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.

2023 Spot­light on the Fu­ture of Drug De­vel­op­ment for Small and Mid-Sized Biotechs

In the context of today’s global economic environment, there is an increasing need to work smarter, faster and leaner across all facets of the life sciences industry.  This is particularly true for small and mid-sized biotech companies, many of which are facing declining valuations and competing for increasingly limited funding to propel their science forward.  It is important to recognize that within this framework, many of these smaller companies already find themselves resource-challenged to design and manage clinical studies themselves because they don’t have large teams or in-house experts in navigating the various aspects of the drug development journey. This can be particularly challenging for the most complex and difficult to treat diseases where no previous pathway exists and patients are urgently awaiting breakthroughs.

Albert Bourla, Pfizer CEO (Efren Landaos/Sipa USA/Sipa via AP Images)

Pfiz­er makes an­oth­er bil­lion-dol­lar in­vest­ment in Eu­rope and ex­pands again in Michi­gan

Pfizer is continuing its run of manufacturing site expansions with two new large investments in the US and Europe.

The New York-based pharma giant’s site in Kalamazoo, MI, has seen a lot of attention over the past year. As a major piece of the manufacturing network for Covid-19 vaccines and antivirals, Pfizer is gearing up to place more money into the site. Pfizer announced it will place $750 million into the facility, mainly to establish “modular aseptic processing” (MAP) production and create around 300 jobs at the site.

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Vas Narasimhan, Novartis CEO (Thibault Camus/AP Images, Pool)

No­var­tis bol­sters Plu­vic­to's case in prostate can­cer with PhI­II re­sults

The prognosis is poor for metastatic castration-resistant prostate cancer (mCRPC) patients. Novartis wants to change that by making its recently approved Pluvicto available to patients earlier in their course of treatment.

The Swiss pharma giant unveiled Phase III results Monday suggesting that Pluvicto was able to halt disease progression in certain prostate cancer patients when administered after androgen-receptor pathway inhibitor (ARPI) therapy, but without prior taxane-based chemotherapy. The drug is currently approved for patients after they’ve received both ARPI and chemo.

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Up­dat­ed: FDA re­mains silent on or­phan drug ex­clu­siv­i­ty af­ter last year's court loss

Since losing a controversial court case over orphan drug exclusivity last year, the FDA’s Office of Orphan Products Development has remained entirely silent on orphan exclusivity for any product approved since last November, leaving many sponsors in limbo on what to expect.

That silence means that for more than 70 orphan-designated indications for more than 60 products, OOPD has issued no public determination on the seven-year orphan exclusivity in the Orange Book, and no new listings of orphan exclusivity appear in OOPD’s searchable database, as highlighted recently by George O’Brien, a partner in Mayer Brown’s Washington, DC office.

Yuling Li, Innoforce CEO

In­no­force opens new man­u­fac­tur­ing site in Chi­na

Innoforce is off to the races at its new site in the city of Hangzhou, China.

The Chinese CDMO announced last week that it has started manufacturing at the new facility, which was built to offer process development and manufacturing operations for RNA, plasmid DNA, viral vectors and other cell therapeutics. It will also serve as Innoforce’s corporate HQ.

The company said it’s investing more than $200 million in the 550,000-square-foot manufacturing base for advanced therapies. The GMP manufacturing facility features space for producing plasmids with three 30-liter bioreactors. For viral vector manufacturing, Innoforce also has 200- and 500-liter bioreactors at its disposal, along with eight suites to make cell therapies. The site also includes several labs and warehouse spaces.

FDA grants or­phan drug des­ig­na­tion to Al­ger­non's ifen­prodil, while ex­clu­siv­i­ty re­mains un­clear

As the FDA remains silent on orphan drug exclusivity in the wake of a controversial court case, the agency continues to hand out new designations. The latest: Algernon Pharmaceuticals’ experimental lung disease drug ifenprodil.

The Vancouver-based company announced on Monday that ifenprodil received orphan designation in idiopathic pulmonary fibrosis (IPF), a chronic lung condition that results in scarring of the lungs.  Most IPF patients suffer with a dry cough, and breathing can become difficult.

‘Catchy’ de­sign tops big ad buys on­line for grab­bing on­col­o­gists’ at­ten­tion — sur­vey

The cancer drug ads that get oncologists’ attention online are informative and use clear, eye-catching designs. That’s ZoomRx’s assessment in its most recent tracking survey, and while not necessarily surprising, the details in the research do break a few common misconceptions.

One of those is frequency, also known as the number of impressions an ad gets. No matter how many times oncologists saw a particular cancer drug ad, effectiveness prevailed in the survey across five drug brands. ZoomRx measured effectiveness as a combination of most attention-getting, relevant information and improved perception as reported by the doctors.

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Tim Walbert, Horizon Therapeutics CEO (via YouTube)

And then there were two: Janssen bows out of Hori­zon takeover ne­go­ti­a­tions

Horizon Therapeutics announced last week that it was in talks with three pharmaceutical giants that could take over the company. You can now remove one of them from the equation.

J&J’s Janssen, after Horizon reported its initial involvement in early discussions to acquire the rare disease biotech, issued a statement Saturday that said Janssen “does not intend to make an offer for Horizon,” and that Janssen is bound by restrictions set in Rule 2.8 of the Irish Takeover Rules. These rules are in place for any company interested in taking over Irish companies, with Horizon Therapeutics currently based in Dublin.

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Rick Modi, Affinia Therapeutics CEO

Ver­tex-part­nered gene ther­a­py biotech Affinia scraps IPO plans

Affinia Therapeutics has ditched its plans to go public in a relatively closed-door market that has not favored Nasdaq debuts for the drug development industry most of this year. A pandemic surge in 2020 and 2021 opened the doors for many preclinical startups, which caught Affinia’s attention and gave the gene therapy biotech confidence in the beginning days of 2022 to send in its S-1.

But on Friday, Affinia threw in the S-1 towel and concluded now is not the time to step onto Wall Street. The biotech has put out few public announcements since the spring of this year. Endpoints News picked the startup as one of its 11 biotechs to watch last year.

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