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.

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His­toric drug pric­ing re­forms pass; Pfiz­er ac­quires GBT; The long search for non-opi­oid pain drugs; 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.

The Endpoints Weekly has officially crossed the 60,000 mark on subscribers — thanks to all of your support. As the editorial team grows, we’ve been able to do a lot more, with many of those on display this week. Be sure to check out Lei Lei Wu’s deep dive on pain R&D. If you missed it, you may also rewatch her companion panel here.

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Gold for adults, sil­ver for in­fants: Pfiz­er's Pre­vnar 2.0 head­ed to FDA months af­ter Mer­ck­'s green light

Pfizer was first to the finish line for the next-gen pneumococcal vaccine in adults, but Merck beat its rival with a jab for children in June.

Now, two months after Merck’s 15-valent Vaxneuvance won the FDA stamp of approval for kids, Pfizer is out with some late-stage data on its 20-valent shot for infants.

Known as Prevnar 20 for adults, Pfizer’s 20vPnC will head to the FDA by the end of this year for an approval request in infants, the Big Pharma said Friday morning. Discussions with the FDA will occur first and more late-stage pediatric trials are expected to read out soon, informing the regulatory pathway in other countries and regions.

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Seagen interim CEO Roger Dansey and Daiichi Sankyo CEO Sunao Manabe

Paving the way for Mer­ck­'s buy­out, Seagen los­es ar­bi­tra­tion dis­pute with Dai­ichi over ADC tech

As Seagen awaits a final buyout offer from Merck that could be in the territory of $40 billion, Seagen revealed Friday afternoon that it lost an arbitration dispute with Daiichi Sankyo relating to the companies’ 2008 collaboration around the use of antibody-drug conjugate (ADC) technology.

But that loss likely won’t matter much when it comes to Merck’s deal.

After breaking off its pact with Daiichi in mid-2015, the two companies battled over “linker” tech — a chemical bridge between an ADC’s antibody component and the cytotoxic payload — that Seagen claims Daiichi would improve upon and implement in its current generation of ADCs.

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Winselow Tucker, Eli Lilly's new Loxo unit chief commercial officer

Eli Lil­ly plucks a new com­mer­cial chief from Bris­tol My­ers in man­age­ment shuf­fle as HR chief re­tires

Eli Lilly has found a new chief commercial officer from among the ranks at Bristol Myers Squibb, as it says farewell to its longtime head of human resources Stephen Fry.

Fry announced on Thursday his plans to retire after more than 35 years with Lilly. He’ll vacate his seat as SVP of human resources and diversity at the end of the year, and current Loxo CCO Eric Dozier is slated to take his place. As a result, BMS’ Winselow Tucker is joining the team as Loxo CCO at the end of the month.

Senate Finance Committee Chair Ron Wyden (D-OR) (Francis Chung/E&E News/POLITICO via AP Images)

Sen­ate Fi­nance chair con­tin­ues his in­ves­ti­ga­tion in­to phar­ma tax­es with re­quests for Am­gen

Amgen is the latest pharma company to appear on the radar of Senate Finance Committee Chair Ron Wyden (D-OR), who is investigating the way pharma companies are using subsidiaries in low- or zero-tax countries to lower their tax bills.

Like its peers Merck, AbbVie and Bristol Myers Squibb, Wyden notes how Amgen uses its Puerto Rico operations to consistently pay tax rates that are substantially lower than the U.S. corporate tax rate of 21%, with an effective tax rate of 10.7% in 2020 and 12.1% in 2021.

FDA ap­proves sec­ond in­di­ca­tion for As­traZeneca and Dai­ichi's En­her­tu in less than a week

AstraZeneca and Daiichi Sankyo’s antibody-drug conjugate Enhertu scored its second approval in less than a week, this time for a subset of lung cancer patients.

Enhertu received accelerated approval on Thursday to treat adults with unresectable or metastatic non-small cell lung cancer (NSCLC) whose tumors have activating HER2 (ERBB2) mutations, and who have already received a prior systemic therapy.

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J&J to re­move talc prod­ucts from shelves world­wide, re­plac­ing with corn­starch-based port­fo­lio

After controversially spinning out its talc liabilities and filing for bankruptcy in an attempt to settle 38,000 lawsuits, Johnson & Johnson is now changing up the formula for its baby powder products.

J&J is beginning the transition to an all cornstarch-based baby powder portfolio, the pharma giant announced on Thursday — just months after a federal judge ruled in favor of its “Texas two-step” bankruptcy to settle allegations that its talc products contained asbestos and caused cancer. An appeals court has since agreed to revisit that case.

CSL is gathering its four business units under a unified brand identity strategy (Credit: CSL company site)

CSL brings Se­qirus, Vi­for un­der par­ent um­brel­la brand in iden­ti­ty re­vamp

CSL is gathering its brands under the family name umbrella, renaming its vaccine and newly acquired nephrology specialty businesses with the parent initials.

CSL Seqirus and CSL Vifor join CSL Plasma and CSL Behring as the four now uniformly branded business units of the global biopharma. The Seqirus vaccine division was formed in 2015 with the combination of bioCSL and its purchase of Novartis’ flu vaccine business. CSL picked up Vifor Pharma late last year in an $11.7 billion deal for the nephrology, iron deficiency and cardio-renal drug developer.

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