Ronald Crys­tal's Lex­eo Ther­a­peu­tics pulls in a $100M megaround just nine months af­ter launch

About a month af­ter Lex­eo Ther­a­peu­tics ac­quired Ste­lios Ther­a­peu­tics — bring­ing its 18-pro­gram gene ther­a­py pipeline to 21 — in­vestors are reach­ing a lit­tle deep­er in­to their pock­ets to en­sure the New York City-based biotech is up to the task.

Lex­eo un­veiled a $100 mil­lion Se­ries B round on Thurs­day morn­ing, led by D1 Cap­i­tal Part­ners and Even­tide As­set Man­age­ment. The funds will be used to sup­port the com­pa­ny’s “next phase of growth,” ac­cord­ing to CEO Nolan Townsend, with a spe­cif­ic fo­cus on three lead­ing gene ther­a­pies — one for CLN2 Bat­ten dis­ease, one for Alzheimer’s, and one for a rare con­di­tion called Friedre­ich’s atax­ia.

Ronald Crys­tal

The com­pa­ny’s roots trace back to gene ther­a­py long hauler Ronald Crys­tal, who’s been work­ing in the field since the 1980s. He took his ideas to Weill Cor­nell Med­i­cine in 1993, where he helped build a large gene ther­a­py pro­gram and spent more than a decade de­vel­op­ing po­ten­tial can­di­dates.

Af­ter de­cid­ing to splin­ter off, Crys­tal as­sem­bled a sea­soned brain trust to lead Lex­eo, in­clud­ing Townsend, who spent 12 years at Pfiz­er; chair­man Steven Altschuler, who was pre­vi­ous­ly chair­man of gene ther­a­py pi­o­neer Spark Ther­a­peu­tics; and ex­ec­u­tive VP and CMO Jay Barth, who hails from  Am­i­cus Ther­a­peu­tics and PTC Ther­a­peu­tics. Crys­tal has al­so kept his po­si­tion at Weill Cor­nell.

Nolan Townsend

The pipeline cur­rent­ly in­cludes eight can­di­dates for car­diac and CNS dis­or­ders, plus an­oth­er 13 undis­closed pro­grams. How­ev­er, on­ly two have reached the clin­ic: LX1004, which re­cent­ly com­plet­ed a Phase I/II study and is head­ed for a piv­otal tri­al in 2022 for CLN2 Bat­ten dis­ease, an au­to­so­mal re­ces­sive lyso­so­mal stor­age dis­ease; and LX100, cur­rent­ly in Phase I for APOE4-as­so­ci­at­ed Alzheimer’s dis­ease.

Townsend’s al­so lin­ing up a third can­di­date for the clin­ic, dubbed LX2006, for Friedre­ich’s atax­ia. FA is a rare, de­gen­er­a­tive mul­ti-sys­tem dis­or­der caused by a gene mu­ta­tion that dis­rupts the nor­mal pro­duc­tion of the pro­tein fratax­in, crit­i­cal to the func­tion of mi­to­chon­dria in a cell. Lex­eo is plan­ning on sub­mit­ting an IND for that in­di­ca­tion lat­er this year.

Lex­eo in­tends to main­tain an on­go­ing re­search col­lab­o­ra­tion with Weill Cor­nell to bol­ster their pre­clin­i­cal pipeline — and with the Ste­lios ac­qui­si­tion back in Ju­ly, they ac­quired a new aca­d­e­m­ic part­ner in UC San Diego’s de­part­ment of car­di­ol­o­gy.

And while Lex­eo re­mains well-fund­ed (the lat­est round brings the start­up’s to­tal raise to $185 mil­lion), Townsend says he isn’t rul­ing out an IPO: “We can cre­ate a lot of val­ue and reach clear val­ue cre­ation mile­stones with­out an IPO or an ad­di­tion­al fi­nanc­ing here. So it’s not nec­es­sary, but ob­vi­ous­ly for any com­pa­ny we’re al­ways think­ing in­to the fu­ture and that is, you know, one of the con­sid­er­a­tions that we have.”

In ad­di­tion to D1 and Even­tide, a slate of new and old in­vestors chipped in­to the round, in­clud­ing CAM Cap­i­tal, Veri­tion Fund Man­age­ment, Lau­ri­on Cap­i­tal Man­age­ment, Gray’s Creek Cap­i­tal Part­ners, Lon­gi­tude Cap­i­tal, Omega Funds, Lund­beck­fonden Ven­tures, PBM Cap­i­tal, Janus Hen­der­son In­vestors, Wood­line Part­ners LP, In­vus Cap­i­tal, and Alexan­dria Ven­ture In­vest­ments.

This sto­ry has been up­dat­ed to clar­i­fy that Ronald Crys­tal still holds his po­si­tion at Weill Cor­nell. 

Biotech in­vestors and CEOs see two paths to growth, but are they equal­ly vi­able?

The dynamic in the biotech market has been highly volatile in the last few years, from the high peaks immediately after the COVID vaccine in 2021, to the lowest downturns of the last 20 years in 2022. This uncertainty makes calling the exact timing of the market’s turn something of a fool’s errand, according to Dr. Chen Yu, Founder and Managing Partner of TCG Crossover (TCG X). He speaks with RBC’s Noël Brown, Head of US Biotechnology Investment Banking, about the market’s road ahead and two possible paths for growth.

Vlad Coric, Biohaven CEO

Vlad Coric charts course for 'New Bio­haven' with neu­ro­science push and Big Phar­ma vets on board

What’s Biohaven without its CGRP portfolio? That’s what CEO Vlad Coric is tasked with deciding as he maps out the “New Biohaven” post-Pfizer takeover.

Pfizer officially scooped up Biohaven’s CGRP assets on Monday, including blockbuster migraine drug Nurtec and the investigational zavegepant, for $11.6 billion. As a result, Coric spun the broader pipeline into an independent company on Tuesday — with the same R&D team behind Nurtec but about 1,000 fewer staffers and a renewed focus on neuroscience and rare disease.

In AstraZeneca's latest campaign, wild eosinophils called Phils personify the acting up often seen in uncontrolled asthma

As­traZeneca de­buts an­noy­ing pur­ple ‘Phil’ crea­tures, per­son­i­fied asth­ma eosinophils ‘be­hav­ing bad­ly’

There are some odd-looking purple creatures lurking around the halls of AstraZenca lately. The “Phil” character cutouts are purple, personified eosinophils with big buggy eyes and wide mouths, and they’re a part of AZ’s newest awareness effort to help people understand eosinophilic asthma.

The “Asthma Behaving Badly” characters aren’t only on the walls at AZ to show the new campaign to employees, however. The “Phils” are also showing up online on the campaign website, and in digital and social ads and posts on Facebook and Instagram.

Casey McPherson shows his daughters Rose (left) and Weston around Everlum Bio, a lab that he co-founded to spark a treatment for Rose and others with ultra-rare conditions. (Ilana Panich-Linsman)

Fa­ther starts lab af­ter in­tel­lec­tu­al prop­er­ty is­sues stymie rare dis­ease drug de­vel­op­ment

Under bright lab lights, Casey McPherson holds his 6-year-old daughter, Rose. His free hand directs Rose’s gaze toward a computer screen with potential clues in treating her one-of-a kind genetic condition.

Gray specks on the screen show her cells that scientists reprogrammed with the goal of zeroing in on a custom medicine. McPherson co-founded the lab, Everlum Bio, to spark a treatment for Rose — and others like her. A regarded singer-songwriter, McPherson never imagined going into drug development.

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Ying Huang, Legend CEO

Lentivi­ral vec­tor ramp-up: J&J and Leg­end to in­vest $500M in New Jer­sey man­u­fac­tur­ing to sup­port Carvyk­ti

In response to a question on manufacturing scale at Legend Biotech’s R&D day yesterday, the company’s top exec said its partnership with Johnson & Johnson will be doubling its investment in its New Jersey manufacturing center and will be investing a total of $500 million.

With an eye on their BCMA-directed CAR-T therapy Carvykti (cilta-cel), approved in February as a fifth-line treatment for multiple myeloma, Legend CEO Ying Huang said that the ramp-up in production and the decision to manufacture its own lentiviral vectors — currently in shortage worldwide — means they won’t have to deal with that shortage.

Kite Phar­ma gets FDA to sign off on new Cal­i­for­nia-based vec­tor man­u­fac­tur­ing fa­cil­i­ty

Kite Pharma just got FDA approval to kick off operations at a new manufacturing campus.

The cancer-focused, CAR-T cell therapy player made the announcement Monday, saying that the federal regulatory agency gave the green light to Kite’s 100,000 square-foot, retroviral vector manufacturing facility in Oceanside, CA.

Kite’s global head of technical operations Chris McDonald tells Endpoints News that the facility has been in the works for about four years, after Kite teamed up with its parent company Gilead. Gilead acquired Kite Pharma for just shy of $12 billion in 2017.

Mar­ket­ingRx roundup: No­var­tis re­cruits NFL coach for Leqvio cam­paign; Pfiz­er pro­motes ‘Sci­ence’ merch on so­cial me­dia

Novartis is turning to a winning coach to talk about Leqvio and the struggles of high cholesterol — including his own. Bruce Arians, the retired NFL head coach of the Arizona Cardinals and Super Bowl-winning Tampa Bay Buccaneers, is partnering with the pharma for its “Coaching Cholesterol” digital, social and public relations effort.

In the campaign, Arians talks about the potential for “great comebacks” in football and heart health. Once nicknamed a “quarterback whisperer,” he is now retired from fulltime coaching (although still a front-office consultant for Tampa Bay), and did a round of media interviews for Novartis, including one with People and Forbes.

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Amy West, Novo Nordisk head of US digital innovation and transformation (Illustration: Assistant Editor Kathy Wong for Endpoints News)

Q&A: No­vo Nordisk dig­i­tal in­no­va­tion chief Amy West dis­cuss­es phar­ma pain points and a health­care 'easy but­ton’

Amy West joined Novo Nordisk more than a decade ago to oversee marketing strategies and campaigns for its US diabetes portfolio. However, her career path shifted into digital, and she hasn’t looked back. West went from leading Novo’s first digital health strategy in the US to now heading up digital innovation and transformation.

She’s currently leading the charge at Novo Nordisk to not only go beyond the pill with digital marketing and health tech, but also test, pilot and develop groundbreaking new strategies needed in today’s consumerized healthcare world.

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Marc Dunoyer, Alexion CEO (AstraZeneca via YouTube)

Up­dat­ed: As­traZeneca nabs a small rare dis­ease gene ther­a­py play­er for 667% pre­mi­um

AstraZeneca is kicking off the fourth quarter with a little M&A Monday for a gene editing player recently overcoming a second clinical hold to its only program in human studies.

The Big Pharma and its subsidiary Alexion are buying out little LogicBio for $2.07 per share. That’s good for a massive 667% premium over its Friday closing price, when it headed into the weekend at 27 cents and just weeks after Nasdaq said LogicBio would have to delist, which has been put on hold as the biotech requests a hearing. It’s one of two biotech deals to commence October, alongside the news of Incyte buying a vitiligo-focused biotech.

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