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.

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.

Big week for Alzheimer’s da­ta; As­traZeneca buys cell ther­a­py start­up; Dig­i­tal ther­a­peu­tics hits a pay­er wall; and more

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Am­gen, years be­hind ri­vals, says PhI obe­si­ty drug shows dura­bil­i­ty signs

While NBC ran “The Biggest Loser” for 17 seasons, deemed toxic by critics for the reality show’s punishing exercise and diet upheavals, researchers in pharmaceutical labs have been attempting to create prescription drugs that induce weight loss — and one pharma betting it can require less frequent dosing is out with a new crop of data.

Amgen was relatively late to the game compared to its approved competitor Novo Nordisk and green light-approaching rival Eli Lilly. But early data suggested Amgen’s AMG 133 led to a 14.5% weight reduction in the first few months of dosing, buoying shares earlier this fall, and now the California pharma is out with its first batch of durability data showing that figure fell slightly to 11.2% about 150 days after the last dose. Amgen presented at the 20th World Congress on Insulin Resistance, Diabetes & Cardiovascular Disease on Saturday afternoon.

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Illustration: Assistant Editor Kathy Wong for Endpoints News

As mon­ey pours in­to dig­i­tal ther­a­peu­tics, in­sur­ance cov­er­age crawls

Talk therapy didn’t help Lily with attention deficit hyperactivity disorder, or ADHD. But a video game did.

As the 10-year-old zooms through icy waters and targets flying creatures on the snow-capped planet Frigidus, she builds attention skills, thanks to Akili Interactive Labs’ video game EndeavorRx. She’s now less anxious and scattered, allowing her to stay on a low dose of ADHD medication, according to her mom Violet Vu.

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Eli Lil­ly’s Alzheimer’s drug clears more amy­loid ear­ly than Aduhelm in first-ever head-to-head. Will it mat­ter?

Ahead of the FDA’s decision on Eli Lilly’s Alzheimer’s drug donanemab in February, the Big Pharma is dropping a first cut of data from one of the more interesting trials — but less important in a regulatory sense — at an Alzheimer’s conference in San Francisco.

In the unblinded 148-person study, Eli Lilly pitted its drug against Aduhelm, Biogen’s drug that won FDA approval but lost Medicare coverage outside of clinical trials. Notably, the study didn’t look at clinical outcomes, but rather the clearance of amyloid, a protein whose buildup is associated with Alzheimer’s disease, in the brain.

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US month­ly costs for biosim­i­lars 'sub­stan­tial­ly high­er' than Ger­many or Switzer­land, JA­MA re­search finds

As the global biologics market is expected to hit nearly the half-trillion-dollar mark this year, new JAMA research points to the importance of timely biosimilar entry, particularly as fewer biosimilars are entering the US than in Europe, and as monthly treatment costs for biosimilars were “substantially higher” in the US compared with Germany and Switzerland.

Among the three countries, biosimilar market share at launch was highest in Germany, but increased at the fastest rate in the US, the authors from the University of Zurich’s Institute of Law wrote in JAMA Network Open today.

Kirk Myers is shown in a still image from a new film series showcasing the efforts of HIV advocates funded by Gilead.

Gilead spot­lights HIV projects and the com­mu­ni­ty lead­ers dri­ving them in new mi­ni-doc­u­men­tary films

Gilead is going behind the scenes of some of the HIV initiatives it funds through grants in a new film series narrated by the people helming the projects.

The first four films and leaders come from across the US — Arianna Lint in Florida and Puerto Rico, Cleve Jones in San Francisco, June Gipson in Mississippi and Kirk Myers in Texas. Their HIV-focused efforts range from addressing unmet needs of the transgender community to delivering social services and high-quality health care in underserved communities.

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EMA pulls an opi­oid from the 1950s used to treat dry cough

The European Medicines Agency said Friday that it’s pulling from all European markets pholcodine-containing medicines, which are an opioid used in adults and children for the treatment of dry cough and in combo with other drugs as a treatment for cold and flu.

The decision to pull the medicines comes as the EMA points to the results from the recent ALPHO study, which show that use of pholcodine during the 12 months preceding anesthesia is linked to a risk of an anaphylactic reaction related to the neuromuscular blocking agents (NMBAs) used (with an adjusted OR of 4.2, and a 95% confidence interval of 2.5 to 6.9).

Immunocore CEO Bahija Jallal (L) and Gadeta CEO Marcel Zwaal

In search for a 'u­ni­ver­sal T cell en­gager', Im­muno­core inks deal with Gilead-backed Gade­ta

Immunocore, which became the first company to win approval for a TCR therapy earlier this year, has penned a deal with gamma delta T cell specialist Gadeta to develop treatments for solid tumors, starting with colorectal cancer.

Immunocore R&D head David Berman told Endpoints News the deal is part of the company’s efforts to develop a “universal T cell engager.” TCR therapies made from typical T cells, like Immunocore’s TCR therapy Kimmtrak, are restricted to patients with certain HLA types, a kind of marker that helps the body separate friend and foe. Kimmtrak, for instance, is indicated only for patients with unresectable or metastatic uveal melanoma, a rare form of cancer that happens in the eye, who have the tissue type HLA-A*02:01, one of the more common HLA types which by Berman’s estimate includes around 40-50% of the population.