CymaBay flash­es pos­i­tive re­sults from the tri­al they have to re­launch

Two weeks af­ter the FDA lift­ed its clin­i­cal hold on their lead drug, CymaBay said it showed pos­i­tive re­sults in an abort­ed Phase III tri­al.

The drug, a small mol­e­cule known as se­ladel­par, had been in de­vel­op­ment for three dif­fer­ent liv­er con­di­tions be­fore an in­de­pen­dent re­view of a NASH study last year showed that it might ac­tu­al­ly be dam­ag­ing pa­tient’s liv­er cells. The FDA slapped a clin­i­cal hold across all three tri­als, on­ly lift­ing it last month when an FDA re­view de­ter­mined that the drug hadn’t caused liv­er dam­age.

Su­jal Shah

That hold meant Su­jal Shah-led CymaBay couldn’t com­plete an in­tend­ed 52-week tri­al in pri­ma­ry bil­iary cholan­gi­tis (PBC), a chron­ic dis­or­der that slow­ly de­stroys a pa­tient’s bile ducts. Still, the biotech said Mon­day that the 12-week and 26-week da­ta they col­lect­ed be­fore the tri­al was halt­ed for the drug were pos­i­tive. That da­ta cov­ered 167 pa­tients out of the 265 pa­tients the tri­al was orig­i­nal­ly sched­uled to en­roll.

 “We couldn’t be hap­pi­er with what we’ve seen in this da­ta set, de­spite the fact that this Phase III study was halt­ed ear­ly,” Shah told End­points News. 

Af­ter 12 weeks, CymaBay said, 78% on the high dose of se­ladel­par had achieved the pri­ma­ry end­point — a com­pos­ite met­ric of liv­er func­tion. That com­pared with 12.5% on place­bo, lead­ing to a p val­ue less than 0.0001.

On a sec­ondary end­point, 27.3% of pa­tients on high dose se­ladel­par achieved nor­mal lev­els of a liv­er en­zyme known as ALP, which is of­ten used as a mark­er of dis­ease. Ze­ro pa­tients on place­bo achieved that end­point.

Shah ar­gued the re­sults look bet­ter than what In­ter­cept saw in its Phase III study for Ocali­va, which was ap­proved for PBC in 2016, al­though CymaBay did not put the ther­a­pies head-to-head in the same tri­al, mak­ing com­par­isons dif­fi­cult.

Un­for­tu­nate­ly for CymaBay, the clin­i­cal hold means the da­ta can’t sup­port an ap­pli­ca­tion for ap­proval, as the tri­al was orig­i­nal­ly in­tend­ed to do. In­stead, the re­sults sim­ply bur­nish the com­pa­ny’s de­ci­sion, an­nounced last month, to restart the study, hop­ing this time to gath­er the 52-week da­ta they need to sup­port an NDA. En­roll­ment for the new tri­al should be­gin at the start of next year, Shah said. He added they’ll even­tu­al­ly look to restart tri­als in pri­ma­ry scle­ros­ing cholan­gi­tis as well.

CymaBay’s stock was up 20%, or $0.74, on the news.

Be­fore the NASH im­plo­sion, CymaBay was one of a hand­ful of com­pa­nies that gath­ered in­vestor in­ter­est for its po­ten­tial to treat the fat­ty liv­er con­di­tion. Their fail­ure, though, was one of a string to hit the field over the last 18 months.

The com­pa­ny hasn’t left NASH en­tire­ly, Shah said, but they have put it on the back­burn­er. He said that al­though the com­pa­ny is well fi­nanced, they’re con­strained by a small staff – they went from around 60 em­ploy­ees to 20 af­ter the tri­al fail­ure – and they be­lieve that a study will ul­ti­mate­ly be treat­ed by com­bi­na­tion ther­a­pies. So they’ll wait un­til a part­ner presents it­self.

“I think the da­ta we had from our Phase II study, par­tic­u­lar­ly on NASH res­o­lu­tion and fi­bro­sis, is quite com­pelling,” Shah said.

Mi­no­ryx and Sper­o­genix ink an ex­clu­sive li­cense agree­ment to de­vel­op and com­mer­cial­ize lerigli­ta­zone in Chi­na

September 23, 2020 – Hong Kong, Beijing, Shanghai (China) and Mataró, Barcelona (Spain)  

Minoryx will receive an upfront and milestone payments of up to $78 million, as well as double digit royalties on annual net sales 

Sperogenix will receive exclusive rights to develop and commercialize leriglitazone for the treatment of X-linked adrenoleukodystrophy (X-ALD), a rare life-threatening neurological condition

FDA chief Stephen Hahn on Capitol Hill earlier this week (Getty Images)

As FDA buck­les un­der the strain of a pan­dem­ic work­load, Trump again ac­cus­es the agency of a po­lit­i­cal hit job

Peter Marks appeared before a virtual SVB Leerink audience yesterday and said that his staff at FDA’s CBER is on the verge of working around the clock. Manufacturing inspections, policy work and sponsor communications have all been pushed down the to-do list so that they can be responsive to Covid-related interactions. And the agency’s objective right now? “To save as many lives as we can,” Marks said, likening the mortality on the current outbreak as equivalent to “a nuclear bomb on a small city.”

Daniel O'Day, Gilead CEO (Kevin Dietsch/UPI/Bloomberg via Getty Images)

Play-by-play of Gilead­'s $21B Im­munomedics buy­out de­tails a fren­zied push — and mints a new biotech bil­lion­aire

Immunomedics had not really been looking for a buyout when the year began. Excited by its BLA for Trodelvy, submitted to the FDA in late 2019, executive chairman Behzad Aghazadeh started off looking for potential licensing deals and zeroed in on four potential partners, including Gilead, following January’s JP Morgan Healthcare Conference in San Francisco. Such talks advanced throughout the year, with discussions advancing to the second round in mid-August.

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The win­dow is wide open as four more biotechs join the go-go IPO class of 2020

It’s another day of hauling cash in the biopharma world as four more IPOs priced Friday and a fifth filed its initial paperwork.

The biggest offering comes from PMV Pharma, an oncology biotech focusing on p53 mutations, which raised $211.8 million after pricing shares at $18 apiece. Prelude Therapeutics, developing PRMT5 inhibitors for rare cancers, was next with a $158 million raise, pricing shares at $19 each. Graybug Vision raised $90 million after pricing at $16 per share for its wet AMD candidates, and breast cancer biotech Greenwich Lifesciences brought up the rear with a small, $7 million raise after pricing shares at $5.75.

J&J of­fers PhI/IIa da­ta show­ing its sin­gle-dose vac­cine can stir up suf­fi­cient im­mune re­sponse

Days after J&J dosed the first participants of its Phase III ENSEMBLE trial, the pharma giant has detailed the early-stage data that gave them confidence in a single-dose regimen.

Testing two dose levels either as a single dose or in a two-dose schedule spaced by 56 days in, the scientists from Janssen, the J&J subsidiary developing its vaccine, reported that the low dose induced a similar immune response as the high dose. The interim Phase I/IIa results were posted in a preprint on medRxiv.

President Donald Trump reacts after signing an executive order following his remarks on his healthcare policies yesterday in Charlotte, North Carolina (Getty Images)

Op-ed: Will phar­ma re­al­ly pay for Trump’s lat­est law­less promise to 33 mil­lion Medicare ben­e­fi­cia­ries? Not like­ly

Sitting atop the executive branch, President Donald Trump is the ultimate authority at the FDA. He can fast track any vaccine to approval himself. If it came to that, of course.

What he can’t do is unilaterally order the legislative branch to loosen the Treasury’s coffers for $6.6 billion. Nor can he command pharmaceutical companies to pay for $200 vouchers sent to 33 million Medicare beneficiaries for prescription drugs before the election.

Pal­la­dio bags $20 mil­lion Se­ries B to top­ple a prob­lem­at­ic kid­ney dis­ease drug

Palladio Biosciences just took one step further in its quest to topple Otsuka’s Samsca with its own — it hopes safer — autosomal dominant polycystic kidney disease (ADPKD) drug.

The Pennsylvania-based biotech announced a $20 million Series B on Friday, which will fund a 10-person Phase III trial of its vasopressin V2 receptor agonist, lixivaptan. CEO Alex Martin expects to read out data in the first half of next year, then launch straight into a larger pivotal Phase III study with about 1,200 participants.

New York governor Andrew Cuomo (AP Images)

An­drew Cuo­mo says New York will un­der­take its own vac­cine re­view process, and wouldn’t rec­om­mend trust­ing the fed­er­al gov­ern­ment

The concerns keep mounting over President Donald Trump’s politicization of the FDA and other federal agencies guiding the development of a safe and effective vaccine. And today, the telegenic New York governor Andrew Cuomo appeared to introduce even more politics into the matter — latest in an ongoing series of incidents that have cast the proudly independent FDA in starkly political terms.

During his daily press conference Cuomo said that the state will review any coronavirus vaccines approved by the federal government, citing a lack of trust in the Trump administration. The announcement comes one day after Trump accused the FDA of making an “extremely political” move in proposing stricter vaccine guidance.

PhII Alzheimer's fail­ure deals new blow to Roche, AC Im­mune — but the tau hy­poth­e­sis is far from dead

The leading anti-tau antibody has failed its first Phase II testing, casting a shadow on a popular target (just trailing amyloid beta) for Alzheimer’s disease.

Roche and AC Immune are quick to acknowledge disappointment in the topline readout, which suggested that semorinemab did not reduce cognitive decline among patients with early Alzheimer’s disease, who are either just starting to have symptoms or have mild manifestations.

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