Shkre­li out, Cuban in: Twit­ter proves es­sen­tial for biotech rebel Ethan Perl­stein

Im­age: Ethan Perl­stein


En­tre­pre­neurs in the tech world of­ten tell fundrais­ing founders to look to Twit­ter for mak­ing con­nec­tions with in­vestors. It’s less com­mon to see biotech in­vest­ments arise from wit­ty tweets.

Not true for Ethan Perl­stein, the founder of a small biotech in San Fran­cis­co called Per­lara. To­day, he shared de­tails of a ran­dom Twit­ter en­counter with celebri­ty in­vestor Mark Cuban that led to Cuban’s fund Rad­i­cal In­vest­ments chip­ping in $250,000 to Per­lara’s $7.4 mil­lion Se­ries A round.

Cuban’s con­tri­bu­tion was the re­sult of a con­ver­sa­tion on Twit­ter with Perl­stein, who’s quite ac­tive on the bio­phar­ma twit­ter­sphere. As Perl­stein re­calls, he had teased the Shark Tank in­vestor for Cuban’s tweet about drug pric­ing dur­ing the de­bate over Spin­raza’s price tag.

The con­ver­sa­tion turned se­ri­ous af­ter Perl­stein shared more de­tails about ear­ly-stage drug dis­cov­ery in bio­phar­ma.

“His tweet in­di­cat­ed that he hadn’t spent a lot of time think­ing about the ins and outs of bio­phar­ma and tech trans­fer,” Perl­stein said. “The next thing I know, I get a mes­sage from him ask­ing for my pitch deck. I was like, ‘is this a joke?’”

A month lat­er, Cuban’s in­vest­ment firm was on board. Ac­cord­ing to Cuban, he likes the idea of sup­port­ing drug dis­cov­ery for rare dis­ease.

“I want to see more peo­ple helped by or­phan drugs,” Cuban said in an email.

The Mar­tin Shkre­li dol­lars

But this wasn’t the first time a Twit­ter con­ver­sa­tion led to an in­vest­ment for Perl­stein’s com­pa­ny. Be­fore Mar­tin Shkre­li’s pub­lic flay­ing (and felony con­vic­tion), Perl­stein and Shkre­li were al­so ex­chang­ing tweets.

“I wasn’t ask­ing peo­ple for in­vest­ment on Twit­ter or any­thing, I was just com­ment­ing on sci­ence for rare dis­ease,” Perl­stein said. “It caught Mar­tin’s at­ten­tion.”

Af­ter the Twit­ter con­ver­sa­tion, Shkre­li in­vest­ed in Per­lara and was in­volved with the com­pa­ny for a short time. But Perl­stein said he asked Shkre­li to ex­tri­cate him­self from the busi­ness in ear­ly 2016.

“I asked him to be bought out by oth­er share­hold­ers,” Perl­stein said. “Now he’s out of the cap ta­ble and out of the com­pa­ny. He can de­stroy your rep­u­ta­tion just by as­so­ci­a­tion. Plus, I had learned he wasn’t a good per­son to do busi­ness with.”

A biotech born from Twit­ter

Perl­stein said Twit­ter has ac­tu­al­ly played an in­te­gral role in the de­vel­op­ment of Per­lara.

“Af­ter the post­do­ca­lypse, I left acad­e­mia,” Perl­stein said. “Twit­ter was the place I was re­born pro­fes­sion­al. I can hon­est­ly say that Per­lara wouldn’t be here if it weren’t for Twit­ter.”

Per­lara is work­ing on six drug pro­grams for ul­tra-rare dis­eases, in­clud­ing lyso­so­mal stor­age dis­eases. Since the com­pa­ny’s 2014 in­cep­tion, it’s large­ly re­lied on part­ner­ships with pa­tient groups. Last year, how­ev­er, it land­ed a re­search part­ner­ship with No­var­tis. Af­ter hit­ting its mile­stones on that deal, Per­lara just re­ceived some ex­tra fi­nan­cial sup­port from No­var­tis in its lat­est Se­ries A round, an­nounced this morn­ing.

The to­tal eq­ui­ty round ($7.4 mil­lion) in­clud­ed with cash from in­vestors Piv­otal Cap­i­tal Al­pha, Al-Ham­ra Group, Home­brew Ven­tures, Haystack Fund and ex­ist­ing in­vestors.

Martin Shkreli [via Getty]

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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.

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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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Brian Kaspar. AveXis via Twitter

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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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Bob Smith, Pfizer

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And now they’re really going for it.

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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

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Early Wednesday the pharma giant announced that their NEPTUNE study had failed, with the combination unable to beat standard chemo at overall survival in high TMB cases of advanced non-small cell lung cancer. We won’t get hard data until later in the year, but the drumbeat of failures will call into question what — if any — future this combination can have left.

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

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