Play­ing catch-up, Roche out­lines plans for a PhI­II he­mo­phil­ia A pro­gram — next year

For now, the big fo­cus in the he­mo­phil­ia A gene ther­a­py race has been cen­tered on fron­trun­ner Bio­Marin, with an ex­pect­ed OK just weeks away, and the crew from Sang­amo/Pfiz­er, which has been look­ing to play catch-up with a piv­otal study in the works.

But now Roche’s new gene ther­a­py di­vi­sion at Spark has up­dat­ed its own sched­ule, say­ing they’re look­ing at a Phase III start some­time next year as they op­ti­mize the dose and im­munomod­u­la­to­ry reg­i­men in or­der to avoid any fu­ture mishaps that could de­rail the pro­gram.

We al­ready knew that 2 of 9 par­tic­i­pants in the 2×1012 dose co­hort “lost FVI­II ex­pres­sion like­ly due to a cap­sid-based im­mune re­sponse,” says the com­pa­ny. “The sev­en oth­er par­tic­i­pants in the 2×1012 dose co­hort and the five to­tal par­tic­i­pants in the 5×1011 vg/kg and 1×1012 vg/kg dose co­horts con­tin­ue to show sta­ble and durable fac­tor FVI­II ex­pres­sion. These da­ta rep­re­sent the longest sta­ble ex­pres­sion of FVI­II fol­low­ing in­ves­ti­ga­tion­al gene ther­a­py and re­in­force the abil­i­ty of AAV gene ther­a­py tar­get­ing he­pa­to­cytes to achieve sta­ble and durable FVI­II ex­pres­sion.”

SPK-8011 was linked to a 91% re­duc­tion in an­nu­al­ized bleed rate and a 96% drop in fac­tor VI­II in­fu­sions. And they have da­ta stretch­ing out past 3 years.

With such small num­bers, though, any set­backs can af­flict these pro­grams with a lin­ger­ing sting. And with sev­er­al gene ther­a­pies in the mix now, an­a­lysts won­der which will be the dom­i­nant play­er.

5AM Ven­tures: Fu­el­ing the Next Gen­er­a­tion of In­no­va­tors

By RBC Capital Markets
With Andy Schwab, Co-Founder and Managing Partner at 5AM Ventures

Key Points

Prescription Digital Therapeutics, cell therapy technologies, and in silico medicines will be a vital part of future treatment modalities.
Unlocking the potential of the microbiome could be the missing link to better disease diagnosis.
Growing links between academia, industry, and venture capital are spinning out more innovative biotech companies.
Biotech is now seen by investors as a growth space as well as a safe haven, fuelling the recent IPO boom.

Biohaven CEO Vlad Coric (Photo Credit: Andrew Venditti)

Pssst: That big Bio­haven Alzheimer's study? It was a bust. Even the sub­group analy­sis ex­ecs tout­ed was a flop

You know it’s bad when a biopharma player plucks out a subgroup analysis for a positive take — even though it was way off the statistical mark for success, like everything else.

So it was for Biohaven $BHVN on MLK Monday, as the biotech reported on the holiday that their Phase II/III Alzheimer’s study for troriluzole flunked both co-primary endpoints as well as a key biomarker analysis.

The drug — a revised version of the ALS drug riluzole designed to regulate glutamate — did not “statistically differentiate” from placebo on the Alzheimer’s Disease Assessment Scale-Cognitive Subscale 11 (ADAS-cog) and the Clinical Dementia Rating Scale Sum of Boxes (CDR-SB).  The “hippocampal volume” assessment by MRI also failed to distinguish itself from placebo for all patients fitting the mild-to-moderate disease profile they had established for the study.

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Janet Woodcock and Joshua Sharfstein (AP, Images)

Poll: Should Joshua Sharf­stein or Janet Wood­cock lead the FDA from here?

It’s time for a new FDA commissioner to come on board, a rite of passage for Joe Biden’s administration that should help seal the new president’s rep on seeking out the experts to lead the government over the next 4 years.

As of now, the competition for the top job appears to have narrowed down to 2 people: The longtime CDER chief Janet Woodcock and Joshua Sharfstein, the former principal deputy at the FDA under Peggy Hamburg. Both were appointed by Barack Obama.

Dan Skovronsky, Eli Lilly CSO (Lilly via Facebook)

Eli Lil­ly tees up dis­cov­ery pact worth more than $1.6B with Merus for T cell-fo­cused bis­pe­cif­ic an­ti­bod­ies

Under science chief Dan Skovronsky, Eli Lilly has taken some big swings at next-gen therapies, including trying to find the next big thing in oncology. Now, after one early failure in the field, Lilly is going back to the bispecific antibody well with a new deal with a Dutch biotech.

Lilly will pay $40 million upfront with an additional $20 million equity stake in Merus NV to identify and develop three bispecific antibodies looking to engage the CD3 antigen on T cells and redirect immune cells, the Indianapolis pharma giant said Tuesday.

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Sekar Kathiresan (Verve)

'Bluest of blue-chip in­vestors' pump $94M in­to Sekar Kathire­san's quest to bring CRISPR to the heart

Going into his first JP Morgan conference, Sekar Kathiresan had some major news to share. His ambitious cardiovascular-focused startup, Verve Therapeutics, had designated the PCSK9 base editor as its lead program, homed in on severe heterozygous familial hypercholesterolemia as the first indication, and released animal data suggesting the in vivo edits to the monkey livers last up to six months. For a company that had just been in the public for less than a year, “it was wonderful.”

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Andrew Allen (Gritstone)

As coro­n­avirus vari­ants trig­ger new alarms, the NIH is putting an un­der-the-radar ‘next-gen’ vac­cine in­to PhI

Over the past year, the world has been transfixed by the development of new vaccines to fight SARS-CoV-2. In a frenzy of activity, the new mRNA approach has delivered pioneering emergency approvals in record time. And with some setbacks, the more traditional big players are coming along with added jabs as the most affluent nations in the world begin to vaccinate large portions of their populations.

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The IPO queue adds 5 more biotechs hop­ing to ring in 2021 by blitz­ing Nas­daq

Following a record year for IPOs — in terms of both proceeds and count — there’s already a long lineup of biotechs ready to jump onto Nasdaq in the new year. The companies are likely looking for much higher raises than they initially projected on their S-1s. Now it’s time to see if investors are still hungry for another round of biotech stocks.

Sana helped set the pace early on, as its founders look to divvy up a fortune from their IPO. And late last week 5 more biotechs crowded in, looking to pick up the pace where 2020 left off. Here they are:

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News brief­ing: Beam bags a $260M pri­vate place­ment; mi­Ra­gen re­brands to Virid­i­an Ther­a­peu­tics

Agios vet John Evans has demonstrated how to raise big money for a little biotech.

The Beam Therapeutics CEO — and ARCH partner — has pieced together a $260 million private placement from a group of backers that includes Perceptive Advisors, Farallon Capital, Casdin Capital, Redmile Group and Cormorant Asset Management. And there are 3 main goals they’ll pursue with it: clinical development, strategic partnerships and general corporate purposes.

As­traZeneca keeps the ball rolling on Dai­ichi-part­nered En­her­tu, pick­ing up 2nd in­di­ca­tion in gas­tric can­cer

AstraZeneca’s big gamble on Daiichi Sankyo’s antibody-drug conjugate Enhertu has already paid off with a big approval in breast cancer more than a year ago. But the partners have big plans for their blockbuster in the making, and a new nod in gastric cancer will raise their spirits even higher.

The FDA on Friday approved Enhertu to treat locally advanced or metastatic HER2-positive gastric or gastroesophageal junction adenocarcinoma in patients who have previously undergone at least one round of treatment with a Herceptin-based regimen, AstraZeneca said in a release.