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.

Scoop: Boehringer qui­et­ly shut­ters a PhII for one of its top drugs — now un­der re­view

Boehringer Ingelheim has quietly shut down a small Phase II study for one of its lead drugs.

The private pharma player confirmed to Endpoints News that it had shuttered a study testing spesolimab as a therapy for Crohn’s patients suffering from bowel obstructions.

A spokesperson for the company tells Endpoints:

Taking into consideration the current therapeutic landscape and ongoing clinical development programs, Boehringer Ingelheim decided to discontinue our program in Crohn’s disease. It is important to note that this decision is not based on any safety findings in the clinical trials.

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Alex­ion puts €65M for­ward to strength­en its po­si­tion on the Emer­ald Isle

Ireland has been on a roll in 2022, with several large pharma companies announcing multimillion-euro projects. Now AstraZeneca’s rare disease outfit Alexion is looking to get in on the action.

Alexion on Friday announced a €65 million ($68.8 million) investment in new and enhanced capabilities across two sites in the country, including at College Park in the Dublin suburb of Blanchardstown and the Monksland Industrial Park in the central Irish town of Athlone, according to the Industrial Development Agency of Ireland.

Members of the G7 from left to right: Prime Minister of Italy Mario Draghi, European Commission President Ursula von der Leyen, President Joe Biden, German Chancellor Olaf Scholz, British Prime Minister Boris Johnson, Canadian Prime Minister Justin Trudeau, Prime Minister of Japan Fumio Kishida, French President Emmanuel Macron and European Council President Charles Michel (AP Photo/Susan Walsh)

Biden and G7 na­tions of­fer funds for vac­cine and med­ical prod­uct man­u­fac­tur­ing project in Sene­gal

Amidst recently broader vaccine manufacturing initiatives from the EU and European companies, the G7 summit in the mountains of Bavaria has brought about some positive news for closing vaccine and medical product manufacturing gaps around the globe.

According to a statement from the White House, the G7 leaders have formally launched the partnership for global infrastructure, PGII. The effort will aim to mobilize hundreds of billions of dollars to deliver infrastructure projects in several sectors including the medical and pharmaceutical manufacturing space.

State bat­tles over mifepri­s­tone ac­cess could tie the FDA to any post-Roe cross­roads

As more than a dozen states are now readying so-called “trigger” laws to kick into effect immediate abortion bans following the overturning of Roe v. Wade on Friday, these laws, in the works for more than a decade in some states, will likely kick off even more legal battles as states seek to restrict the use of prescription drug-based abortions.

Since Friday’s SCOTUS opinion to overturn Americans’ constitutional right to an abortion after almost 50 years, reproductive rights lawyers at Planned Parenthood and other organizations have already challenged these trigger laws in Utah and Louisiana. According to the Guttmacher Institute, other states with trigger laws that could take effect include Arkansas, Idaho, Kentucky, Mississippi, Missouri, North Dakota, Oklahoma, South Dakota, Tennessee, Texas, and Wyoming.

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Deborah Dunsire, Lundbeck CEO

Af­ter a 5-year re­peat PhI­II so­journ, Lund­beck and Ot­su­ka say they're fi­nal­ly ready to pur­sue OK to use Rex­ul­ti against Alzheimer's ag­i­ta­tion

Five years after Lundbeck and their longtime collaborators at Otsuka turned up a mixed set of Phase III data for Rexulti as a treatment for Alzheimer’s dementia-related agitation, they’ve come through with a new pivotal trial success they believe will finally put them on the road to an approval at the FDA. And if they’re right, some analysts believe they’re a short step away from adding more than $500 million in annual sales for the drug, already approved in depression and schizophrenia.

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A Mer­ck part­ner is sucked in­to the fi­nan­cial quag­mire as key lender calls in a note

Another biotech standing on shaky financial legs has fallen victim to the bears.

Merck partner 4D Pharma has reported that a key lender, Oxford Finance, shoved the UK company into administration after calling in a $14 million loan they couldn’t immediately make good on. Trading in their stock was halted with a market cap that had fallen to a mere £30 million.

“Despite the very difficult prevailing market conditions,” 4D reported on Friday, the biotech had been making progress on finding some new financing and turned to Oxford with an alternative late on Thursday and then again Friday morning.

Fed­er­al judge de­nies Bris­tol My­er­s' at­tempt to avoid Cel­gene share­hold­er law­suit

Some Celgene shareholders aren’t happy with how Bristol Myers Squibb’s takeover went down.

On Friday, a New York federal judge ruled that they have a case against the pharma giant, denying a request to dismiss allegations that it purposely slow-rolled Breyanzi’s approval to avoid paying out $6.4 billion in contingent value rights (CVR).

When Bristol Myers put down $74 billion to scoop up Celgene back in 2019, liso-cel — the CAR-T lymphoma treatment now marketed as Breyanzi — was supposedly one of the centerpieces of the deal. After going back and forth on negotiations for about six months, BMS put $6.4 billion into a CVR agreement that required an FDA approval for Zeposia, Breyanzi and Abecma, each by an established date.

Chris Anzalone, Arrowhead CEO

Take­da, Ar­row­head spot­light da­ta from small tri­al show­ing RNAi works in a rare liv­er con­di­tion

Almost two years after Takeda wagered $300 million cash to partner with Arrowhead on an RNAi therapy for a rare disease, the companies are spelling out Phase II data that they believe put them one step closer to their big dreams.

In a small, open label study involving only 16 patients who had liver disease associated with alpha-1 antitrypsin deficiency (AATD), Arrowhead’s candidate — fazirsiran, previously ARO-AAT — spurred substantial reductions in accumulated mutant AAT protein in the liver, a hallmark of the condition. Investigators also tracked improvements in symptoms, with seven out of 12 who received the high, 200 mg dose seeing regression of liver fibrosis.

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Faheem Hasnain at Endpoints' #BIO22 panel (J.T. MacMillan Photography for Endpoints News)

Bat­tle-test­ed biotech ex­ecs turn to an ex­pe­ri­enced in­dus­try god­fa­ther for some board­room guid­ance

Little Sling Therapeutics out of Ann Arbor, MI may be just a few days out of the starting gate from the announcement of its $35 million launch round, but execs are positioning themselves within clear sight of a key clinical finish line. And they’re revving up with an experienced player to help guide them on the way.

The biotech is putting out word this morning that Faheem Hasnain has agreed to godfather the drug developer as chairman of the board, adding a measured dash of seasoning at a point when most biotechs are still struggling with the embryo stage.

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