4D Molecular: Updated Data From Currently Paused Fabry Disease Cardiomyopathy Study

 

• 4D Molecular Therapeutics Inc 
  FDMT+1.01%+ Free Alerts
   announced updated interim safety and efficacy data from the two 4D-310 INGLAXA Phase 1/2 trials for Fabry disease cardiomyopathy. 
• All three patients with 12 months of follow-up demonstrated improved cardiac contractility, exercise capacity, and quality of life endpoints. 
• Treatment was generally well tolerated, with transient acute aHUS being the only significant adverse event. 
• Following a detailed investigation into the etiology of aHUS, 4DMT is engaging with the FDA to resume enrollment based on updated exclusion criteria and the highly effective rituximab/sirolimus immunosuppressive regimen. 
• aHUS (atypical hemolytic uremic syndrome) is a rare disease that causes tiny blood clots in the small blood vessels.
• The company voluntarily paused enrollment in two INGLAXA trials in January 2023 after observing an aHUS dose-limiting toxicity, and FDA subsequently put the program on clinical hold. 
• 4DMT anticipated that any future clinical development of 4D-310 would be with rituximab-sirolimus under a new clinical protocol(s) and amended or new INDs.
• The company is confident that the rituximab and sirolimus immunosuppressive regimen and updated exclusion criteria will mitigate safety risks and improve patient benefits.

https://www.benzinga.com/general/biotech/23/02/31023425/4d-molecular-discloses-updated-data-from-currently-paused-fabry-disease-cardiomyopathy-study

Sangamo: Fabry Gene Therapy Update Shows Evidence Of Clinical Benefit

 

• Sangamo Therapeutics Inc 
  SGMO+8.08%+ Free Alerts
   announced updated preliminary data as of October 20, 2022, the cutoff date from the Phase 1/2 STAAR study of isaralgagene civaparvovec, or ST-920, a wholly owned gene therapy product candidate for Fabry disease. 
• As of the November 15, 2022, supplemental cutoff date, 13 patients exhibited supraphysiological levels of α-Gal A activity, sustained for over two years for the patient with the longest follow-up. 
• All five patients who began the dose escalation phase on enzyme replacement therapy (ERT) had been successfully withdrawn from ERT and continued to exhibit supraphysiological levels of α-Gal A activity following withdrawal. .
• No patient has required the resumption of ERT treatment to date.
• One patient achieved 78% globotriaosylceramide (Gb3) substrate clearance at six months and a 77% reduction in urine podocyte loss in one of the first kidney biopsies.
• Gb3 is a fatty substrate that accumulates in the cells of Fabry disease patients and can damage multiple organs.
• Since the cutoff date, four additional patients have been dosed in the expansion phase, and two more patients have been withdrawn from ERT.
• The Phase 1/2 STAAR study expansion phase is ongoing, and preparations for a potential Phase 3 trial are underway, anticipated to start by the end of 2023. Dosing could begin as early as the first part of 2024.

https://www.benzinga.com/general/biotech/23/02/31023691/sangamo-therapeutics-shares-updated-data-from-fabry-gene-therapy-study-says-it-shows-evidence-of-

Ukraine-China meeting 'would be desirable' - Zelenskiy

 

Ukrainian President Volodymyr Zelenskiy said on Thursday he had not seen any Chinese plan for ending Russia's war on Ukraine, but that "it would be desirable" for Chinese and Ukrainian representatives to meet.

He said on the eve of the first anniversary of Russia's invasion of Ukraine that Kyiv had already communicated the desire for such a meeting.

"We would like to meet with China," he said during a news briefing in Kyiv with visiting Spanish Prime Minister Pedro Sanchez. "This is in the interests of Ukraine today."

China's top diplomat has said Beijing will set out its position on settling the Ukraine conflict through political means in a document that will reference principles from the United Nations' founding charter and take into account territorial integrity, sovereignty and security concerns.

Chinese President Xi Jinping is also expected to deliver a "peace speech" on the Feb. 24 anniversary of Moscow's invasion.

Zelenskiy said he had heard only "general things" about China's proposals through Ukrainian diplomats, but that it was encouraging that China was considering brokering peace.

https://www.marketscreener.com/quote/currency/US-DOLLAR-RUSSIAN-ROUBL-2370597/news/Ukraine-China-meeting-would-be-desirable-Zelenskiy-43068540/

Teladoc upped to Outperform from Market Perform by SVB

 Target $34

https://finviz.com/quote.ashx?t=TDOC&p=d

VBL Therapeutics and Notable Labs to merge

 Merger will create Nasdaq-listed, clinical-stage therapeutic platform company developing new precision medicines for patient populations selected through Notable’s proprietary Predictive Precision Medicines Platform (“PPMP”)

Notable’s validated PPMP combines multi-dimensional biological assays and machine learning to bio-simulate a patient’s cancer treatment and predict their clinical response to the actual treatment

Notable has applied its PPMP to selecting and developing two clinical-stage therapeutic candidates in acute myeloid leukemia (“AML”); Phase 2a results for lead asset Volasertib in adult AML in platform-predicted responding patients expected in 3Q 2024

Leading healthtech-focused investors have committed an additional $10.3 million in a private placement, led by Builders VC, B Capital Group, Y Combinator, First Round Capital, and Founders Fund; Aggregate net transaction proceeds expected to fund planned operations into 2025

https://finance.yahoo.com/news/vbl-therapeutics-notable-labs-announce-120000861.html