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.

Grow­ing ac­cep­tance of ac­cel­er­at­ed path­ways for nov­el treat­ments: but does reg­u­la­to­ry ap­proval lead to com­mer­cial suc­cess?

By Mwango Kashoki, MD, MPH, Vice President-Technical, and Richard Macaulay, Senior Director, of Parexel Regulatory & Access

In recent years, we’ve seen a significant uptake in the use of regulatory options by companies looking to accelerate the journey of life-saving drugs to market. In 2018, 73% of the novel drugs approved by the U.S. Federal Drug Administration (FDA) were designated under one or more expedited development program categories (Fast Track, Breakthrough Therapy, Priority Review, and Accelerated Approval).ᶦ

Sanofi out­lines big API plans as coro­n­avirus out­break re­port­ed­ly threat­ens short­age of 150 drugs

As the world becomes increasingly dependant on Asia for the ingredients of its medicines, Sanofi sees business to be done in Europe.

The French drugmaker said it’s creating the world’s second largest active pharmaceutical ingredients (API) manufacturer by spinning out its six current sites into a standalone company: Brindisi (Italy), Frankfurt Chemistry (Germany), Haverhill (UK), St Aubin les Elbeuf (France), Újpest (Hungary) and Vertolaye (France). They have mapped out €1 billion in expected sales by 2022 and 3,100 employees for the new operations headquartered in France.

UP­DAT­ED: NGM Bio takes leap for­ward in crowd­ed NASH field

South San Francisco-based NGM Bio may have underwhelmed with its interim analysis of a key cohort from a mid-stage NASH study last fall — but stellar topline data unveiled on Monday showed the compound induced significant signs of antifibrotic activity, NASH resolution and liver fat reduction, sending the company’s stock soaring.

There are an estimated 50+ companies focused on developing drugs for non-alcoholic steatohepatitis, or NASH, a common liver disease that has long flummoxed researchers. The first wave of NASH drug developers struggled with efficacy as well as safety — and companies big and small have crashed and burned.

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Mickey Kertesz, KidsandArtOrg via YouTube

Soft­Bank's newest, $165M biotech in­vest­ment looks for in­fec­tious traces in the blood

SoftBank has found its newest biotech investment.

The Japanese bank has invested $165 million into Karius, a company that uses blood tests to diagnose infectious diseases, as part of its new Vision Fund 2. The full scope of the new fund has yet to be announced, but the first and newly-beleaguered Vision Fund poured $100 billion into technology companies, including the biotechs Vir Biotechnology and Roivant and the sequencing company 10x Genomics.

Methicillin-resistant Staph aureus (Shutterstock)

FDA grants ‘break­through’ sta­tus to an­tibi­ot­ic al­ter­na­tive as Con­tra­Fect rush­es to join fight against su­per­bug

An experimental drug that promises to be the first anti-infective agent to prove superior to vancomycin — an antibiotic approved in 1958 — has notched the FDA’s “breakthrough” status.

ContraFect said the designation was based on Phase II data in which exebacase was tested against a superbug known as methicillin-resistant Staph aureus, or MRSA. In a subgroup analysis, the clinical responder rate at day 14 was 42.8% higher than that among those treated with standard of care, the company said (p=0.010).

Zhong Nanshan, CGTN via YouTube

Har­vard joins coro­n­avirus fight with $115 mil­lion and a high-pro­file Chi­nese part­ner

For two months, as the novel coronavirus swelled from a few early cases tied to a Wuhan market to a global epidemic, most of the world’s focus and dollars have flowed toward emergency initiatives: building vaccines at a record pace, plucking experimental antivirals out of freezers to see what sticks and immunizing mice for new antibodies.

Now a new and well-funded collaboration between Harvard and a top Chinese research institute will play the long game. In a 5-year, $115 million initiative backed by China Evergrande Group, researchers from the Harvard Medical School, Harvard T.H. Chan School of Public Health and Guangzhou Institute for Respiratory Health will study the virus in an effort to develop therapies against infections by the novel coronavirus, known as SARS–CoV-2, and to prevent new ones.

No­var­tis gets a boost in block­buster mul­ti­ple scle­ro­sis race with Roche

In the first step of what’s likely to be a long and uphill battle for the drugmaker, the FDA has accepted Novartis’s BLA submission for a new multiple sclerosis drug and given it priority review. The PDUFA date for the potential blockbuster drug is in June.

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Juergen Horn

An­i­mal health vet Juer­gen Horn makes new an­ti­body play for pets, rak­ing $15M in Se­ries A haul

Zoetis forked over $85 million in 2017 to acquire Nexvet Biopharma and its pipeline of monoclonal antibodies. Juergen Horn, Nexvet’s former chief product development officer, has now secured $15 million for his own biologic company for animals: Invetx.

Buoyed by emerging advances in gene therapies for humans, scientists have started looking at harnessing the technology for animals setting up companies such as Penn-partnered Scout Bio and George Church-founded Rejuvenate Bio. But akin to Nexvet, Invetx is working on leveraging the time-tested science of monoclonal antibodies to treat chronic diseases that afflict man’s best friend.

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As the coronavirus originating out of Wuhan spreads to South Korea, Italy and Iran, stoking already intense fears of a pandemic, GlaxoSmithKline has found another pair of trusted hands to place its adjuvant system. China’s Clover Biopharmaceuticals will add the adjuvant to its preclinical, protein-based vaccine candidate against SARS-CoV-2.

Clover, which is based in the inland city of Chengdu, boasts of a platform dubbed Trimer-Tag that produces covalently-trimerized fusion proteins. Its candidate, COVID-19 S-Trimer, resembles the viral spike (S)-protein found in the virus.