EMA en­dors­es Teva's mi­graine drug; Dis­as­ter prone CTI Bio­Phar­ma re­scinds EU ap­pli­ca­tion for pa­cri­tinib

→ The EMA was busy on Fri­day. The Eu­ro­pean drug reg­u­la­tor, which is cur­rent­ly in the process of shift­ing its head­quar­ters to Am­s­ter­dam from Lon­don, rec­om­mend­ed the ap­proval of Te­va’s mi­graine-pre­ven­tion drug, Ajovy. A fi­nal de­ci­sion is ex­pect­ed in the first half of this year, and if ap­proved the treat­ment drug will be the first and on­ly an­ti-CGRP med­i­cine in the EU with both quar­ter­ly and month­ly dos­ing op­tions, the Is­raeli drug­mak­er said. CTI Bio­Phar­ma, how­ev­er, had bad news to re­port on the Eu­ro­pean front for its myelofi­bro­sis drug pa­cri­tinib. The no­to­ri­ous­ly fail­ure-prone drug de­vel­op­er an­nounced it was with­draw­ing an ap­pli­ca­tion to mar­ket the drug in the EU, fol­low­ing dis­cus­sions with the EMA. Last year, Shire backed away from a part­ner­ship with the ill-fat­ed com­pa­ny, fol­low­ing a mixed da­ta read­out from a piv­otal study that was pre­vi­ous­ly put on a full clin­i­cal hold — un­der the be­hest of the FDA — af­ter the death of sev­er­al pa­tients. The com­pa­ny’s shares $CTIC tum­bled more than 15% in ear­ly Fri­day trad­ing.

→ Sep­a­rate­ly on Fri­day, the EMA al­so laid out guid­ance to help bio­phar­ma com­pa­nies pre­pare for Brex­it, so as to en­sure sup­ply is not dis­rupt­ed — on the as­sump­tion the UK will be­come a third coun­try as of 30 March 2019. For pre-sub­mis­sion meet­ings re­gard­ing hu­man and an­i­mal drugs re­quest­ed from last Oc­to­ber, the EMA will en­gage with man­u­fac­tur­ers via tele­con­fer­ence or vir­tu­al meet­ings. Be­tween 11 Feb­ru­ary and 15 March 2019, no such meet­ings for ini­tial mar­ket­ing au­tho­ri­sa­tion ap­pli­ca­tions will take place, while the EMA moves to its new home in Am­s­ter­dam, it said.

→ On Thurs­day at a par­lia­men­tary com­mit­tee meet­ing, UK health and so­cial care sec­re­tary Matt Han­cock said the British gov­ern­ment was pri­or­i­tiz­ing med­i­cines over food if a no-deal Brex­it would make it tricky to im­port prod­ucts in ei­ther cat­e­go­ry of goods. Last year, Han­cock  asked drug sup­pli­ers to keep six weeks worth of med­i­cines stock­piled in ad­di­tion to their buffer stocks and last month the UK’s Med­i­cines and Health­care prod­ucts Reg­u­la­to­ry Agency (MHRA) said that in the event of a no-deal, the UK would be no longer be part of the EMA um­brel­la, and that reg­u­la­to­ry sub­mis­sions for drugs would be made di­rect­ly to the MHRA.

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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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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Lian­Bio an­nounces terms for IPO next week; NIH and White House of­fi­cials dis­cuss hy­po­thet­i­cal bio-med re­search agency

LianBio, a biotech that has operations in both the US and China, announced the terms yesterday for its initial public offering.

The biotech plans to raise $325 million by offering 20.3 million shares priced between $15 and $17.

At the midpoint of the proposed range, LianBio could command a fully diluted market value of $1.8 billion, based on a number of expected outstanding shares.

The two year old biotech has focused on in-licensing and commercialization of therapeutics in China, Hong Kong, Taiwan, Macau and other Asian markets. The company currently has nine programs across five therapeutic areas, including oncology, cardiovascular, and inflammatory diseases.

Sen. Richard Durbin (D-IL, foreground) and Sen. Richard Blumenthal (D-CT) (Patrick Semansky/AP Images)

Sen­a­tors back FDA's plan to re­quire manda­to­ry pre­scriber ed­u­ca­tion for opi­oids

Three Senate Democrats are backing an FDA plan to require mandatory prescriber education for opioids as overdose deaths have risen sharply over the past decade, with almost 97,000 American opioid-related overdose deaths in the past year alone.

While acknowledging a decline in overall opioid analgesic dispensing in recent years, the FDA said it’s reconsidering the need for mandatory prescriber training through a REMS given the current situation with overdoses, and is seeking input on the aspects of the opioid crisis that mandatory training could potentially mitigate.

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.

Boost­er bo­nan­za: FDA en­dors­es 'mix-and-match' scheme, and Mod­er­na and J&J too

The FDA late Wednesday signed off on authorizing the use of heterologous — or what FDA calls a “mix and match” of a primary vaccine series and different booster doses — for all currently available Covid-19 vaccines, in addition to separately authorizing Moderna and J&J boosters.

On the mix-and-match approach, which FDA officials insisted isn’t too confusing in a press conference, the agency offered the example of an 18-year-old who received the J&J shot at least two months ago and may now receive a single booster of the J&J, a half dose of the Moderna, or the Pfizer-BioNTech booster.