc-wainwright-initiates-coverage-on-this-nash-focused-stock

Oak Street cut to Neutral from Buy by Goldman

 Target to $29 from $32

https://finviz.com/quote.ashx?t=OSH&ty=c&ta=1&p=d

Ventyx surges after Bristol rival's approval without black box warning

 Ventyx Biosciences Inc. shares rose 59% to $36.80 after analysts mentioned favorable signs related to the approval of Bristol Myers Squibb Co.'s psoriasis drug Sotyktu.

Stifel, Evercore ISI and Oppenheimer analysts noted that the U.S. Food and Drug Administration approval of the TYK2 inhibitor didn't include a black-box warning.

Jeff Jones and Leland Gershell at Oppenheimer wrote that "we see this as a significant win for deucravacinitib, VTYX and other TYK2 developers, substantially differentiating the TYK2 inhibitors from the JAK 1-3 inhibitors."

https://www.marketscreener.com/quote/stock/VENTYX-BIOSCIENCES-INC-128506325/news/Ventyx-Shares-Surge-59-After-Bristol-Myers-Drug-Approved-Without-Black-Box-Warning-41754939/