Af­ter a bruis­ing turn as Sarep­ta CEO, Chris Garabe­di­an is back in biotech with a new role as god­fa­ther to a bunch of star­tups

Chris Garabe­di­an is back.

Two years af­ter the for­mer Sarep­ta CEO end­ed a tur­bu­lent reign marked by a roller coast­er ride for in­vestors — which is still not over, even af­ter the con­tro­ver­sial FDA OK of eteplirsen — Garabe­di­an has won $15 mil­lion from some loy­al back­ers at Per­cep­tive Ad­vi­sors to es­tab­lish a com­pa­ny that promis­es to guide biotech star­tups through that first crit­i­cal stage of de­vel­op­ment lead­ing to proof-of-con­cept da­ta.

This is brand new and its called Xon­toge­ny. Now that he has the first tranche of a $25 mil­lion com­mit­ment in hand, Garabe­di­an is putting to­geth­er a small team that will be tasked with god­fa­ther­ing new drug de­vel­op­ment for a group of would-be en­tre­pre­neurs who ei­ther can’t or re­al­ly don’t want to go the tra­di­tion­al VC route to fund a biotech start­up.

“I was get­ting a num­ber of calls from en­tre­pre­neurs who had an idea, had their hands on some­thing of val­ue, in­creas­ing­ly look­ing for some­one to part­ner with but with­out a lot of deep in­dus­try ex­pe­ri­ence,” Garabe­di­an tells me. VCs ei­ther weren’t all that in­ter­est­ed or want­ed way too much eq­ui­ty. These were “unique and good op­por­tu­ni­ties that would nev­er see a path­way for­ward un­less they found some­one to help them find a way for­ward.”

For about a half dozen to 10 new star­tups, Garabe­di­an and his team plan to play that role.

At one point, Per­cep­tive’s stake in Sarep­ta amount­ed to one of its largest in­vest­ments. And that gave Garabe­di­an time to get to know CEO Joseph Edel­man and the oth­er ex­ecs at Per­cep­tive, a fund which backs pub­lic biotechs and of­ten plays a crossover role in the lead up to an IPO. But Garabe­di­an stress­es that Xon­toge­ny isn’t a fund. They may be able to put up a few hun­dred thou­sand for seed cash to get things start­ed at a promis­ing new ven­ture, but most of Per­cep­tive’s mon­ey will go to cre­at­ing the team and fund­ing their op­er­a­tion through the start­up pe­ri­od.

The group will shep­herd new drugs through a proof-of-con­cept event, pro­vid­ing the in­fra­struc­ture on in­dus­try spe­cial­ists that each new biotech doesn’t re­al­ly need to start with from scratch. And then af­ter that point, they can con­sid­er what to do next, with all the usu­al choic­es of in­dus­try sale, a ven­ture round or IPO. And they plan to take eq­ui­ty in pay­ment, with Garabe­di­an fill­ing a role as ex­ec­u­tive chair­man or board mem­ber.

“It will be a rel­a­tive­ly small team,” says Garabe­di­an, “like a typ­i­cal se­nior biotech op­er­at­ing team, about 12 with sup­port staff” and ex­per­tise in things like pre­clin­i­cal tox and reg­u­la­to­ry work. And they can play a vir­tu­al role in man­ag­ing a range of star­tups. Garabe­di­an plans to work with aca­d­e­mics as well as some deeply pas­sion­ate dis­ease ad­vo­cates who are look­ing to launch a com­pa­ny around a new drug prospect, per­haps li­censed in. And he ex­cit­ed­ly told me — in gen­er­al terms — about sev­er­al he’s al­ready in talks with.

“What I’ve ob­served,” says Garabe­di­an, who com­plet­ed stints at Gilead and Cel­gene be­fore tak­ing the helm at Sarep­ta, “is that noth­ing has beat­en the val­ue of clin­i­cal proof-of-con­cept da­ta.”

That’s still true to­day, he adds, even with biotech val­u­a­tions run­ning high. He watched Sarep­ta shares shoot up from $45 to $55 a share on Phase II da­ta. To­day, with a drug on the mar­ket, it’s $35.

So what did he learn at Sarep­ta that would help guide him at Xon­toge­ny?

“I think Sarep­ta found it­self and I found my­self at the cen­ter of many things the in­dus­try and the FDA was grap­pling with,” he tells me. Maybe he was too trans­par­ent, he adds, though com­mu­ni­cat­ing with the pa­tient com­mu­ni­ty at every turn al­so pro­vid­ed a foun­da­tion of pas­sion­ate sup­port that was ul­ti­mate­ly crit­i­cal to the FDA ap­proval process. But per­haps the best les­son is that run­ning a pub­lic com­pa­ny is not his best role.

“I can be that pub­lic com­pa­ny CEO,” he says, “but that’s not what dri­ves me. I in­her­it­ed a30-year-old com­pa­ny that need­ed a lot of fix­ing, with min­i­mal fi­nanc­ing, no top tier board or sci­en­tif­ic founders.”

With Xon­toge­ny he can “work with peo­ple who want to work with me.” He can fo­cus on de­vel­op­ment, in­stead of the dis­trac­tions of man­ag­ing a larg­er com­pa­ny in the pub­lic eye.

“A lot of peo­ple love the spot­light,” he says. “I re­al­ly want to fo­cus on de­vel­op­ment. I feel I can beat the in­dus­try av­er­ages.”

Now he has the mon­ey to find out.

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.

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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.

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Hal Barron. GSK

GSK's Hal Bar­ron her­alds their sec­ond pos­i­tive piv­otal 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.

We don’t know what the data are yet, but DREAMM-2 falls on the heels of a promising set of data delivered 5 months ago for DREAMM-1. There investigators noted that complete responses among treatment-resistant patients rose to 15% in the extra year’s worth of data to look over, with a median progression-free survival rate of 12 months, up from 7.9 months reported earlier. The median duration of response was 14.3 months.

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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.

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Why would Am­gen want to buy Alex­ion? An­a­lysts call hot­ly ru­mored takeover un­like­ly, but seize the mo­ment

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Mizuho analyst Salim Syed first lent credence to the report out of the Spanish news outlet Intereconomía, which said Amgen is bidding as much as $200 per share. While the source may be questionable, “the concept of this happening doesn’t sound too crazy to me,” he wrote.

FDA asks why No­var­tis took two months to launch for­mal in­ter­nal probe, af­ter AveX­is flagged da­ta ma­nip­u­la­tion

And the plot thickens. Novartis $NVS officials are reportedly now scrambling to explain to the FDA why it took them two months to open an internal investigation into data discrepancies for their $2.1 million gene-therapy for spinal muscular dystrophy — the world’s most expensive drug.

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Build­ing on suc­cess­ful PD-1 pact, Eli Lil­ly li­cens­es di­a­betes drug to Chi­nese part­ners at In­novent

Eli Lilly is expanding its partnership with China’s Innovent in a deal involving a diabetes drug sitting in its Phase I reserves.

The two companies had jointly developed one of China’s first homegrown PD-1 agents, scoring an approval for Tyvyt (sintilimab) late last year for relapsed/refractory classical Hodgkin’s lymphoma. This time around, Lilly is out-licensing a piece of its diabetes pipeline, a leading franchise that has historically produced the top-selling Trulicity and Humalog.

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.

Trump ad­min­is­tra­tion re­vives bid to get drug list prices on TV ads

The Trump administration is not giving up just yet. On Wednesday, the HHS filed an appeal against a judge’s decision in July to overturn a ruling obligating drug manufacturers to disclose the list price of their therapies in television adverts — hours before it was stipulated to go into effect.

In May, the HHS published a final ruling requiring drugmakers to divulge the wholesale acquisition cost— of a 30-day supply of the drug — in tv ads in a bid to enhance price transparency in the United States. The pharmaceutical industry has vehemently opposed the rule, asserting that list prices are not what a typical patient in the United States pays for treatment — that number is typically determined by the type of (or lack thereof) insurance coverage, deductibles and out-of-pocket costs. Although there is truth to that claim, the move was considered symbolic in the Trump administration’s healthcare agenda to hold drugmakers accountable in a climate where skyrocketing drug prices have incensed Americans on both sides of the aisle.