Ver­tex strikes agree­ment with Aus­tralia for cys­tic fi­bro­sis pill, end­ing high-pro­file stand­off

Ver­tex reached an agree­ment on Sun­day to sell its newest cys­tic fi­bro­sis drug in Aus­tralia, end­ing a high-pro­file stand­off over ac­cess to a life-al­ter­ing but ex­pen­sive rare dis­ease drug.

As with most agree­ments be­tween drug­mak­ers and na­tion­al pay­ment sys­tems, Ver­tex and the Aus­tralian Phar­ma­ceu­ti­cal Ben­e­fits Scheme didn’t say how much. But they said it will give 2,200 CF pa­tients in the coun­try ac­cess to Trikaf­ta, the pill shown to dra­mat­i­cal­ly im­prove lung func­tion in 90% of CF pa­tients, in­clud­ing 700 who weren’t el­i­gi­ble for pre­vi­ous Ver­tex drugs be­cause of their par­tic­u­lar ge­net­ic mu­ta­tions.

Ver­tex has clashed re­peat­ed­ly with na­tion­al gov­ern­ments over ac­cess to its CF drugs, in­clud­ing a news­pa­per ink-spilling clash with the UK in which for­mer CEO Jef­frey Lei­den ac­cused the NHS of  “lack[ing] of com­mit­ment to chil­dren and young peo­ple with this dev­as­tat­ing dis­ease.”

By the ad­vent of Trikaf­ta, in 2019, the stand­off with the UK and oth­er gov­ern­ments’ pay­ers — in­clud­ing in the US —  had been re­solved. Trikaf­ta was al­so sig­nif­i­cant­ly more ef­fec­tive for most CF pa­tients than pre­vi­ous Ver­tex drugs, help­ing mol­li­fy ob­jec­tions to the rough­ly $300,000 price tag the com­pa­ny has put on each of their drugs.

In Aus­tralia and oth­er coun­tries, though, the fight dragged on. Last year, Aus­tralian reg­u­la­tors ap­proved Trikaf­ta, but an in­de­pen­dent pric­ing com­mit­tee rec­om­mend­ed the gov­ern­ment hold off on cov­er­ing it, say­ing “cost-ef­fec­tive­ness … had not sat­is­fac­to­ri­ly been es­tab­lished.”

Cys­tic Fi­bro­sis Aus­tralia, a lead­ing lo­cal pa­tient group, ap­peared to slam both Ver­tex and the Aus­tralian gov­ern­ment for the stand­off, ask­ing Aus­tralians to con­tact their rep­re­sen­ta­tives while ac­cus­ing Ver­tex of “craven com­mer­cial­ism.”

“Hav­ing seen the out­stand­ing clin­i­cal tri­al da­ta we can on­ly sur­mise that once again ‘mon­ey’ is the stum­bling block,” they wrote, urg­ing Ver­tex to give wise com­pas­sion­ate use ac­cess while ne­go­ti­a­tions played out.

The group wel­comed the new agree­ment, writ­ing on Twit­ter that they were “on Cloud 9.” Cov­er­age will be­gin on April 1.

Trikaf­ta, how­ev­er, re­mains in­ac­ces­si­ble in many oth­er coun­tries around the globe, in­clud­ing large swaths of Latin Amer­i­ca, Africa and Asia, where cys­tic fi­bro­sis is less com­mon but still present.

Re­searchers and ad­vo­cates have pushed the com­pa­ny to vol­un­tar­i­ly al­low gener­ic man­u­fac­ture and sell the drug to low and mid­dle-in­come coun­tries, as Pfiz­er and Mer­ck have done for their Covid-19 pills and Gilead did for its he­pati­tis C cure.

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.

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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In­cyte hit by CRL on ex­tend­ed-re­lease JAK tablets, mud­dy­ing plans for Jakafi fran­chise ex­pan­sion

The FDA has rejected Incyte’s extended-release formulation of ruxolitinib tablets, in a surprise setback for the company’s plans to build on its blockbuster Jakafi franchise.

The ruxolitinib XR tablets are designed to be taken once a day, whereas Jakafi is indicated for twice daily dosage (although some patients can take it once daily).

According to Incyte, the FDA acknowledged in its complete response letter that the study submitted in the NDA “met its objective of bioequivalence based on area under the curve (AUC) parameters but identified additional requirements for approval.”

Zhi Hong, Brii Biosciences CEO

Brii Bio­sciences stops man­u­fac­tur­ing Covid-19 an­ti­body com­bo, plans to with­draw EUA re­quest

Brii Biosciences said it will stop manufacturing its Covid-19 antibody combination, sold in China, and is working to withdraw its emergency use authorization request in the US, which it started in October 2021.

The Beijing and North Carolina biotech commercially launched the treatment in China last July but is now axing the work and reverting resources to other “high-priority programs,” per a Friday update. The focus now is namely hepatitis B viral infection, postpartum depression and major depressive disorders.

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Eu­ro­pean Com­mis­sion de­lays pro­pos­al for ma­jor changes to phar­ma leg­is­la­tion

The European Commission has once again delayed the release of its proposal for an overhaul of the continent’s pharmaceutical legislation.

The release, previously anticipated on March 29, will occur “slightly later” than expected due to the “very busy College agendas of the last few weeks,” a Commission spokesperson told Endpoints News via email.

While the agency hasn’t provided an updated timeline, the spokesperson said the agenda is “always indicative and adoption dates of Commission proposals may change any time, especially when these proposals concern reforms of complex legislations of major importance.”

Sergio Traversa, Relmada Therapeutics CEO

Rel­ma­da makes 'crit­i­cal changes' to PhI­II tri­al to try and save de­pres­sion drug

Relmada Therapeutics is making changes to its Phase III study of its lead drug for major depressive disorder, in an attempt to avoid problems with a prior trial that showed little difference between the drug and a placebo.

That failure in October wiped 80% from Relmada’s stock price, and was followed by another negative readout a few months later. In both cases, the company said that there had been trial sites that were associated with what it called surprising placebo effects that skewed the results compared with the drug, REL-1017.

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Paul Song, NKGen Biotech CEO

NK cell ther­a­py-fo­cused biotech eyes SPAC deal

A small, Santa Ana-based biotech created in 2017 is looking to enter a SPAC deal as it lays out plans to begin trials in its lead cell therapy candidates and bring on new executives.

Graf Acquisition Corp. IV and NKGen Biotech announced Thursday, with few other details, that the two companies signed a non-binding letter of intent to “pursue a business combination.” Graf Acquisition II and III withdrew their IPOs last year.

Peter Hecht, Cyclerion Therapeutics CEO

Hard pressed for cash, Cy­cle­ri­on looks for help fund­ing rare dis­ease drug

Cyclerion Therapeutics may have the design of a Phase IIb study ready to go, but it’s scrambling for a way to fund it.

The company said in a press release that it’s “actively evaluating the best combination of capital, capabilities, and transactions available to it to advance the development of zagociguat,” its lead candidate for a rare, genetic mitochondrial disease known as MELAS.

In a separate SEC filing, Cyclerion once again flagged “substantial doubt about (its) ability to continue as a going concern.” As of the end of 2022, it had cash and cash equivalents of only $13.4 million.