With decades in gene ther­a­py un­der his belt, Ronald Crys­tal launch­es new ven­ture with up to 18 can­di­dates in the pipe

Ronald Crys­tal be­gan work­ing in gene ther­a­py in the 1980s, long be­fore the first wave of ap­provals shook the in­dus­try. 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.

Now, the gene ther­a­py long hauler is launch­ing his own com­pa­ny, Lex­eo Ther­a­peu­tics, with an $85 mil­lion Se­ries A to dri­ve three of the com­pa­ny’s AAV-ad­min­is­tered can­di­dates to mar­ket, it said Thurs­day. Crys­tal will take the role of chief sci­en­tif­ic ad­vis­er with Pfiz­er vet­er­an Nolan Townsend join­ing as CEO.

Ronald Crys­tal

“These three clin­i­cal pro­grams are re­al­ly the fo­cus of the com­pa­ny and … the Se­ries A fi­nanc­ing,” Townsend told End­points News. How­ev­er, Lex­eo has even more can­di­dates wait­ing in the wings.

Townsend, who hails from Pfiz­er’s rare dis­ease pro­gram, was in­tro­duced to Crys­tal via a mu­tu­al con­tact. He served in a va­ri­ety of roles in his 12 years at the phar­ma gi­ant, in­clud­ing as pres­i­dent of rare dis­ease in North Amer­i­ca and oth­er de­vel­oped mar­kets, coun­try man­ag­er in sev­er­al na­tions, and di­rec­tor of busi­ness op­er­a­tions for Asia-es­tab­lished prod­ucts. But what at­tract­ed him to Lex­eo was the op­por­tu­ni­ty to go af­ter both rare and com­mon dis­eases.

“I saw the po­ten­tial of this re­search plat­form to ad­dress a num­ber of rare dis­eases that do not have ad­e­quate ther­a­pies to­day, but al­so the po­ten­tial of this plat­form to ad­dress non-rare dis­eases,” he said.

To lead the fledg­ling team, Crys­tal and Townsend as­sem­bled a sea­soned brain trust, in­clud­ing chair­man Steven Altschuler, who pre­vi­ous­ly served as chair­man of gene ther­a­py pi­o­neer Spark Ther­a­peu­tics, and PTC Ther­a­peu­tics vet Jay Barth as ex­ec­u­tive VP and CMO.

The New York City-based biotech, whose name is a nod to its Lex­ing­ton Av­enue roots, al­ready has two can­di­dates in the clin­ic: LX1004, which has com­plet­ed a Phase I/II study and is head­ed for a piv­otal tri­al in 2022, in CLN2 Bat­ten dis­ease, an au­to­so­mal re­ces­sive lyso­so­mal stor­age dis­ease; and LX100, cur­rent­ly in Phase I for APOE4-as­so­ci­at­ed Alzheimer’s dis­ease. Lex­eo’s third high­light­ed can­di­date, an IV-ad­min­is­tered treat­ment for Friedre­ich’s atax­ia (FA) dubbed LX2006 is ex­pect­ed to en­ter Phase I this year. 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.

Nolan Townsend

The up­start has up to 15 more undis­closed gene ther­a­py pro­grams at dif­fer­ent stages of de­vel­op­ment, ac­cord­ing to Townsend and Crys­tal. Plus, they in­tend to main­tain an on­go­ing re­search col­lab­o­ra­tion with Weill Cor­nell to bol­ster their pre­clin­i­cal pipeline.

“Lex­eo’s AAV me­di­at­ed gene ther­a­py pro­grams have the po­ten­tial for broad ap­plic­a­bil­i­ty across a range of ther­a­peu­tic in­di­ca­tions, and in a sin­gle com­pa­ny pipeline present an op­por­tu­ni­ty for the nat­ur­al evo­lu­tion of gene ther­a­py from rare ge­net­ic con­di­tions to more com­mon dis­eases,” Crys­tal said in a state­ment.

The founder still serves as chair­man of Weill’s De­part­ment of Ge­net­ic Med­i­cine and will look to con­tin­ue build­ing Lex­eo’s team in the near fu­ture.

“We have less than 10 peo­ple in the com­pa­ny to­day. I think we’re ex­pand­ing rapid­ly, so that that num­ber will in­crease sig­nif­i­cant­ly over the next 12 months,” Townsend said.

The Se­ries A was led by Lon­gi­tude Cap­i­tal and Omega Funds, and joined by Lund­beck­fonden Ven­tures, PBM Cap­i­tal, Janus Hen­der­son In­vestors, In­vus, Wood­line Part­ners, the Alzheimer’s Drug Dis­cov­ery Foun­da­tion and Alexan­dria Ven­ture In­vest­ments.

Da­ta Lit­er­a­cy: The Foun­da­tion for Mod­ern Tri­al Ex­e­cu­tion

In 2016, the International Council for Harmonisation (ICH) updated their “Guidelines for Good Clinical Practice.” One key shift was a mandate to implement a risk-based quality management system throughout all stages of a clinical trial, and to take a systematic, prioritized, risk-based approach to clinical trial monitoring—on-site monitoring, remote monitoring, or any combination thereof.

Mer­ck scraps Covid-19 vac­cine pro­grams af­ter they fail to mea­sure up on ef­fi­ca­cy in an­oth­er ma­jor set­back in the glob­al fight

After turning up late to the vaccine development game in the global fight against Covid-19, Merck is now making a quick exit.

The pharma giant is reporting this morning that it’s decided to drop development of 2 vaccines — V590 and V591 — after taking a look at Phase I data that simply don’t measure up to either the natural immune response seen in people exposed to the virus or the vaccines already on or near the market.

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Bahija Jallal, Immunocore

Buried in Im­muno­core's IPO fil­ings? A kick­back scheme from a now for­mer em­ploy­ee

Immunocore spent much of 2019 dealing with the fallout of the Neil Woodford scandal, as the former star investor’s fall crashed the biotech’s valuation out of unicorn range. Now it turns out that the company spent 2020 dealing with another internal scandal.

The longtime UK biotech darling disclosed in their IPO filing last week that they had fallen victim to an alleged kickback scheme involving one of their employees. After a whistleblower came forward, they said in their F-1, they spent the summer and spring investigating, finding fraud on the part of an employee and two outside vendors.

IPO track­er: 2021 gets start­ed with a flur­ry of new of­fer­ings

A global pandemic couldn’t slow down what turned out to be a record year for biotech IPOs. With the calendar turning toward 2021, the Endpoints News team is prepped to track each new filing this year, and the outcome. We’re off to another hot start at least.

Below, you’ll find the companies that have filed to go public, in addition to those that have already priced. Through the first two business weeks of January, there have already been 9 biotechs that have filed or priced, and the number is only expected to grow. We’ll keep the tracker updated as it does.

Matt Gline (L) and Vivek Ramaswamy

Scoop: Vivek Ra­maswamy is hand­ing the CEO job to a top lieu­tenant at Roivant — but he’s not ex­act­ly leav­ing the biotech scene

Over the past 7 years since founding Roivant, Vivek Ramaswamy has been a constant blur of biotech building motion.

He launched his first biotech with an Alzheimer’s drug he picked up cheap, and watched the experiment implode in one of the highest profile pivotal disasters seen in the last decade. But it didn’t slow the 30-something exec down; if anything, he hit the accelerator. Ramaswamy blazed global paths and went on to raise billions to spur the creation of a large lineup of little Vants promising big things at a fast pace. He sold off a section of the Vant brigade to Sumitomo Dainippon for $3 billion. And more recently the relentless dealmaker has been building a computational discovery arm to add an AI-driven approach to kicking up new programs and companies, supplementing the in-licensing drive while pursuing advances that have created more than 700 jobs at Roivant, with $2 billion in reserves.

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Ron Cooper, Albireo CEO

Al­bireo just ad­vanced down to the 10-yard line at the FDA. And Ron Coop­er’s team is get­ting prepped for the next big play

When Albireo Pharma’s board $ALBO moved to bring in Ron Cooper as the CEO more than 5 years ago, the development-stage company went with an experienced commercial player who had a big-time position on his resume after running Bristol Myers’ commercial ops in Europe.

Now, after successfully navigating a pivotal study, putting them in a foot race with a rival toward an FDA OK, Cooper is getting a boost from regulators on the last drive back to an arena he understands completely.

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David Southwell (L) and Christoph Westphal

Har­vard spin­out kicks off 2021 with a crossover round and sights set on the clin­ic

Several months after striking an alliance with Novartis, TCR therapy-focused TScan Therapeutics has reeled in a crossover round that should hold it over for the next two years as it eyes a public debut.

The Christoph Westphal portfolio company had been arranging the crossover for the last few months, CEO David Southwell said. Just before Christmas, they nailed down what he called a “really blue-chip” syndicate of four new investors, including BlackRock, RA Capital Management and two undisclosed funds. They closed on the $100 million Series C just over a week ago, and waited until Monday morning to announce it.

Hal Barron, GSK via YouTube

What does $29B buy you in Big Phar­ma? In Glax­o­SmithK­line’s case, a whole lot of un­com­fort­able ques­tions about the pipeline

Talk about your bad timing.

A little over a week ago, GSK R&D chief Hal Barron marked his third anniversary at the research helm by taking a turn at the virtual podium during JP Morgan to make the case that he and his team had built a valuable late-stage pipeline capable of churning out more than 10 blockbusters in the next 5 years.

And then, just days later, one of the cancer drugs he bet big on as a top prospect — bintrafusp, partnered with Merck KGaA — failed its first pivotal test in non-small cell lung cancer.

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Can strug­gling Iterum turn the cor­ner to an an­tibi­ot­ic suc­cess sto­ry? They will know in six months

More than five years after Corey Fishman and Michael Dunne dusted sulopenem off Pfizer’s shelves — the second castoff antibiotic they’ve brought out of the pharma giant — and founded Iterum Therapeutics around that single drug, they have lined up a quick shot at approval with priority review from the FDA.

The decision, six months from now, will mark a make-or-break moment for a struggling biotech that has just enough cash to keep the lights on until the third quarter.