→ 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.

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.

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.

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.