Jim Wilson (WuXi Global Forum at JPM20)

Mod­er­na sur­faces as the first part­ner for Jim Wilson's rare dis­ease non­prof­it, do­nat­ing an mR­NA pro­gram at no cost

A week af­ter Jim Wil­son and Alex Kar­nal de­buted their Tachi Ya­ma­da-in­spired non­prof­it aimed at treat­ing ul­tra-rare dis­eases, the part­ner pro­vid­ing the In­sti­tute for Life Chang­ing Med­i­cines with its first pipeline pro­gram has emerged from the shad­ows.

Alex Kar­nal

Mod­er­na jumped in­to the spot­light Tues­day, an­nounc­ing it had do­nat­ed an mR­NA-based ther­a­py for Crigler-Na­j­jar syn­drome free of charge. In a press re­lease, CEO Stéphane Ban­cel said Mod­er­na would have had to charge a for­tune if the com­pa­ny had gone on to de­vel­op it for prof­it giv­en the ul­tra-rare na­ture of the dis­ease.

But that’s ex­act­ly the kind of pro­gram IL­CM wants, Kar­nal told End­points News.

“They share our recog­ni­tion that there’s a mar­ket fail­ure in the for-prof­it world lead­ing to Crigler-Na­j­jar pa­tients be­ing left be­hind,” Kar­nal said of Mod­er­na. “It’s un­der­stand­able from an eco­nom­ic per­spec­tive for that to hap­pen … it speaks to the mar­ket to­day where these amaz­ing tech­nolo­gies are not read­i­ly avail­able for the peo­ple who need them.”

The can­di­date is called mR­NA-3351, and Wil­son says it orig­i­nal­ly emerged from a part­ner­ship he had with Alex­ion sev­er­al years ago. Alex­ion ul­ti­mate­ly ex­it­ed the mR­NA space and gave the full slate of pro­grams to Mod­er­na, with whom Wil­son con­tin­ued the col­lab­o­ra­tion.

What piqued Wil­son’s in­ter­est here was the use of an mR­NA ther­a­py in the event of an acute cri­sis. Crigler-Na­j­jar pa­tients tend to need about 12 hours of pho­tother­a­py every day,  large­ly char­ac­ter­ized by sleep­ing un­der bright blue lights, in or­der not to de­vel­op jaun­dice. Some­times, if a pa­tient gets less of the ther­a­py than need­ed, emer­gency in­ter­ven­tion may be need­ed.

The In­sti­tute’s new pro­gram can step in here, Wil­son says, as one po­ten­tial ap­pli­ca­tion could be to stock phar­ma­cies with an ap­proved drug, al­low­ing pa­tients to re­ceive treat­ment should the need arise. If pa­tients al­so need to trav­el long dis­tances on a plane, or through an­oth­er method where bring­ing along their pho­tother­a­py beds is cum­ber­some, they can head to the phar­ma­cy ahead of time and not have to wor­ry about their dis­ease.

“This would be in the phar­ma­cy of hos­pi­tals or avail­able quick­ly in stores, and if a pa­tient gets in trou­ble or re­quires a ma­jor surgery or needs to trav­el, they get their in­fu­sion and they would then be main­tained on their stan­dard of care, which is blue lights,” Wil­son said.

The oth­er use Wil­son en­vi­sions for the drug is as a main­te­nance ther­a­py, re­plac­ing the blue lights al­to­geth­er. Da­ta from the pro­gram have shown in mouse mod­els the ther­a­py could prove ef­fec­tive for al­most three weeks, he said, pos­si­bly al­low­ing pa­tients to vis­it a clin­ic at about the same rate.

Kar­nal al­so shed some more light on the pri­or­i­ty re­view vouch­er sys­tem the In­sti­tute will uti­lize to bring in funds. The non­prof­it plans on rais­ing mon­ey to de­vel­op the Crigler-Na­j­jar pro­gram sole­ly through phil­an­thropic do­na­tions, and be­cause the can­di­date has re­ceived the FDA’s rare pe­di­atric dis­ease des­ig­na­tion, Mod­er­na and the In­sti­tute are el­i­gi­ble to re­ceive a vouch­er should it be ap­proved.

Whether or not Mod­er­na de­cides to use or pass on the vouch­er, the In­sti­tute will re­ceive funds tied to the val­ue of the PRV or its proxy, which will then be used to bring in more pipeline pro­grams el­i­gi­ble for the same des­ig­na­tion as the Crigler-Na­j­jar treat­ment. Once the In­sti­tute has seen mul­ti­ple ap­provals, it will start sell­ing con­tracts tied to the vouch­ers to con­tin­ue bring­ing in more pro­grams, Kar­nal said, cre­at­ing a cy­cle of “self-sus­tain­ing in­de­pen­dence.”

Ul­ti­mate­ly, Kar­nal and Wil­son are hope­ful the Crigler-Na­j­jar pro­gram is just the start of things to come. Wil­son has re­searched this spe­cif­ic dis­ease for a while and be­lieves the tri­al de­sign can win over reg­u­la­tors by us­ing biliru­bin — the com­pound im­pli­cat­ed in jaun­dice — as a bio­mark­er.

“We think there’s a good chance we could get the prod­uct reg­is­tered by demon­strat­ing a de­crease in biliru­bin with­out the need for clin­i­cal end­points, as it’s as close to a val­i­dat­ed clin­i­cal bio­mark­er as cho­les­terol would be for fa­mil­ial hy­per­c­ho­les­terolemia be­cause it’s biliru­bin that ac­tu­al­ly caus­es the tox­i­c­i­ty in the brain,” Wil­son said.

“We are poised to be­gin tox­i­col­o­gy stud­ies and sub­mit brief­ing docs as we speak,” he added.

Has the mo­ment fi­nal­ly ar­rived for val­ue-based health­care?

RBC Capital Markets’ Healthcare Technology Analyst, Sean Dodge, spotlights a new breed of tech-enabled providers who are rapidly transforming the way clinicians deliver healthcare, and explores the key question: can this accelerating revolution overturn the US healthcare system?

Key points

Tech-enabled healthcare providers are poised to help the US transition to value, not volume, as the basis for reward.
The move to value-based care has policy momentum, but is risky and complex for clinicians.
Outsourced tech specialists are emerging to provide the required expertise, while healthcare and tech are also converging through M&A.
Value-based care remains in its early stages, but the transition is accelerating and represents a huge addressable market.

Lat­est on ul­tra-rare dis­ease ap­proval; Pos­i­tive, if mixed, signs for Bio­gen's ALS drug; Clay Sie­gall finds a new job; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

Over the last four years, we’ve honored 80 women whose extraordinary accomplishments have changed the game in biopharma R&D. You can now nominate someone to be highlighted in this year’s special report. Details are here.

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Clay Siegall, Morphimmune CEO

Up­dat­ed: Ex-Seagen chief Clay Sie­gall emerges as CEO of pri­vate biotech

Clay Siegall will be back in the CEO seat, taking the helm of a private startup working on targeted cancer therapies.

It’s been almost a year since Siegall resigned from Seagen, the biotech he co-founded and led for more than 20 years, in the wake of domestic violence allegations by his then-wife. His eventual successor, David Epstein, sold the company to Pfizer in a $43 billion deal unveiled last week.

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FDA ad­vi­sors unan­i­mous­ly rec­om­mend ac­cel­er­at­ed ap­proval for Bio­gen's ALS drug

A panel of outside advisors to the FDA unanimously recommended that the agency grant accelerated approval to Biogen’s ALS drug tofersen despite the drug failing the primary goal of its Phase III study, an endorsement that could pave a path forward for the treatment.

By a 9-0 vote, members of the Peripheral and Central Nervous System Drugs Advisory Committee said there was sufficient evidence that tofersen’s effect on a certain protein associated with ALS is reasonably likely to predict a benefit for patients. But panelists stopped short of advocating for a full approval, voting 3-5 against (with one abstention) and largely citing the failed pivotal study.

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No­vo Nordisk oral semaglu­tide tri­al shows re­duc­tion in blood sug­ar, plus weight loss

Novo Nordisk is testing higher levels of its oral version of its GLP-1, semaglutide, and its type 2 diabetes trial results released today show reductions in blood sugar as well as weight loss.

In the Phase IIIb trial, Novo compared its oral semaglutide in 25 mg and 50 mg doses with the 14 mg version that’s currently the maximum approved dose. The trial looked at how the doses compared when added to a stable dose of one to three oral antidiabetic medicines in people with type 2 diabetes who were in need of an intensified treatment.

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FDA spells out how can­cer drug de­vel­op­ers can use one tri­al for both ac­cel­er­at­ed and full ap­provals

The FDA’s Oncology Center of Excellence has been a bright spot within the agency in terms of speeding new treatments to patients. That flexibility was on full display this morning as FDA released new draft guidance spelling out exactly how oncology drug developers can fulfill both the accelerated and full approval’s requirements with just a single randomized controlled trial.

While Congress recently passed legislation that will allow FDA to require confirmatory trials to be recruiting and ongoing prior to granting an accelerated approval, the agency is now making clear that the initial trial used to win the AA, if designed appropriately, can also serve as the trial for converting the accelerated approval into a full approval.

Eu­ro­pean doc­tors di­al up dig­i­tal com­mu­ni­ca­tion with phar­mas, but still lean to­ward in-per­son med meet­ings, study finds

As in-person sales rep access declines in the big five European countries, a corresponding uptick in virtual rep access is happening. It’s not surprising, but it does run counter to pharma companies’ assessment – along with long-held sales rep sway in Europe – that in-person access hadn’t changed.

CMI Media Group and Medscape’s recent study reports that 75% of physicians in the EU5 countries of Spain, Germany, Italy, France and the UK already limit engagements with pharma sales reps, and 25% of those surveyed plan to decrease time with reps.

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Judge al­lows ex­pert tes­ti­mo­ny in GSK tri­al al­leg­ing Zan­tac link to can­cer

A California judge will allow a plaintiff in a state court case to introduce expert testimony connecting a potential carcinogen in former blockbuster medicine Zantac to cancer.

The order was handed down on Thursday from state judge Evelio Grillo, who is now allowing both parties to introduce expert testimony in an upcoming trial after what’s known as a Sargon hearing, where a judge determines the admissibility of expert witnesses and expert opinions.

Mark Womack, BioCina CEO

Q&A: BioCi­na’s new CEO Mark Wom­ack on the CD­MO he says is 'worth trav­el­ing over'

A handful of CDMOs have made changes at the top over the past few weeks, including Genezen and Curia.

That also includes Australian CDMO BioCina, which announced last week that Mark Womack would be taking the helm. Womack previously served as chief business officer at AGC Biologics, CEO of Indian manufacturer Stelis Biopharma and most recently, CEO at CDMO KBI Biopharma and Selexis SA.

BioCina completed the takeover of a Pfizer manufacturing facility in Adelaide in 2021 and is now prepping for wider growth. Endpoints News sat down with Womack to discuss his new role, plans for the future, and how to compete in the wider CDMO market. This interview has been edited for brevity and clarity.