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.

 

 

 

Tar­get­ing a Po­ten­tial Vul­ner­a­bil­i­ty of Cer­tain Can­cers with DNA Dam­age Re­sponse

Every individual’s DNA is unique, and because of this, every patient responds differently to disease and treatment. It is astonishing how four tiny building blocks of our DNA – A, T, C, G – dictate our health, disease, and how we age.

The tricky thing about DNA is that it is constantly exposed to damage by sources such as ultraviolet light, certain chemicals, toxins, and even natural biochemical processes inside our cells.¹ If ignored, DNA damage will accumulate in replicating cells, giving rise to mutations that can lead to premature aging, cancer, and other diseases.

Fol­low biotechs go­ing pub­lic with the End­points News IPO Track­er

The Endpoints News team is continuing to track IPO filings for 2021, and we’ve designed a new tracker page for the effort.

Check it out here: Biopharma IPOs 2021 from Endpoints News

You’ll be able to find all the biotechs that have filed and priced so far this year, sortable by quarter and listed by newest first. As of the time of publishing on Feb. 25, there have already been 16 biotechs debuting on Nasdaq so far this year, with an additional four having filed their S-1 paperwork.

Steve Cutler, Icon CEO (Icon)

In the biggest CRO takeover in years, Icon doles out $12B for PRA Health Sci­ences to fo­cus on de­cen­tral­ized clin­i­cal work

Contract research M&A had a healthy run in recent years before recently petering out. But with the market ripe for a big buyout and the Covid-19 pandemic emphasizing the importance of decentralized trials, Wednesday saw a tectonic shift in the CRO world.

Icon, the Dublin-based CRO, will acquire PRA Health Sciences for $12 billion in a move that will shake up the highest rungs of a fragmented market. The merger would combine the 5th- and 6th-largest CROs by 2020 revenue, according to Icon, and the merger will set the newco up to be the second-largest global CRO behind only IQVIA.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 102,000+ biopharma pros reading Endpoints daily — and it's free.

Tom Barnes (Orna)

The mR­NA era is here. MPM be­lieves the fu­ture be­longs to oR­NA — and Big Phar­ma wants a seat at the ta­ble

If the ultra-fast clinical development of Covid-19 vaccines opened the world’s eyes to the promises of messenger RNA, the subsequent delays in supply offered a crash course on the ultra-complex process of producing them. Even before the formulation and fill-finish steps, mRNA is the precious end product from an arduous journey involving enzyme-aided transcription, modification and purification.

For Bristol Myers Squibb, Novartis Institutes for Biomedical Research, Gilead’s Kite and Astellas, it’s time to rethink the way therapeutic RNA is engineered.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 102,000+ biopharma pros reading Endpoints daily — and it's free.

Xen­cor, UCLA to part­ner for new ther­a­peu­tic an­ti­bod­ies; Schrödinger ex­pands part­ner­ship with Google Cloud

Seeking to build on the 20 clinical-stage drug candidates already in its pipeline, California oncology and autoimmune disease-focused biotech Xencor announced on Thursday a partnership with nearby UCLA and the university’s Technology Development Group.

The two will collaborate to develop novel therapeutic antibodies, pairing novel targets proposed by scientists at UCLA with Xencor’s XmAb technology platforms that engineer monoclonal antibodies to create new protein structures. No financial terms of the agreement were disclosed, but any selected drug candidates resulting from the partnership will use a framework of “predefined terms to enter sponsored research agreements and potential license agreements,” the organizations said.

S&P ex­pects steady ero­sion in Big Phar­ma's cred­it pro­file in 2021 as new M&A deals roll in — but don't un­der­es­ti­mate their un­der­ly­ing strength

S&P Global has taken a look at the dominant forces shaping the pharma market and come to the conclusion that there will be more downgrades than upgrades in 2021 — the 8th straight year of steady decline.

But it’s not all bad news. Some things are looking up, and there’s still plenty of money to be made in an industry that enjoys a 30% to 40% profit margin, once you factor in steep R&D expenses.

Tal Zaks, Moderna CMO (AP Photo/Rodrique Ngowi, via still image from video)

CMO Tal Zaks bids Mod­er­na a sur­prise adieu as biotech projects $18.4B in rev­enue, plots post-Covid ex­pan­sion

How do you exit a company after six years in style? Developing one of the most lucrative and life-saving products in pharma history is probably not the worst way to go.

Tal Zaks, Moderna’s CMO since 2015, will leave the mRNA biotech in September, the biotech disclosed in their annual report this morning. The company has already retained the recruitment firm Russell Reynolds to find a replacement.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 102,000+ biopharma pros reading Endpoints daily — and it's free.

Ken Frazier, Merck CEO (Bess Adler/Bloomberg via Getty Images)

UP­DAT­ED: Mer­ck takes a swing at the IL-2 puz­zle­box with a $1.85B play for buzzy Pan­dion and its au­toim­mune hope­fuls

When Roger Perlmutter bid farewell to Merck late last year, the drugmaker perhaps best known now for sales giant Keytruda signaled its intent to take a swing at early-stage novelty with the appointment of discovery head Dean Li. Now, Merck is signing a decent-sized check to bring an IL-2 moonshot into the fold.

Merck will shell out roughly $1.85 billion for Pandion Pharmaceuticals, a biotech hoping to gin up regulatory T cells (Tregs) to treat a range of autoimmune disorders, the drugmaker said Thursday.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 102,000+ biopharma pros reading Endpoints daily — and it's free.

Glax­o­SmithK­line re­thinks strat­e­gy for Covid-19 an­ti­body — not the Vir ones — af­ter tri­al flop. Is there hope in high-risk pa­tients?

In the search for a better Covid-19 therapeutic, GlaxoSmithKline and Vir have partnered up on two antibodies they hope have a chance. GSK is also testing its own in-house antibody, and early results may have shut the door on its widespread use.

A combination of GSK’s monoclonal antibody otilimab plus standard of care couldn’t best standard of care alone in preventing death and respiratory failure in hospitalized Covid-19 patients after 28 days, according to data from the Phase IIa OSCAR study unveiled Thursday.