Rat­tle and hum: Roche, PTC post up­date on their SMA drug as a shak­en Bio­gen faces yet an­oth­er fran­chise buster

Right on the heels of No­var­tis’ lat­est da­ta on its SMA gene ther­a­py for spinal mus­cu­lar at­ro­phy, PTC and its part­ners at Roche/Genen­tech post­ed their own pos­i­tive take on the lat­est da­ta for their oral ri­val ris­diplam. And a shaky Bio­gen will have to take it in­to ac­count as well as they as­sess the fu­ture for their block­buster drug Spin­raza, which has led the way on treat­ing this dis­ease for more than 2 years.

Tot­ting up their lat­est scores, the ris­diplam team chalked up:

  • 9 in­fants (53%) were able to main­tain an up­right head po­si­tion (HINE-2, n=17)
  • 10 in­fants (59%) demon­strat­ed rolling (rolling to the side, prone to supine, or supine to prone) (HINE-2, n=17)
  • 7 in­fants (41%) were able to sit with­out sup­port for at least 5 sec­onds (BSID-III, n=17)
  • 1 in­fant (6%) was able to stand (HINE-2, n=17)
  • 85.7% (18/21) ba­bies were event-free over­all

Three pa­tients had died, though. And while the re­searchers were quick to note that none of the in­fants died as a re­sult of ther­a­py, both Spin­raza and No­var­tis’ treat­ment have racked up pro­longed pe­ri­ods with no deaths.

That sets the stage for an FDA ap­pli­ca­tion lat­er this year, as No­var­tis awaits word from the agency any day now on its pitch for a gene ther­a­py.

SVB Leerink’s Mani Foroohar looked it over and pro­nounced him­self sat­is­fied.

Topline ris­diplam da­ta re­leased by Roche pri­or to the pre­sen­ta­tions at AAN lat­er this af­ter­noon con­tin­ue to demon­strate an ef­fi­ca­cy and safe­ty pro­file that ap­pears sup­port­ive of reg­is­tra­tion across spinal mus­cu­lar at­ro­phy (SMA) sub­types.

The ju­ry is still out on how this ri­val­ry will play out, but the prospect of a gene ther­a­py as well as an oral drug hit­ting the mar­ket may well have a pro­found ef­fect on Spin­raza. Bio­gen sells this drug for $750,000 for the first year, and then half that for each year there­after. That gives No­var­tis plen­ty of head­room to look for around $2 mil­lion for a claimed once-and-done ther­a­py. And then Roche along­side PTC could cause se­ri­ous dis­rup­tion with what­ev­er price they pick.

Mani Forhoor


What­ev­er par­ents pre­fer, in­sur­ers will have a lot of say over who gets what.

None of that bodes well for Bio­gen, which can ill af­ford to see its fran­chise eat­en up by ri­vals af­ter its big late-stage Alzheimer’s drug ad­u­canum­ab just crashed and burned.

“Bot­tom line — we could see a 1/3, 1/3, 1/3 split of mar­ket over time, Jef­feries an­a­lysts re­cent­ly said that the three drugs could end up di­vid­ing things even­ly. Based on their dis­cus­sions with physi­cians, gene ther­a­py “could be 30-33% share of new SMA Type I-III pa­tients over time. An es­ti­mat­ed 20-25% could look to switch from Spin­raza to gene ther­a­py (de­pends on weight of pa­tient). 2) Sim­i­lar­ly, docs sug­gest 1/3 share for oral ris­diplam, too, af­ter 3-5 years; 3) Com­men­tary by docs sug­gests an even split of opin­ions on the mar­ket as well — from oral pills ‘very ap­peal­ing’ and ‘game chang­er’ and ‘es­pe­cial­ly at­trac­tive for teens/adults’ to oth­er ther­a­pies need more da­ta and like­ly com­bo.”

Am­gen lays off about 300 work­ers, cit­ing 'in­dus­try head­wind­s'

Amgen has laid off about 300 employees, a company spokesperson confirmed to Endpoints News via email Sunday night.

Employees posted to LinkedIn in recent days about layoffs hitting Amgen last week. The Thousand Oaks, CA-based biopharma, which employs about 24,000 people, said the reduction “mainly” impacted US-based workers on its commercial team.

Drug developers of all sizes, including small upstarts and pharma giants, have let employees go in recent months as the biopharma market drags through a quarters-long winter doldrum.

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Bob Bradway, Amgen CEO (Stephen Lam/Reuters)

Am­gen launch­es the first US Hu­mi­ra biosim­i­lar at two dif­fer­ent list prices

The bizarre dynamics of the US prescription drug market were on full display once again this morning as Amgen announced that it would launch the first US biosimilar for Humira, the best-selling drug of all time, at two completely different list prices.

One price for Amgen’s Amjevita (adalimumab-atto) will be 55% below the current Humira list price, which is about $84,000 per year, and another at a list price 5% below the current Humira list price, but presumably (pharma companies don’t disclose rebates) with high rebates to attract PBMs and payers.

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Credit: Shutterstock

New York City in­vests $20M in­to biotech 'in­no­va­tion space' at the Brook­lyn Navy Yard

New York City is investing $20 million in biotech this year in the form of a 50,000-square-foot “innovation space” at the Brooklyn Navy Yard, complete with offices, research laboratories and events and programming space to grow biotech startups and companies.

Mayor Eric Adams said during his State of The City Address last Thursday that there will be an “emphasis” on making more opportunities for women and people of color to further diversify the industry. The City first reported the news.

Boehringer In­gel­heim touts pre­ven­tion re­sults in rarest form of pso­ri­a­sis

Boehringer Ingelheim uncorked some positive results suggesting that Spevigo can help prevent flare-ups in patients with a severe form of psoriasis, months after the drug was approved to treat existing flares.

Spevigo, an IL-36R antibody also known as spesolimab, met its primary and a key secondary endpoint in the Phase IIb EFFISAYIL 2 trial in patients with generalized pustular psoriasis (GPP), Boehringer announced on Monday. While the company is keeping the hard numbers under wraps until later this year, it said in a news release that it anticipates sharing the results with regulators.

As­traZeneca, No­vo Nordisk and Sanofi score 340B-re­lat­ed ap­peals court win over HHS

AstraZeneca, Novo Nordisk, and Sanofi won an appeals court win on Monday, as the US Court of Appeals for the Third Circuit found that the companies cannot be forced to provide 340B-discounted drugs purchased by hospitals from an unlimited number of community and specialty pharmacies.

“Legal duties do not spring from silence,” the decision says as the court makes clear that the federal government’s interpretation of the “supposed requirement” that the 340B program compels drugmakers to supply their discounted drugs to an unlimited number of contract pharmacies is not correct, noting:

Ap­peals court toss­es J&J's con­tro­ver­sial 'Texas two-step' bank­rupt­cy case

A US appeals court has ruled against Johnson & Johnson’s use of bankruptcy to deal with mounting talc lawsuits, deciding that doing so would “create a legal blind spot.”

The Third Circuit Court of Appeals reversed a previous bankruptcy court decision on Monday, calling for the dismissal of a Chapter 11 filing by J&J’s subsidiary LTL Management.

Faced with more than 38,000 lawsuits alleging its talc-based products caused cancer, J&J spun its talc liabilities into a separate company called LTL Management back in October 2021 and filed for bankruptcy, a controversial move colloquially referred to as a “Texas two-step” bankruptcy. Claimants argued that the strategy is a misuse of the US bankruptcy code — and on Monday, a panel of judges agreed.

Chad Mirkin, Flashpoint co-founder

‘The field is at a flash­point’: New Chad Mirkin-found­ed biotech hopes to make more ef­fec­tive can­cer vac­cines

Following the success of the mRNA Covid vaccines, cancer vaccines are seeing renewed interest after years of middling results. But a group of researchers suggests that more attention needs to be paid not to what goes into those vaccines, but how the parts are put together.

In a recent paper published in Nature Biomedical Engineering, researchers led by Northwestern University’s Chad Mirkin describe how the placement of different antigens in a cancer vaccine impacts its efficacy. The paper builds on past work done by Mirkin’s lab that suggests the structure, or how the parts of a vaccine are arranged, impact a vaccine’s efficacy, not just its components.

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Dirk Thye, Quince Therapeutics CEO

Af­ter piv­ot­ing from Alzheimer's to bone con­di­tions, biotech piv­ots again — and halves its head­count

When troubled public biotech Cortexyme bought a private startup named Novosteo and handed the keys to its executive team, the company — which changed its name to Quince Therapeutics — said it would shift its focus from an unorthodox Alzheimer’s approach to Novosteo’s bone-targeting drug platform.

Less than a year later, Quince is pivoting again.

The biotech has decided to out-license its bone-targeting drug platform and its lead drug, NOV004, and instead look for clinical-stage programs to in-license or acquire, according to a press release.

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Richard Gonzalez, AbbVie CEO (Chris Kleponis/picture-alliance/dpa/AP Images)

Up­dat­ed: $100B+ in sav­ings? Why the in­com­ing Hu­mi­ra biosim­i­lars will take time to catch on

The 20-year reign of AbbVie’s best-selling biologic of all time — the autoimmune disease biologic Humira (adalimumab) that has brought in upwards of $200 billion during its monopoly — is coming to an end tomorrow with the launch of Amgen’s biosimilar Amjevita.

The launch comes more than four years after Europe saw the exact same competition, leading to steep discounts in price, higher uptake, and big cost savings across the board.

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