Achao­gen hits, then miss­es: FDA OKs Zem­dri for cUTIs, but re­jects sec­ond in­di­ca­tion

Fol­low­ing an ad­vi­so­ry com­mit­tee’s sug­ges­tions back in May, the FDA has hand­ed one ap­proval — and one re­jec­tion — to Achao­gen’s lead an­tibi­ot­ic.

Blake Wise

Al­though some­what ex­pect­ed, the news ap­pears to be dis­ap­point­ing Achao­gen’s in­vestors. The com­pa­ny’s stock $AKAO is down 11% in pre-mar­ket trad­ing.

Achao­gen’s drug, which now goes by the brand name Zem­dri (pla­zomicin), has been OK’ed to treat com­pli­cat­ed uri­nary tract in­fec­tions. How­ev­er, the agency is­sued a com­plete re­sponse let­ter (CRL) in re­sponse to Achao­gen’s bid to treat blood­stream in­fec­tions, cit­ing lack of ef­fec­tive­ness of the drug in a clin­i­cal study. The com­pa­ny said it plans to meet with the FDA to see if there’s a “fea­si­ble res­o­lu­tion” to ad­dress the CRL.

A large part of what seems to have doomed Achao­gen’s plans for the blood­stream in­fec­tions in­di­ca­tion was a small­er-than-ex­pect­ed sam­ple size that com­pli­cat­ed sta­tis­ti­cal analy­sis. The biotech had to amend pro­to­cols for its Phase III study (ACHN-490-007) be­cause it could on­ly en­roll 37 pa­tients out of the planned 286. That re­sult­ed in sta­tis­ti­cal lim­i­ta­tions prov­ing Zem­dri’s su­pe­ri­or ef­fi­ca­cy to col­istin in the com­para­tor arm — a point of dis­cus­sion among the FDA’s ad­vi­so­ry com­mit­tee on the drug ear­li­er this year.

For cU­TI, on the oth­er hand, com­mit­tee mem­bers had no prob­lem unan­i­mous­ly en­dors­ing Zem­dri based on a study that showed it was non­in­fe­ri­or to meropen­em, even con­sid­er­ing a larg­er mar­gin of 15% (ver­sus a con­ven­tion­al 10%) the FDA sanc­tioned.

“The ap­proval of Zem­dri marks a sig­nif­i­cant mile­stone for Achao­gen and we are ex­cit­ed to of­fer health­care prac­ti­tion­ers a new treat­ment op­tion for pa­tients with cer­tain se­ri­ous bac­te­r­i­al in­fec­tions,” said Achao­gen’s CEO Blake Wise in a state­ment. “Zem­dri is de­signed to re­tain its po­tent ac­tiv­i­ty in the face of cer­tain dif­fi­cult-to-treat mul­tidrug re­sis­tant in­fec­tions, in­clud­ing CRE and ES­BL- pro­duc­ing En­ter­obac­te­ri­aceae.”

Im­age: Mc­Far­lene Ar­chi­tects

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.

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 is 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 is 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 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.

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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Bris­tol My­ers pledges to sell its Ac­celeron shares as ac­tivist in­vestors cir­cle Mer­ck­'s $11.5B buy­out — re­port

Just as Avoro Capital’s campaign to derail Merck’s proposed $11.5 billion buyout of Acceleron gains steam, Bristol Myers Squibb is leaning in with some hefty counterweight.

The pharma giant is planning to tender its Acceleron shares, Bloomberg reported, which add up to a sizable 11.5% stake. Based on the offer price, the sale would net Bristol Myers around $1.3 billion.

To complete its deal, Merck needs a majority of shareholders to agree to sell their shares.

Leen Kawas (L) has resigned as CEO of Athira and will be replaced by COO Mark Litton

Ex­clu­sive: Athi­ra CEO Leen Kawas re­signs af­ter in­ves­ti­ga­tion finds she ma­nip­u­lat­ed da­ta

Leen Kawas, CEO and founder of the Alzheimer’s upstart Athira Pharma, has resigned after an internal investigation found she altered images in her doctoral thesis and four other papers that were foundational to establishing the company.

Mark Litton, the company’s COO since June 2019 and a longtime biotech executive, has been named full-time CEO. Kawas, meanwhile, will no longer have ties to the company except for owning a few hundred thousand shares.

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Some can­cer pa­tients now have to find oth­er op­tions as Bris­tol My­er­s' Abrax­ane falls in­to short­age from man­u­fac­tur­ing woes

When Beth Hogan, a metastatic pancreatic cancer patient, showed up for her infusion at Yale’s Smilow Cancer Hospital in New Haven, CT on Oct. 11, she said she was informed that day that she would not be receiving Bristol Myers Squibb’s Abraxane, part of her combo treatment, because of a shortage.

“I was told we don’t know when you can have it,” she told Endpoints News via email, adding that she doesn’t expect to receive any Abraxane this coming Monday at her treatment appointment either, and she doesn’t know when things will change.

Michel Vounatsos, Biogen CEO (Credit: World Economic Forum/Valeriano Di Domenico)

Up­dat­ed: 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 reimbursement, 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. That’s a far cry from the peak Wall Street sales estimate of about $9 billion in annual sales, and even a ways away from the sell-side consensus of about $17 million in Q3 sales.

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Eli Lil­ly or­dered to pay roy­al­ties on block­buster di­a­betes drugs, though ex­act dam­ages are un­clear

A federal court found Eli Lilly in breach of a royalty agreement with an Arizona company, likely sending the case — which deals with Lilly’s blockbuster diabetes drugs — to a trial.

The Arizona District Court ordered Lilly to pay the royalties to Tucson, AZ-based Research Corporation Technologies, per an opinion delivered Tuesday, stemming from a 1990 agreement involving materials used in manufacturing Lilly’s insulin products. Lilly had agreed to pay a 2% royalty on worldwide sales, and the exact amount of damages will be determined in a trial, Judge Scott Rash wrote.