Treve­na ham­mered again as the FDA spurns their con­tro­ver­sial pain drug — but Acel­Rx gets a green light

The FDA ev­i­dent­ly man­aged to sur­prise the die-hard sup­port­ers of Treve­na to­day by re­ject­ing their con­tro­ver­sial pain drug oliceri­dine — send­ing their shares $TRVN down 34% in a deep plunge in­to pen­ny stock ter­ri­to­ry.

Ac­cord­ing to the biotech — which gets to out­line what the agency is de­mand­ing ahead of its next re­view — the FDA is de­mand­ing a lot more safe­ty da­ta. That in­cludes more da­ta on QT pro­lon­ga­tion and a big­ger safe­ty data­base for the drug. The “FDA al­so re­quest­ed cer­tain ad­di­tion­al non­clin­i­cal da­ta and val­i­da­tion re­ports.”

Max­ine Gowen, ex-CEO of Treve­na

The FDA wasn’t say­ing no to every new pain drug, though, de­spite its con­cerns about the opi­oid epi­dem­ic that has spurred alarm from coast to coast. Acel­Rx came up with their long-await­ed ap­proval for Dsu­via (ARX-04), dri­ving the stock $ACRX up 16%.

Their drug won a lop­sided pan­el re­view vote, but still drew flak as crit­ics pounced on the in­tro­duc­tion of an­oth­er pow­er­ful pain med that could be ripe for abuse. In a state­ment, FDA chief Scott Got­tlieb coun­tered his crit­ics by not­ing the need for a non-IV opi­oid as well as the de­fense de­part­ment’s de­mand for a ther­a­py that would be ben­e­fi­cial on the bat­tle­field.

Treve­na plans to con­tin­ue to plug along in search of an OK, but the pen­ny stock price in­di­cates the fierce head­winds they face af­ter the agency’s in­ter­nal re­view of­fered a harsh as­sess­ment of the drug’s safe­ty and mixed ef­fi­ca­cy re­sults.

A pan­el re­view fol­lowed up with a vote against ap­proval, of­fer­ing all the writ­ing on the wall need­ed for most an­a­lysts.

Treve­na still faces ac­cu­sa­tions that the com­pa­ny mis­led in­vestors about their in­ter­ac­tions with the FDA over their de­vel­op­ment pro­gram.

At­tor­neys at Bern­stein Lieb­hard ac­cused Treve­na ex­ecs, specif­i­cal­ly ex-CEO Max­ine Gowen — who stepped down a few days ago — of mis­lead­ing in­vestors about an end-of-Phase II meet­ing with the FDA. At the time, they not­ed, Gowen said in a May 2, 2016 re­lease that she was “very pleased” with the out­come of its dis­cus­sions with the FDA about oliceri­dine (TRV130) and that they had reached a gen­er­al agree­ment about the Phase III de­sign. The agency, though, out­lined some deep prob­lems the FDA had with that study in their re­view of the drug.

Grow­ing ac­cep­tance of ac­cel­er­at­ed path­ways for nov­el treat­ments: but does reg­u­la­to­ry ap­proval lead to com­mer­cial suc­cess?

By Mwango Kashoki, MD, MPH, Vice President-Technical, and Richard Macaulay, Senior Director, of Parexel Regulatory & Access

In recent years, we’ve seen a significant uptake in the use of regulatory options by companies looking to accelerate the journey of life-saving drugs to market. In 2018, 73% of the novel drugs approved by the U.S. Federal Drug Administration (FDA) were designated under one or more expedited development program categories (Fast Track, Breakthrough Therapy, Priority Review, and Accelerated Approval).ᶦ

Sanofi out­lines big API plans as coro­n­avirus out­break re­port­ed­ly threat­ens short­age of 150 drugs

As the world becomes increasingly dependant on Asia for the ingredients of its medicines, Sanofi sees business to be done in Europe.

The French drugmaker said it’s creating the world’s second largest active pharmaceutical ingredients (API) manufacturer by spinning out its six current sites into a standalone company: Brindisi (Italy), Frankfurt Chemistry (Germany), Haverhill (UK), St Aubin les Elbeuf (France), Újpest (Hungary) and Vertolaye (France). They have mapped out €1 billion in expected sales by 2022 and 3,100 employees for the new operations headquartered in France.

Bio­gen touts new ev­i­dence from the gene ther­a­py com­pa­ny it wa­gered $800M on

A year ago, Biogen made a big bet on a small gene therapy company. Now they have new evidence one of their therapies could work.

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Anthony Fauci (AP Images)

UP­DAT­ED: NIH-part­nered Mod­er­na ships off its PhI-ready coro­n­avirus vac­cine can­di­date to a sea of un­cer­tain­ty

Off it goes.

Moderna has shipped the first batch of its mRNA vaccine against SARS-CoV-2 from its manufacturing facility in Norwood, Massachusetts, to the National Institute of Allergy and Infectious Diseases in Bethesda, Maryland, for a pioneering Phase I study.

It’s a hectic race against time. In the 42 days since Moderna selected the sequence they would use to develop their vaccine — a record time, no less — the number of confirmed cases around the world has surged astronomically from a few dozen to over 80,000, per WHO and Johns Hopkins estimates.

The candidate that they came up with, mRNA-1273, encodes for a prefusion stabilized form of the spike protein, which gives the virus its crown shape and plays a key role in transmission. The Coalition for Epidemic Preparedness Innovations, the Oslo-based group better known as CEPI, funded the manufacture of this batch.

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In fi­nal re­port, ICER ap­pears to have a change of heart on new acute mi­graine ther­a­pies

ICER appears to have reversed course on the fresh crop of acute migraine therapies.

The cost-effectiveness watchdog in November issued a draft report suggesting that existing generic medicines are more effective and cheaper than Allergan’s December-approved CGRP ubrogepant, Biohaven rival molecule, rimegepant (which is under FDA review), and Lilly’s October-sanctioned lasmiditan, which binds to 5-HT1F receptors.

Bi­cy­cle Ther­a­peu­tics takes Roche's Genen­tech on an up to $2B im­muno-on­col­o­gy ride

Bicycle Therapeutics — which is developing a new class of chemically synthesized drugs designed to be pharmacologically as active as biologics, yet manufactured as small molecules —  has scored another big partner: Roche’s Genentech.

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When drug val­ue as­sess­ment meets re­al-world ev­i­dence: ICER en­lists Ae­tion in pric­ing eval­u­a­tion

In a union of two of the hottest trends in the US biopharma world, ICER is teaming up with a high-profile company to integrate real-world evidence in their assessment of treatment value.

The drug pricing watchdog — formally the Institute for Clinical and Economic Review — said it will utilize Aetion’s evidence platform in “select upcoming assessments” and their new 24-month re-evaluations of drugs granted accelerated approval by the FDA.

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First US Covid-19 tri­als set to get un­der­way in Ne­bras­ka and Wash­ing­ton, backed by NIH

The first US clinical trials on the novel coronavirus are scheduled to get underway next month at the University of Nebraska Medical Center, where American passengers were taken after being evacuated from the Diamond Princess cruise ship, and at the Kaiser Permanente Washington Health Research Institute. Both trials are sponsored by the NIH’s National Institute for Allergy and Infectious Diseases, which has led the US’s medical response to the outbreak.

Mallinck­rodt, once the na­tion’s largest oxy­codone pro­duc­er, an­nounces ten­ta­tive $1.6B set­tle­ment

Three years after it first paid out fines for its role in the US opioid abuse epidemic, Mallinckrodt has announced an agreement-in-principle that will see the company pay out $1.6 billion and place its generics unit in bankruptcy.

The tentative deal would settle hundreds of lawsuits from state and local governments over Mallinckrodt’s role in the epidemic, while also helping address the company’s increasingly mountainous debt. Although Purdue Pharma has drawn the bulk of both public and legal acrimony for opioid sales, documents made public earlier this year showed that Mallinckrodt subsidiary SpecGx, along with the generic subsidiaries of Teva and Endo Pharmaceuticals, accounted for the vast majority of the 76 billion opioid pills distributed from 2006 to 2012. Mallinckrodt was at the top of that list.