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.

Biotech Half­time Re­port: Af­ter a bumpy year, is biotech ready to re­bound?

The biotech sector has come down firmly from the highs of February as negative sentiment takes hold. The sector had a major boost of optimism from the success of the COVID-19 vaccines, making investors keenly aware of the potential of biopharma R&D engines. But from early this year, clinical trial, regulatory and access setbacks have reminded investors of the sector’s inherent risks.

RBC Capital Markets recently surveyed investors to take the temperature of the market, a mix of specialists/generalists and long-only/ long-short investment strategies. Heading into the second half of the year, investors mostly see the sector as undervalued (49%), a large change from the first half of the year when only 20% rated it as undervalued. Around 41% of investors now believe that biotech will underperform the S&P500 in the second half of 2021. Despite that view, 54% plan to maintain their position in the market and 41% still plan to increase their holdings.

UP­DAT­ED: Boehringer nabs FDA's first in­ter­change­abil­i­ty des­ig­na­tion for its Hu­mi­ra com­peti­tor — but will it mat­ter?

The FDA late Friday awarded Boehringer Ingelheim the first interchangeability designation for its Humira biosimilar Cyltezo, meaning that when it launches in July 2023, pharmacists will be able to automatically substitute the Boehringer’s version for AbbVie’s mega-blockbuster without a doctor’s input.

The designation will likely give Boehringer, which first won approval for Cyltezo in 2017, the leg up on a crowded field of Humira competitors.

Bio­gen hit by ALS set­back with PhI­II fail­ure for tofersen — but fol­lows a fa­mil­iar strat­e­gy high­light­ing the pos­i­tive

Patients and analysts waiting to hear Sunday how Biogen’s SOD1-ALS drug tofersen fared in Phase III didn’t have to wait long for the top-line result they were all waiting for. The drug failed the primary endpoint on significantly improving the functional and neurologic decline of patients over 28 weeks as well as the extension period for continued observation.

In fact, there was very little difference in response.

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Scott Struthers, Crinetics CEO

Cri­net­ics spins out ra­dio­phar­ma ef­forts in­to a new com­pa­ny, high­light­ing the grow­ing field­'s al­lure

Largely known for its nonpeptide small molecule research, Crinetics has been keeping its radiopharma work comparatively under wraps. But that changed Monday afternoon as the California biotech spun out a new company focused solely on the burgeoning field.

Crinetics launched Radionetics after the closing bell Monday, the company announced, seeding the new entity with $30 million raised from 5AM Ventures and Frazier Healthcare Partners. Radionetics will start with its own radiopharma-centric platform and a pipeline of 10 programs aimed at solid tumors.

Sheldon Koenig, Esperion CEO

Es­pe­ri­on gets out the bud­get ax, chop­ping 170 staffers as its big drug launch sput­ters

Esperion’s executive team spent years insisting that they had found the sweet spot in the market for their cholesterol drug. But that strategy has soured badly, and after struggling to sell its heart disease pill for more than a year, the biotech says it will cut about 40% of its staff over the next few weeks.

The layoffs will take place across the board, from sales and marketing to R&D, CEO Sheldon Koenig told Endpoints News on Monday. While the chief executive declined to elaborate on how many employees will be affected, an SEC filing stated that approximately 170 staffers are on the chopping block.

Two drug­mak­ers hit with PDU­FA date de­lays from FDA amid back­log of in­spec­tions

As the FDA is weighed down with more and more pandemic responsibilities, the agency is beginning to miss PDUFA dates with more frequency too. Two different companies on Monday said they received notices that the FDA has not completed their drug reviews on time.

The review of an NDA for Avadel Pharmaceuticals’ candidate treatment for narcolepsy is not coming this month, the company said, and the review of UCB’s BLA for bimekizumab, used to treat moderate to severe plaque psoriasis, will miss its target date as well.

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Reshma Kewalramani, Vertex CEO (YouTube)

Ver­tex gets much-need­ed win with ‘ex­tra­or­di­nary’ first pa­tient re­sults on po­ten­tial di­a­betes cure

Vertex said Monday that the first patient dosed with its cell therapy for type 1 diabetes saw their need for insulin injections vanish almost entirely, a key early step in the decades-long effort to develop a curative treatment for the chronic disease.

The patient, who had suffered five potentially life-threatening hypoglycemic — or low blood sugar — episodes in the year before the therapy, was injected with synthetic insulin-producing cells. After 90 days, the patient’s new cells produced insulin steadily and ramped up their insulin production after a meal like normal cells do, as measured by a standard biomarker for insulin production.

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Covid-19 vac­cine boost­ers earn big thumbs up, but Mod­er­na draws ire over world sup­ply; What's next for Mer­ck’s Covid pill?; The C-suite view on biotech; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

You may remember that at the beginning of this year, Endpoints News set a goal to go broader and deeper. We are still working towards that, and are excited to share that Beth Snyder Bulik will be joining us on Monday to cover all things pharma marketing. You can sign up for her weekly Endpoints MarketingRx newsletter in your reader profile.

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No­var­tis de­vel­op­ment chief John Tsai: 'We go deep in the new plat­form­s'

During our recent European Biopharma Summit, I talked with Novartis development chief John Tsai about his experiences over the 3-plus years he’s been at the pharma giant. You can read the transcript below or listen to the exchange in the link above.

John Carroll: I followed your career for quite some time. You’ve had more than 20 years in big pharma R&D and you’ve obviously seen quite a lot. I really was curious about what it was like for you three and a half years ago when you took over as R&D chief at Novartis. Obviously a big move, a lot of changes. You went to work for the former R&D chief of Novartis, Vas Narasimhan, who had his own track record there. So what was the biggest adjustment when you went into this position?

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