FDA green­lights sec­ond Ebo­la treat­ment in three months, hand­ing new OK to Ridge­back­'s Eban­ga

A hus­band-and-wife biotech that li­censed a Covid-19 an­tivi­ral be­fore quick­ly part­ner­ing on it with Mer­ck ear­li­er this sum­mer has a new ap­proval in an­oth­er high­ly in­fec­tious dis­ease — Ebo­la.

Wendy Hol­man

Ridge­back Bio­ther­a­peu­tics, co-found­ed by CEO Wendy Hol­man and her hus­band Wayne Hol­man, a hedge fund man­ag­er who used to work for a com­pa­ny in­volved in one of the biggest in­sid­er trad­ing scan­dals in his­to­ry, has clinched an FDA OK for an­su­vimab-zykl to treat the dead­ly Ebo­la virus in adults and chil­dren. The drug, a mon­o­clon­al an­ti­body, will be mar­ket­ed as Eban­ga.

It’s the sec­ond-ever Ebo­la treat­ment to gain US ap­proval, both of which came over the last three months. Re­gen­eron’s an­ti­body cock­tail In­mazeb marked the first back in Oc­to­ber and was cho­sen by BAR­DA for the na­tion­al stock­pile in case of an out­break.

Both treat­ments were part of an NIH-spon­sored study in the De­mo­c­ra­t­ic Re­pub­lic of the Con­go dur­ing the coun­try’s 2018-19 out­break. Launched in Nov 2018, the study looked at four dif­fer­ent Ebo­la ther­a­pies and was stopped less than a year lat­er due to the over­whelm­ing­ly pos­i­tive re­sults pro­duced by Re­gen­eron and Ridge­back’s drugs.

The study it­self was ran­dom­ized and open-la­bel, with 174 pa­tients in the Eban­ga arm and 168 in the con­trol arm. Re­searchers honed in on 28-day mor­tal­i­ty rates as the pri­ma­ry end­point and found that, of those who re­ceived Eban­ga, 35.1% died af­ter 28 days. That fig­ure com­pared to 49.4% of those tak­ing the con­trol.

Reg­u­la­tors ad­vised against tak­ing both Eban­ga and an Ebo­la vac­cine at the same time. They not­ed that most of the com­mon side ef­fects from Eban­ga can al­so be con­fused with Ebo­la symp­toms them­selves, and rec­om­mend­ed dis­con­tin­u­a­tion should pa­tients de­vel­op a hy­per­sen­si­tiv­i­ty re­ac­tion.

Al­so in the study were a ZMapp an­ti­body and Gilead’s an­tivi­ral remde­sivir, which has since been re­pur­posed for Covid-19 and en­dorsed with a full FDA ap­proval de­spite lin­ger­ing ques­tions.

It’s been an event­ful year for the Mi­a­mi-based biotech. At the out­set of the Covid-19 pan­dem­ic in March, Hol­man struck a deal with Emory Uni­ver­si­ty to li­cense an ex­per­i­men­tal coro­n­avirus pill in­vent­ed by re­searchers there with $16 mil­lion in gov­ern­ment grants. Two months lat­er, Mer­ck gained world­wide rights to the pro­gram, dubbed EI­DD-2801, from Ridge­back for an undis­closed sum as part of their col­lab­o­ra­tion.

The quick turn­around drew con­cerns that Ridge­back at­tempt­ed to cash in big on a tax­pay­er-fund­ed pro­gram, with promi­nent Har­vard bioethi­cist Aaron Kessel­heim blast­ing the move in the Wash­ing­ton Post at the time. But in a Na­ture study pub­lished ear­li­er this month, re­sults showed that EI­DD-2801 com­plete­ly sup­pressed SARS-CoV-2 trans­mis­sion with­in 24 hours in fer­rets.

For Eban­ga, it’s not im­me­di­ate­ly clear where and how the drug will be used, giv­en Re­gen­eron’s pres­ence in the US strate­gic stock­pile and the DRC.

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.

David Livingston (Credit: Michael Sazel for CeMM)

Renowned Dana-Far­ber sci­en­tist, men­tor and bio­phar­ma ad­vi­sor David Liv­ingston has died

David Livingston, the Dana-Farber/Harvard Med scientist who helped shine a light on some of the key molecular drivers of breast and ovarian cancer, died unexpectedly last Sunday.

One of the senior leaders at Dana-Farber during his nearly half century of work there, Livingston was credited with shedding light on the genes that regulate cell growth, with insights into inherited BRCA1 and BRCA2 mutations that helped lay the scientific foundation for targeted therapies and earlier detection that have transformed the field.

With hun­dreds of mil­lions spent on failed ac­cel­er­at­ed ap­provals, re­searchers call for faster FDA with­drawals

Between 2017 and 2019, Medicare spent more than $220 million on cancer drugs for which the indications were either voluntarily pulled by their applicants or FDA’s oncology adcomm had recommended their withdrawal.

That kind of massive spending on cancer drugs lacking overall survival benefit is wasteful and risks harming people’s health, a research letter published in JAMA Internal Medicine on Monday said. The researchers from Harvard and the London School of Economics called on the FDA to move faster in both requiring timely postmarketing trials and accelerating the speed in pulling these dangling approvals when the confirmatory studies fail.

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No­vo CEO Lars Fruer­gaard Jør­gensen on R&D risk, the deal strat­e­gy and tar­gets for gen­der di­ver­si­ty

 

I kicked off our European R&D summit last week with a conversation involving Novo Nordisk CEO Lars Fruergaard Jørgensen. Novo is aiming to launch a new era of obesity management with a new approval for semaglutide. And Jørgensen had a lot to say about what comes next in R&D, how they manage risk and gender diversity targets at the trendsetting European pharma giant.

John Carroll: I’m here with Lars Jørgensen, the CEO of Novo Nordisk. Lars, it’s been a really interesting year so far with Novo Nordisk, right? You’ve projected a new era of growing sales. You’ve been able to expand on the GLP-1 franchise that was already well established in diabetes now going into obesity. And I think a tremendous number of people are really interested in how that’s working out. You have forecast a growing amount of sales. We don’t know specifically how that might play out. I know a lot of the analysts have different ideas, how those numbers might play out, but that we are in fact embarking on a new era for Novo Nordisk in terms of what the company’s capable of doing and what it’s able to do and what it wants to do. And I wanted to start off by asking you about obesity in particular. Semaglutide has been approved in the United States for obesity. It’s an area of R&D that’s been very troubled for decades. There have been weight loss drugs that have come along. They’ve attracted a lot of attention, but they haven’t actually ever gained traction in the market. My first question is what’s different this time about obesity? What is different about this drug and why do you expect it to work now whereas previous drugs haven’t?

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Man­u­fac­tur­ing woes for No­vavax’s Covid jab bad­ly dis­rupt plans for roll­out to the poor — re­port

Production problems at a Novavax facility in Maryland have led to delays in the Covax vaccine sharing program. Now, a shortage of 1 billion doses is expected, as the supplier tries to navigate producing a shot up to regulators’ standards, Politico reported Tuesday.

The company has run into trouble with the purity of the vaccine. Novavax has had trouble proving it can produce a shot consistently up to standards, and it has caused significant delays in the rollout to low- and middle-income countries. This follows several delays at Novavax that has put the executive crew on the defensive.

Sur­geons suc­cess­ful­ly at­tach pig kid­ney to a hu­man for the first time, us­ing tech from Unit­ed's Re­vivi­cor

In a first, researchers reportedly successfully transplanted a pig kidney into a human without triggering an immediate immune response this week. And the technology came from the biotech United Therapeutics.

Surgeons spent three days attaching the kidney to the patient’s blood vessels, but when all was said and done, the kidney appeared to be functioning normally in early testing, Reuters and the New York Times were among those to report. The kidney came from a genetically altered pig developed through United’s Revivicor unit.

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Break­ing: Bio­gen sells just $300K worth of Aduhelm in Q3, as ques­tions on long term vi­a­bil­i­ty re­main

Barely anyone is accessing Biogen’s controversial Alzheimer’s treatment, with the company reporting just $0.3 million in Aduhelm sales in the third quarter. Although investors will be looking to the longer term, when CMS may decide to cover the drug and open the floodgates for more coverage, use of the drug is currently stalled.

Since June, when the FDA first signed off on the drug under its accelerated pathway, Biogen said Wednesday that it’s sold a total of $2 million worth of Aduhelm. The total sales numbers indicate that likely about 100 Alzheimer’s patients have so far received the drug, which is priced at $56,000 annually.

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Raymond Stevens, ShouTi Pharma CEO

A new Schrödinger-backed start­up emerges from the sci­en­tist who mapped the first hu­man GPCR

One of the most popular targets in drug development, representing about a third of existing drugs, are G-protein coupled receptors — the tiny but integral membrane proteins responsible for recognizing things like light, taste, smell, hormones and pain.

But due to challenges in mapping their structure, the protein family remains largely unexplored.

A slate of companies has emerged over the last few years to change that. If one can figure out the structure of these elusive membrane receptors, it might be possible to create small molecule drugs that overcome the limitations of, say, biologic and peptide therapies. That promise is what gets serial entrepreneur Raymond Stevens out of bed in the morning.

Carl June (Brian Ach/Getty Images for TIME 100 Health Summit)

Carl June lends 'wings' to Chi­nese CAR-T start­up led by for­mer post­doc, pur­su­ing off-the-shelf ap­proach with CRISPR fla­vor

Carl June still has plenty of energy to bring forth new iterations of CAR-T technology — wherever they’re coming from.

Adding another role to his already lengthy list of titles, June is joining the scientific advisory board at Nanjing Bioheng Biotech, where he will serve as chairman.

The appointment, if slightly out of the ordinary, is both a testament to the fruitfulness of June’s lab at the University of Pennsylvania and China’s increasing appeal to biotech entrepreneurs educated overseas.