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.

UP­DAT­ED: The FDA sets a reg­u­la­to­ry speed record, pro­vid­ing a snap OK for Ver­tex's break­through triplet for cys­tic fi­bro­sis

The FDA has approved Vertex’s new triplet for cystic fibrosis at a record-setting speed.

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UP­DAT­ED: Clay Sie­gall’s $614M wa­ger on tu­ca­tinib pays off with solid­ly pos­i­tive piv­otal da­ta and a date with the FDA

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That $335M JV Bay­er set up on CRISPR/Cas9? They’re let­ting the biotech part­ner car­ry on

Bayer committed $300 million to set up a joint venture on CRISPR/Cas9 tech with CRISPR Therapeutics $CRSP. But they’re handing off control now to the smaller biotech while retaining a couple of opt-ins for programs nearing an IND.

Bayer $BAY made much of the fact that they were going all-in on gene editing when they did their deal 3 years ago with CRISPR Therapeutics, which pitched $35 million in on their end. This was the cornerstone of their plan to set up new JVs that could make some serious leap forwards in hot new R&D spaces. Now CRISPR will have full management control of Casebia as they pursue programs in hemophilia, ophthalmology and autoimmune diseases.
Samarth Kulkarni, the CEO at CRISPR, made it sound like a natural progression.

IM­brave150: Roche’s reg­u­la­to­ry crew plans a glob­al roll­out of Tecen­triq com­bo for liv­er can­cer as PhI­II scores a hit

Just weeks after Bristol-Myers Squibb defended its failed pivotal study pitting Opdivo against Nexavar in liver cancer, Roche says it’s beat the frontline challenge with a combination of their PD-L1 Tecentriq with Avastin. And now they’re rolling their regulatory teams in the US, Europe and China in search of a new approval — badly needed to boost a trailing franchise effort.
Given their breakthrough and Big Pharma status as well as the use of two approved drugs, FDA approval may well prove to be something of a formality. And the Chinese have been clear that they want new drugs for liver cancer, where lethal disease rates are particularly high.
Researchers at their big biotech sub, Genentech, say that the combo beat Bayer’s Nexavar on both progression-free survival as well as overall survival — the first advance in this field in more than a decade. We won’t get the breakdown in months of life gained, but it’s a big win for Roche, which has lagged far, far behind Keytruda and Opdivo, the dominant PD-1s that have captured the bulk of the checkpoint market so far.
Researchers recruited hepatocellular carcinoma — the most common form of liver cancer — patients for the IMbrave150 study who weren’t eligible for surgery ahead of any systemic treatment of the disease.
Roche has a fairly low bar to beat, with modest survival benefit for Nexavar, approved for this indication 12 years ago. But they also plan to offer a combo therapy that could have significantly less toxicity, offering patients a much easier treatment regimen.
Cowen’s Steven Scala recently sized up the importance of IMbrave150, noting:

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J&J's block­buster Ste­lara wins US ap­proval for ul­cer­a­tive col­i­tis

J&J’s Stelara, which is set to be in the top ten list of blockbusters come 2025, is now cleared by the FDA for use in ulcerative colitis (UC), an inflammatory disease of the large intestine.

The biologic targets interleukin (IL)-12 and IL-23 cytokines, which are known to play a key role in inflammatory and immune responses. Stelara, which generated about $4.7 billion in the first nine months of 2019, is a key player in the crowded marketplace of drugs to treat autoimmune disorders such as psoriasis, rheumatoid arthritis and Crohn’s disease. AbbVie’s star therapy, Humira, continues to dominate, despite its looming patent cliff in the United States, while others including J&J’s $JNJ own anti-IL23 Tremfya, Lilly’s $LLY anti-IL-17 Taltz and AbbVie’s $ABBV recently approved anti-IL-23 antibody Skyrizi carve out a slice of market share.

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→ Hours before the first federal opioid trial was set to begin, three drug distributors and an opioid manufacturer agreed to a $260 million agreement settlement, the Wall Street Journal was the first to report. The deal — which will see McKesson, Cardinal Health and AmerisourceBergen pay $215 million to Summit and Cuyahoga counties, and Teva deal out $35 million in cash and addiction treatments — does not resolve the pending, nationwide litigation that may result in a settlement worth upwards of $40 billion. Negotiators in that case, brought by 2,300 tribes, counties and cities nationwide and led by several states’ attorneys general, worked through much of Friday without success. Josh Stein, the attorney general for North Carolina, said they were trying to put together a $48 billion deal.

GSK of­floads two vac­cines in $1.1B deal as it works to re­vive the pipeline

GlaxoSmithKline is leaving the deep dark woods and its viruses behind.

GSK has agreed to divest its vaccines for rabies, RabAvert, and tick-born encephalitis vaccine, Encepur, to Bavarian Nordic, part of the company’s broader efforts to narrow its pipeline and focus on oncology and immunology.

The deal is worth up to nearly $1.1 billion, with a $336 million upfront payment. GSK acquired the vaccines from Novartis as part of an exchange for their late-stage oncology programs in 2015 under former chief Sir Andrew Witty.

Pfiz­er gets some en­cour­ag­ing PhI­II news on a fran­chise sav­ior, but is a dos­ing ad­van­tage worth the $295M up­front?

Close to 3 years after Opko tried to defend itself as shares tumbled on the news that its long-acting growth hormone had failed to outperform a placebo, the Pfizer partner $PFE is back. And this time they’re pitching Phase III data that demonstrate their drug is non-inferior — or maybe a tad better — than their well-known but fading standard in the field.
The comparator drug here is Genotropin, which earned a marginal $142 million for Pfizer last year — down 9% from the year before. Approved 24 years ago, biosimilars are now in development that Pfizer would like to stay out in front of. The market leader here is Norditropin, a growth hormone from Novo Nordisk that uses the same basic ingredient as Genotropin, which the Danish company sells with a kid-friendly self-injectable pen. That would also present some big competition if the new therapy from Opko/Pfizer makes it to the market.
The new data, says researchers, underscore that a weekly injection of somatrogon performed as well or slightly better than Genotropin (somatropin) in young children with growth hormone deficiency. Investigators tracked height velocity at 10.12 cm/year, edging out the older drug’s 9.78 cm/year. That 0.33 difference may not prove compelling to payers, though, who have been known to overlook dosing advantages in favor of lower costs.
That message may have weighed on the stock reaction this morning, with a 30%-plus hike $OPK giving way to more marginal gains.
Back in late 2016, Opko had to defend itself against a devastating Phase III setback as their initial late-stage trial failed against a sugar pill. Opko later blamed that setback on outliers in the study, though it wasn’t able to expunge the failure.

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As­traZeneca's Farx­i­ga scores FDA nod to cut risk of hos­pi­tal­iza­tion for heart fail­ure in di­a­bet­ics

While the FDA recently spurned an application to allow AstraZeneca’s blockbuster drug Farxiga for type 1 diabetes that cannot be controlled by insulin, citing safety concerns — the US regulator has endorsed the use of the SGLT2 treatment to reduce the risk of hospitalisation for heart failure in patients with type-2 diabetes and established cardiovascular disease or multiple CV risk factors.