Sean Nolan, RA Session II

Crossover round? Check. Top team? Check. Taysha sails to an IPO with $100M ini­tial ask

On the same day No­var­tis erased the AveX­is brand from the com­pa­ny, the ex-AveX­is crew says their new gene ther­a­py start­up is ready for an IPO.

Taysha Gene Ther­a­pies has left enough bread crumbs for biotech sleuths to fig­ure out that it’s go­ing down this path. CEO RA Ses­sion II, who hap­pi­ly chat­ted to mul­ti­ple re­porters at launch, de­clined in­ter­views for the re­cent Se­ries B (and pre­sumed crossover round); the biotech brought in a gene ther­a­py pro­gram de­vel­oped by its co-founder Steven Gray from Abeona; and days ago it tapped the ex-CMO and ex-CFO of AveX­is to join the board, chaired by their for­mer boss Sean Nolan.

What’s still re­mark­able is the speed at which Dal­las-based Taysha has moved. It’s been just a year since the com­pa­ny was formed, a lit­tle over four months since Taysha launched with $30 mil­lion and a slate of 15 pro­grams, and bare­ly a month af­ter it closed a $95 mil­lion Se­ries B. The ac­cu­mu­lat­ed deficit so far is just $27.8 mil­lion.

That casts a unique spot­light on Taysha com­pared to the 48 play­ers that have rid­den to a pub­lic list­ing on the biotech boom so far this year. Even Pas­sage Bio, the Philadel­phia-based start­up that has a sim­i­lar pact with gene ther­a­py gu­ru Jim Wil­son, took a year from Se­ries A to go pub­lic.

For now Taysha is pen­cil­ing in a $100 mil­lion raise, but these days the place­hold­er fig­ure could mean lit­tle.

Oth­er than lay­ing out the clin­i­cal tri­al time­lines for the lead pro­grams — the first study, a Phase I/II of TSHA-101 for GM2 gan­gliosi­do­sis, is planned in Cana­da for lat­er this year — the S-1 re­veals some in­ter­est­ing de­tails about the part­ner­ships that are core to the biotech.

To get UT South­west­ern on board, it turned out Taysha didn’t pay a pen­ny. In­stead, it is­sued 2 mil­lion shares of its stock to the uni­ver­si­ty, with no fu­ture mile­stones or roy­al­ty promis­es at­tached oth­er than costs of main­tain­ing patents. Gray, an ex­pert in AAV-based gene ther­a­pies for CNS dis­or­ders, is lead­ing the col­lab­o­ra­tion along­side child neu­rol­o­gy di­vi­sion chief Berge Mi­nass­ian.

By the end of 2021, Taysha al­so ex­pects to file INDs in the US for four pro­grams, all pack­aged in an AAV9 vec­tor and span­ning the three pil­lars of neu­rode­gen­er­a­tive, neu­rode­vel­op­men­tal and epilep­tic dis­or­ders:

TSHA-101, a bi­cistron­ic HexBP2A-HexA trans­gene pack­aged in­to an AAV9 vec­tor un­der the con­trol of a CAG pro­mot­er de­signed for con­di­tions like Tay-Sachs and Sand­hoff dis­ease. TSHA-102, which com­bines a neu­ronal spe­cif­ic pro­mot­er, MeP426, with the min­iMECP2 trans­gene, plus a miR­NA-Re­spon­sive Au­to-Reg­u­la­to­ry El­e­ment for the treat­ment of Rett syn­drome. TSHA-103, which tar­gets a ge­net­ic form of epilep­sy caused by SLC6A1 hap­loin­suf­fi­cien­cy dis­or­der, is con­struct­ed from a codon-op­ti­mized ver­sion of the hu­man SLC6A1 gene un­der the con­trol of a JeT pro­mot­er. TSHA-104, which ad­dress­es SURF1 de­fi­cien­cy, adopts a ver­sion of the hu­man SURF1 gene un­der the con­trol of a mod­i­fied ver­sion of the pro­mot­er CBA hy­brid in­tron, or CBh.

With 18 pro­grams now in the pipeline re­flect­ing a port­fo­lio ap­proach, Taysha is en­ti­tled to an op­tion for four more pro­grams un­der the re­search pact with UT South­west­ern, which ex­pires in No­vem­ber 2021.

Then there’s TSHA-118, a po­ten­tial treat­ment of in­fan­tile Bat­ten dis­ease in­vent­ed by Gray that Taysha re­cent­ly li­censed from Abeona. Taysha is paid on­ly $7 mil­lion up­front to get its hands on the clin­ic-ready drug, then known as ABO-202, with $3 mil­lion in li­cense fees and $4 mil­lion for the in­ven­to­ry — in­clud­ing clin­i­cal-grade CLN1 plas­mid. Mile­stones add up to $56 mil­lion.

In its fil­ing, Taysha dis­closed that it’s al­so struck a deal with Queen’s Uni­ver­si­ty at Kingston to ac­cess some of its tech­nolo­gies, in­clud­ing plas­mid pro­duc­tion, for next-gen­er­a­tion gene ther­a­pies. In ad­di­tion to $3 mil­lion up­front, it re­im­bursed around $220,000 in costs and agreed to pay an­oth­er $20 mil­lion for mile­stones.

Secretary of health and human services Alex Azar speaking in the Rose Garden at the White House (Photo: AFP)

Trump’s HHS claims ab­solute au­thor­i­ty over the FDA, clear­ing path to a vac­cine EUA

The top career staff at the FDA have vowed not to let politics get in the way of science when looking at vaccine data this fall. But Alex Azar, who happens to be their boss’s boss, apparently won’t even give them a chance to stand in the way.

In a new memorandum issued Tuesday last week, the HHS chief stripped health agencies under his purview — including the FDA — of their rulemaking ability, asserting all such power “is reserved to the Secretary.” Sheila Kaplan of the New York Times first obtained and reported the details of the September 15 bulletin.

Dan Skovronsky, Eli Lilly CSO

UP­DAT­ED: An­a­lysts are quick to pan Eli Lil­ly's puz­zling first cut of pos­i­tive clin­i­cal da­ta for its Covid-19 an­ti­body

Eli Lilly spotlighted a success for one of 3 doses of their closely-watched Covid-19 antibody drug Wednesday morning. But analysts quickly highlighted some obvious anomalies that could come back to haunt the pharma giant as it looks for an emergency use authorization to launch marketing efforts.

The pharma giant reported that LY-CoV555, developed in collaboration with AbCellera, significantly reduced the rate of hospitalization among patients who were treated with the antibody. The drug arm of the study had a 1.7% hospitalization rate, compared to 6% in the control group, marking a 72% drop in risk.

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Eli Lilly CSO Dan Skovronsky (file photo)

#ES­MO20: Eli Lil­ly shows off the da­ta for its Verzenio suc­cess. Was it worth $18 bil­lion?

The press release alone, devoid of any number except for the size of the trial, added nearly $20 billion to Eli Lilly’s market cap back in June. Now investors and oncologists will get to see if the data live up to the hype.

On Sunday at ESMO, Eli Lilly announced the full results for its Phase III MonarchE trial of Verzenio, showing that across over 5,000 women who had had HR+, HER2- breast cancer, the drug reduced the odds of recurrence by 25%. That meant 7.8% of the patients on the drug arm saw their cancers return within 2 years, compared with 11.3% on the placebo arm.

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Greg Friberg (File photo)

#ES­MO20: Am­gen team nails down sol­id ear­ly ev­i­dence of AMG 510’s po­ten­tial for NSCLC, un­lock­ing the door to a wave of KRAS pro­grams

The first time I sat down with Amgen’s Greg Friberg to talk about the pharma giant’s KRAS G12C program for sotorasib (AMG 510) at ASCO a little more than a year ago, there was high excitement about the first glimpse of efficacy from their Phase I study, with 5 of 10 evaluable non-small cell lung cancer patients demonstrating a response to the drug.

After decades of failure targeting KRAS, sotorasib offered the first positive look at a new approach that promised to open a door to a whole new approach by targeting a particular mutation to a big target that had remained “undruggable” for decades.

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#ES­MO20: Out to beat Tagris­so, J&J touts 100% ORR for EGFR bis­pe­cif­ic/TKI com­bo — fu­el­ing a quick leap to PhI­II

J&J’s one-two punch on EGFR-mutant non-small cell lung cancer has turned up some promising — although decidedly early — results, fueling the idea that there’s yet room to one up on third-generation tyrosine kinase inhibitors.

Twenty out of 20 advanced NSCLC patients had a response after taking a combination of an in-house TKI dubbed lazertinib and amivantamab, a bispecific antibody targeting both EGFR and cMET engineered on partner Genmab’s platform, J&J reported at ESMO. All were treatment-naïve, and none has seen their cancer progress at a median follow-up of seven months.

#ES­MO20: As­traZeneca aims to spur PRO­found shift in prostate can­cer treat­ment with Lyn­parza OS da­ta

AstraZeneca has unveiled the final, mature overall survival data that cemented Lynparza’s first approval in prostate cancer approval — touting its lead against rivals with the only PARP inhibitor to have demonstrated such benefit.

But getting the Merck-partnered drug to the right patients remains a challenge, something the companies are hoping to change with the new data cut.

The OS numbers on the subgroup with BRCA1/2 or ATM-mutated metastatic castration-resistant prostate cancer are similar to the first look on offer when the FDA expanded the label in May: Lynparza reduced the risk of death by 31% versus Xtandi and Zytiga. Patients on Lynparza lived a median of 19.1 months, compared to 14.7 months for the anti-androgen therapies (p = 0.0175).

Exelixis CEO Michael Morrissey (file photo)

#ES­MO20: Look out Mer­ck. Bris­tol My­ers and Ex­elix­is stake out their com­bo’s claim to best-in-class sta­tus for front­line kid­ney can­cer

Now that the PD-(L)1 checkpoints are deeply entrenched in the oncology market, it’s time to welcome a wave of combination therapies — beyond chemo — looking to extend their benefit to larger numbers of patients. Bristol Myers Squibb ($BMY} and Exelixis {EXEL} are close to the front of that line.

Today at ESMO the collaborators pulled the curtain back on some stellar data for their combination of Opdivo (the PD-1) and Cabometyx (the TKI), marking a significant advance for the blockbuster Bristol Myers franchise while offering a big leg up for the team at Exelixis.

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Donald Trump and White House chief of staff Mark Meadows, before boarding Marine One (Getty Images)

Pric­ing deal col­laps­es over Big Phar­ma's re­fusal to is­sue $100 'cash card­s' be­fore the elec­tion — re­port

Late in August, as negotiations on a pricing deal with President Trump reached a boiling point, PhRMA president Stephen Ubl sent an email update to the 34 biopharma chiefs that sit on his board. He wrote that if the industry did not agree to pay for a $100 “cash card” sent to seniors before November, White House chief of staff Mark Meadows was going to tell the news media Big Pharma was refusing to “share the savings” with the elderly — and that all of the blame for failed drug pricing negotiations would lie squarely on the industry.

#ES­MO20: It’s not just Keytru­da any­more — Mer­ck spot­lights 3 top ear­ly-stage can­cer drugs

Any $12 billion megablockbuster in the portfolio tends to overshadow everything else in the pipeline. Which is something Merck can tell you a little bit about.

Keytruda not only dominates the PD-(L)1 field, it looms over everything Merck does, to the point some analysts wonder if Merck is a one-trick pony.

There’s no shortage of Keytruda data on display at ESMO this weekend, but now the focus is shifting to the future role of new drugs and combos in maintaining that lead position for years to come. And the pharma giant has a special focus for 3 early-stage efforts where Roger Perlmutter’s oncology team is placing some big bets.

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