Richard Pops, Alkermes CEO (Patrick T. Fallon/Bloomberg via Getty Images)

FDA doc­u­ments raise opi­oid abuse po­ten­tial but pave a path to­ward bad­ly-need­ed ap­proval for Alk­er­mes

Alk­er­mes’ lat­est at­tempt to get an in-house drug past the FDA seems to be go­ing bet­ter than the pre­vi­ous one.

The agency re­leased their as­sess­ment of ALKS-3831, the Irish biotech’s ex­per­i­men­tal drug for schiz­o­phre­nia and bipo­lar I dis­or­der, ahead of an ad­vi­so­ry com­mit­tee hear­ing Fri­day. And while FDA re­view­ers flagged at least one key is­sue, the doc­u­ments over­all point to a far eas­i­er path to ap­proval than Alk­er­mes faced the last time they went to reg­u­la­tors, when the re­jec­tion of a con­tro­ver­sial opi­oid for de­pres­sion helped trig­ger lay­offs and a strate­gic shift at the Richard Pops-led com­pa­ny.

“They read fair­ly be­nign,” Ever­core ISI’s Umer Raf­fat wrote in a note to in­vestors. “This bodes very well for ALKS head­ing in­to the Ad­Com.”

ALKS-3831 be­longed to a long-run­ning Alk­er­mes strat­e­gy to repack­age ex­ist­ing block­buster med­ica­tions in ways that made them safer or more ap­peal­ing. In this case, that meant com­bin­ing the Eli Lil­ly an­ti-psy­chot­ic Zyprexa with an opi­oid an­tag­o­nist in an ef­fort to mit­i­gate the weight gain the cur­rent drug can cause.

Po­ten­tial sales for the drug are mod­er­ate – SVB Leerink’s Marc Good­man pegged peak sales be­tween $250 mil­lion and $300 mil­lion – but an ap­proval would be key for a com­pa­ny whose stock has fall­en 75% over the last two years amid FDA re­jec­tions and mar­ket­ing prac­tices the agency said vi­o­lat­ed stan­dards.

Among the key ques­tions that mix posed for the FDA was what it would mean for pa­tients who used opi­oids. Re­view­ers not­ed that it could lead to with­draw­al in opi­oid-de­pen­dent pa­tients, or block the pain-re­liev­ing ef­fects of opi­oids in set­tings and for peo­ple who need it. It could, they wrote, even lead to over­dose if some­one tried to over­come the ef­fects of the an­tag­o­nist.

Alk­er­mes ex­clud­ed opi­oid users from their tri­als and have pro­posed slap­ping a la­bel that tells doc­tors not to pre­scribe the drug to those tak­ing opi­oids. The FDA, though, point­ed to da­ta that showed oth­er drugs not sup­posed to be giv­en to opi­oid users were in fact pre­scribed to them as much as 11% of the time, “sug­gest­ing that de­spite the pro­posed la­bel­ing in olan­za­p­ine/sami­dor­phan, con­cur­rent use of opi­oids could oc­cur in the pa­tient pop­u­la­tion.”

The FDA didn’t take a de­fin­i­tive stand on whether good la­bel­ing could re­solve these con­cerns, but an­a­lysts saw their dis­cus­sion as en­cour­ag­ing for Alk­er­mes.

The com­ments “were fair­ly bal­anced and not as neg­a­tive as it could have been,” SVB Leerink’s Marc Good­man wrote in a note to in­vestors, adding that the agency ap­pears to see the risk as con­fined to just a sub­set of the pa­tient pop­u­la­tion.

There were two oth­er con­cerns, as well, in­clud­ing whether Alk­er­mes ad­e­quate­ly char­ac­ter­ized the rest of the safe­ty pro­file for the drug. The agency large­ly agreed that there ap­peared to be no new safe­ty is­sues not seen in Zyprexa.

More press­ing, Good­man wrote, was the ques­tion of how the FDA would view the weight gain da­ta. The FDA has clear bench­marks for what war­rants ap­proval of a weight loss drug. Alk­er­mes, though, was not try­ing to show that ALKS-3831 in­duced weight loss but rather that it didn’t cause weight gain, and it was un­clear how the FDA would treat their da­ta.

The FDA ac­knowl­edged that the two goals were dif­fer­ent, telling ad­vi­sors they can use the weight loss stan­dards as a “ref­er­ence” but that it “does not rep­re­sent the stan­dard for es­tab­lish­ing the con­tri­bu­tion of” ALKS-3831. Still, they left open ques­tions about how ben­e­fi­cial the drug was, not­ing that, while it curbed weight gain in stud­ies, it missed on oth­er meta­bol­ic end­points.

The com­mit­tee will con­vene Fri­day to dis­cuss the ques­tion, with an FDA de­ci­sion due by Nov. 15.

Charles Baum, Mirati CEO

Mi­rati plots a march to the FDA for its KRAS G12C drug, breath­ing down Am­gen’s neck with bet­ter da­ta

Mirati Therapeutics $MRTX took another closely-watched step toward a now clearly defined goal to file for an approval for its KRAS G12C cancer drug adagrasib (MRTX849), scoring a higher response rate than the last readout from the class-leading rival at Amgen but still leaving open a raft of important questions about its future.

Following a snapshot of the first handful of responses, where the drug scored a tumor response in 3 of 5 patients with non-small cell lung cancer, the response rate has now slid to 45% among a pooled group of 51 early-stage and Phase II patients, 43% — 6 of 14 — when looking solely at the Phase I/Ib. Those 14 patients had a median treatment duration of 8.2 months, with half still on therapy and 5 of 6 responders still in response.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 92,400+ biopharma pros reading Endpoints daily — and it's free.

In his­toric Covid-19 ad­comm, vac­cine ex­perts de­bate a sea of ques­tions — but of­fer no clear an­swers

The most widely anticipated and perhaps most widely watched meeting in the FDA’s 113-year history ended late Thursday night with a score of questions and very few answers.

For nearly 9 hours, 18 different outside experts listened to public health agencies and foundations present how the United States’ Covid-19 vaccine program developed through October, and they debated where it should go from there: Were companies testing the right metrics in their massive trials? How long should they track patients before declaring a vaccine safe or effective? Should a vaccine, once authorized, be given to the volunteers in the placebo arm of a trial?

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 92,400+ biopharma pros reading Endpoints daily — and it's free.

Pascal Soriot, AstraZeneca CEO (Zach Gibson/Bloomberg via Getty Images)

UP­DAT­ED: FDA gives As­traZeneca the thumbs-up to restart PhI­II Covid-19 vac­cine tri­als, and J&J is prepar­ing to re­sume its study

Several countries had restarted their portions of AstraZeneca’s global Phase III Covid-19 vaccine trial after the study was paused worldwide in early September, but the US notably stayed on the sidelines — until now. Friday afternoon the pharma giant announced the all clear from US regulators. And on top of that, J&J announced Friday evening that it’s preparing to resume its own Phase III vaccine trial.

Stephen Hahn, FDA commissioner (AP Images)

As FDA sets the stage for the first Covid-19 vac­cine EUAs, some big play­ers are ask­ing for a tweak of the guide­lines

Setting the stage for an extraordinary one-day meeting of the Vaccines and Related Biological Products Advisory Committee this Thursday, the FDA has cleared 2 experts of financial conflicts to help beef up the committee. And regulators went on to specify the safety, efficacy and CMC input they’re looking for on EUAs, before they move on to the full BLA approval process.

All of this has already been spelled out to the developers. But the devil is in the details, and it’s clear from the first round of posted responses that some of the top players — including J&J and Pfizer — would like some adjustments and added feedback. And on Thursday, the experts can offer their own thoughts on shaping the first OKs.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 92,400+ biopharma pros reading Endpoints daily — and it's free.

Michel Vounatsos, Biogen CEO (via YouTube)

Bio­gen spot­lights a pair of painful pipeline set­backs as ad­u­canum­ab show­down looms at the FDA

Biogen has flagged a pair of setbacks in the pipeline, spotlighting the final failure for a one-time top MS prospect while scrapping a gene therapy for SMA after the IND was put on hold due to toxicity.

Both failures will raise the stakes even higher on aducanumab, the Alzheimer’s drug that Biogen is betting the ranch on, determined to pursue an FDA OK despite significant skepticism they can make it with mixed results and a reliance on post hoc data mining. And the failures are being reported as Biogen was forced to cut its profit forecast for 2020 as a generic rival started to erode their big franchise drug.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 92,400+ biopharma pros reading Endpoints daily — and it's free.

Dan O'Day, Gilead CEO (Andrew Harnik, AP Images)

UP­DAT­ED: FDA anoints Gilead­'s remde­sivir as the Covid-19 treat­ment win­ner, hand­ing down full ap­proval — de­spite some deep skep­ti­cism

Seven months into the Covid-19 pandemic, the race to develop a treatment for the disease that’s proved to be the biggest health crisis in a century has an officially designated winner: Gilead. And they’re picking up the prize — worth billions in peak sales — despite a major study that concluded the drug was no help in reducing the number of people who die from the virus.

The FDA handed down a thumbs-up for remdesivir, the company announced Thursday afternoon, as the drug becomes the first fully approved treatment for Covid-19 in the US. Remdesivir, to be marketed as Veklury, will come with a label for treatment in adults and children older than 12 in Covid-19 cases that require hospitalization.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 92,400+ biopharma pros reading Endpoints daily — and it's free.

Stephen Hoge, Moderna president (Moderna)

On morn­ing of FDA Covid-19 ad­comm, Mod­er­na com­pletes PhI­II en­roll­ment, putting them neck-and-neck with Pfiz­er

Weeks away from a potential EUA application, Moderna announced they have completed enrollment in their 30,000-person Phase III Covid-19 vaccine trial, with over a third of volunteers non-white and a quarter over the age of 65.

The announcement caps what has been the most closely-watched recruitment race in the history of drug development, as Pfizer and Moderna rushed to get enough volunteers to prove whether or not experimental vaccines could actually protect people from contracting Covid-19. Pfizer reached that mark on Sept. 15. Moderna said around the same time that they would slow down enrollment to ensure they enrolled enough participants from minority and at-risk groups.

Biond­Vax stock im­plodes af­ter a big PhI­II gam­ble for its uni­ver­sal flu vac­cine fails

After flying high on Wall Street for the last few months of a pandemic, BiondVax’s stock and dreams of getting approval for its universal flu vaccine hit the windshield.

The Jerusalem-based biotech announced on Friday that its only clinical candidate, M-001, failed both primary and secondary endpoints in a Phase III study. There was no statistically significant difference in reduction of flu illness and severity between the vaccine and placebo groups, according to the company. The vaccine did prove safe, if ineffective, BiondVax said.

Adrian Gottschalk, Foghorn CEO (Foghorn)

Foghorn hits Nas­daq in $120M de­but as the biotech IPO boom shows no sign of slow­ing

It’s been a record year for biotech IPOs, and the execs at Nasdaq would like nothing better than to see that momentum continue into the first half of next year.

Since January, 72 biotech and biopharma companies have hit Wall Street, according to Nasdaq head of healthcare listings Jordan Saxe, together raising $13.2 billion.

The latest is Flagship’s Foghorn Therapeutics, which priced its shares last night at $16 apiece, the midpoint of a $15 to $17 range. The Cambridge, MA-based biotech — which initially filed for a $100 million raise on Oct. 2 — is netting $120 million from a 7.5 million-share offering. The proceeds will go right into its gene traffic control platform, including two lead preclinical oncology candidates.