Japan­ese biotech pitch­ing iP­SC-de­rived med for the heart gains $26M; An­dreessen Horowitz leads $4.1M seed fund­ing for AI up­start

→ A long­time re­searcher in car­diac re­gen­er­a­tive med­i­cine has closed a $26 mil­lion Se­ries B to ad­vance his de­vel­op­ment of iP­SC-de­rived car­diomy­ocytes as a treat­ment for heart fail­ure. Kei­ichi Fuku­da con­vinced a num­ber of new in­vestors, in­clud­ing SBI In­vest­ment, JMDC, Gene Tech­no Sci­ence, Nis­say Cap­i­tal and SM­BC Cap­i­tal, to join Astel­las Ven­ture Man­age­ment in back­ing Tokyo-based Heart­seed. The biotech ex­pects to launch its own Phase I/II for the lead drug in HFrEF late next year, while sup­port­ing an­oth­er tri­al for di­lat­ed car­diomy­opa­thy ini­ti­at­ed by Keio Uni­ver­si­ty.

Gen­e­sis Ther­a­peu­tics — a spin­out of Vi­jay Pande’s lab at Stan­ford join­ing the rush of putting AI tech to work on drug dis­cov­ery and de­vel­op­ment — has bagged $4.1 mil­lion in seed fund­ing led by An­dreessen Horowitz with Fe­li­cis Ven­tures as a ma­jor in­vestor.

Gen­e­sis founder Evan Fein­berg told End­points News they dif­fer­en­ti­ate them­selves from oth­er AI com­pa­nies by build­ing a neur­al net­work specif­i­cal­ly tai­lored to­wards bio­phar­ma, rather than one based from im­age pro­cess­ing or oth­er forms of AI.

“For many years, both aca­d­e­mics and com­pa­nies work­ing on ma­chine learn­ing for small mol­e­cule drug dis­cov­ery “cut and past­ed” AI tech­niques from these oth­er do­mains of com­put­er vi­sion and nat­ur­al lan­guage and ap­plied them to chem­istry, and many still do,” He wrote in an email to End­points.  “How­ev­er, in our re­search, we re­al­ized that small mol­e­cule drug can­di­dates, which are formed of atoms and bonds, could be more rich­ly rep­re­sent­ed, and those rep­re­sen­ta­tions could be bet­ter lever­aged, by de­vis­ing neur­al net­works that di­rect­ly re­flect sym­me­tries of na­ture”

→ Af­ter pro­vid­ing an up­date on its on­go­ing Phase II study of vosori­tide in chil­dren with achon­dropla­sia, Bio­Marin Phar­ma­ceu­ti­cal has an­nounced that they’ve sub­mit­ted a mar­ket­ing au­tho­riza­tion ap­pli­ca­tion (MAA) to the EMA for its in­ves­ti­ga­tion­al gene ther­a­py, val­oc­toco­gene rox­a­parvovec, for adults with se­vere he­mo­phil­ia A. The com­pa­ny an­tic­i­pates the re­view to take place in Jan­u­ary 2020 un­der ac­cel­er­at­ed as­sess­ment.

 

 

 

Health­care Dis­par­i­ties and Sick­le Cell Dis­ease

In the complicated U.S. healthcare system, navigating a serious illness such as cancer or heart disease can be remarkably challenging for patients and caregivers. When that illness is classified as a rare disease, those challenges can become even more acute. And when that rare disease occurs in a population that experiences health disparities, such as people with sickle cell disease (SCD) who are primarily Black and Latino, challenges can become almost insurmountable.

The End­points 11: They've got mad mon­ey and huge am­bi­tions. It's time to go big or go home

These days, selecting a group of private biotechs for the Endpoints 11 spotlight begins with a sprint to get ahead of IPOs and the M&A teams at Big Pharma. I’ve had a couple of faceplants earlier this year, watching some of the biotechs on my short list choose a quick leap onto Nasdaq or into the arms of a buyer.

Vividion, you would have been a great pick for the Endpoints 11. I’m sorry I missed you.

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Dave Lennon, former president of Novartis Gene Therapies

So what hap­pened with No­var­tis Gene Ther­a­pies? Here's your an­swer

Over the last couple of days it’s become clear that the gene therapy division at Novartis has quietly undergone a major reorganization. We learned on Monday that Dave Lennon, who had pursued a high-profile role as president of the unit with 1,500 people, had left the pharma giant to take over as CEO of a startup.

Like a lot of the majors, Novartis is an open highway for head hunters, or anyone looking to staff a startup. So that was news but not completely unexpected.

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Jacob Van Naarden (Eli Lilly)

Ex­clu­sives: Eli Lil­ly out to crash the megablock­buster PD-(L)1 par­ty with 'dis­rup­tive' pric­ing; re­veals can­cer biotech buy­out

It’s taken 7 years, but Eli Lilly is promising to finally start hammering the small and affluent PD-(L)1 club with a “disruptive” pricing strategy for their checkpoint therapy allied with China’s Innovent.

Lilly in-licensed global rights to sintilimab a year ago, building on the China alliance they have with Innovent. That cost the pharma giant $200 million in cash upfront, which they plan to capitalize on now with a long-awaited plan to bust up the high-price market in lung cancer and other cancers that have created a market worth tens of billions of dollars.

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Who are the women su­per­charg­ing bio­phar­ma R&D? Nom­i­nate them for this year's spe­cial re­port

The biotech industry has faced repeated calls to diversify its workforce — and in the last year, those calls got a lot louder. Though women account for just under half of all biotech employees around the world, they occupy very few places in C-suites, and even fewer make it to the helm.

Some companies are listening, according to a recent BIO survey which showed that this year’s companies were 2.5 times more likely to have a diversity and inclusion program compared to last year’s sample. But we still have a long way to go. Women represent just 31% of biotech executives, BIO reported. And those numbers are even more stark for women of color.

Blue­bird sends blood dis­or­der drug to FDA for ap­proval; CG On­col­o­gy en­ters col­lab­o­ra­tion with Roche for Tecen­triq

Bluebird bio announced it completed the rolling submission of its BLA to the FDA for betibeglogene autotemcel gene therapy.

The therapy, designed for patients with beta-thalassemia who require regular red blood cell transfusions, was previously granted breakthrough therapy designation for treating transfusion-dependent beta-thalassemia (TDT). If approved, beti-cel will be the first hematopoietic stem cell ex-vivo gene therapy for patients in the US.

FDA+ roundup: Bs­U­FA III ready for show­time, court tells FDA to re-work com­pound­ing plan, new guid­ance up­dates and more

The FDA has now spelled out what exactly will be included in the third iteration of Biosimilar User Fee Act (BsUFA) from 2023 through 2027, which similarly to the prescription drug deal, sets fees that industry has to pay for submitting applications, in exchange for firm timelines that the agency must meet.

This latest deal includes several sweeteners for the biosimilar industry, which has yet to make great strides in the US market, with shorter review timelines for safety labeling updates and updates to add or remove an indication that does not contain efficacy data.

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Jean Bennett (Brent N. Clarke/Invision/AP Images)

Lux­tur­na in­ven­tor Jean Ben­nett starts a new gene ther­a­py com­pa­ny to tack­le rare dis­eases left be­hind by phar­ma, VCs

A few years ago Jean Bennett found herself in a surprising place for a woman who invented the first gene therapy ever approved in the United States: No one, it seemed, wanted her work.

Bennett, who designed and co-developed Luxturna, approved in 2018 for a rare form of blindness, had kept building new gene therapies for eye diseases at her University of Pennsylvania lab. But although the results in animals looked promising, pharma companies and investors kept turning down the pedigreed ophthalmology professor.

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David Meek, new Mirati CEO (Marlene Awaad/Bloomberg via Getty Images)

Fresh off Fer­Gene's melt­down, David Meek takes over at Mi­rati with lead KRAS drug rac­ing to an ap­proval

In the insular world of biotech, a spectacular failure can sometimes stay on any executive’s record for a long time. But for David Meek, the man at the helm of FerGene’s recent implosion, two questionable exits made way for what could be an excellent rebound.

Meek, most recently FerGene’s CEO and a past head at Ipsen, has become CEO at Mirati Therapeutics, taking the reins from founding CEO Charles Baum, who will step over into the role of president and head of R&D, according to a release.