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.

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

Back at the beginning of 2018, Clay Siegall snagged a cancer drug called tucatinib with a $614 million cash deal to buy Cascadian. It paid off today with a solid set of mid-stage data for HER2 positive breast cancer that will in turn serve as the pivotal win Siegall needs to seek an accelerated approval in the push for a new triplet therapy.

And if all the cards keep falling in its favor, they’ll move from 1 drug on the market to 3 in 2020, which is shaping up as a landmark year as Seattle Genetics prepares for its 23rd anniversary on July 15.

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Med­ical an­i­ma­tion: Mak­ing it eas­i­er for the site and the pa­tient to un­der­stand

Medical animation has in recent years become an increasingly important tool for conveying niche information to a varied audience, particularly to those audiences without expertise in the specialist area. Science programmes today, for example, have moved from the piece-to-camera of the university professor explaining how a complex disease mechanism works, to actually showing the viewer first-hand what it might look like to shrink ourselves down to the size of an ant’s foot, and travel inside the human body to witness these processes in action. Effectively communicating a complex disease pathophysiology, or the novel mechanism of action of a new drug, can be complex. This is especially difficult when the audience domain knowledge is limited or non-existent. Medical animation can help with this communication challenge in several ways.
Improved accessibility to visualisation
Visualisation is a core component of our ability to understand a concept. Ask 10 people to visualise an apple, and each will come up with a slightly different image, some apples smaller than others, some more round, some with bites taken. Acceptable, you say, we can move on to the next part of the story. Now ask 10 people to visualise how HIV’s capsid protein gets arranged into the hexamers and pentamers that form the viral capsid that holds HIV’s genetic material. This request may pose a challenge even to someone with some virology knowledge, and it is that inability to effectively visualise what is going on that holds us back from fully understanding the rest of the story. So how does medical animation help us to overcome this visualisation challenge?

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.

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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Alex­ion clinch­es aHUS ap­proval for Ul­tomiris as the clock ticks on Soliris con­ver­sion

Alexion has racked up a second approval for Ultomiris, the successor therapy to Soliris, as its mainstay blockbuster therapy faces a patent review process that could drastically shorten its patent exclusivity.

The FDA OK for atypical hemolytic uremic syndrome (aHUS) on Friday was widely expected after Alexion posted a full slate of positive Phase III data in January. But regulators also flagged concerns about serious meningococcal infections, slapping a black box warning on the label and mandating a REMS.

FDA ap­proval lets Foamix set its maid­en ac­ne ther­a­py on course for US mar­ket launch

Months ago, Foamix leaned on its biggest shareholders — Perceptive Advisors and OrbiMed — to financially grease its wheels, ahead of the FDA decision date for its acne therapy. On Friday, that approval came in — and the topical formulation of the antibiotic minocycline is set for a January launch.

The therapy, Amzeeq (formerly known as FMX101), was approved to treat inflammatory lesions of non-nodular moderate-to-severe acne vulgaris in patients aged 9 and older.

Hal Barron, GSK's president of R&D and CSO, speaks to Endpoints News founder and editor John Carroll in London at Endpoints' #UKBIO19 summit on October 8, 2019

[Video] Cel­e­brat­ing tri­al fail­ures, chang­ing the cul­ture and al­ly­ing with Cal­i­for­nia dream­ers: R&D chief Hal Bar­ron talks about a new era at GSK

Last week I had a chance to sit down with Hal Barron at Endpoints’ #UKBIO19 summit to discuss his views on R&D at GSK, a topic that has been central to his life since he took the top research post close to 2 years ago. During the conversation, Barron talked about changing the culture at GSK, a move that involves several new approaches — one of which involves celebrating their setbacks as they shift resources to the most promising programs in the pipeline. Barron also discussed his new alliances in the Bay Area — including his collaboration pact with Lyell, which we covered here — frankly assesses the pluses and minuses of the UK drug development scene, and talks about his plans for making GSK a much more effective drug developer.

This is one discussion you won’t want to miss. Insider and Enterprise subscribers can log-in to watch the video.

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Christine Bunt, Robert Langer. Verseau

Armed with Langer tech and $50M, Verseau hails new check­point drugs un­leash­ing macrophages against can­cer

The rising popularity of CD47 has propelled the “don’t-eat-me” signal to household name status in the immuno-oncology world: By blocking that protein, the theory goes, one can stop cancer cells from fooling macrophages. But just as PD-(L)1 merely represents the most fruitful of all checkpoints regulating T cells, Verseau Therapeutics is convinced that CD47 is one of many regulators one can modulate to stir up or tame the immune system.

Alice Shaw, Lung Cancer Foundation of America

Top ALK ex­pert and can­cer drug re­searcher Al­ice Shaw bids adieu to acad­e­mia, hel­lo to No­var­tis

Jay Bradner has recruited a marquee oncology drug researcher into the ranks of the Novartis Institutes for BioMedical Research. Alice Shaw is jumping from prestigious posts intertwined through Mass General, Harvard and Dana-Farber to take the lead of NIBR’s translational clinical oncology group.

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