MyoKar­dia steps clos­er to the launch of a ground­break­ing heart tri­al af­ter find­ing its low-dose start­ing point

MyoKar­dia $MYOK is one step clos­er to launch­ing a piv­otal tri­al for its ge­net­i­cal­ly tar­get­ed lead heart drug.

In the sec­ond part of their two-step Phase II tri­al, the South San Fran­cis­co-based biotech says it land­ed pos­i­tive da­ta for a slate of end­points with their low-dose ap­proach to ob­struc­tive hy­per­trophic car­diomy­opa­thy. That’s en­cour­ag­ing them to drop off the top and bot­tom dos­es from the full scale up and fo­cus on the 5 mg, 10 mg and 15 mg steps in the range.

But there’s a catch. The low-dose 2 mg-step-up-to-5 mg arm did hit goals for most end­points, but at that lev­el their drug mava­camten al­so failed to achieve a sta­tis­ti­cal­ly sig­nif­i­cant in­crease in peak VO2 (ex­er­cise ca­pac­i­ty as mea­sured by an in­crease in oxy­gen con­sump­tion), which the com­pa­ny out­lined ear­li­er as the in­tend­ed pri­ma­ry end­point for their piv­otal study, dubbed EX­PLOR­ER-HCM.

In fact, that was the on­ly end­point that didn’t achieve sta­tis­ti­cal sig­nif­i­cance with a p val­ue of 0.121.

“That is the one mea­sure that did not hit sta­tis­ti­cal sig­nif­i­cance, but that is not sur­pris­ing,” MyoKar­dia CEO Tas­sos Gi­anakakos tells me. The re­searchers want­ed to find the bot­tom of an ef­fec­tive range, and they have plen­ty of proof-of-con­cept da­ta from about 20 pa­tients in both co­horts to con­vince them they have good rea­son to be­lieve it will work when they move to a place­bo-con­trolled reg­is­tra­tion study with a dose that starts out at 5 mg and can rise to 15 mg. They add that you al­so shouldn’t just dis­miss the pos­i­tive trend that they saw at the low dose.

MyoKar­dia is break­ing new ground in this study in many ways, and not just with the first new drug to be stud­ied for this con­di­tion. It’s a small com­pa­ny tak­ing on heart dis­ease, a field dom­i­nat­ed by gi­ants like No­var­tis, which have the means to mount huge, long-run­ning stud­ies that cost a for­tune. But the biotech isn’t tak­ing on mass car­dio con­di­tions — it’s look­ing to break up its re­search pro­gram in­to tight­ly con­tained buck­ets of ge­net­i­cal­ly de­fined pa­tient groups. Its lead drug tar­gets a mu­ta­tion in heart pro­teins which forces the heart to squeeze more, thick­en­ing heart mus­cles and cre­at­ing a cas­cade of ef­fects and symp­toms that can lead to afib­ril­la­tion and death.

So they’ve been look­ing for the Goldilocks for­mu­la­tion that can achieve the right serum con­cen­tra­tions of the drug — per­mit­ting physi­cians to step up from a low dose that may well prove ef­fec­tive for a large chunk of pa­tients be­fore go­ing on to find the right lev­el for the rest, while not over­step­ping their in­tend­ed im­pact on the heart.

MyoKar­dia — which has seen its hot stock more than triple in val­ue since its first read­out from their mid-stage study — had out­lined a time­line for launch­ing the reg­is­tra­tion study be­fore the end of 2017. Now dos­ing is ex­pect­ed to get start­ed in the sec­ond quar­ter.

That’s lat­er than what they had en­vi­sioned last sum­mer, con­cedes the CEO. But the ex­ec­u­tive team wants to take time for two sit-downs with the FDA, fol­low­ing up to make sure reg­u­la­tors are com­fort­able with the da­ta they have, the de­sign of the piv­otal tri­al and the piv­otal goals they’ll be us­ing, stick­ing with peak VO2 for the pri­ma­ry.

MyoKar­dia is hold­ing back from pro­vid­ing a time­line on the read­out from the EX­PLOR­ER study. They want to get the FDA’s sign off and start dos­ing pa­tients, judg­ing how fast they can re­cruit the pa­tients they need. Then they’ll tell the world what to ex­pect.

If they’re right, MyoKar­dia could well be on the way to pi­o­neer­ing some­thing rad­i­cal­ly new — and that’s no easy task.

Im­age: MyoKar­dia CEO Tas­sos Gi­anakakos. MYOKAR­DIA

Eli Lilly CEO David Ricks at the Rose Garden, May 26, 2020 (Evan Vucci/AP Images)

Eli Lil­ly lines up a block­buster deal for Covid-19 an­ti­body, right af­ter it failed a NI­AID tri­al

Two days after Eli Lilly conceded that its antibody bamlanivimab was a flop in hospitalized patients, the US government is preparing to make it a blockbuster.

The pharma giant reported early Wednesday that it struck a deal to supply the feds with 300,000 vials of the drug at a cost of $375 million — once it gets an EUA stamp from the FDA. And once that 2-month supply deal is done, the government has an option on another 650,000 doses on the same terms — which could potentially add another $812 million.

En­her­tu picks up an­oth­er win for As­traZeneca and Dai­ichi Sankyo, join­ing the pri­or­i­ty re­view lane for gas­tric can­cer

Five months after Enhertu received twin breakthrough therapy designations, AstraZeneca and Daiichi Sankyo are one step closer to nabbing another approval for their potential blockbuster drug.

The companies announced Wednesday morning that their billion-dollar antibody-drug conjugate has received priority review for HER2 positive metastatic gastric cancer. Already approved in the US for third-line metastatic breast cancer patients that are HER2 positive, Enhertu’s gastric cancer PDUFA date is scheduled for the first quarter of 2021.

Patrick Soon-Shiong at the JP Morgan Healthcare Conference, Jan. 13, 2020 (David Paul Morris/Bloomberg via Getty Images)

Af­ter falling be­hind the lead­ers, dissed by some ex­perts, biotech show­man Patrick Soon-Sh­iong fi­nal­ly gets his Covid-19 vac­cine ready for a tri­al. But can it live up to the hype?

In January, when dozens of scientists rushed to start making a vaccine for the then-novel coronavirus, they were joined by an unlikely compatriot: Patrick Soon-Shiong, the billionaire doctor most famous for making big, controversial promises on cancer research.

Soon-Shiong had spent the last 4 years on his “Cancer Moonshot,” but part of his project meant buying a small Seattle biotech that specialized in making common-cold vectors, called adenoviruses, to train the immune system. The billionaire had been using those vectors for oncology, but the company had also developed vaccine candidates for H1N1, Lassa fever and other viruses. When the outbreak began, he pivoted.

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Jude Samulski, Marianne De Backer

Bay­er buys a biotech ‘race horse’ with a $4B deal — $2B in cash — aimed at go­ing big in­to gene ther­a­py

In the latest sign that Big Pharma wants a leading place in the push to develop a new generation of cell and gene therapies, Bayer is stepping up today with a $2 billion cash deal to buy out one of the fast-moving pioneers in the field, while adding up to $2 billion more in milestones if the new pharma subsidiary can deliver the goods.

As part of a continuing series of deals engineered by Bayer BD chief Marianne De Backer, the pharma player has snapped up Asklepios, more commonly referred to in more casual fashion as AskBio. And they are paying top dollar for a Research Triangle Park-based company that raised $225 million a little more than a year ago to back the brainchild of Jude Samulski, the gene therapy pioneer out of the University of North Carolina Gene Therapy Center.

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Sci­en­tists warn Amer­i­cans are ex­pect­ing too much from a coro­n­avirus vac­cine

The White House and many Americans have pinned their hopes for defeating the Covid-19 pandemic on a vaccine being developed at “warp speed.” But some scientific experts warn they’re all expecting too much, too soon.

“Everyone thinks COVID-19 will go away with a vaccine,” said William Haseltine, chair and president of Access Health International, a foundation that advocates for affordable care.

Christian Rommel (via Roche)

Bay­er fol­lows R&D deal spree by raid­ing Roche's can­cer group for its new re­search chief

The day after Bayer signed off on a $4 billion deal designed to put the company among the leaders in gene therapy development, the pharma giant has recruited a new chief for its R&D division. And they opted for an expert in the cancer field.

Christian Rommel, Roche’s head of discovery and early-stage oncology development, has been tapped to take over the job. Joerg Moeller, who got the top research post after early- and late-stage development roles were combined 2 years ago, is hitting the exit “to pursue other career opportunities.”

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Albert Bourla, AP

UP­DAT­ED: Where's the Pfiz­er ef­fi­ca­cy read­out? CEO Bourla says 'soon,' but you're go­ing to have to wait for it

Pfizer CEO Albert Bourla had promised repeatedly that the pharma giant would know if its leading Covid-19 vaccine is effective by the end of this month — now just a few days away.

Instead, the company reported early Tuesday that it has yet to conduct any interim efficacy analyses. And it won’t now until sometime next month.

The news was included in a slide for their Q3 report.

In the morning Q3 call with analysts, Bourla says that they expect efficacy data “soon,” but noted that they wouldn’t be able to say anything until all the administrative work was done on the interim, which would take about a week. And he added that Pfizer isn’t going to say anything else about that hot topic until they have the data in hand.

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Charles Baum, Mirati CEO

UP­DAT­ED: Mi­rati plots a march to the FDA for its KRAS G12C drug, breath­ing down Am­gen’s neck with bet­ter da­ta

Mirati Therapeutics $MRTX took another closely-watched step toward a now clearly defined goal to file for an approval for its KRAS G12C cancer drug adagrasib (MRTX849), scoring a higher response rate than the last readout from the class-leading rival at Amgen but still leaving open a raft of important questions about its future.

Following a snapshot of the first handful of responses, where the drug scored a tumor response in 3 of 5 patients with non-small cell lung cancer, the response rate has now slid to 45% among a pooled group of 51 early-stage and Phase II patients, 43% — 6 of 14 — when looking solely at the Phase I/Ib. Those 14 patients had a median treatment duration of 8.2 months, with half still on therapy and 5 of 6 responders still in response.

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No­var­tis CEO Vas Narasimhan signs off on a $231M deal to try some­thing new in the R&D fight against SARS-CoV-2

Patrick Amstutz was baptized by pandemic fire early on.

He and colleagues attended the notorious Cowen conference in early March that included some of the top Biogen execs who helped trigger a superspreader event in Boston. Heading back to his post as CEO of Molecular Partners in Switzerland, the outbreak was sweeping through Italy, triggering near panic in some quarters and creeping into the voices of people he knew, including one friend on the Italian side of the country.

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