Backed by neu­ro en­thu­si­ast Arch, up­start Black­Thorn tack­les some tough tar­gets with $40M round

Mark Cor­ri­g­an

Arch Ven­ture Part­ners has fos­tered a new neu­ro­sciences biotech com­pa­ny that will now fol­low a sim­i­lar trail to the one be­ing blazed by the high-pro­file start­up De­nali. Work­ing with John­son & John­son In­no­va­tion, Al­ti­tude Life Sci­ence Ven­tures, Mer­cury Fund, Alexan­dria Re­al Es­tate Eq­ui­ties as well as an “undis­closed crossover fund,” the VC is un­veil­ing Black­Thorn Ther­a­peu­tics to­day, which will now have a $40 mil­lion Se­ries A to play with as it looks to help re­vive a com­plex and chal­leng­ing field.

Black­Thorn in­tends to tar­get neu­robe­hav­ioral dis­or­ders such as autism spec­trum dis­or­der, ob­ses­sive com­pul­sive dis­or­der, de­pres­sion and schiz­o­phre­nia, fields that de­fied a gen­er­a­tion of Big Phar­ma clin­i­cal ef­forts and saw many of the biggest play­ers like As­traZeneca or Glax­o­SmithK­line ei­ther drop out or scale far back.

Big Phar­ma’s re­treat, though, was fol­lowed by rapid progress in ge­nomics. And Black­Thorn, de­scribed by Ex­ec­u­tive Chair­man Mark Cor­ri­g­an as a “broth­er com­pa­ny to De­nali,” plans to fol­low up on that progress by ad­vanc­ing new tech­nolo­gies that can help iden­ti­fy spe­cif­ic pa­tient pop­u­la­tions that can be helped by spe­cif­ic ther­a­pies. And it’s start­ing out with a well ad­vanced pro­gram, pick­ing up a failed drug from Eli Lil­ly with plans to launch Phase II study.

The treat­ment – a no­ci­ceptin re­cep­tor an­tag­o­nist – is now dubbed BTRX- 246040.

Lil­ly “had put it in a cou­ple of ear­ly Phase II pro­grams,” says Cor­ri­g­an. “It failed in ma­jor de­pres­sive dis­or­ders,” but there were al­so in­trigu­ing re­sults to fol­low up on.

Cor­ri­g­an is care­ful to note, though, that this is just the be­gin­ning of build­ing a se­ri­ous pipeline of new drugs for neu­robe­hav­ioral con­di­tions. The big idea at the com­pa­ny is that they can ad­vance new tools for imag­ing and as­sess­ment that will al­low the biotech to do a much bet­ter job at track­ing the im­pact of a new drug on pa­tients. Done the right way, Black­Thorn ex­pects it can skirt the pit­falls that claimed so many projects be­fore.

At the same time, Cor­ri­g­an says that reg­u­la­tors will be asked to look at these dis­eases and the ways they can be treat­ed dif­fer­ent­ly, adapt­ing to the progress in or­der to avoid see­ing a re­peat of the same mis­takes. But at this stage, he adds, “I think the FDA is open to some of these dis­cus­sions.”

The biotech has a cast of sci­en­tif­ic ad­vis­ers in its cor­ner with an im­pres­sive set of re­sumes. The Scripps team of Ed­ward Roberts, PhD, and Hugh Rosen, MD, PhD, who co-found­ed Re­cep­tos, are on board. And the ad­vi­so­ry group in­cludes Steven Hy­man, MD, di­rec­tor of the Stan­ley Cen­ter for Psy­chi­atric Re­search and core mem­ber at the Broad In­sti­tute of MIT and Har­vard Uni­ver­si­ty, and Matt State, MD, PhD, chair of the UCSF De­part­ment of Psy­chi­a­try and di­rec­tor of the Lan­g­ley Porter Psy­chi­atric In­sti­tute.

For­mer Cel­gene re­search ex­ec Tom Daniel is ad­vis­ing the com­pa­ny.

Cor­ri­g­an says you can ex­pect to see Black­Thorn start adding staffers to the 13-mem­ber team which has al­ready signed on. And there are plans afoot to link up with tech part­ners, much like De­nali has done, as it builds its plat­form. That’s some­thing that Daniel al­so knows some­thing about.

Com­mu­ni­cat­ing the val­ue of pre­ci­sion med­i­cine

By Natasha Cowan, Content Marketing Manager at Blue Latitude Health.
Many stakeholders are confused by novel precision medicines, including patients and healthcare professionals. So, how can industry help them to navigate this complexity?

Precision medicine represents a new paradigm in healthcare. It embodies the shift from treating many patients with the same therapy, to having the tools to identify the best treatment for every patient.

Spe­cial re­port: Twen­ty ex­tra­or­di­nary women in bio­phar­ma R&D who worked their way to the top

What differentiates a woman leader in biopharma R&D from a man?

Not much, except there are fewer of them in senior posts. Data suggest women are not more risk-averse, family-oriented or less confident than their male counterparts — indeed the differences between the two sexes are negligible. But a glance at the top R&D positions in Big Pharma leaves little doubt that upward migration in the executive ranks of biopharma R&D is tough.

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GSK's asth­ma bi­o­log­ic Nu­cala scores in rare blood dis­or­der study

GlaxoSmithKline’s asthma drug Nucala, which received a resounding FDA rejection for use in chronic obstructive pulmonary disease (COPD) last year, has shown promise in a rare blood disorder.

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Mer­ck buys a fledg­ling neu­rode­gen­er­a­tive biotech spawned by an old GSK dis­cov­ery al­liance. What’s up with that?

Avalon Ventures chief Jay Lichter has a well-known yen for drug development programs picked up in academia. And what he found in Haoxing Xu’s lab at the University of Michigan pricked his interest enough to launch one of his umbrella biotechs in San Diego.
Xu’s work laid the foundation for Avalon to launch Calporta, which has been working on finding small molecule agonists of TRPML1 (transient receptor potential cation channel, mucolipin subfamily, member 1) for lysosomal storage disorders. And that pathway, they believe, points to new approaches on major market neurodegenerative diseases like Parkinson’s, ALS and Alzheimer’s.

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FDA slaps a hold on an AML tri­al as Mark­er scraps a fail­ing ovar­i­an can­cer pro­gram, sink­ing shares

The FDA has placed a hold on a Phase II AML trial from the small immuno-oncology biotech Marker Therapeutics. Marker disclosed the issue two weeks after responding to FDA concerns, adding it to the Q3 release Tuesday. The company also announced it was scrapping a Phase II ovarian cancer program it determined was unlikely to succeed.

The agency’s concern centers around two reagents used in manufacturing for their trial for acute myeloid leukemia patients who have received a stem cell transplant. The reagents are from third parties and not present in the final product, Marker said.

Eli Lil­ly-backed biotech grabs $100M to dis­patch an­ti­body-oligonu­cleotide con­ju­gates af­ter mus­cu­lar dy­s­tro­phy

Hold up your hand. Make a fist. Now open it. And again.

If you can do it fully and with ease, then the proteins in your hand are likely working properly. If you can’t then they may not be. In people with myotonic muscular dystrophy, something more atomic is going on.

In those folks, the problem is RNA. Certain base pairs repeat far beyond normal, up to 11,000 superfluous letters in some cases. The extended strands form “clumps.” Proteins misform and can’t function properly. They often allow one movement but not the reverse, a condition called myotonia that gives the dystrophy its name.

As­traZeneca sets stage for mar­ket­ing ap­pli­ca­tion with promis­ing piv­otal lu­pus drug da­ta

After fumbling in its first late-stage lupus study, AstraZeneca disclosed that a second pivotal trial testing its experimental drug, anifrolumab, had met the main goal, in August. Earlier this week, the British drugmaker broke out the numbers from its successful study.

Last year, anifrolumab failed to meet the main goal of diminishing disease activity in the 460-patient TULIP I study, a 52-week trial that tested two doses of the drug versus a placebo. But in the 373-patient TULIP II study, the higher dose (300 mg) was compared to patients given a placebo — patients in both arms were on baseline standard care.

FDA Vas­cepa re­view spot­lights new safe­ty sig­nals, pos­si­ble min­er­al oil spoil­er as Amarin hunts a block­buster ap­proval

An in-house FDA review of Amarin’s Vascepa raises a set of hurdles the biotech will have to clear if the biotech expects to get the long-awaited FDA approval that could set it on a path to superstar status. But it appears that Amarin has survived another potential setback without introducing a major new threat to its prospects.

The stakes don’t get much higher, with analysts saying a win this week for Amarin could lead to billions in new sales — provided the agency stamps it with an OK. And investors liked what they say in the FDA review this morning, bumping the stock $AMRN 17%.

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FDA puts Sol­id Bio’s lead gene ther­a­py pro­gram on hold — again — af­ter an­oth­er pa­tient is hurt by SGT-001

Solid Biosciences continues to be plagued by safety issues.

Close to 18 months after the gene therapy biotech was able to quickly shed an FDA hold on their lead Duchenne muscular dystrophy program for SGT-001, regulators have stepped back in to force another halt after another patient was hit hard by a set of serious adverse events remarkably similar to the first set.

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