Miki Kapoor

Re­al-world da­ta get re­al dol­lars: GV, Bain help in­fuse $100M in­to Ve­r­ana Health as prag­mat­ic tri­al space heats up

As the ap­pli­ca­tion of re­al-world da­ta in clin­i­cal prac­tice and drug de­vel­op­ment be­comes the re­al­i­ty for a grow­ing num­ber of physi­cians as well as drug­mak­ers, GV and a mar­quee syn­di­cate are bet­ting some re­al dol­lars on one San Fran­cis­co play­er.

The crew for­mer­ly known as Google Ven­tures is lead­ing a $100 mil­lion round for Ve­r­ana Health, which has just ac­quired a small­er com­pa­ny spe­cial­iz­ing in large-scale da­ta ar­chi­tec­ture so­lu­tions dubbed PYA An­a­lyt­ics. Bain Cap­i­tal Ven­tures, Cas­din Cap­i­tal and De­fine Ven­tures al­so chipped in.

Ve­r­ana be­gan two years ago fo­cused on two big dis­ease ar­eas: oph­thal­mol­o­gy and neu­rol­o­gy. Part­ner­ing with the Amer­i­can Acad­e­my of Oph­thal­mol­o­gy and the Amer­i­can Acad­e­my of Neu­rol­o­gy, the com­pa­ny agrees to foot the bill for their reg­istries — where physi­cians can both re­search de-iden­ti­fied elec­tron­ic health records da­ta and re­port their own ob­ser­va­tions — in ex­change for ac­cess to the datasets.

“Our dif­fer­en­tia­tor is our close col­lab­o­ra­tion with med­ical as­so­ci­a­tions and physi­cians — and our fo­cus on da­ta depth and our ex­per­tise in spe­cial­ty ar­eas,” CEO Mi­ki Kapoor told End­points News. “When we go deep on a dataset, we’re talk­ing about 70% of the coun­try’s records in oph­thal­mol­o­gy, for ex­am­ple.”

As med­ical record shar­ing pacts be­tween hos­pi­tal net­works and tech gi­ants come un­der scruti­ny, it al­so helps that Ve­r­ana is work­ing with au­thor­i­ta­tive, trust­ed stew­ards of da­ta.

The new fi­nanc­ing al­lows them to ex­pand in­to new ther­a­peu­tic cat­e­gories and in­te­grate imag­ing, ge­nomics and claims da­ta sources, Ve­r­ana said, with new da­ta analy­sis and link­ing ca­pa­bil­i­ties from the PYAA team. Ve­r­ana has al­so signed on with a third large med­ical reg­istry, Kapoor said.

Cur­rent­ly, these da­ta are locked in sep­a­rate repos­i­to­ries, he added, cre­at­ing a kind of siloed in­for­ma­tion sys­tems that have been fre­quent­ly crit­i­cized by oth­ers work­ing on re­al-world ev­i­dence.

While Ve­r­ana bills it­self as a “provider-fo­cused” op­er­a­tion — with ser­vices help­ing doc­tors see ag­gre­gat­ed prac­tice trends and learn about clin­i­cal tri­als — Kr­ish­na Yesh­want, gen­er­al part­ner at GV, not­ed its po­ten­tial to “un­lock deep clin­i­cal in­sights that sup­port the de­vel­op­ment of new treat­ments while in­creas­ing our un­der­stand­ing of how these treat­ments can ben­e­fit pa­tients more broad­ly.”

Kapoor of­fered an il­lus­tra­tion in a blog post:

For ex­am­ple, through the Ax­on Reg­istry, we want to be able to re­search trends in pro­gres­sion and track the im­pact of spe­cif­ic treat­ments over time for con­di­tions such as mul­ti­ple scle­ro­sis, mi­graine and epilep­sy. We aim to im­prove treat­ments and dis­cov­er cures for the one in six peo­ple af­fect­ed by neu­ro­log­ic dis­ease.

Un­like some of its coun­ter­parts, though, Ve­r­ana doesn’t boast about its Big Phar­ma col­lab­o­ra­tions — at least not yet.

But Ve­r­ana does work with a num­ber of life sci­ences and di­ag­nos­tics play­ers, Kapoor said, of­fer­ing ser­vices in four ar­eas: pro­to­col op­ti­miza­tion, pa­tient re­cruit­ment, site en­gage­ment and re­al-world drug per­for­mance. And a fifth, pro­vid­ing reg­u­la­to­ry-grade da­ta for a prag­mat­ic con­trol arm in clin­i­cal tri­als that drug­mak­ers can take to reg­u­la­tors, is in the works — which in­cludes an FDA-part­nered project com­ing in a few months.

“If we were to fol­low the time­lines of our phar­ma cus­tomers, and reg­u­la­tor part­ners, we would be do­ing it soon­er,” he said. “In fact, we would be do­ing it soon­er than we want to. But what we have de­cid­ed is that over the course of this year, 2020, we’re get­ting our­selves ready for a world in which we can ac­tu­al­ly do that. So by the end of this year, I hope to be do­ing prag­mat­ic work.”

PYAA will re­main and grow in Knoxville, TN, it added, while the par­ent com­pa­ny scales up its San Fran­cis­co head­quar­ters and New York of­fice, hir­ing 45 to com­ple­ment the 85-strong work­force.

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.

Lessons for biotech and phar­ma from a doc­tor who chased his own cure

After being struck by a rare disease as a healthy third year medical student, David Fajgenbaum began an arduous journey chasing his own cure. Amidst the hustle of this year’s JP Morgan conference, the digital trials platform Medable partnered with Endpoints Studio to share Dr. Fajgenbaum’s story with the drug development industry.

What follows is an edited transcript of the conversation between Medable CEO Dr. Michelle Longmire and Dr. Fajgenbaum, and it is full of lessons for biotech executives charged with bringing the next generation of medicines to patients.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 72,900+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 72,900+ biopharma pros reading Endpoints daily — and it's free.

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.

As coro­n­avirus out­break reach­es 'tip­ping point,' GSK lends ad­ju­vant tech to Chi­nese part­ner armed with pre­clin­i­cal vac­cine

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.