AM-Phar­ma gath­ers €116M for PhI­II pro­gram on kid­ney drug Pfiz­er passed on

Erik van den Berg AM-Phar­ma

Back in 2015, Pfiz­er put a spot­light on Dutch biotech AM-Phar­ma and its drug can­di­date for sep­sis-as­so­ci­at­ed acute kid­ney in­jury, an­te­ing up $87.5 mil­lion for a mi­nor­i­ty stake in ad­di­tion to an op­tion to gob­ble the whole op­er­a­tion up. The Phase II tri­al that the gi­ant part­ner had bet on end­ed up de­liv­er­ing a mixed win, and Pfiz­er walked away from the deal cit­ing “in­ter­nal strate­gic rea­sons” — leav­ing the small play­er on its own to pull off a large Phase III study and pon­der com­mer­cial­iza­tion.

A year and a half af­ter that da­ta read­out, AM-Phar­ma is go­ing for it. In lieu of one deep-pock­et­ed al­ly, it’s as­sem­bled a group of top-tier Eu­ro­pean in­vestors to back its fi­nal stretch in the clin­ic.

LSP and An­dera Part­ners co-led the €116 mil­lion ($133 mil­lion) round, join­ing found­ing in­vestor For­bion and oth­er ex­ist­ing sup­port­ers: Ys­ios Cap­i­tal, Kur­ma Part­ners, ID In­vest Part­ners, BB Pure­os Bioven­tures and Gilde Health­care.

The Phase III pro­gram will re­cruit 1,400 pa­tients across 12 coun­tries to test whether AM-Phar­ma’s re­com­bi­nant hu­man al­ka­line phos­phatase (re­cAP) can in­deed re­duce the mor­tal­i­ty rate due to kid­ney dam­age in­duced by the body’s over­whelm­ing re­sponse to in­fec­tions.

The 40% re­duc­tion not­ed in the pre­vi­ous Phase II was what gave van den Berg con­fi­dence de­spite the drug not hit­ting the pri­ma­ry end­point of im­prov­ing kid­ney func­tion in 7 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,” he said. “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.”

And it’s not hard to see why. With mil­lions of pa­tients suf­fer­ing from acute kid­ney in­jury each oth­er — half of which are as­so­ci­at­ed with sep­sis — and 700,000 deaths around the world an­nu­al­ly, mak­ing a dent in that would mean a block­buster op­por­tu­ni­ty.

Re­cAP works by de­phos­pho­ry­lat­ing sub­stances that can trig­ger the im­mune sys­tem in­to dev­as­tat­ing the kid­ney, thus dex­o­ti­fy­ing them and re­duc­ing the in­flam­ma­tion.

“From our in­ter­ac­tions with FDA and EMA last year, we just need to con­duct one sin­gle pos­i­tive piv­otal study to file for mar­ket ap­proval,” he tells me, adding that the drug has se­cured fast track des­ig­na­tion in the US.

He plans to bring the com­pa­ny’s head­count up from 15 to as many as 30 to com­plete the clin­i­cal work and per­haps be­gin lay­ing some ground­work for launch.

Fol­low­ing the fi­nanc­ing the board will al­so ex­pand, com­pris­ing Mar­ti­jn Klei­jwegt and Fe­lice Ver­duyn van Wee­gen from LSP, Raphael Wis­niews­ki from An­dera, Re­mi Droller from Kur­ma and Geert-Jan Mul­der from For­bion.

Biotech Half­time Re­port: Af­ter a bumpy year, is biotech ready to re­bound?

The biotech sector has come down firmly from the highs of February as negative sentiment takes hold. The sector had a major boost of optimism from the success of the COVID-19 vaccines, making investors keenly aware of the potential of biopharma R&D engines. But from early this year, clinical trial, regulatory and access setbacks have reminded investors of the sector’s inherent risks.

RBC Capital Markets recently surveyed investors to take the temperature of the market, a mix of specialists/generalists and long-only/ long-short investment strategies. Heading into the second half of the year, investors mostly see the sector as undervalued (49%), a large change from the first half of the year when only 20% rated it as undervalued. Around 41% of investors now believe that biotech will underperform the S&P500 in the second half of 2021. Despite that view, 54% plan to maintain their position in the market and 41% still plan to increase their holdings.

NYU surgeon transplants an engineered pig kidney into the outside of a brain-dead patient (Joe Carrotta/NYU Langone Health)

No, sci­en­tists are not any clos­er to pig-to-hu­man trans­plants than they were last week

Steve Holtzman was awoken by a 1 a.m. call from a doctor at Duke University asking if he could put some pigs on a plane and fly them from Ohio to North Carolina that day. A motorcyclist had gotten into a horrific crash, the doctor explained. He believed the pigs’ livers, sutured onto the patient’s skin like an external filter, might be able to tide the young man over until a donor liver became available.

UP­DAT­ED: Agenus calls out FDA for play­ing fa­vorites with Mer­ck, pulls cer­vi­cal can­cer BLA at agen­cy's re­quest

While criticizing the FDA for what may be some favoritism towards Merck, Agenus on Friday officially pulled its accelerated BLA for its anti-PD-1 inhibitor balstilimab as a potential second-line treatment for cervical cancer because of the recent full approval for Merck’s Keytruda in the same indication.

The company said the BLA, which was due for an FDA decision by Dec. 16, was withdrawn “when the window for accelerated approval of balstilimab closed,” thanks to the conversion of Keytruda’s accelerated approval to a full approval four months prior to its PDUFA date.

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How to col­lect and sub­mit RWD to win ap­proval for a new drug in­di­ca­tion: FDA spells it out in a long-await­ed guid­ance

Real-world data are messy. There can be differences in the standards used to collect different types of data, differences in terminologies and curation strategies, and even in the way data are exchanged.

While acknowledging this somewhat controlled chaos, the FDA is now explaining how biopharma companies can submit study data derived from real-world data (RWD) sources in applicable regulatory submissions, including new drug indications.

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David Lockhart, ReCode Therapeutics CEO

Pfiz­er throws its weight be­hind LNP play­er eye­ing mR­NA treat­ments for CF, PCD

David Lockhart did not see the meteoric rise of messenger RNA and lipid nanoparticles coming.

Thanks to the worldwide fight against Covid-19, mRNA — the genetic code that can be engineered to turn the body into a mini protein factory — and LNPs, those tiny bubbles of fat carrying those instructions, have found their way into hundreds of millions of people. Within the biotech world, pioneers like Alnylam and Intellia have demonstrated just how versatile LNPs can be as a delivery vehicle for anything from siRNA to CRISPR/Cas9.

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René Russo, Xilio CEO (Alicia Petitti for Xilio)

Xilio Ther­a­peu­tics goes pub­lic as bio­phar­ma IPOs rum­ble for­ward in Q4

It’s been a busy fall on Wall Street — and this week, we’ve got another biotech going public after Ventyx yesterday.

Massachusetts solid-tumor biotech Xilio Therapeutics made its public debut today, joining 149 other biotechs that have gone public so far this year.

Back on October 1st, Xilio filed its S-1 with the SEC to make its IPO debut for $100 million — the next step in financing after completing a $95 million Series C back in February.

Luc Boblet, Egle Therapeutics CEO

A new Treg play­er emerges with $46M and back­ing from Take­da

In recent years, the chorus of biotechs and Big Pharma backers targeting regulatory T cells — also known as “Tregs” — for cancer and autoimmune diseases has only grown louder.

The newest voice is from Egle Therapeutics, which sang out a $46.4 million Series A round on Friday led by LSP and Bpifrance through their InnoBio 2 fund. Takeda’s venture arm also chipped in, about a year after the pharma struck a research pact with the Paris-based upstart.

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Marty Duvall, Oncopeptides CEO

On­copep­tides stock craters as it pulls can­cer drug Pepax­to from the mar­ket

Shares of Oncopeptides crashed more than 70% in early Friday trading after the company said it’s pulling its multiple myeloma drug Pepaxto (melphalan flufenamide) from the US market after failing a confirmatory trial. The move will force the company to close its US and EU business units and enact significant layoffs.

The FDA had scheduled an adcomm meeting next Thursday to discuss Pepaxto, which first won accelerated approval in February and costs about $19,000 per course of treatment. The committee was to weigh in on whether the confirmatory trial demonstrated a worse overall survival in the treatment arm compared to the control arm.

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Pfiz­er pitch­es its Covid-19 vac­cine for younger chil­dren ahead of ad­comm next week

Pfizer will present its case to the FDA’s vaccine adcomm next week, seeking authorization for a lower-dose version of its Covid-19 vaccine for kids ages 5 through 12, which the Biden administration said will likely begin rolling out early next month.

Two primary doses of the 10 µg vaccine (the dose for those ages 12 and up is 30 μg) given 3 weeks apart in this group of children “have shown a favorable safety and tolerability profile, robust immune responses against all variants of concern including Delta, and vaccine efficacy of 90.7% against laboratory-confirmed symptomatic COVID-19,” the company said in briefing documents ahead of next Tuesday’s meeting of the FDA’s Vaccines and Related Biological Products Advisory Committee.