Taysha buys clin­ic-ready gene ther­a­py from Abeona, bring­ing Steven Gray's port­fo­lio back in one place

Less than four months af­ter Taysha launched with a suite of AAV gene ther­a­pies de­vel­oped at UT South­west­ern — and two weeks af­ter the ex-AveX­is team raised $95 mil­lion in fresh cash — the biotech is look­ing to its chief sci­en­tif­ic ad­vi­sor for an­oth­er as­set.

The new pro­gram is ABO-202, which is de­signed to treat CLN1 dis­ease (or in­fan­tile Bat­ten dis­ease) by de­liv­er­ing a cor­rect copy of the PPT1 gene with a mod­i­fied AAV9 vec­tor.

Steven Gray

Steven Gray de­vel­oped the ther­a­py while at UNC-Chapel Hill, where he had stud­ied un­der Jude Samul­s­ki years ago. Abeona li­censed it in 2016, built out the clin­i­cal tri­al pro­to­col and man­u­fac­tur­ing process, and has since had an IND for a Phase I/II clin­i­cal tri­al cleared with the FDA.

Taysha is pay­ing on­ly $7 mil­lion up­front to get its hands on ABO-202, with $3 mil­lion in li­cense fees and $4 mil­lion for the in­ven­to­ry — in­clud­ing clin­i­cal-grade CLN1 plas­mid. Abeona is el­i­gi­ble to re­ceive up to $56 mil­lion down the road for de­vel­op­ment and sales mile­stones, plus roy­al­ties.

The deal puts Taysha on track to be­gin two clin­i­cal stud­ies next year: both the CLN1 pro­gram and an­oth­er gene ther­a­py for Tay-Sachs dis­ease. Three more INDs are on the dock­et for 2021.

RA Ses­sion

“CLN1 is a pro­gres­sive mono­genic CNS dis­ease with sig­nif­i­cant un­met med­ical need, and we be­lieve the ABO-202 da­ta gen­er­at­ed thus far demon­strate great trans­la­tion­al po­ten­tial and of­fer hope to chil­dren suf­fer­ing from this dev­as­tat­ing dis­or­der,” CEO RA Ses­sion II said.

Gray, who now leads 50 trans­la­tion­al sci­en­tists and man­ages a GMP man­u­fac­tur­ing site at UT South­west­ern’s vi­ral vec­tor core, will con­tin­ue to ad­vise the com­pa­ny along­side Berge Mi­nass­ian, the di­vi­sion chief of child neu­rol­o­gy.

Rather than shep­herd­ing one as­set at a time like the first gen­er­a­tion of gene ther­a­py play­ers, Taysha had opt­ed for a port­fo­lio ap­proach to ad­vance mul­ti­ple gene ther­a­pies in par­al­lel. It’s look­ing to build three big fran­chis­es: ge­net­ic forms of epilep­sy, neu­rode­vel­op­men­tal dis­or­ders and neu­rode­gen­er­a­tive dis­or­ders. And al­though the crew has eyes on new­er tech­nolo­gies like re­dos­ing, bi­cistron­ic plas­mids, mi­croR­NA knock­down, hair­pin tech­nol­o­gy to turn on a silent al­lele and de­liv­ery through the va­gus nerve, the cor­ner­stone re­mains ade­no-as­so­ci­at­ed virus (AAV).

“If it’s not broke, you don’t fix it,” Ses­sion told End­points News back in April.

Sean Nolan

Taysha, which is chaired by for­mer AveX­is CEO Sean Nolan, has re­served rights to tap four more pro­grams out of Gray’s lab.

For Abeona, the deal with Taysha gives them time and some cash to fo­cus on its more ad­vanced pro­grams in re­ces­sive dy­s­troph­ic epi­der­mol­y­sis bul­losa, San­fil­ip­po syn­drome type A and San­fil­ip­po syn­drome type B as it re­cov­ers from a some­what tu­mul­tuous 2019.

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.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 90,400+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 90,400+ biopharma pros reading Endpoints daily — and it's free.

#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).

#ES­MO20: Trodelvy da­ta show that Gilead­'s $21B buy­out may have been worth the big pre­mi­um

Gilead CEO Dan O’Day has been on a shopping spree. And while some analysts gawked at the biotech’s recent $21 billion Immunomedics buyout, new data released at virtual ESMO 2020 suggest the acquisition may have been worth the hefty price.

The deal, announced last weekend, will give California-based Gilead $GILD Trodelvy, which was recently approved for metastatic triple-negative breast cancer (mTNBC).

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 90,400+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 90,400+ biopharma pros reading Endpoints daily — and it's free.

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.

Dan Skovronsky, Eli Lilly CSO

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 90,400+ biopharma pros reading Endpoints daily — and it's free.