Gene ther­a­py up­start re­cruits more ex-AveX­is tal­ent; I-Mab re­veals first-half re­sults

It hasn’t been hard to see where Sean Nolan went look­ing for the tal­ent need­ed to make Taysha in­to a lead­ing play­er in the bustling gene ther­a­py field.

Sean Nolan

Mon­day morn­ing the com­pa­ny, where Nolan holds the chair­man’s post, put out word that Taysha re­cruit­ed the ex-CMO and ex-CFO at AveX­is to come on­to the board in the wake of a $94 mil­lion crossover raise.

In this en­vi­ron­ment, it’s yet an­oth­er sign that Nolan and the top crew could have their sights set on an IPO as the biotech up­start com­pletes the full team.

Tapped for the board are Suku­mar Na­gen­dran, who had been CMO at AveX­is when No­var­tis stepped in to buy out the SMA com­pa­ny for $8.7 bil­lion. Then there’s the num­bers guy, Phillip Do­nen­berg, who gained ex­pe­ri­ence work­ing with the SEC that could soon come in handy.

They’re join­ing a full boat of ex­ecs lead­ing the way at the Dal­las-based biotech, in­clud­ing: Bio­Marin vet Suyash Prasad as CMO; an­oth­er AveX­is alum Kam­ran Alam as CFO; AveX­is’ for­mer BD chief Dan Ja­ni­ak and Sean McAu­li­ffe, who helped bring out Zol­gens­ma and now holds the top com­mer­cial post at Taysha.

They’re all work­ing with CEO RA Ses­sion II, who came out of the en­tre­pre­neur-in-res­i­dence pro­gram at UT South­west­ern, which is pro­vid­ing the gene ther­a­py can­di­dates for Taysha. — John Car­roll

I-Mab re­leas­es first half 2020 re­sults with much tri­al da­ta soon ex­pect­ed

The Chi­nese biotech I-Mab Bio­phar­ma $IMAB re­leased its first half 2020 fi­nan­cial re­sults, giv­ing a win­dow in­to the op­er­a­tions of the com­pa­ny.

Among the most news­wor­thy as­pects of the up­date in­clud­ed the pre­view of sev­er­al tri­al read­outs to come be­fore the end of the year, in­clud­ing an ex­pect­ed IND ap­proval in Chi­na for a hor­mone ther­a­py in pe­di­atric growth hor­mone de­fi­cien­cy. The com­pa­ny will like­ly ini­ti­ate a Phase III tri­al for the can­di­date, ef­tan­so­ma­t­ropin, soon af­ter­wards.

I-Mab al­so di­vulged that its cash po­si­tion was $221 mil­lion at the end of June, as op­posed to about $175 mil­lion at the start of the year. The biotech re­port­ed a loss of $82.5 mil­lion over the last six months, as op­posed to about $125 mil­lion in the first six months of 2019.

The com­pa­ny’s busi­ness mod­el is rather un­usu­al, as it con­ducts proof-of-con­cept tri­als in the US, and then us­es the da­ta for tri­als in Chi­na. Once the drug is clin­i­cal­ly val­i­dat­ed in the US, I-Mab re­tains Chi­nese rights for fur­ther de­vel­op­ment and glob­al out-li­cens­ing.

It’s a bet that’s paid off so far, as I-Mab raised over $400 mil­lion in pri­vate fund­ing be­fore go­ing pub­lic in Jan­u­ary with a $107.3 mil­lion IPO raise. I-Mab’s stock price has since risen over 150% from a $14 open­ing price to over $31 in ear­ly Mon­day trad­ing. — Max Gel­man

Ar­rakis us­es pho­toaffin­i­ty la­bel­ing to iden­ti­fy RNA bind­ing sites

Ar­rakis Ther­a­peu­tics read out da­ta on the abil­i­ty of its PEARL-seq tech­nol­o­gy to iden­ti­fy bind­ing sites in RNA mol­e­cules — a step for­ward for the de­vel­op­ment of its pipeline of RNA-tar­get­ed drugs.

Jen­nifer Pet­ter

“PEARL-seq rep­re­sents our in­no­v­a­tive ap­proach to es­tab­lish tar­get en­gage­ment, iden­ti­fy bind­ing sites on the RNA, and learn about se­lec­tiv­i­ty across the tran­scrip­tome,” Ar­rakis founder and CSO Jen­nifer Pet­ter said in a state­ment. “As part of Ar­rakis’ mis­sion to drug RNA, we are build­ing and re­fin­ing the tools need­ed to dis­cov­er and de­sign rSMs against im­por­tant RNA tar­gets in­volved in dis­ease.”

The da­ta show that the tech — which stands for pho­toaffin­i­ty eval­u­a­tion of RNA Lig­a­tion-se­quenc­ing — can iden­ti­fy lig­and bind­ing sites and se­lec­tiv­i­ty “across RNAs in a sin­gle ex­per­i­ment,” ac­cord­ing to the com­pa­ny. Ar­rakis’ pipeline in­cludes var­i­ous RNA-tar­get­ing mech­a­nisms, in­clud­ing ones for can­cer, car­dio­vas­cu­lar con­di­tions, neu­rode­gen­er­a­tion and rare dis­eases.

“The re­search showed that pho­toaffin­i­ty la­bels could be teth­ered to rSMs with­out dis­rupt­ing the bind­ing in­ter­ac­tions be­tween the rSM and the RNA. Up­on ac­ti­va­tion with UV light, the rSM ir­re­versibly crosslinks to the RNA, en­abling map­ping of the in­ter­ac­tion by se­quenc­ing analy­sis,” the com­pa­ny stat­ed. — Nicole De­Feud­is

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.

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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.

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.

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#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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