Ar­a­digm los­es CEO, CMO and CFO fol­low­ing reg­u­la­to­ry dis­as­ter; Omeros shares go up on pos­i­tive re­sults for blood clot treat­ment

→ As Ar­a­digm $ARDM con­tends with the af­ter­math of a dis­as­trous reg­u­la­to­ry jour­ney, it has let go of three of its top ex­ec­u­tives in an ap­par­ent mea­sure to cut costs. CEO Ig­or Gon­da, CMO Juer­gen Froehlich and CFO Nan­cy Peco­ta have re­signed from all of their po­si­tions at the trou­bled biotech. John Siebert, cur­rent chair­man of the board, will step in as ex­ec­u­tive chair­man and in­ter­im prin­ci­pal ex­ec­u­tive of­fi­cer. If Ar­a­digm wants an­oth­er chance at the FDA for its in­haled an­tibi­ot­ic Lin­haliq, which it in­di­cat­ed it would like to do, it would need fresh cash to fu­el a whole new PhI­II pro­gram.

→ By com­par­ing the per­for­mance of its drug against his­tor­i­cal da­ta in a Phase II study, Omeros has de­clared that OMS721 leads to a sig­nif­i­cant in­crease in me­di­an over­all sur­vival for pa­tients with a type of blood clot as­so­ci­at­ed with stem cell trans­plant. Among a to­tal of 19 hematopoi­et­ic stem cell trans­plant-as­so­ci­at­ed throm­bot­ic mi­croan­giopa­thy (HCT-TMA) pa­tients, the me­di­an OS was 347 days, a big im­prove­ment com­pared to the 21 days record­ed in lit­er­a­ture. The bio­mark­ers al­so looked pos­i­tive to re­searchers. Four deaths oc­curred, dur­ing the study, but the com­pa­ny said on­ly one was pos­si­bly drug-re­lat­ed. Omeros’ shares $OMER edged up 12% in pre-mar­ket trad­ing, slow­ly re­cov­er­ing from a Wednes­day slump that some say were spurred by re­ports that their oth­er drug will lose re­im­burse­ment un­der the new fed­er­al bud­get.

→  Ver­tex Phar­ma $VRTX says its Phase II study of the nono­pi­oid pain drug VX-150 hand­i­ly beat out a place­bo in treat­ing post-sur­gi­cal pain. The suc­cess in Phase II helps clar­i­fy re­searchers’ plans for the next round of stud­ies as it looks to ad­vance a new ap­proach to pain in the mid­dle of an opi­oid ad­dic­tion epi­dem­ic. Their drug clear­ly fell short of a com­para­tor arm of pa­tients giv­en the com­mon­ly pre­scribed opi­oid med­i­cine hy­drocodone+ac­eta­minophen, but may prove good enough as a sub­sti­tute in many cas­es.

→  Switzer­land’s San­thera Phar­ma­ceu­ti­cals (SIX: $SANN) struck a deal with Polyphor to in-li­cense POL6014, a clin­i­cal-stage ther­a­py for cys­tic fi­bro­sis and oth­er neu­trophilic pul­monary dis­eases such as non-cys­tic fi­bro­sis bronchiec­ta­sis, al­pha-1 an­tit­rypsin de­fi­cien­cy and pri­ma­ry cil­iary dysk­i­ne­sia.

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.