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FDA green­lights Mar­ius' oral hy­pog­o­nadism pill; VBL Ther­a­peu­tics makes cuts to its work­force

The FDA has approved Marius’ testosterone replacement treatment for adult males with hypogonadism, or testosterone deficiency.

The oral drug, to be marketed as Kyzatrex, will be available at three different doses — 100, 150 and 200mg — and the pill would be taken twice daily. The drug was approved on the back of a Phase III program with 155 males with hypogonadism. Of the men who got Kyzatrex, 88% had an average testosterone level within a normal range by the three-month mark.

Pri­or­i­ty re­view for Gami­da's cell ther­a­py; PhI­II shut­tered at Akari; Im­mutep drops da­ta on LAG3 study

Eight weeks after completing the rolling BLA for its blood cancer stem cell therapy, Gamida Cell says the FDA has accepted it for priority review.

The agency will make a decision on omidubicel by Jan. 30 of next year, the Boston-based biotech said Monday morning. The cell therapy is being evaluated as an allogeneic hematopoietic stem cell (bone marrow) transplant for patients with blood cancers.

An advisory committee meeting is not expected, the company said. The FDA has previously tagged the cell therapy with breakthrough therapy and orphan drug designations.

Io­n­is, Cy­cle­ri­on tout pos­i­tive da­ta read­outs; MA­IA prices first biotech IPO in over two months

Ionis put out positive topline results from a Phase IIb trial on end-stage renal disease, investigating patients on dialysis.

The drug candidate, fesomersen, achieved its primary goal of “no increase in incidence of major bleeding and clinically relevant non-major bleeding,” compared to placebo. The study, named RE-THINC ESRD, was conducted by Bayer, which licensed the drug from Ionis.

FDA ac­cepts Lex­i­con's heart fail­ure re­sub­mis­sion; Bris­tol My­ers buys out mava­camten roy­al­ties

Just two weeks after the FDA again denied Lexicon Pharmaceuticals’ attempt to overturn a CRL in type 1 diabetes, the Texas biotech said the agency has accepted the drug’s resubmission for heart failure.

A decision date is slated for sometime in May 2023. The regulator will consider two Phase III trials, one in which worsening heart failure sent type 2 diabetes patients into the hospital (SOLOIST-WHF) and another that includes patients with type 2 diabetes, chronic kidney disease and risks for cardiovascular disease (SCORED). In total, sotagliflozin has been tested in 14 Phase III trials in about 20,000 people.

Galectin gets $60M cred­it line from bil­lion­aire chair; In­flam­ma­to­ry dis­ease biotech reach­es SPAC deal

The chair of Galectin Therapeutics, billionaire and Jan. 6 pro-Trump rally financier Richard Uihlein, is offering up $60 million in an unsecured credit facility to the biotech.

Almost 15 years ago, Uihlein began investing in the company. With his latest batch of financial support, the largest in the company’s history, Galectin can keep the lights on through 2024, which will be key as the biotech’s Phase IIb NAVIGATE trial is slated to read out in mid-2024.

No­var­tis gener­ics arm files for MS biosim­i­lar, chal­leng­ing Bio­gen; Pluris­tem short­ens name

Novartis’ generics arm Sandoz said it has submitted applications for a multiple sclerosis and Crohn’s disease biosimilar.

The drug is a biosimilar for Biogen’s Tysabri, known generically as natalizumab. According to Sandoz, in patients with relapsing-remitting MS, the biosimilar matched Tysabri in both efficacy and safety. Its BLA for the biosimilar has been accepted by both US and EU authorities, Sandoz said.

Ver­tex wins 'break­through' nod for non-opi­oid pain drug; Af­ter US with­draw­al, EMA re­stricts Clo­vis' Rubra­ca

Vertex won a breakthrough therapy designation for its non-opioid pain drug, the company announced Friday.

Regulators bestowed the designation upon VX-548, an experimental pill targeting nerve receptors, for the treatment of moderate-to-severe acute pain. The move comes about four months after Vertex said the program achieved its primary goals in two Phase II studies.

With the BTD, Vertex added that the FDA has also signed off on its plan for a pivotal trial program.

F-star lines up Take­da pact weeks af­ter M&A ex­it; En route to Eu­rope de­ci­sion, Gen­Sight touts 5-year da­ta

Weeks after wrapping up a monthslong search for a new owner, F-star Therapeutics has signed a licensing agreement with Takeda for a new immuno-oncology bispecific antibody.

The deal terms are far from the big figures floated by other Big Pharma-biotech tie-ups in recent months, with Takeda doling out $1 million to start and offering up to $40 million more in back-end payments if all milestones are met, the companies said Wednesday morning.

Apel­lis nabs pri­or­i­ty re­view for ge­o­graph­ic at­ro­phy pitch; Bris­tol My­ers ex­tends of­fer time­line on Turn­ing Point buy­out

The FDA will decide by Nov. 26 whether to approve the first treatment for geographic atrophy secondary to age-related macular degeneration, an irreversible disease that leads to vision loss. And the agency won’t hold an adcomm for it.

The maker of the potential treatment is Apellis, which put out long-term data on the drug in March and quashed some market concerns after mixed data last year, with one analyst pegging the chance for approval at 80%.

An old epilep­sy med gets oral liq­uid for­mu­la­tion nod; PMV pairs up AS­CO stand­out with Keytru­da

The FDA approved an oral liquid formulation of the drug zonisamide, originally made by Eton Pharmaceuticals and currently owned by Azurity Pharmaceuticals.

The nod gives Azurity the ability to market Zonisade as an adjunctive therapy for treating partial seizures in adults and kids 16 years and older who have epilepsy. The green light comes after three double-blind and placebo-controlled clinical studies of the drug, which is administered once or twice a day. Zonisamide was originally approved in the early 2000s as a capsule for treating partial epileptic seizures and has been marketed by a variety of companies, including the former Irish biotech Elan Pharmaceuticals, Japanese drugmaker Eisai and Sumitomo Dainippon. In 2010, Elan and Eisai agreed to pay $214.5 million to resolve allegations of off-label marketing of Zonegran.