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

George Scangos (L) and Marianne De Backer

Pi­o­neer­ing biotech icon George Scan­gos hands in his re­tire­ment pa­pers — and this time it’s for re­al

George Scangos, one of the all-time great biotech CEOs, says the time has come to turn over the reins one last time.

The 74-year-old biotech legend spent close to three decades in a CEO post. The first was at Exelixis — which is still heavily focused on a drug Scangos advanced in the clinic. The second “retirement” was at Biogen, where he and his team were credited with a big turnaround with the now fading MS blockbuster Tecfidera. And the third comes at Vir, where he traded in his Big Biotech credentials for a marquee founder’s role back on the West Coast, hammering out a Covid-19 alliance with Hal Barron — then R&D chief at GSK — and breaking new ground on infectious diseases with some high-powered venture players.

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Jeanne Loring, director of the Center for Regenerative Medicine (Credit: Jamie Scott Lytle)

A stem cell pi­o­neer sent an ex­per­i­ment in­to space. Pa­tients are the next fron­tier

Last July, Jeanne Loring stood on a dirt road surrounded by Florida swampland and watched as a nearby SpaceX rocket blasted into the sky. The payload included a very personal belonging: cell clusters mimicking parts of her brain.

For more than two decades, Loring has been at the forefront of a stem cell field that always seems on the brink of becoming the next thing in medicine, but has been slow to lift off.

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FDA re­ports ini­tial 'no sig­nal' for stroke risk with Pfiz­er boost­ers, launch­es con­comi­tant flu shot study

The FDA hasn’t detected any potential safety signals, including for stroke, in people aged 65 years and older who have received Pfizer’s bivalent Covid booster, one senior official told members of the agency’s Vaccines and Related Biological Products Advisory Committee (VRBPAC) on Thursday.

The update comes as the FDA and CDC investigate a “preliminary signal” that may indicate an increased risk of ischemic stroke in older Americans who received Pfizer’s updated shot.

FDA cuts off use for As­traZeneca’s Covid-19 ther­a­py Evusheld

The FDA has stopped use of another drug as a result of the new coronavirus variants. On Thursday, the agency announced that AstraZeneca’s antibody combo Evusheld, which was an important prevention option for many immunocompromised people and others, is no longer authorized.

The FDA said it made its decision based on the fact that Evusheld works on fewer than 10% of circulating variants.

Evusheld was initially given emergency authorization at the end of 2021. However, as Omicron emerged, so did studies that showed Evusheld might not work against the dominant Omicron strain. In October, the FDA warned healthcare providers that Evusheld was useless against the Omicron subvariant BA.4.6. It followed that up with another announcement earlier this month that it did not think Evusheld would work against the latest Omicron subvariant XBB.1.5.

Evotec inks li­cense agree­ment with J&J; On­colyt­ic virus biotech prices $15M IPO

Drug discovery and development player Evotec entered another licensing deal with Big Pharma — this time with J&J’s Janssen.

The companies put out word that they entered into a strategic collaboration and license agreement with each other that focuses on targeted immune-based cancer therapies, to be commercialized by Janssen.

According to a statement, the collaboration will hinge on Evotec’s integrated drug discovery and manufacturing capabilities. During the pre-clinical R&D phase, the companies will collaborate closely — and then Janssen will take on full responsibility for both clinical development and commercialization.

In­vestor 'misalign­men­t' leads to tR­NA biotech's shut­ter­ing

A small biotech looking to carve a lane in the tRNA field has folded, an investor and a co-founder confirmed to Endpoints News.

Similar to Flagship’s Alltrna and other upstarts like Takeda-backed hC Bioscience, the now-shuttered Theonys was attempting to go after transfer RNA, seen as a potential Swiss Army knife in the broader RNA therapeutics space. The idea is that one tRNA drug could be used across a galaxy of disorders and diseases.

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#JPM23: What's re­al­ly dri­ving the cost of health­care and drugs in 2023?

Executive Editor Drew Armstrong spoke with PhRMA CEO Steve Ubl, EmsanaRx CEO Greg Baker and ICER President Steve Pearson about how the debate over drug costs has changed (or not) in the last decade, the shifting payer landscape and why there seems to be so little movement on drug rebates. This transcript has been edited for brevity and clarity.

Drew Armstrong:

So first of all, thank you to everybody for being here and for our panel for being here. Incredibly excited to have this discussion on the cost of healthcare and drugs and what’s driving that. We’re here with Steve Ubl, the head of PhRMA. Thank you so much. Steve Pearson from ICER, and Greg Baker from EmsanaRx. I want to start this conversation with a little bit of a personal reminiscence. So about almost 10 years exactly. I was a reporter back in my previous job and I was covering drug pricing and Gilead had just launched their hepatitis C drug and I was having a conversation with another Steve over at Express Scripts and he made some comments essentially about how they intended to launch a price war over hepatitis C therapies.

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Pa­tient death spurs tri­al halt for Ma­gen­ta Ther­a­peu­tics

Magenta Therapeutics is pausing an early-stage clinical trial after a patient died. The death was deemed to be possibly related to its drug, MGTA-117.

The biotech said the pause of the Phase I/II trial is voluntary and gives it time to review all available data before deciding what to do next. It’s also reported the known information to the FDA.

The dose-escalation trial was designed to test whether MGTA-117, an antibody-drug conjugate, could serve as a more targeted alternative to high-intensity chemotherapy as a conditioning agent for cancer patients who are set to receive a stem cell transplant. It recruited patients with relapsed/refractory acute myeloid leukemia and myelodysplastic syndrome.

In a win for Re­gen­eron, No­var­tis' sy­ringe for AMD drug de­clared 'un­patentable'

Regeneron has won a patent case against Swiss pharma giant Novartis over the delivery system for its eye drug Eylea.

The US Patent Trial and Appeal Board ruled that Novartis’ pre-filled syringe for injecting its eye medication Lucentis was “unpatentable” and handed the victory to Regeneron and its AMD drug Eylea.

In the initial complaint in 2020, Novartis alleged to the US International Trade Commission that certain pre-filled syringes for the intravitreal injection, and ultimately Regeneron’s delivery system for Eylea, were infringing on Novartis’ patent. Regeneron filed a petition to review Novartis’ claims in 2021.