Erik van den Berg, AM-Pharma CEO

Years af­ter Pfiz­er passed on a buy­out op­tion, AM-Phar­ma finds a new part­ner for its kid­ney drug

A cou­ple of years ago, Pfiz­er passed on an op­tion to buy out AM-Phar­ma and its re­com­bi­nant hu­man al­ka­line phos­phatase for kid­ney fail­ure on the heels of some mixed Phase II da­ta. Now, the pro­gram has at­tract­ed a new part­ner — and an ap­proval could mean a big pay­out for AM-Phar­ma.

Tokyo-based Ky­owa Kirin is putting down $23.6 mil­lion up­front for ex­clu­sive de­vel­op­ment and com­mer­cial­iza­tion rights to ilo­fo­tase al­fa in Japan. There’s an­oth­er $148 mil­lion in biobucks on the line, adding up to a $290 mil­lion deal with “tiered dou­ble-dig­it roy­al­ties” and a drug sup­ply fee.

AM-Phar­ma will be re­spon­si­ble for an on­go­ing Phase III study, which seeks to en­roll up to 1,600 pa­tients with sep­sis-as­so­ci­at­ed acute kid­ney in­jury and is ex­pect­ed to pro­duce da­ta in 2023. The com­pa­ny’s al­so on the hook for a Phase I PK, safe­ty and tol­er­a­bil­i­ty study in Japan. Af­ter that, Ky­owa Kirin will take the can­di­date to reg­u­la­tors.

Sep­sis is a life-threat­en­ing or­gan dys­func­tion that’s caused by an im­prop­er host re­sponse to in­fec­tion. The kid­ney is the most com­mon­ly af­fect­ed or­gan, re­sult­ing in AKI, which oc­curs in 40% to 60% of crit­i­cal-care ad­mis­sions, ac­cord­ing to the part­ners.

Ilo­fo­tase al­fa works by de­phos­pho­ry­lat­ing sub­stances that can trig­ger the im­mune sys­tem in­to dev­as­tat­ing the kid­ney, thus detox­i­fy­ing them and re­duc­ing the in­flam­ma­tion. In a Phase II tri­al, a 1.6 mg/kg dose showed a 46% rel­a­tive re­duc­tion in mor­tal­i­ty com­pared to the place­bo group (p=0.022). How­ev­er, the drug missed its pri­ma­ry end­point of im­prov­ing kid­ney func­tion in sev­en days.

“Short term kid­ney func­tion im­prove­ment was re­al­ly more a de­sign for the Phase II study to be able to pick the most op­ti­mal dose,” CEO Erik van den Berg told End­points News back in 2019. “It’s kind of a bio­mark­er if you like. For Phase III one has to choose hard clin­i­cal end­points, and the most pre­ferred end­point by the reg­u­la­tors is the mor­tal­i­ty in this set­ting.”

Pfiz­er had paid $87.5 mil­lion for a mi­nor­i­ty stake in AM-Phar­ma and an ex­clu­sive op­tion to buy out the rest of the com­pa­ny back in 2015. The phar­ma walked away from the deal fol­low­ing the Phase II read­out, cit­ing “in­ter­nal strate­gic rea­sons.” But AM-Phar­ma pushed on with a Phase III tri­al any­way, en­rolling the first pa­tient in its piv­otal RE­VIVAL study back in No­vem­ber. The tri­al was planned with the help of a small boost from Cowen Health­care In­vest­ments and a loan from the Eu­ro­pean In­vest­ment Bank last March.

“Based on the num­ber of suc­cess­ful in­ter­na­tion­al part­ner­ships they have, they are the ide­al part­ner to sup­port the com­mer­cial­iza­tion of ilo­fo­tase al­fa in Japan,” van den Berg said of Ky­owa Kirin in a state­ment.

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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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.

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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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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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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Geoff McDonough, Generation Bio president and CEO

Mod­er­na part­ners on non-vi­ral gene ther­a­py with Gen­er­a­tion Bio af­ter swing­ing gene edit­ing deals

Moderna has inked a five-year partnership with gene therapy biotech Generation Bio, it announced Thursday morning, wading deeper into the genetic medicines space as it navigates beyond its vaccine work.

Moderna will pay Generation Bio $40 million upfront and invest another $36 million into the gene therapy biotech. In exchange, Moderna can license Generation Bio’s non-viral gene therapy platforms for two immune cell programs and two liver programs, with an option for a fifth program. Moderna will fund all the research work under the partnership, and could be on the hook for milestone, fee and royalty payments totaling up to $1.8 billion, a company spokesperson tells Endpoints News.

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Steven James, Pionyr Immunotherapeutics CEO

Gilead pass­es on ful­ly ac­quir­ing Pi­o­nyr, as eyes now turn to Tizona, a fel­low sum­mer 2020 buy­out op­tion

Gilead and Pionyr Immunotherapeutics, a biotech trying to follow up on the first generation of checkpoint inhibitors, have “mutually agreed” on a rewrite to their 2020 terms, with Gilead deciding not to buy out the company.

The California biopharma waived its option to acquire the remaining 50.1% of Pionyr, which would have triggered a $315 million upfront payment and up to $1.15 billion down the road. Had Gilead waited to decide, the drugmaker would have had a potential payment to make in the near term under their agreement, a spokesperson said in an email to Endpoints News.

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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.”