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The pri­ma­ry failed. The key sec­ondary failed. But this biotech still be­lieves it can win an FDA OK in ALS

Two years after the executive team at BrainStorm Cell Therapeutics decided to back off a controversial attempt to sell their stem cell therapy for ALS under the new ‘Right to Try’ legislation, the biotech is back with the top-line data from Phase III. And the data aren’t good.

Researchers say the drug — with a once-proposed price of $300,000 — failed the primary endpoint as well as the key secondaries on disease progression. But the executive crew still thinks it’s approvable. And in fact, the biotech also insists the FDA is eager to review it.

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Richard Pops, Alkermes CEO (Patrick T. Fallon/Bloomberg via Getty Images)

Alk­er­mes is hand­ed a CRL for ‘3831, mark­ing an­oth­er set­back on the R&D front

Alkermes $ALKS is going to have to wait some more before launching its anti-psychotic combo drug ALKS-3831. The FDA has handed the biotech a CRL, rejecting the application in the company’s latest setback in R&D.

After getting a majority of the outside experts that reviewed the drug for the FDA to provide a thumbs up on approval, the biotech reported today that their manufacturing operations didn’t pass muster during their remote inspection of records — an alternative to a formal survey the agency adopted last summer as a result of the pandemic.

Faheem Hasnain on stage at the Endpoints News / PharmCube #BIIS18 summit in Shanghai (Endpoints News)

Fa­heem Has­nain is go­ing back to run­ning a biotech start­up. And he has his work cut out for him

After founding Gossamer Bio $GOSS and handing the CEO’s reins to his longtime colleague and co-founder Sheila Gujrathi, biotech entrepreneur Faheem Hasnain is stepping back in to run the company at a critical juncture.

Monday morning Gossamer reported that Hasnain has been re-appointed CEO of Gossamer, weeks after their lead drug failed 2 key mid-stage trials. Now it will be up to Hasnain to work through talks with the FDA to see if there’s a path forward for the drug into a Phase III asthma study — and a partner out there in the industry who would like to carry this forward.

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Stephané Bancel (Endpoints at JPM20)

Mod­er­na says its vac­cine is 94.5% ef­fec­tive at pre­vent­ing Covid-19, ap­pears to stop se­vere dis­ease

Seven days ago, no one had any data to prove a Covid-19 vaccine could work. Now, two appear to be effective in large scale trials.

Moderna announced on Monday that its vaccine was 94.5% effective at preventing Covid-19, matching or beating the results Pfizer and their partner BioNTech released last week and potentially giving the world a second key weapon against a virus that is now surging to record levels. Pfizer said their vaccine was “more than 90%” effective.

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Cy­to­ki­net­ics cheers a PhI­II read­out for their heart drug de­vel­oped with Am­gen, but an­a­lysts and in­vestors aren't join­ing the par­ty

Fifteen years after it entered the clinic, Amgen $AMGN and Cytokinetics’ closely watched heart drug popped out the other side, meeting the primary endpoint in a Phase III study. But it missed key secondary endpoints including reduction in cardiovascular death — and Cytokinetics CEO Robert Blum said whether they’ll approach regulators is still up for discussion.

“It seems reasonable to assume that that will occur. But we’ve not made any decisions on that,” Blum told Endpoints News. While the companies celebrated the results at this year’s AHA, Cytokinetics’ stock $CYTK slipped just over 5% before the bell.

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Short­age of mon­keys slowed down Eli Lil­ly's Covid-19 an­ti­body part­ner. And it's a prob­lem for every­one

It may not be surprising that Shanghai Junshi Biosciences has pushed back some clinical trials to prioritize its Eli Lilly-partnered Covid-19 antibody. But what exactly was sucking up their time is almost certain to raise some eyebrows: The company spent months finding enough monkeys on which to test the experimental drug.

Junshi’s plight highlights a serious, if oft-underlooked, challenge that’s beset the whole biomedical research enterprise. Testing a molecule on non-human primates is often a crucial final step before it can be moved into the clinic, but a confluence of factors have resulted in a shortage in both the US and Europe just as drugmakers were scrambling to put their experimental programs through trials at record speed.

Jean Jacques Bienaimé, BioMarin via YouTube

Bio­Marin CEO Bi­en­aimé beats a re­treat from Eu­rope, yank­ing their ap­pli­ca­tion for he­mo­phil­ia A gene ther­a­py

There will be no near-term OK for BioMarin’s hemophilia A gene therapy valrox in Europe.

Already sent back by the FDA to gather more data on durability after researchers flagged a waning response over several years, the EMA had also pinned a request for extended results — though they were willing to take less than the US agency.

The EMA posted a November 4 letter from BioMarin $BMRN saying they were pulling their application — for now — because they wouldn’t have the added data within the time allowed for it.

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In­ter­fer­on be­ta-1a can­di­date for COPD ad­vances to PhI­II to test its po­ten­tial against Covid-19

Another repurposed drug is entering Phase III to see if it passes the test against Covid-19.

In a small Phase II trial, Synairgen says hospitalized Covid-19 patients who took its inhalable interferon beta-1a formulation, SNG001, were twice as likely to recover within 14 days than patients on a placebo. CEO Richard Marsden said the Southampton, UK-based biotech is launching the candidate — which was already in development for COPD and asthma — into a 900-person Phase III study in the coming weeks.

Franz-Werner Haas, CureVac CEO

Pfiz­er’s cel­e­brat­ed Covid-19 vac­cine freezes many peo­ple out — but a ri­val mR­NA team says they thawed out a so­lu­tion

Once the euphoria began to wane a bit following Pfizer’s dramatic unveiling of a 90%-plus effectiveness rate from its pivotal Covid-19 vaccine study earlier this week, less affluent countries and regions around the world began to wonder if they would ever be able to get in line for it.

The vaccine has to be shipped in -70 degrees celsius temps and then used within 5 days of being thawed. For distributors in China, that means building a unique distribution system to deliver the first dose and then a booster. And there are plenty of countries that say they can’t afford to go that route — adding a big obstacle in front of a huge logistical challenge.

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Jim Wilson (WuXi Global Forum at JPM20)

Jim Wil­son of­fers a safer so­lu­tion to some of the in­dus­try's gene ther­a­py woes

Jim Wilson has been warning for a while about the dangers of the viral vectors used in most gene therapies. Two years ago he walked away from Solid Biosciences as he published a paper showing the potential adverse neurological effects of AAV vectors. Last year, he called the field’s progress on vectors “embarrassing.”

Now the gene therapy pioneer, whose tragic 1999 trial showed the deadly risks of the first generation of vectors, is offering a solution. On Wednesday, he and a team of researchers at the University of Pennsylvania published a paper in Science Translational Medicine showing a way of modifying the vectors so they correct genes without hitting a delicate clusters of neurons that, when disturbed, can cause dangerous side effects.

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