Cheer­ful mid-stage de­pres­sion drug da­ta send Ax­some stock fly­ing

On the same day Sage rolled out $SAGE tremen­dous oral drug da­ta in post­par­tum de­pres­sion, Ax­some Ther­a­peu­tics has an­nounced that its lead ex­per­i­men­tal CNS drug in­duced sta­tis­ti­cal­ly sig­nif­i­cant im­prove­ments in pa­tients with ma­jor de­pres­sive dis­or­der (MDD) in a mid-stage study, prompt­ing the com­pa­ny’s stock $AXSM to sky­rock­et more than 150% in pre-mar­ket trad­ing on Mon­day.

In gen­er­al, tri­als test­ing de­pres­sion drugs are no­to­ri­ous­ly tricky and prone to fail­ure, in ma­jor part due to high rates of the place­bo ef­fect.

The pill AXS-05, which con­sists of a com­bi­na­tion of dex­tromethor­phan (DM) and bupro­pi­on, was test­ed against bupro­pi­on alone in 80 MDD pa­tients for 6 weeks in the mid-stage AS­CEND study. Da­ta showed the com­pa­ny’s drug in­duced a sta­tis­ti­cal­ly sig­nif­i­cant re­duc­tion on the Mont­gomery-Ås­berg de­pres­sion rat­ing scale, av­er­aged over the 6-week treat­ment pe­ri­od ver­sus bupro­pi­on (p<0.001), and its su­pe­ri­or­i­ty over the com­para­tor treat­ment had be­gun to emerge from week 1, cul­mi­nat­ing in sta­tis­ti­cal sig­nif­i­cance in week 2, the com­pa­ny said.

At week 6, AXS-05 demon­strat­ed a 17.2 point re­duc­tion on the scale, com­pared to a 12.1 point re­duc­tion for bupro­pi­on (p=0.013).

Raghu­ram Sel­vara­ju

These da­ta bode well for the pill, which is cur­rent­ly be­ing eval­u­at­ed in a Phase III tri­al — dubbed STRIDE-1 — for treat­ment-re­sis­tant de­pres­sion (TRD), wrote H.C. Wain­wright’s Raghu­ram Sel­vara­ju in a note, rais­ing his prob­a­bil­i­ty of TRD ap­proval from 30% from the pri­or 15%.

We view these da­ta as high­ly en­cour­ag­ing, par­tic­u­lar­ly giv­en the rel­a­tive­ly small size of the tri­al; AXS-05’s clear sep­a­ra­tion from bupro­pi­on un­der­scores the dif­fer­en­ti­at­ed pro­file of Ax­some’s can­di­date…the pos­i­tive ef­fi­ca­cy seen in the AS­CEND study should bol­ster in­vestors’ con­fi­dence in the out­come of STRIDE-1, which could po­si­tion AXS-05 fur­ther as a dif­fer­en­ti­at­ed next-gen­er­a­tion ther­a­peu­tic ap­proach for de­pres­sion.

In the AS­CEND study, AXS-05 was found to be su­pe­ri­or to bupro­pi­on across all sec­ondary end­points, with sta­tis­ti­cal­ly sig­nif­i­cant ef­fects demon­strat­ed on most mea­sures. Over­all, the rate of ad­verse events was sim­i­lar in ei­ther arm, and there were no side-ef­fects of weight gain or sex­u­al dys­func­tion — typ­i­cal­ly ob­served with an­ti­de­pres­sant use — as­so­ci­at­ed with AXS-05.

Topline re­sults from the on­go­ing STRIDE-1 are an­tic­i­pat­ed lat­er this quar­ter. The drug, which has been grant­ed fast track sta­tus by the FDA, is al­so be­ing test­ed as a treat­ment for ag­i­ta­tion as­so­ci­at­ed with Alzheimer’s and smok­ing ces­sa­tion.

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.