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

Janet Woodcock (AP Images)

Janet Wood­cock is in the run­ning for FDA com­mis­sion­er — what does that mean for the agen­cy's fu­ture?

Just a day after reports emerged that Janet Woodcock will serve as interim chief of the FDA, word has gotten out that she is also in the running for the permanent job.

The decision, as the initial wave of reactions suggest, could have dramatic implications for where the agency is headed in the next four years — if not beyond.

Woodcock, the longtime CDER director, is being vetted alongside former FDA principal deputy commissioner Joshua Sharfstein, Bloomberg reported. Already tapped as acting head of the agency, she’s set to take over from Stephen Hahn right after Biden’s inauguration next week.

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Janet Woodcock (AP Images)

Janet Wood­cock to be act­ing FDA com­mis­sion­er while Biden team fi­nal­izes nom­i­nee — re­ports

Janet Woodcock is set to be the most powerful person at the FDA in less than a week.

The veteran regulator and longtime director of the Center for Drug Evaluation and Research has been tapped as acting commissioner of the FDA, according to reports by BioCentury’s Steve Usdin and Pink Sheet’s Sarah Karlin-Smith.

The appointment was requested by the incoming Biden team, Karlin-Smith added, as they sort out the nomination of a permanent successor to Stephen Hahn — whose one-year tenure has been defined by Covid-19.

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News brief­ing: Five Prime fi­nal­izes PhI­II plans for gas­tric can­cer; AI di­ag­nos­tics-fo­cused Paige ex­pands staff

Five Prime Therapeutics has finalized a plan to take their comeback gastric cancer drug into late-stage studies.

The South San Francisco-based biotech released full Phase II data for bemarituzumab on Friday, which Five Prime said in November met all of its pre-specified efficacy endpoints in a topline readout. Now, the company is announcing it plans to launch a Phase III trial for the program in 2021. Following November’s readout, the future of bemarituzumab had not yet been finalized.

Steve Harr (L) and Hans Bishop

Paint­ing by the num­bers, Sana founders carve up a gi­ant uni­corn-sized IPO — for a biotech that has­n't quite made it to the clin­ic

Sana Biotechnology is one of those startups that was sketched in on the chalkboard day one in the shape of a unicorn.

A giant unicorn.

And from the numbers the cell therapy 2.0 play spelled out in their S-1 $SANA, it’s clear that the company founders — led by a pair of major VCs aligned with some high-profile industry figures — are hunting a big chunk of that value for themselves.

The raise they penciled in — $150 million — isn’t likely what they actually have in mind, and it doesn’t do justice to the size of their ambitions.

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CEO Brett Monia (Ionis)

Can Brett Mo­nia push Io­n­is be­yond Spin­raza?

For 30 years, Brett Monia struggled as one of Ionis’ top scientists to get their antisense technology to work. Now, as CEO, he’s trying to use it to turn Ionis into one of the industry’s biggest biotechs.

Monia, one of the handful of young scientists who in 1989 followed Stanley Crooke across the country from SmithKline (now GSK) in Philadelphia to found Ionis in Northern California, replaced Crooke as CEO last January. By then, they had proven antisense, an RNA-based method for manipulating gene expression, could work dramatically well in at least some instances, transforming spinal muscular atrophy with the Biogen-partnered blockbuster Spinraza.

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David Kessler in April 2009 (Eric Risberg/AP Images)

Covid-19 roundup: Hack­ers start re­leas­ing 'ma­nip­u­lat­ed' Covid-19 vac­cine docs; Ex-FDA com­mish David Kessler to re­place Mon­cef Slaoui as Op­er­a­tion Warp Speed chief — re­port

There’s a new twist on the EMA Covid-19 hacking story.

Friday the European agency put out the 5th in a series of statements about the hackers who broke into their system, noting that some of the information on vaccines that was gleaned in the attack is showing up online — altered to raise questions about the Covid-19 vaccines now in use.

This included internal/confidential email correspondence dating from November, relating to evaluation processes for COVID-19 vaccines. Some of the correspondence has been manipulated by the perpetrators prior to publication in a way which could undermine trust in vaccines.

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Nadim Ahmed (Bristol Myers Squibb)

Bris­tol My­er­s' top hema­tol­ogy ex­ec is on his way out — right on the heels of a $6B CVR im­plo­sion

Fourteen days after the $6.3 billion CVR tied to the approval of liso-cel went up in smoke, one of the top execs in charge of the work at Bristol Myers Squibb is preparing to step out of his job.

Mizuho analyst Salim Syed, who’s been following every twist and turn in the CVR saga, told investors on Thursday morning that Nadim Ahmed is on his way out. Syed’s note:

Recall, Ahmed is EVP and President of Hematology at BMY (i.e. JCAR017 and bb2121 are both hematological drugs). He’s still listed on the BMY management page. This is true — he’s still technically there. However, I have confirmed w/ BMY that his last day is tomorrow, Friday 1/15. To my best knowledge, Ahmed does not have another job lined up post his departure tomorrow.

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UP­DAT­ED: Am­gen tops cost watch­dog's price gougers list based on 'un­sup­port­ed' in­creas­es for En­brel with­out mean­ing­ful da­ta

In a top 10 ranking of the most egregious price gougers from 2019, Amgen’s Enbrel topped US cost watchdog ICER’s naughty list with “unsupported” markups that added as much as $403 million to the nation’s drug spend during that time.

Price increases for some of pharma’s most popular drugs have long been a focus of consumer ire, but the industry has argued those increases are routine and meant to cover the cost of R&D innovation. Without meaningful guidance at the state or federal level, ICER looked to connect how much a drug had progressed in the clinic compared with its increase in both wholesale and net price in 2019.

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News brief­ing: Pre­ci­sion Med­i­cine Group ac­quires cell and gene ther­a­py-fo­cused ser­vices firm Pro­ject Far­ma; Roost­er­Bio and Sar­to­rius col­lab on gene ther­a­py man­u­fac­tur­ing

As more and more players pursue cell and gene therapies, companies are looking for ways to keep up on the manufacturing end.

On Thursday, Precision Medicine Group took the wraps off a deal to buy out Project Farma, a bioengineering services firm that supports the manufacturing and scale-up of cell and gene therapies. Project Farma will join Precision for Medicine, PMG’s research and development services arm.