Bill Anderson, Roche

Roche aims to 'un­der­whelm' with ris­diplam price as it en­ters SMA ri­val­ry with Bio­gen, No­var­tis — re­port

With their third-to-mar­ket spinal mus­cu­lar at­ro­phy drug ris­diplam now cruis­ing on the FDA’s VIP lane to­ward a May de­ci­sion, Roche is think­ing through its pric­ing strat­e­gy.

You can ex­pect them to take a page out of their Hem­li­bra play­book as they tried to lure pa­tients away from No­vo Nordisk and Take­da, Bill An­der­son — CEO of the phar­ma group — told Reuters.

“With Hem­li­bra, we priced at about half of by­pass­ing agent,” An­der­son said in an in­ter­view with the news agency. “We aim to un­der­whelm with our price” with ris­diplam, he added.

They wouldn’t have to go far. No­var­tis’ gene ther­a­py, Zol­gens­ma, is the world’s most ex­pen­sive med­i­cine by one-time cost at $2.1 mil­lion. And while Bio­gen’s Spin­raza has a list price of $750,000 in the first year and $375,000 in sub­se­quent years, the an­nu­al prices could quick­ly add up to the mil­lions for an an­ti­sense oligonu­cleotide pa­tients are sup­posed to take for life.

Un­like the two ri­val ther­a­pies, which are ad­min­is­tered in­tra­venous­ly and in­trathe­cal­ly re­spec­tive­ly, ris­diplam is an oral med­ica­tion to be tak­en dai­ly. By mod­i­fy­ing how the SMN2 gene is spliced, the drug sup­pos­ed­ly in­creas­es func­tion­al SMN pro­tein lev­els in both the cen­tral ner­vous sys­tem and pe­riph­er­al tis­sues.

Zol­gens­ma’s ap­proval is al­so lim­it­ed to in­fants up to 2 years old, and an at­tempt to ex­pand in­to old­er pa­tients through in­trathe­cal ad­min­is­tra­tion has been stalled by a par­tial clin­i­cal hold.

Roche’s drug, which it first li­censed from PTC Ther­a­peu­tics, is be­ing tout­ed as ap­plic­a­ble for a broad age range from new­borns to 60-year-olds, though the piv­otal tri­al sub­mit­ted to the FDA en­rolled pa­tients be­tween 2 and 25.

In ad­di­tion to pri­or­i­ty re­view — which gives it a PDU­FA date of May 24 — the drug al­so en­joys the FDA’s or­phan and fast track des­ig­na­tions.

Pi­o­neer­ing Click Chem­istry in Hu­mans

Reimagining cancer treatments

Cancer is a leading cause of death worldwide, accounting for nearly 10 million deaths in 2020, which is nearly one in six deaths. Recently, we have seen incredible advances in novel cancer therapies such as immune checkpoint inhibitors, cell therapies, and antibody-drug conjugates that have revamped cancer care and improved survival rates for patients.

Despite this significant progress in therapeutic targeting, why are we still seeing such a high mortality rate? The reason is that promising therapies are often limited by their therapeutic index, which is a measure of the effective dose of a drug, relative to its safety. If we could broaden the therapeutic indices of currently available medicines, it would revolutionize cancer treatments. We are still on the quest to find the ultimate cancer medicine – highly effective in several cancer types, safe, and precisely targeted to the tumor site.

Ivan Cheung, Eisai US chairman and CEO

Bio­gen, Ei­sai re­fresh amy­loid hy­poth­e­sis with PhI­II show­ing Alzheimer's med slows cog­ni­tive de­cline

In the first look at Phase III data for lecanemab, Eisai and Biogen’s follow-up Alzheimer’s drug to the embattled Aduhelm launch, results show the drug passed with flying colors on a test looking at memory, problem solving and other dementia metrics.

One of the most-watched Alzheimer’s therapies in the clinic, lecanemab met the study’s primary goal on the CDR-SB — Clinical Dementia Rating-Sum of Boxes — giving the biotech the confidence to ask for full approval in the US, EU and Japan by next March 31. The experimental drug reduced clinical decline on the scale by 27% compared to placebo at 18 months, the companies said Tuesday night Eastern time and Wednesday morning in Japan.

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Gilead names 'k­ing­pin­s' in coun­ter­feit HIV med law­suit

Gilead is mounting its counterfeit drug lawsuit, naming two “kingpins” and a complex network of conspirators who allegedly sold imitation bottles of its HIV meds, some of which ended up in US pharmacies.

The pharma giant on Wednesday provided an update on what it called a “large-scale, sophisticated counterfeiting conspiracy,” accusing two new defendants of “leading and orchestrating” a scheme to sell hundreds of millions of dollars in illegitimate drugs posing as meds such as Biktarvy and Descovy.

Nooman Haque, head of life sciences and healthcare at Silicon Valley Bank, and John Carroll

I’m head­ed to Lon­don soon for #EU­BIO22. Care to join me?

It was great getting back to a live ESMO conference/webinar in Paris followed by a live pop-up event for the Endpoints 11 in Boston. We’re staying on the road in October with our return for a live/streaming EUBIO22 in London.

Silicon Valley Bank’s Nooman Haque and I are once again jumping back into the thick of it with a slate of virtual and live events on October 12. I’ll get the ball rolling with a virtual fireside chat with Novo Nordisk R&D chief Marcus Schindler, covering their pipeline plans and BD work.

Tar­sus looks to raise aware­ness of eye­lid mite dis­ease in cam­paign aimed at eye­care spe­cial­ists

Eyelid mite disease may be “gross” but it’s also fairly common, affecting about 25 million people in the US.

Called demodex blepharitis, it’s a well-known condition among eyecare professionals, but they often don’t always realize how common it is. Tarsus Pharmaceuticals wants to change that with a new awareness campaign called “Look at the Lids.”

The campaign and website debut Thursday — just three weeks after Tarsus filed for FDA approval for a drug that treats the disease.

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Work taking place in the clean rooms at Vor (Credit: Vor)

Vor Bio opts to keep man­u­fac­tur­ing op­er­a­tions in-house for de­vel­op­ing stem cell, CAR-T ther­a­pies

While it is not uncommon for a biotech to go down the route of having the product manufactured by a contract organization, one small biotech is looking to keep its card close to its chest.

Vor Biopharma has started manufacturing operations at an in-house facility at its HQ in Cambridge, MA after beginning construction last summer.

According to the biotech, the facility aims to develop Vor’s hematopoietic stem cells (eHSCs) and CAR-T therapies for patients with blood cancers. The site will initially manufacture a clinical supply of its candidate VCAR33allo to support its IND, which is slated to be submitted in the first half of next year. It also plans to transfer the production of VOR33 to the facility. Vor is getting to work quickly as engineering runs for VCAR33allo has started this week.

An­oth­er Avastin biosim­i­lar joins the par­ty as Roche's grip on the mar­ket loosens

Avastin has attracted quite the crowd of biosimilars in recent years, and one more pharma company has elbowed its way in.

Regulators on Wednesday approved Celltrion’s biosimilar Vegzelma in six cancer types, including metastatic colorectal cancer; recurrent or metastatic non-squamous non-small cell lung cancer (nsNSCLC); recurrent glioblastoma; metastatic renal cell carcinoma; persistent, recurrent, or metastatic cervical cancer; and epithelial ovarian, fallopian tube or primary peritoneal cancer.

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Elisabeth Stampa, Medicines for Europe president

As win­ter ap­proach­es, a Eu­ro­pean gener­ics group rais­es alarm over en­er­gy prices for man­u­fac­tur­ers

While colder temperatures are fast approaching, the situation surrounding the rise of energy prices in Europe is hitting businesses of every kind, including generic drug manufacturers.

Medicines for Europe, a group that represents the generic industry on the continent, sent a letter addressed to energy ministers and commissioners concerning inflation and the costs of energy on the supply of generic medicines.

Vlad Coric, Biohaven CEO (Photo Credit: Andrew Venditti)

As Amy­lyx de­ci­sion waits in the wings, Bio­haven’s ALS drug sinks (again) in plat­form tri­al

The FDA’s decision on Amylyx’s ALS drug is set to come out sometime Thursday. In a space with few drugs, any approval would be a major landmark.

But elsewhere in the ALS field, things are a bit more tepid.

Thursday morning, Biohaven announced that its drug verdiperstat failed its arm of an ALS platform trial led by Massachusetts General Hospital. According to a press release, the drug did not meet its primary endpoint — improvement on an ALS functional status test — or any key secondary endpoints at 24 weeks. The trial had enrolled 167 patients, giving them either verdiperstat or placebo twice a day.

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