Genen­tech takes aim at Bio­gen and No­var­tis, high­light­ing an ear­ly look at pos­i­tive re­sults for their oral SMA drug from PTC

Af­ter get­ting hit with a set­back that forced the phar­ma gi­ant Roche to aban­don its lead drug for spinal mus­cu­lar at­ro­phy, com­pa­ny ex­ecs are back with a new drug in-li­censed from PTC $PTCT that they be­lieve has legs.

It’s an in­ter­im, up­dat­ed look at a tiny da­ta set for ris­diplam (or RG7916), but re­searchers spot­light­ed ev­i­dence that the drug could help sig­nif­i­cant per­cent­ages of in­fants sit, kick and con­trol their head move­ments — all el­e­ments that the dis­ease de­stroys as it re­lent­less­ly push­es these chil­dren to an ear­ly death.

San­dra Horn­ing

This is a sin­gle-arm study be­ing pur­sued by Genen­tech, the same kind of ap­proach that AveX­is scored on in the sum­mer of 2017 for the gene ther­a­py AVXS-101, when not­ed in­ves­ti­ga­tor Jer­ry Mendell and his team high­light­ed their own im­pres­sive re­sults in a small study. That set of da­ta pro­pelled the biotech to out­line plans for an ac­cel­er­at­ed ap­proval, as well as in­spir­ing No­var­tis’ Vas Narasimhan to buy the com­pa­ny for $8.7 bil­lion. It’s still wide­ly con­sid­ered the top con­tender.

No­var­tis al­so has an oral drug sim­i­lar to Roche’s called LMI070 (branaplam), which got back in­to the clin­ic last fall af­ter it was forced in­to a 2-year hia­tus trig­gered by safe­ty fears raised by an­i­mal stud­ies that had high­light­ed the risk of nerve dam­age.

Roche turned to its oral SMN2 splic­ing mod­i­fi­er last sum­mer af­ter reg­u­la­tors on both sides of the At­lantic de­mand­ed new late-stage work be­fore they would con­sid­er ap­prov­ing their drug ole­soxime. Roche punt­ed the drug in­stead.

Both No­var­tis and Roche are now hot on the trail of Spin­raza, the pi­o­neer­ing drug in the field from Bio­gen, which was priced at $750,000 — mak­ing it one of the most ex­pen­sive drugs on the plan­et. It’s al­so ripe for be­ing over­tak­en by oth­ers. And Baird’s Bri­an Sko­r­ney likes Roche’s chances with this drug. He not­ed:

The da­ta is com­pelling so far, and sug­gests that ris­diplam could im­pact Spin­raza’s mar­ket share, if it comes to mar­ket, giv­en its con­ve­nience as an oral small mol­e­cule.

In the FIRE­FISH study, re­searchers re­port­ed that a key score on dis­ease pro­gres­sion showed more than half of the in­fants with type 1 SMA hit 40-plus. They not­ed:

The me­di­an CHOP-IN­TEND scores in­creased over time (37.5 at 6 months [n=20] com­pared to 41.5 at eight months [n=14]). The me­di­an age at first dose in FIRE­FISH was 6.7 months and me­di­an treat­ment du­ra­tion was 9.5 months. Nine­teen out of 21 in­fants en­rolled (90 per­cent) re­main alive with two hav­ing dis­con­tin­ued due to the fa­tal pro­gres­sion of their dis­ease. Three pa­tients are now over 24 months old. No in­fant has re­quired a tra­cheosto­my or per­ma­nent ven­ti­la­tion since study ini­ti­a­tion, and no in­fant has lost the abil­i­ty to swal­low.

In the Sun­fish study for type 2 and 3 SMA, they found:

Of the pa­tients treat­ed with ris­diplam for at least one year (n=30), the me­di­an change from base­line in Mo­tor Func­tion Mea­sure (MFM), the pri­ma­ry end­point in the con­fir­ma­to­ry part of SUN­FISH and a scale used to as­sess mo­tor func­tion in neu­ro­mus­cu­lar dis­eases, was a 3.1-point im­prove­ment. Six­ty-three per­cent of pa­tients ex­pe­ri­enced an im­prove­ment in MFM over base­line of three points or more af­ter one year. Such im­prove­ments were seen both in pa­tients un­der 12 years old (76 per­cent; n=17) and over 12 years old (46 per­cent; n=13).

“SMA ther­a­pies that pro­duce a sus­tained in­crease in SMN pro­tein in both the CNS and pe­riph­ery may pro­vide com­pre­hen­sive ben­e­fits to peo­ple di­ag­nosed with SMA, and we look for­ward to shar­ing ad­di­tion­al da­ta on ris­diplam as the clin­i­cal pro­gram pro­gress­es,” said CMO San­dra Horn­ing in a state­ment.

Tar­get­ing a Po­ten­tial Vul­ner­a­bil­i­ty of Cer­tain Can­cers with DNA Dam­age Re­sponse

Every individual’s DNA is unique, and because of this, every patient responds differently to disease and treatment. It is astonishing how four tiny building blocks of our DNA – A, T, C, G – dictate our health, disease, and how we age.

The tricky thing about DNA is that it is constantly exposed to damage by sources such as ultraviolet light, certain chemicals, toxins, and even natural biochemical processes inside our cells.¹ If ignored, DNA damage will accumulate in replicating cells, giving rise to mutations that can lead to premature aging, cancer, and other diseases.

Roivant par­lays a $450M chunk of eq­ui­ty in biotech buy­out, grab­bing a com­pu­ta­tion­al group to dri­ve dis­cov­ery work

New Roivant CEO Matt Gline has crafted an all-equity upfront deal to buy out a Boston-based biotech that has been toiling for several years now at building a supercomputing-based computational platform to design new drugs. And he’s adding it to the Erector set of science operations that are being built up to support their network of biotech subsidiaries with an eye to growing the pipeline in a play to create a new kind of pharma company.

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Fol­low biotechs go­ing pub­lic with the End­points News IPO Track­er

The Endpoints News team is continuing to track IPO filings for 2021, and we’ve designed a new tracker page for the effort.

Check it out here: Biopharma IPOs 2021 from Endpoints News

You’ll be able to find all the biotechs that have filed and priced so far this year, sortable by quarter and listed by newest first. As of the time of publishing on Feb. 25, there have already been 16 biotechs debuting on Nasdaq so far this year, with an additional four having filed their S-1 paperwork.

Ken Frazier, Merck CEO (Bess Adler/Bloomberg via Getty Images)

UP­DAT­ED: Mer­ck takes a swing at the IL-2 puz­zle­box with a $1.85B play for buzzy Pan­dion and its au­toim­mune hope­fuls

When Roger Perlmutter bid farewell to Merck late last year, the drugmaker perhaps best known now for sales giant Keytruda signaled its intent to take a swing at early-stage novelty with the appointment of discovery head Dean Li. Now, Merck is signing a decent-sized check to bring an IL-2 moonshot into the fold.

Merck will shell out roughly $1.85 billion for Pandion Pharmaceuticals, a biotech hoping to gin up regulatory T cells (Tregs) to treat a range of autoimmune disorders, the drugmaker said Thursday.

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Roche and Genen­tech re­searchers plot $53M dis­cov­ery quest aimed at spark­ing a 'Holy moly' piv­ot in neu­ro R&D

Roche and Genentech have committed $53 million to back a 10-year quest aimed at going back to the drawing board to use new technology and fresh scientific insights to generate a pipeline of drugs for neurological diseases.

Researchers from both Roche and its big South San Francisco hub — mixing teams from gRED and pRED this time — will mix it up with the scientists drawn together for the Weill Neurohub — formed in 2019 as a joint research partnership involving UCSF, Berkeley and the University of Washington — in an exploration of the field to develop new therapies for some of the toughest diseases in drug R&D: Alzheimer’s, Parkinson’s, Huntington’s, ALS and autism.

Am­gen, As­traZeneca speed to­ward fil­ing next-gen an­ti­body for asth­ma af­ter un­cork­ing full late-stage da­ta

On the hunt for a novel competitor to Sanofi and Regeneron’s Dupixent in severe asthma, Amgen and AstraZeneca posted “exciting” results from their next-gen antibody late last year. Now, the partners are showing their hands, and the results look good enough for approval.

Amgen and AstraZeneca’s tezepelumab plus standard of care cut the rate of severe asthma attacks by 56% at the one-year mark compared with SOC alone, according to full data from the Phase III NAVIGATOR study presented Friday at the virtual American Academy of Allergy, Asthma & Immunology meeting. And those significant results were consistent regardless of patients’ baseline eosinophil counts.

Tal Zaks, Moderna CMO (AP Photo/Rodrique Ngowi, via still image from video)

CMO Tal Zaks bids Mod­er­na a sur­prise adieu as biotech projects $18.4B in rev­enue, plots post-Covid ex­pan­sion

How do you exit a company after six years in style? Developing one of the most lucrative and life-saving products in pharma history is probably not the worst way to go.

Tal Zaks, Moderna’s CMO since 2015, will leave the mRNA biotech in September, the biotech disclosed in their annual report this morning. The company has already retained the recruitment firm Russell Reynolds to find a replacement.

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Glax­o­SmithK­line re­thinks strat­e­gy for Covid-19 an­ti­body — not the Vir ones — af­ter tri­al flop. Is there hope in high-risk pa­tients?

In the search for a better Covid-19 therapeutic, GlaxoSmithKline and Vir have partnered up on two antibodies they hope have a chance. GSK is also testing its own in-house antibody, and early results may have shut the door on its widespread use.

A combination of GSK’s monoclonal antibody otilimab plus standard of care couldn’t best standard of care alone in preventing death and respiratory failure in hospitalized Covid-19 patients after 28 days, according to data from the Phase IIa OSCAR study unveiled Thursday.

With dust set­tled on ac­tivist at­tack, Lau­rence Coop­er leaves Zio­pharm to a new board

Laurence Cooper has done his part.

In the five years since he left a tenured position at Houston’s MD Anderson Cancer Center to become CEO of Boston-based Ziopharm, he’s steered the small-cap immunotherapy player through patient deaths in trials, clinical holds, short attacks and, most recently, an activist attack on the board.

So when the company has “fantastic news” like an IND clearance for a TCR T cell therapy program, he’s ready to pass on the baton.

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