Sanofi walks away from MyoKar­dia heart drug part­ner­ship, long be­fore key da­ta read­out

Last June, MyoKar­dia launched its keen­ly an­tic­i­pat­ed piv­otal tri­al for its ge­net­i­cal­ly tar­get­ed lead heart drug. Da­ta are ex­pect­ed next year, but part­ner Sanofi is not wait­ing around. On Wednes­day, the San Fran­cis­co-based biotech said its French part­ner had elect­ed to cease their part­ner­ship that was forged in 2014.

The drug, mava­camten, is ex­pect­ed to break new ground in heart dis­ease, a field mo­nop­o­lized by phar­ma ma­jors large­ly due to the long, ar­du­ous and ex­pen­sive tri­als that are com­mon­place in heart drug de­vel­op­ment. Un­like oth­er com­pa­nies fo­cus­ing on com­mon heart dis­or­ders, MyoKar­dia’s lead ex­per­i­men­tal treat­ment is al­so tar­get­ing a so-far un­tapped con­di­tion — ob­struc­tive hy­per­trophic car­diomy­opa­thy — in which a heart pro­tein mu­ta­tion forces the or­gan to squeeze more, thick­en­ing heart mus­cles and cre­at­ing a cas­cade of con­se­quences that can cul­mi­nate in death.

In the sec­ond tranche of the drug’s Phase II tri­al in a small group of pa­tients re­port­ed in March, the com­pa­ny said its low dose ap­proach was large­ly suc­cess­ful across a crop of end­points, ex­cept one for peak VO2 (ex­er­cise ca­pac­i­ty mea­sured by an in­crease in oxy­gen con­sump­tion).

MyoKar­dia’s ap­proach to re­search is to de­vel­op drugs for ge­net­i­cal­ly de­fined pa­tient groups, which is al­so re­flect­ed in its sec­ond pro­gram, MYK-491, un­der de­vel­op­ment for di­lat­ed car­diomy­opa­thy. The Sanofi part­ner­ship inked in 2014 in­volved the de­vel­op­ment of up to three pro­grams through dis­cov­ery and in­to clin­i­cal de­vel­op­ment for the treat­ment of hy­per­trophic car­diomy­opa­thy (HCM) and di­lat­ed car­diomy­opa­thy (DCM).

On Wednes­day, MyoKar­dia said it had re­gained the glob­al rights to all the pro­grams (in­clud­ing mava­camten and MYK-491) un­der the re­search and col­lab­o­ra­tion agree­ment with Sanofi, which will not be ex­tend­ed be­yond the ini­tial re­search term, which end­ed on De­cem­ber 31, 2018. The biotech’s $MYOK shares were down about 8.5% in ear­ly trad­ing.

As part of the deal, MyoKar­dia re­ceived rough­ly $230 mil­lion in fund­ing from Sanofi, and has ad­vanced mava­camten from pre­clin­i­cal de­vel­op­ment in­to a late-stage piv­otal study for the treat­ment of HCM, and MYK-491 from dis­cov­ery to a Phase II proof-of-con­cept study in pa­tients with DCM.

Mean­while, Phase II da­ta for mava­camten in non-ob­struc­tive HCM are ex­pect­ed in the sec­ond half of this year and Phase IIa proof-of-con­cept da­ta for MYK-491 in DCM are ex­pect­ed be­fore the end of 2019.

UP­DAT­ED: Bio­gen pulls the plug on prized IPF drug from $562M+ Stromedix buy­out

One of Biogen’s attempts to branch out has flopped as the biotech scraps a mid-stage program for idiopathic pulmonary fibrosis.

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Warts for the win: Aclar­is' lead drug clears piv­otal study

Aclaris Therapeutics has found a way to get rid of the warts and all.

The company — which earlier this month decided to focus on its arsenal of kinase inhibitors — on Monday unveiled positive data from a pivotal study testing its lead experimental drug for use in common warts.

The drug, A-101, was tested in a 502-patient study called THWART-2 — patients enrolled had one to six warts before qualifying for the trial. Patients either self-administered A-101 topical solution or a vehicle twice a week over a two-month period. A higher proportion of patients on the drug (a potent hydrogen peroxide topical solution) saw their warts disappear at day 60, versus the vehicle (p<0.0001) — meeting the main goal of the study.  Each secondary endpoint also emerged in favor of A-101, the company said.

Deborah Dunsire. Lundbeck

UP­DAT­ED: Deb­o­rah Dun­sire is pay­ing $2B for a chance to leap di­rect­ly in­to a block­buster show­down with a few of the world's biggest phar­ma gi­ants

A year after taking the reins as CEO of Lundbeck, Deborah Dunsire is making a bold bid to beef up the Danish biotech’s portfolio of drugs in what will likely be a direct leap into an intense rivalry with a group of giants now carving up a growing market for new migraine drugs.

Bright and early European time Monday morning the company announced that it will pay up to about $2 billion to buy Alder, a little biotech that is far along the path in developing a quarterly IV formulation of a CGRP drug aimed at cutting back the number of crippling migraines patients experience each month. In a followup call, Dunsire also noted that the company will likely need 200 to 250 reps for this marketing task on both sides of the Atlantic. And analysts were quick to note that the dealmaking at Lundbeck isn’t done, with another $2 billion to $3 billion available for more deals to beef up the pipeline.

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Tower Bridge in London [Shutterstock]

#UK­BIO19: Join GSK’s Hal Bar­ron and a group of top biotech ex­ecs for our 2nd an­nu­al biotech sum­mit in Lon­don

Over the past 10 years I’ve made a point of getting to know the Golden Triangle and the special role the UK biopharma industry plays there in drug development. The concentration of world class research institutes, some of the most accomplished scientists I’ve ever seen at work and a rising tide of global investment cash leaves an impression that there’s much, much more to come as biotech hubs are birthed and nurtured.

Charles Nichols, LSU School of Medicine

Could psy­che­delics tack­le the obe­si­ty cri­sis? A long­time re­searcher in the field says his lat­est mouse study sug­gests po­ten­tial

Psychedelics have experienced a renaissance in recent years amid a torrent of preclinical and clinical research suggesting it might provide a path to treat mood disorders conventional remedies have only scraped at. Now a preclinical trial from a young biotech suggests at least one psychedelic compound has effects beyond the mind, and — if you believe the still very, very early hype — could provide the first single remedy for some of the main complications of obesity.

It’s fi­nal­ly over: Bio­gen, Ei­sai scrap big Alzheimer’s PhI­I­Is af­ter a pre­dictable BACE cat­a­stro­phe rais­es safe­ty fears

Months after analysts and investors called on Biogen and Eisai to scrap their BACE drug for Alzheimer’s and move on in the wake of a string of late-stage failures and rising safety fears, the partners have called it quits. And they said they were dropping the drug — elenbecestat — after the independent monitoring board raised concerns about…safety.

We don’t know exactly what researchers found in this latest catastrophe, but the companies noted in their release that investigators had determined that the drug was flunking the risk/benefit analysis.

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Ac­celeron drops a de­vel­op­ment pro­gram as #2 drug fails to spark func­tion­al ben­e­fits in pa­tients with a rare neu­ro­mus­cu­lar ail­ment

Acceleron is scrapping a muscular dystrophy development program underway for its number 2 drug in the pipeline after pouring over some failed mid-stage secondary data.

Gone is the ACE-083 project in patients with facioscapulohumeral muscular dystrophy. Their drug hit the primary endpoint on building muscle but flopped on key secondaries for functional improvements in patients, which execs felt was vital to the drug’s success.

Scott Gottlieb, AP Images

Scott Got­tlieb has a new board po­si­tion to add to the re­sume — and this one is fo­cused on a fa­vorite sub­ject

Scott Gottlieb has another position to add to his lengthy roster of boards and advisory roles in the wake of his departure from the helm of the FDA.

He’ll be joining the advisory board of FasterCures, a think tank which former junk bond king Michael Milken set up to help drive more drugs to the market, looking to accelerate drug R&D. That’s a subject close to the heart of Gottlieb, who blazed a trail at the FDA focused on hustling up the process. That helped endear him to the industry, making him one of the most popular commissioners in FDA history.

It’s also likely to be a much less controversial post than his board position at Pfizer, which stirred criticism from Democratic presidential candidate Elizabeth Warren.

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Karyopharm lines up $150 mil­lion cash in­jec­tion to back con­tro­ver­sial drug launch

Karyopharm has entered into a royalty agreement worth up to $150 million to back the launch of their multiple myeloma drug — recently approved by the FDA over the objections of a majority of the agency’s outside experts.

The deal with HealthCare Royalty Partners, worth $75 million now and $75 million once certain regulatory and commercial milestones have been reached, will fund the commercialization of Karyopharm’s oral SINE compound Xpovio (selinexor) for patients with multiple myeloma who have already had at least four prior therapies. The money will help Karyopharm as it markets its newly approved drug and pushes through clinical trials testing the drug on refractory multiple myeloma patients with one to three therapies and patients with treatment-resistant diffuse large B-cell lymphoma. It will give Karyopharm a cushion through mid-2021.