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.

Biotech Half­time Re­port: Af­ter a bumpy year, is biotech ready to re­bound?

The biotech sector has come down firmly from the highs of February as negative sentiment takes hold. The sector had a major boost of optimism from the success of the COVID-19 vaccines, making investors keenly aware of the potential of biopharma R&D engines. But from early this year, clinical trial, regulatory and access setbacks have reminded investors of the sector’s inherent risks.

RBC Capital Markets recently surveyed investors to take the temperature of the market, a mix of specialists/generalists and long-only/ long-short investment strategies. Heading into the second half of the year, investors mostly see the sector as undervalued (49%), a large change from the first half of the year when only 20% rated it as undervalued. Around 41% of investors now believe that biotech will underperform the S&P500 in the second half of 2021. Despite that view, 54% plan to maintain their position in the market and 41% still plan to increase their holdings.

Covid-19 vac­cine boost­ers earn big thumbs up, but Mod­er­na draws ire over world sup­ply; What's next for Mer­ck’s Covid pill?; The C-suite view on biotech; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

You may remember that at the beginning of this year, Endpoints News set a goal to go broader and deeper. We are still working towards that, and are excited to share that Beth Snyder Bulik will be joining us on Monday to cover all things pharma marketing. You can sign up for her weekly Endpoints MarketingRx newsletter in your reader profile.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 119,800+ biopharma pros reading Endpoints daily — and it's free.

Amit Etkin, Alto Neuroscience CEO (Alto via Vimeo)

A star Stan­ford pro­fes­sor leaves his lab for a start­up out to re­make psy­chi­a­try

About five years ago, Amit Etkin had a breakthrough.

The Stanford neurologist, a soft-spoken demi-prodigy who became a professor while still a resident, had been obsessed for a decade with how to better define psychiatric disorders. Drugs for depression or bipolar disorder didn’t work for many patients with the conditions, and he suspected the reason was how traditional diagnoses didn’t actually get at the heart of what was going on in a patient’s brain.

No­var­tis de­vel­op­ment chief John Tsai: 'We go deep in the new plat­form­s'

During our recent European Biopharma Summit, I talked with Novartis development chief John Tsai about his experiences over the 3-plus years he’s been at the pharma giant. You can read the transcript below or listen to the exchange in the link above.

John Carroll: I followed your career for quite some time. You’ve had more than 20 years in big pharma R&D and you’ve obviously seen quite a lot. I really was curious about what it was like for you three and a half years ago when you took over as R&D chief at Novartis. Obviously a big move, a lot of changes. You went to work for the former R&D chief of Novartis, Vas Narasimhan, who had his own track record there. So what was the biggest adjustment when you went into this position?

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Susan Galbraith, Executive VP, Oncology R&D, AstraZeneca

As­traZeneca on­col­o­gy R&D chief Su­san Gal­braith: 'Y­ou're go­ing to need or­thog­o­nal com­bi­na­tion­s'


Earlier in the week we broadcast our 4th annual European Biopharma Summit with a great lineup of top execs. One of the one-on-one conversations I set up was with Susan Galbraith, the oncology research chief at AstraZeneca. In a wide-ranging discussion, Galbraith reviewed the cancer drug pipeline and key trends influencing development work at the pharma giant. You can watch the video, above, or stick with the script below. — JC

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Roche's Tecen­triq cross­es the fin­ish line first in ad­ju­vant lung can­cer, po­ten­tial­ly kick­ing off gold rush

While falling behind the biggest PD-(L)1 drugs in terms of sales, Roche has looked to carve out a space for its Tecentriq with a growing expertise in lung cancer. The drug will now take an early lead in the sought-after adjuvant setting — but competitors are on the way.

The FDA on Friday approved Tecentriq as an adjuvant therapy for patients with Stage II-IIIA non small cell lung cancer with PD-(L)1 scores greater than or equal to 1, making it the first drug of its kind approved in an early setting that covers around 40% of all NSCLC patients.

FDA ad­comm votes unan­i­mous­ly in sup­port of a J&J Covid-19 boost­er two months af­ter one-dose shot

The FDA’s Vaccines and Related Biological Products Advisory Committee (VRBPAC) on Friday voted 19-0 in favor of authorizing a second shot of J&J’s Covid-19 vaccine to follow at least two months after the initial dose.

Regulators don’t have to follow VRBPAC’s recommendation, but they almost always do. Considering that the CDC’s advisory committee has already been set to review the expanded EUA, VRBPAC’s recommendation is likely to be adopted.

Rajesh Devraj, Rectify Pharmaceuticals CEO

At­las backs a long­time Ver­tex em­ploy­ee’s quest to bring CF suc­cess to nu­mer­ous oth­er dis­eases

One of Vertex’s longest-tenured employees believes he can take the biotech’s biggest medical and scientific accomplishments and use it to develop treatments for more than just cystic fibrosis.

Three years ago, Jonathan Moore, a scientist and then executive at Vertex from 1990 to 2018, founded a company to develop treatments for diseases that, like CF, are caused by mutations in a “super family” of proteins known as ABC transporters.

Yao-Chang Xu, Abbisko Therapeutics founder and CEO

Qim­ing-backed Ab­bisko makes $200M+ Hong Kong de­but, as a SPAC and Agenus spin­out al­so price on Nas­daq

Three new entities priced their public debuts late Thursday and early Friday, including a SPAC, a traditional Nasdaq IPO and a Chinese biotech joining the Hong Kong Index.

Shanghai-based Abbisko Therapeutics raised the most money of the triumvirate, garnering $226 million in its Hong Kong debut and pricing at HK$12.46, or roughly $1.60 in US dollars. The blank check company followed up with a $150 million raise, while MiNK Therapeutics priced on Nasdaq at $12 per share and a $40 million raise.