Neu­ro­crine breaks out proof-of-con­cept da­ta for CAH drug; Ake­bia, MT­PC work to­ward Japan­ese NDA af­ter ane­mia drug scores in twin PhI­I­Is

→ More than a month af­ter its big tick­et deal with Voy­ager Ther­a­peu­tics $VY­GR, Neu­ro­crine Bio­sciences $NBIX an­nounced pos­i­tive in­ter­im da­ta from a Phase II proof-of-con­cept study eval­u­at­ing its ex­per­i­men­tal drug, NBI-74788, in adults with a ge­net­ic dis­or­der that re­sults in an en­zyme de­fi­cien­cy that al­ters the pro­duc­tion of adren­al steroids called clas­sic con­gen­i­tal adren­al hy­per­pla­sia (CAH). Da­ta showed a re­duc­tion of at least 50% from base­line in 17-hy­drox­yprog­es­terone (17-OHP) — used as a di­ag­nos­tic bio­mark­er in CAH — in more than half the treat­ed pa­tients. “But what does a 50% ‘re­spon­der’ rate mean in clin­i­cal terms? This study wasn’t re­al­ly de­signed to an­swer this ques­tion, and while we don’t know the pa­tients’ 17-OHP base­line val­ues in this study, our guess from look­ing at oth­er CAH tri­als (ahead of the da­ta to­day) is that it was prob­a­bly very high. Thus, it is un­clear to us to­day how these bio­mark­er changes will or will not trans­late in­to an im­prove­ment in pa­tient out­comes,” Stifel’s Paul Mat­teis wrote.

Ake­bia, which merged with Au­ryx­ia mak­er Keryx last year, put out pos­i­tive late-stage da­ta from twin Phase III Japan­ese stud­ies eval­u­at­ing its lead ex­per­i­men­tal drug vadadu­s­tat as an ane­mia treat­ment for CKD pa­tients. Part­ner Mit­subishi Tan­abe Phar­ma Corp (MT­PC) ex­pects to sub­mit a Japan­ese mar­ket­ing ap­pli­ca­tion in 2019. Ake­bia $AK­BA is in a fierce race with Fi­bro­Gen $FGEN in both Japan and the Unit­ed States to get their re­spec­tive ane­mia drugs on the mar­ket. Fi­bro­Gen and its part­ner Astel­las re­port­ed pos­i­tive 4th Phase III study of their ri­val rox­adu­s­tat in Japan in 2018.

→ UK’s Ox­ford Bio­med­ica is join­ing forces with tech ma­jor Mi­crosoft in a re­search and de­vel­op­ment col­lab­o­ra­tion to de­vel­op next-gen gene ther­a­py vec­tors us­ing the cloud and ma­chine learn­ing.

Lipocine pro­vid­ed up­dat­ed da­ta from its “liv­er fat” study eval­u­at­ing its ex­per­i­men­tal oral drug — LPCN 1144 — which is be­ing de­vel­oped for NASH. In Jan­u­ary, the com­pa­ny said the drug was be­ing eval­u­at­ing in 36 hy­pog­o­nadal males in a 16-week study. Sev­en of the nine sub­jects had at least 10% base­line liv­er fat, which the com­pa­ny said is in­dica­tive of sub­jects with NAFLD with the po­ten­tial to have NASH. Base­line mean liv­er fat of these sev­en sub­jects was 21.0%, and af­ter treat­ment there was an av­er­age ab­solute mean re­duc­tion from base­line of 7.6% in liv­er fat af­ter 8 weeks of treat­ment. On Tues­day, Lipocine said 62% of the of the eval­u­at­ed sub­jects had NAFLD, de­fined as base­line liv­er fat of at least 5%. At the end, 48% of the treat­ed NAFLD sub­jects had NAFLD res­o­lu­tion, de­fined as liv­er fat <5% post treat­ment for 16 weeks. The com­pa­ny’s shares $LPCN were up 7.3% in morn­ing trad­ing.

Has the mo­ment fi­nal­ly ar­rived for val­ue-based health­care?

RBC Capital Markets’ Healthcare Technology Analyst, Sean Dodge, spotlights a new breed of tech-enabled providers who are rapidly transforming the way clinicians deliver healthcare, and explores the key question: can this accelerating revolution overturn the US healthcare system?

Key points

Tech-enabled healthcare providers are poised to help the US transition to value, not volume, as the basis for reward.
The move to value-based care has policy momentum, but is risky and complex for clinicians.
Outsourced tech specialists are emerging to provide the required expertise, while healthcare and tech are also converging through M&A.
Value-based care remains in its early stages, but the transition is accelerating and represents a huge addressable market.

Clay Siegall, Morphimmune CEO

Up­dat­ed: Ex-Seagen chief Clay Sie­gall emerges as CEO of pri­vate biotech

Clay Siegall will be back in the CEO seat, taking the helm of a private startup working on targeted cancer therapies.

It’s been almost a year since Siegall resigned from Seagen, the biotech he co-founded and led for more than 20 years, in the wake of domestic violence allegations by his then-wife. His eventual successor, David Epstein, sold the company to Pfizer in a $43 billion deal unveiled last week.

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No­vo Nordisk oral semaglu­tide tri­al shows re­duc­tion in blood sug­ar, plus weight loss

Novo Nordisk is testing higher levels of its oral version of its GLP-1, semaglutide, and its type 2 diabetes trial results released today show reductions in blood sugar as well as weight loss.

In the Phase IIIb trial, Novo compared its oral semaglutide in 25 mg and 50 mg doses with the 14 mg version that’s currently the maximum approved dose. The trial looked at how the doses compared when added to a stable dose of one to three oral antidiabetic medicines in people with type 2 diabetes who were in need of an intensified treatment.

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Ly­me vac­cine test com­ple­tion is pushed back by a year as Pfiz­er, Val­ne­va say they'll ad­just tri­al

Valneva and Pfizer have adjusted the end date for the Phase III study of their investigational Lyme disease vaccine, pushing it back by a year after issues at a contract researcher led to thousands of US patients being dropped from the test.

In a March 20 update to, Valneva and Pfizer moved the primary completion date on the trial, called VALOR, from the end of 2024 to the end of 2025.

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Stuart Peltz, former PTC Therapeutics CEO

Stu­art Peltz re­signs as PTC Ther­a­peu­tics CEO af­ter 25 years

Stuart Peltz, the longtime CEO of PTC Therapeutics who’s led the rare disease drug developer since its founding 25 years ago, is stepping down.

Succeeding him in the top job is Matthew Klein, who joined PTC in 2019 and was promoted to chief operating officer in 2022. In a call with analysts, he said the CEO transition has been planned for “quite some time” — in fact, as part of it, he gave the company’s presentation at the JP Morgan healthcare conference earlier this year.

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89bio to net $275M from stock of­fer­ing; As­sem­bly Bio to pause work on one HBV in­hibitor pro­gram

San Francisco-based biotech 89bio announced on Friday that it expects to rake in $275 million on a stock offering. The raise comes after 89bio announced on Tuesday results of a Phase II study showing that its drug was better than placebo at lessening fibrosis without worsening nonalcoholic steatohepatitis, or NASH.

To run a Phase III study, 89bio CEO Rohan Palekar told Endpoints News that the biotech “would need to raise additional capital.” 89bio offered over 16 million shares of its common stock at $16.25 per share, and expects the offering closes on March 28.

Bet­ter Ther­a­peu­tics cuts 35% of staff while await­ing dig­i­tal ther­a­peu­tic ap­proval

Digital therapeutics company Better Therapeutics announced on Thursday that it’s cutting 35% of its staff as it awaits FDA clearance for its first product.

The company, which launched eight years ago, is one of a growing group of companies seeking a digital alternative to traditional medicine. The space saw a record $7.5 billion in investments in 2021, according to Chris Dokomajilar at DealForma, with uses spanning ADHD, PTSD and other indications. However, private insurers have been slow to hop on board.

FDA spells out how can­cer drug de­vel­op­ers can use one tri­al for both ac­cel­er­at­ed and full ap­provals

The FDA’s Oncology Center of Excellence has been a bright spot within the agency in terms of speeding new treatments to patients. That flexibility was on full display this morning as FDA released new draft guidance spelling out exactly how oncology drug developers can fulfill both the accelerated and full approval’s requirements with just a single randomized controlled trial.

While Congress recently passed legislation that will allow FDA to require confirmatory trials to be recruiting and ongoing prior to granting an accelerated approval, the agency is now making clear that the initial trial used to win the AA, if designed appropriately, can also serve as the trial for converting the accelerated approval into a full approval.

Zhi Hong, Brii Biosciences CEO

Brii Bio­sciences stops man­u­fac­tur­ing Covid-19 an­ti­body com­bo, plans to with­draw EUA re­quest

Brii Biosciences said it will stop manufacturing its Covid-19 antibody combination, sold in China, and is working to withdraw its emergency use authorization request in the US, which it started in October 2021.

The Beijing and North Carolina biotech commercially launched the treatment in China last July but is now axing the work and reverting resources to other “high-priority programs,” per a Friday update. The focus now is namely hepatitis B viral infection, postpartum depression and major depressive disorders.

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