Ro­mesh Sub­ra­man­ian births a new biotech with $50M and a plan to steer to­ward the clin­ic with drugs for rare mus­cle dis­eases

The At­las Ven­ture crew has nev­er liked be­ing splashy with mon­ey when it comes to star­tups. They like to get a good, lean team to­geth­er with some seed mon­ey, find an ex­pe­ri­enced helms­man on the bench, gath­er a string of ex­perts to lend ad­vice and of­fer enough cash in the A round — usu­al­ly with syn­di­cate part­ners — to see if they’re re­al­ly on­to some­thing with their lead drug. 

Maybe, the At­las team could even score a pre­clin­i­cal deal, like the one they did with Delinia. Be­cause they are al­ways on the look­out for a great X fac­tor re­turn for an in­vest­ment. The high­er the bet­ter. 10X will get a rous­ing cheer go­ing in this group. A fast 10X-plus works even bet­ter. And if it looks like too long a path to piv­otal da­ta, they’ve been known to bow out as well.

So along those lines, it’s not too sur­pris­ing to see the start­up mod­el Ro­mesh Sub­ra­man­ian is rolling in­to the biotech lane to­day.

Ja­son Rhodes

At­las, For­bion and MPM got to­geth­er to pro­vide the $50 mil­lion launch round, which will al­low the CEO to build up the crew as they build on the pre­clin­i­cal work they’ve been at for the past year or so — cour­tesy of At­las Ven­ture’s in­cu­ba­tion funds.

At­las’ Ja­son Rhodes will be play­ing a key role as ex­ec­u­tive chair­man, help­ing steer a com­pa­ny he co-found­ed.

The biotech is called Dyne Ther­a­peu­tics and they’ve been work­ing on us­ing oligonu­cleotides to de­grade RNA re­spon­si­ble for dis­ease — with a spe­cial fo­cus on mus­cle ail­ments.

Dyne has been de­vel­op­ing its own in-house con­ju­gate tech­nol­o­gy so they can take this ap­proach and care­ful­ly tar­get it to mus­cles. Ze­ro in close enough and you can amp up your dosage and avoid off-tar­get is­sues. 

Their first dis­ease is my­oton­ic dy­s­tro­phy type 1 — or DM1 — a rare, in­her­it­ed ail­ment that caus­es mus­cle weak­ness. The plan is to build a pipeline of ther­a­pies that can kick down gene ex­pres­sion for rare, mono­genic neu­ro­mus­cu­lar dis­eases, start­ing with skele­tal, car­diac and smooth mus­cle. And they want to stay fo­cused on break­through ther­a­pies.

“Fifty mil­lion takes us to the clin­ic,” says Sub­ra­man­ian. He’s not shar­ing any time­lines with me — not un­usu­al in a start­up’s ear­ly days. But with these back­ers, clear, track­able progress is baked in­to every­thing they do.

This is all fa­mil­iar ter­ri­to­ry for Sub­ra­man­ian, a well-known fig­ure in biotech, who did a stint as a se­nior sci­en­tist at Pfiz­er with Art Krieg be­fore mov­ing on to co-found RaNA and then set up trans­la­tion­al re­search groups for rare dis­eases at Alex­ion — up un­til Lud­wig Hantson’s big purge in the fall of 2017.

With­in months, he was build­ing the new com­pa­ny at At­las. These days, you see one door close, you po­ten­tial­ly get to have your pick of doors. 


Left to right: Sud­hir Agraw­al, Charles Thorn­ton, Louis Kunkel, Nan­cy An­drews.

Here’s their im­pres­sive list of sci­en­tif­ic ad­vis­ers help­ing Dyne stay on track:

— Nan­cy An­drews, for­mer dean of the School of Med­i­cine at Duke Uni­ver­si­ty and No­var­tis board mem­ber.

— Louis Kunkel, mem­ber of the Di­vi­sion of Ge­net­ics and Ge­nomics at Boston Chil­dren’s Hos­pi­tal and pro­fes­sor of pe­di­atrics and ge­net­ics at Har­vard Med­ical School.

— Charles Thorn­ton, Saun­ders Dis­tin­guished Pro­fes­sor of Neu­ro­mus­cu­lar Re­search at the Uni­ver­si­ty of Rochester.

— Sud­hir Agraw­al, vis­it­ing pro­fes­sor in the De­part­ment of Med­i­cine at The Uni­ver­si­ty of Mass­a­chu­setts Med­ical School and founder of Idera Phar­ma­ceu­ti­cals.

Martin Shkreli [via Getty]

Pris­on­er #87850-053 does not get to add drug de­vel­op­er to his list of cred­its

Just days after Retrophin shed its last ties to founder Martin Shkreli, the biotech is reporting that the lead drug he co-invented flopped in a pivotal trial. Fosmetpantotenate flunked both the primary and key secondary endpoints in a placebo-controlled trial for a rare disease called pantothenate kinase-associated neurodegeneration, or PKAN.

Endpoints News

Basic subscription required

Unlock this story instantly and join 57,900+ biopharma pros reading Endpoints daily — and it's free.

Hal Barron. GSK

GSK's Hal Bar­ron her­alds their sec­ond pos­i­tive PhI­II for cru­cial an­ti-BC­MA ther­a­py, point­ing to a push for quick OKs in a crowd­ed field

Hal Barron has his second positive round of Phase III data in hand for his anti-BCMA antibody drug conjugate belantamab mafodotin (GSK2857916). And GSK’s research chief says the data paves the way for their drive in search of an FDA approval for treating multiple myeloma. 

It’s hard to overestimate the importance of this drug for GSK, a cornerstone of Barron’s campaign to make a dramatic impact on the oncology market and provide some long-lost excitement for the pharma giant’s pipeline. They’re putting this BCMA program at the front of that charge — looking to lead a host of rivals all aimed at the same target.

UP­DAT­ED: An em­bold­ened As­traZeneca splurges $95M on a pri­or­i­ty re­view vouch­er. Where do they need the FDA to hus­tle up?

AstraZeneca is in a hurry.

We learned this morning that the pharma giant — not known as a big spender, until recently — forked over $95 million to get its hands on a priority review voucher from Sobi, otherwise known as Swedish Orphan Biovitrum.

That marks another step down on price for a PRV, which allows the holder to slash 4 months off of any FDA review time.

Endpoints News

Basic subscription required

Unlock this story instantly and join 57,900+ biopharma pros reading Endpoints daily — and it's free.

We­bi­nar: Re­al World End­points — the brave new world com­ing in build­ing fran­chise ther­a­pies

Several biopharma companies have been working on expanding drug labels through the use of real world endpoints, combing through the data to find evidence of a drug’s efficacy for particular indications. But we’ve just begun. Real World Evidence is becoming an important part of every clinical development plan, in the soup-through-nuts approach used in building franchises.

I’ve recruited a panel of 3 top experts in the field — the first in a series of premium webinars — to look at the practical realities governing what can be done today, and where this is headed over the next few years, at the prodding of the FDA.

ZHEN SU — Merck Serono’s Senior Vice President and Global Head of Oncology
ELLIOTT LEVY — Amgen’s Senior Vice President of Global Development
CHRIS BOSHOFF — Pfizer Oncology’s Chief Development Officer

A premium subscription to Endpoints News is required to attend this webinar. Please upgrade to either an Insider or Enterprise plan for access. Already have Endpoints Premium? Please sign-in below. You can contact our Subscriptions team at help@endpointsnews.com with any issues.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Brian Kaspar. AveXis via Twitter

AveX­is sci­en­tif­ic founder fires back at No­var­tis CEO Vas Narasimhan, 'cat­e­gor­i­cal­ly de­nies any wrong­do­ing'

Brian Kaspar’s head was among the first to roll at Novartis after company execs became aware of the fact that manipulated data had been included in its application for Zolgensma, now the world’s most expensive therapy.

But in his first public response, the scientific founder at AveXis — acquired by Novartis for $8.7 billion — is firing back. And he says that not only was he not involved in any wrongdoing, he’s ready to defend his name as needed.

I reached out to Brian Kaspar after Novartis put out word that he and his brother Allen had been axed in mid-May, two months after the company became aware of the allegations related to manipulated data. His response came back through his attorneys.

Endpoints News

Basic subscription required

Unlock this story instantly and join 57,900+ biopharma pros reading Endpoints daily — and it's free.

Bob Smith, Pfizer

Pfiz­er is mak­ing a $500M state­ment to­day: Here’s how you be­come a lead play­er in the boom­ing gene ther­a­py sec­tor

Three years ago, Pfizer anted up $150 million in cash to buy Bamboo Therapeutics in Chapel Hill, NC as it cautiously stuck a toe in the small gene therapy pool of research and development.

Company execs followed up a year later with a $100 million expansion of the manufacturing operations they picked up in that deal for the UNC spinout, which came with $495 million in milestones.

And now they’re really going for it.

Endpoints News

Basic subscription required

Unlock this story instantly and join 57,900+ biopharma pros reading Endpoints daily — and it's free.

Video: Putting the AI in R&D — with Badhri Srini­vasan, Tony Wood, Rosana Kapeller, Hugo Ceule­mans, Saurabh Sa­ha and Shoibal Dat­ta

During BIO this year, I had a chance to moderate a panel among some of the top tech experts in biopharma on their real-world use of artificial intelligence in R&D. There’s been a lot said about the potential of AI, but I wanted to explore more about what some of the larger players are actually doing with this technology today, and how they see it advancing in the future. It was a fascinating exchange, which you can see here. The transcript has been edited for brevity and clarity. — John Carroll

Am­gen, Al­ler­gan biosim­i­lar of Roche's block­buster Rit­ux­an clears an­oth­er US piv­otal study 

Novartis $NVS may have given up, but Amgen $AMGN and Allergan $AGN are plowing ahead with their knockoff of Roche’s blockbuster biologic Rituxan in the United States.

Their copycat, ABP 798, was found to have a clinically equivalent impact as Rituxan — meeting the main goal of the study involving CD20-positive B-cell non-Hodgkin’s lymphoma patients. This is the second trial supporting the profile of the biosimilar. In January, it came through with positive PK results in patients with rheumatoid arthritis.

BeiGene and Mus­tang nail down spe­cial FDA sta­tus for top drugs; Roche bags added cov­er­age for Hem­li­bra

→ BeiGene $BGNE is getting a boost in its drive to field a rival to Imbruvica. The FDA has offered an accelerated review to zanubrutinib, a BTK inhibitor that has posted positive results for mantle cell lymphoma. The PDUFA date lands on February 27, 2020. The drug scored breakthrough status at the beginning of the year.

→ BeiGene isn’t the only biopharma company to gain special regulatory status today. Mustang Bio $MBIO and St. Jude Children’s Research Hospital announced that MB-107, a lentiviral gene therapy for the treatment of X-linked severe combined immunodeficiency, also known as bubble boy disease, has been granted Regenerative Medicine Advanced Therapy status.