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.

In his­toric Covid-19 ad­comm, vac­cine ex­perts de­bate a sea of ques­tions — but of­fer no clear an­swers

The most widely anticipated and perhaps most widely watched meeting in the FDA’s 113-year history ended late Thursday night with a score of questions and very few answers.

For nearly 9 hours, 18 different outside experts listened to public health agencies and foundations present how the United States’ Covid-19 vaccine program developed through October, and they debated where it should go from there: Were companies testing the right metrics in their massive trials? How long should they track patients before declaring a vaccine safe or effective? Should a vaccine, once authorized, be given to the volunteers in the placebo arm of a trial?

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Pascal Soriot, AstraZeneca CEO (Zach Gibson/Bloomberg via Getty Images)

UP­DAT­ED: FDA gives As­traZeneca the thumbs-up to restart PhI­II Covid-19 vac­cine tri­als, and J&J is prepar­ing to re­sume its study

Several countries had restarted their portions of AstraZeneca’s global Phase III Covid-19 vaccine trial after the study was paused worldwide in early September, but the US notably stayed on the sidelines — until now. Friday afternoon the pharma giant announced the all clear from US regulators. And on top of that, J&J announced Friday evening that it’s preparing to resume its own Phase III vaccine trial.

Ul­tragenyx in­jects $40M to grab Solid's mi­crody­s­trophin trans­gene — while side­step­ping the AAV9 vec­tor that stirred up safe­ty fears

Since before Ilan Ganot started Solid Bio to develop a gene therapy for kids like his son, who has Duchenne muscular dystrophy, Ultragenyx CEO Emil Kakkis has been watching and advising the former investment banker as he navigated the deep waters of drug development.

Just as Solid is getting back up on its feet after a yearlong clinical hold, Kakkis has decided to jump in for a formal alliance.

With a $40 million upfront, Ultragenyx is grabbing 14.45% of Solid’s shares $SLDB and the rights to its microdystrophin construct for use in combination with AAV8 vectors. Solid’s lead program, which utilizes AAV9, remains unaffected. The company also retains rights to other applications of its transgene.

A top drug pro­gram at Bay­er clears a high bar for CKD — open­ing the door to an FDA pitch

Over the past 4 years, Bayer has been steering a major trial through a pivotal program to see if their drug finerenone could slow down the pace of chronic kidney disease in patients suffering from both CKD as well as Type 2 diabetes.

Today, their team jumped on a virtual meeting hosted by the American Society of Nephrology to offer a solid set of pivotal data to demonstrate that the drug can delay dialysis or a kidney replacement as well as cardio disease, while also adding some worrying signs of hyperkalemia among the patients taking the drug. And they’re hustling it straight to regulators in search of an approval for kidney disease and cardio patients — one of the toughest challenges in the book, as demonstrated by repeated past failures.

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Michel Vounatsos, Biogen CEO (via YouTube)

UP­DAT­ED: Bio­gen spot­lights a pair of painful pipeline set­backs as ad­u­canum­ab show­down looms at the FDA

Biogen has flagged a pair of setbacks in the pipeline, spotlighting the final failure for a one-time top MS prospect while scrapping a gene therapy for SMA after the IND was put on hold due to toxicity.

Both failures will raise the stakes even higher on aducanumab, the Alzheimer’s drug that Biogen is betting the ranch on, determined to pursue an FDA OK despite significant skepticism they can make it with mixed results and a reliance on post hoc data mining. And the failures are being reported as Biogen was forced to cut its profit forecast for 2020 as a generic rival started to erode their big franchise drug.

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Adam Koppel and Jeffrey Schwartz, Bain

Bain ex­ecs Adam Kop­pel and Jef­frey Schwartz line up $125M for their first blank check deal as Wall Street con­tin­ues to em­brace biotech

Adam Koppel and Jeffrey Schwartz have jumped into the blank check game, raising $125 million for a stock listing in search of a company.

Their SPAC, BCLS Acquisition Corp, raised $125 million this week, with a line on $25 million more as it scouts for a biotech in search of money and a place on Wall Street.

The two principals at Bain Life Sciences have been on a romp since they set up the Bain operation 4 years ago. Their S-1 spells out a track record of 22 deals totaling $650 million for the life sciences group, which led to 9 IPOs.

Covid-19 roundup: An mR­NA play­er gets a boost out of the lat­est round of an­i­mal da­ta; Phase­Bio pulls the plug on treat­ment tri­al

The big tell for CureVac $CVAC is coming up with a looming early-stage readout on their mRNA Covid-19 vaccine in the clinic. But for now they’ll make do with an upbeat assessment on the preclinical animal data they used to get into the clinic.

Researchers for the German biotech say they got the high antibody titers and T cell activation they were looking for, lining up a hamster challenge to demonstrate — in a simple model — that the vaccine could protect the furry creatures. Like the other mRNA vaccines, the drug sends instructions to spur cells to decorate themselves with the distinctive spike on the virus to elicit an immune response.

Chi­nese in­vestors wa­ger $105M on an IPO-bound biotech look­ing to push RNAi as main­stream can­cer ther­a­py

Shortly after Sirnaomics brought in a $47 million Series C for its small interfering RNA pipeline last year, Patrick Lu — the founder, president and CEO — was asked to outline the scientific advances that will be necessary to make better drugs out of RNA tech.

“The next step in the evolution of RNAi as a leading therapeutic will be the ability to safely target organs outside the liver such as lung, brain, etc,” he had offered. “This will revolutionize disease treatments if the industry can demonstrate similar data sets for non-liver targets as we have seen in liver-based diseases.”

UP­DAT­ED: In­di­v­ior's Shaun Thax­ter heads to prison, join­ing In­sys' John Kapoor among jailed opi­oid ex­ecs

Update: An earlier version of this article misidentified the jailed Insys CEO. Former CEO John Kapoor was sentenced to 5.5 years in prison in January. Endpoints News regrets the error.

The Justice Department’s years-long battle with Indivior has arrived at a rare place: the jailing of a pharmaceutical executive.

A US district court sentenced long-running Indivior CEO Shaun Thaxter to 6 months in federal prison for his role in company efforts to mislead a major healthcare provider about the safety and abusability of their opioid addiction drug Suboxone, which generated billions in revenue over the last decade. Thaxter joins former Insys CEO John Kapoor as one of the only two executives to face prison time for their roles in the opioid epidemic.