A bet­ter ap­proach to growth fac­tors? Schol­ar Rock rais­es $47M on SMA promise

Schol­ar Rock, a biotech based of out Cam­bridge, MA, just got a $47 mil­lion in­fu­sion of cash to de­vel­op what the com­pa­ny con­sid­ers smarter, next-gen­er­a­tion growth fac­tor drugs. Their ap­proach in­volves more tar­get­ed tweak­ing than pre­de­ces­sors in the field, giv­ing pa­tients the up­side with­out some un­want­ed side ef­fects.

That’s the hope, at least, of Schol­ar Rock’s pres­i­dent and CEO Nagesh Ma­han­thap­pa. He tells me the com­pa­ny is tak­ing on spinal mus­cu­lar at­ro­phy (SMA) first, a rare neu­ro­mus­cu­lar dis­ease char­ac­ter­ized by a loss of mo­tor neu­rons and pro­gres­sive mus­cle wast­ing (of­ten lead­ing to ear­ly death).

The field al­ready has some ma­jor play­ers, in­clud­ing Bio­gen’s block­buster Spin­raza. But Ma­han­thap­pa says Schol­ar Rock’s new drug doesn’t com­pete with Spin­raza (ap­proved to treat SMA in 2016), as they’re com­ing at SMA from a dif­fer­ent an­gle.

“[Spin­raza] ad­dress­es the pri­ma­ry is­sue with SMA — the loss of mo­tor neu­rons in the spinal cord,” Ma­han­thap­pa said. “But they’re not ad­dress­ing the at­ro­phy and mus­cle weak­ness that is a con­se­quence of that loss of mo­tor neu­rons.”

That’s where Schol­ar Rock’s lead pro­gram, SRK-015, comes in. This pro­gram aims to boost mus­cle mass and strength in SMA pa­tients by in­hibit­ing the growth fac­tor myo­statin, which breaks down mus­cle mass in the body.

Myo­statin has been stud­ied for decades for its role in mus­cle at­ro­phy, but most com­pa­nies (in­clud­ing Roche/Bio­gen and Ac­celeron Phar­ma) are tar­get­ing ac­tive myo­statin. Schol­ar Rock, how­ev­er, is tar­get­ing la­tent or in­ac­tive myo­statin. By seek­ing out myo­statin be­fore it’s ac­ti­vat­ed, Ma­han­thap­pa said it’s eas­i­er to iden­ti­fy — and dif­fer­en­ti­ate — the growth fac­tor from oth­er growth fac­tors. In turn, Schol­ar Rock can be more se­lec­tive in its mod­u­la­tion, in the­o­ry pre­vent­ing the un­want­ed de­vel­op­men­tal com­pli­ca­tions that re­sult from sys­temic treat­ments that ac­ci­den­tal­ly tweak growth fac­tors un­re­lat­ed to myo­statin.

Ma­han­thap­pa de­scribes the two ap­proach­es this way: “Imag­ine you’re in your home, and you want to turn off the lights in one room. You can do that in two ways, you could turn off the switch in that room or you could cut off the elec­tric­i­ty in the en­tire house. First gen­er­a­tion drugs do the lat­ter, while Schol­ar Rock is at­tempt­ing the for­mer.”

The new cash, a Se­ries C round, will al­low Schol­ar Rock to take SRK-015 in­to the clin­ic in the first half of 2018. The fi­nanc­ing was led by new in­vestor In­vus, with par­tic­i­pa­tion of new in­vestor Red­mile Group and all ex­ist­ing in­vestors. So far, Schol­ar Rock has raised $109 mil­lion in eq­ui­ty fund­ing to­tal, in­clud­ing con­tri­bu­tions from Po­laris Part­ners, Tim­o­thy Springer, ARCH Ven­ture Part­ners, EcoR1 Cap­i­tal, The Kraft Group, Fi­deli­ty Man­age­ment and Re­search Com­pa­ny, and Cor­morant As­set Man­age­ment.

Schol­ar Rock’s plat­form tech­nol­o­gy, which har­ness­es supra­cel­lu­lar ac­ti­va­tion, is be­ing ap­plied in sev­er­al ar­eas oth­er than SMA, in­clud­ing fi­bro­sis, im­muno-on­col­o­gy, and ane­mias as­so­ci­at­ed with iron re­stric­tion.

At the In­flec­tion Point for the Next Gen­er­a­tion of Can­cer Im­munother­a­py

While oncology researchers have long pursued the potential of cellular immunotherapies for the treatment of cancer, it was unclear whether these therapies would ever reach patients due to the complexity of manufacturing and costs of development. Fortunately, the recent successful development and regulatory approval of chimeric antigen receptor-engineered T (CAR-T) cells have demonstrated the significant benefit of these therapies to patients.

All about Omi­cron; We need more Covid an­tivi­rals; GSK snags Pfiz­er’s vac­cine ex­ec; Janet Wood­cock’s fu­ture at FDA; and more

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Usama Malik

Ex-Im­munomedics CFO charged with in­sid­er trad­ing, faces up to 20 years in prison af­ter al­leged­ly tip­ping off girl­friend and rel­a­tives of a PhI­II suc­cess

The former CFO of Immunomedics, who helped steer the company to its $21 billion buyout by Gilead last year, has been charged with insider trading, the Department of Justice announced Thursday.

Usama Malik tipped off his then-girlfriend and four others that a Phase III study for Trodelvy would be stopped early four days before Immunomedics publicly announced the result in April 2020, DoJ alleged in its complaint. The individuals then purchased Immunomedics shares, selling them after the news broke and Immunomedics’ stock price doubled.

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Merck's new antiviral molnupiravir (Quality Stock Arts / Shutterstock)

As Omi­cron spread looms, oral an­tivi­rals ap­pear to be one of the best de­fens­es — now we just need more

After South African scientists reported a new Covid-19 variant — dubbed Omicron by the WHO — scientists became concerned about how effective vaccines and monoclonal antibodies might be against it, which has more than 30 mutations in the spike protein.

“I think it is super worrisome,” Dartmouth professor and Adagio co-founder and CEO Tillman Gerngross told Endpoints News this weekend. Moderna CEO Stéphane Bancel echoed similar concerns, telling the Financial Times that experts warned him, “This is not going to be good.”

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Ab­b­Vie tacks on a new warn­ing to Rin­voq la­bel as safe­ty frets crimp JAK class

The safety problems that continue to plague the JAK class as new data highlight some severe side effects are casting a large shadow over AbbVie’s Rinvoq.

As a result of a recent readout highlighting major adverse cardiac events (MACE), malignancy, mortality and thrombosis with Xeljanz a couple of months ago, AbbVie put out a notice late Friday afternoon that it is adding the new class risks to its label for their rival drug.

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Biospec­i­men M&A: Dis­cov­ery ac­quires Al­bert Li's he­pa­to­cyte project; PhI­II tri­al on Bay­er's Nube­qa reached pri­ma­ry end­point

Discovery Life Sciences has acquired what claims to be the Maryland-based host of the world’s largest hepatocyte inventory, known as IVAL, to help researchers select more effective and safer drug candidates in the future.

The combined companies will now serve a wider range of drug research and development scientists, according to Albert Li, who founded IVAL in 2004 and is set to join the Discovery leadership team as the CSO of pharmacology and toxicology.

Pfiz­er, Am­gen and Janssen seek fur­ther clar­i­ty on FDA's new ben­e­fit-risk guid­ance

Three top biopharma companies are seeking more details from the FDA on how the agency conducts its benefit-risk assessments for new drugs and biologics.

While Pfizer, Amgen and Janssen praised the agency for further spelling out its thinking on the subject in a new draft guidance, including a discussion of patient experience data as part of the assessment, the companies said the FDA could’ve included more specifics in the 20-page draft document.

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Re­searchers move clos­er to de­ci­pher­ing blood clots from As­traZeneca, J&J's Covid-19 vac­cines

Researchers may be nearing an answer for the mysterious and life-threatening blood clots that appeared on very rare occasions in people who received the J&J or AstraZeneca Covid-19 vaccine.

The new work builds on an early hypothesis researchers in Norway put forward last spring, when the cases first cropped up. They proposed the events were similar to blood clots that can occur in a small subset of patients who receive heparin, one of the most commonly used blood thinners.

Janet Woodcock (AP Images)

Janet Wood­cock plots her fu­ture at FDA, with se­nior ad­vi­sor role to fall back on if Califf wins con­fir­ma­tion

Acting FDA commissioner Janet Woodcock has been the face of just about every drug approval decision at the agency since the turn of the century. Since the pandemic began, she’s moved between the top of the drugs center to the head of therapeutics at Operation Warp Speed, leading the drive for work on Covid-targeted mAbs and antivirals.

Looking forward — and pending a quick Senate confirmation to cement Rob Califf’s return to the top of FDA early next year — Woodcock’s role at the agency will again be in flux.

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