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.

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 has vowed not to let politics overrule 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 the FDA and other health agencies under his purview of their rule making 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.

#ES­MO20: Push­ing in­to front­line, Mer­ck and Bris­tol My­ers duke it out with new slate of GI can­cer da­ta

Having worked in parallel for years to move their respective PD-1 inhibitors up to the first-line treatment of gastrointestinal cancers, Merck and Bristol Myers Squibb finally have the data at ESMO for a showdown.

Comparing KEYNOTE-590 and CheckMate-649, of course, comes with the usual caveats. But a side-by-side look at the overall survival numbers also offer some perspective on a new frontier for the reigning checkpoint rivals, both of whom are claiming to have achieved a first.

President Donald Trump (via AP Images)

Signs of an 'Oc­to­ber Vac­cine Sur­prise' alarm ca­reer sci­en­tists

President Donald Trump, who seems intent on announcing a COVID-19 vaccine before Election Day, could legally authorize a vaccine over the objections of experts, officials at the FDA and even vaccine manufacturers, who have pledged not to release any vaccine unless it’s proved safe and effective.

In podcasts, public forums, social media and medical journals, a growing number of prominent health leaders say they fear that Trump — who has repeatedly signaled his desire for the swift approval of a vaccine and his displeasure with perceived delays at the FDA — will take matters into his own hands, running roughshod over the usual regulatory process.

#ES­MO20: Bris­tol My­ers marks Op­di­vo's sec­ond ad­ju­vant win — eye­ing a stan­dard of care gap

Moving into earlier and earlier treatment lines, Bristol Myers Squibb is reporting that adjuvant treatment with Opdivo has doubled the time that esophageal or gastroesophageal junction cancer patients stay free of disease.

With the CheckMate-577 data at ESMO, CMO Samit Hirawat said, the company believes it can change the treatment paradigm.

While a quarter to 30% of patients typically achieve a complete response following chemoradiation therapy and surgery, the rest do not, said Ronan Kelly of Baylor University Medical Center. The recurrence rate is also high within the first year, Hirawat added.

Clay Siegall (Life Science Washington via YouTube)

#ES­MO20: Seat­tle Ge­net­ics eyes 4th ap­proval with new da­ta in a crowd­ed field

Does Seattle Genetics have another approval on its hands?

The last 12 months, not so great for the world, has been great for Seattle Genetics. The company landed two separate FDA approvals, signed a $4.5 billion deal with Merck and watched antibody-drug conjugates — the technology they spent years developing to broad industry skepticism — emerge suddenly as one of the most popular approaches in oncology. And on Monday at ESMO, the company and their partners at Genmab unveiled the data behind the ADC it hopes will provide its next major FDA approval.

Jonathan Rigby, Immune Regulation group CEO

Im­mune Reg­u­la­tion, tak­ing two clin­i­cal pro­grams to 're­set' the im­mune sys­tem, nets $53M+ Se­ries B

A little under two years after a company rebranding, Immune Regulation is taking an even bigger step toward advancing its goals.

Formerly known as Peptinnovate, the British biotech announced a $53.4 million Series B early Monday morning, helping to further advance two clinical programs in rheumatoid arthritis and asthma. Though those are the two initial indications the company is focusing on, CEO Jonathan Rigby told Endpoints News he hopes the candidates can be applied to a broad swath of autoimmune disorders.

Eli Lilly CSO Dan Skovronsky (file photo)

UP­DAT­ED: #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.