Al­ler­gan, Richter steer past an­oth­er late-stage pile­up with cariprazine

Cariprazine’s hit-and-miss record in late-stage de­vel­op­ment has been ex­tend­ed by a sting­ing set­back. In­ves­ti­ga­tors for Al­ler­gan ($AGN) and its part­ner Gedeon Richter say that the drug flopped in a Phase III de­pres­sion study, but will nev­er­the­less go back in­to an­oth­er piv­otal study in search of more promis­ing re­sults.

Per­sis­tence in late-stage work is the on­ly rea­son this drug is still in the spot­light. Back in ear­ly 2015 I cov­ered their small, suc­cess­ful Phase III tri­al for cariprazine in schiz­o­phre­nia, which was good enough for the de­vel­op­ers to send back to the FDA for a lat­er ap­proval (as Vray­lar), even though they had re­ject­ed the drug in 2013. Richter has dogged­ly main­tained its faith in the drug af­ter once pro­ject­ing peak po­ten­tial sales at a block­buster rate of $2 bil­lion a year, which shrinks to $200 mil­lion in the hands of some an­a­lysts.

As the com­pa­nies not­ed in their state­ment this morn­ing, it’s not un­usu­al for a Phase III de­pres­sion study to fail. Alk­er­mes re­cent­ly ran in­to the same is­sue with its first shot at a Phase III. The com­pa­ny failed and saw $4 bil­lion in mar­ket cap dis­solve, though it too be­lieves that a fol­lowup Phase III can still de­liv­er the goods. The track record on Phase III de­pres­sion stud­ies is lit­tered with fail­ure and just the oc­ca­sion­al suc­cess.

Reg­u­la­tors of­ten re­quire sev­er­al late-stage stud­ies for a de­pres­sion drug, large­ly be­cause the place­bo ef­fect can be so strong in a de­pres­sion study it’s the on­ly way to ad­vance new drugs, which of­ten go on to be­come wild­ly hit-and-miss in a ther­a­peu­tic mar­ket that re­quires plen­ty of ex­per­i­men­ta­tion. In­ves­ti­ga­tors point­ed to an­oth­er study (MD-75) in which they say they got a pos­i­tive read­out for de­pres­sion, but this time around the drug arm failed to sep­a­rate from a place­bo, of­fer­ing no more help than a sug­ar pill.

Richter didn’t lose the same per­cent­age of eq­ui­ty as Alk­er­mes, but its stock was still dam­aged on the news.

David Nichol­son, Chief R&D Of­fi­cer, Al­ler­gan

The com­pa­nies are al­so con­duct­ing two Phase III stud­ies for bipo­lar de­pres­sion. Al­ler­gan wound up with rights to the drug af­ter a se­ries of merg­ers, trac­ing its own­er­ship back to a pact by For­est Lab­o­ra­to­ries.

“We are dis­ap­point­ed with the re­sults of this tri­al. How­ev­er, we be­lieve that our plan to move for­ward with an­oth­er Phase III study in (ma­jor de­pres­sion) cou­pled with our pre­vi­ous pos­i­tive clin­i­cal tri­al would pro­vide the two stud­ies need­ed for sub­mis­sion. This is an im­por­tant next step to fur­ther de­vel­op the cariprazine pro­gram,” said David Nichol­son, chief R&D of­fi­cer at Al­ler­gan.

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.

Anthony Fauci, AP Images

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.

UP­DAT­ED: NGM Bio takes leap for­ward in crowd­ed NASH field

South San Francisco-based NGM Bio may have underwhelmed with its interim analysis of a key cohort from a mid-stage NASH study last fall — but stellar topline data unveiled on Monday showed the compound induced significant signs of antifibrotic activity, NASH resolution and liver fat reduction, sending the company’s stock soaring.

There are an estimated 50+ companies focused on developing drugs for non-alcoholic steatohepatitis, or NASH, a common liver disease that has long flummoxed researchers. The first wave of NASH drug developers struggled with efficacy as well as safety — and companies big and small have crashed and burned.

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