Per­lara col­lab­o­rates with Har­vard/Un­di­ag­nosed Dis­eases Net­work; Verona shares soar on pos­i­tive CF da­ta

→ Per­lara, the rare dis­ease com­pa­ny found­ed by Twit­ter’s fa­vorite biotech rebel Ethan Perl­stein, an­nounced two sci­en­tif­ic dis­cov­ery col­lab­o­ra­tions Fri­day with Har­vard Med­ical School and the Un­di­ag­nosed Dis­eases Net­work. The re­search part­ner­ships are for two rare mono­genic neu­rode­vel­op­men­tal dis­or­ders. One is caused by a mu­ta­tion in GNA01 and the oth­er caused by a mu­ta­tion in RPS6KA3 (Cof­fin-Lowry Syn­drome). Per­lara plans to de­vel­op and val­i­date ne­ma­tode and fly “pa­tient avatars” of path­o­gen­ic GNA01 and RPS6KA3 mu­ta­tions for use in high-through­put phe­no­typ­ic drug screens. Perl­stein, CEO of the Per­lara, is best known in the bio­phar­ma world through his out­spo­ken Twit­ter pres­ence, where he’s laid bare the dif­fi­cul­ty of rais­ing cap­i­tal as a young com­pa­ny work­ing on “dis­eases thought too rare to at­tract the in­ter­est of phar­ma­ceu­ti­cal com­pa­nies.” He’s been called rad­i­cal for his trans­paren­cy, and a rebel for chal­leng­ing fundrais­ing norms on a pub­lic stage. In lieu of sig­nif­i­cant ven­ture cap­i­tal funds, Perl­stein has in­stead re­lied heav­i­ly on part­ner­ships with pa­tient groups since the com­pa­ny’s 2014 in­cep­tion. Last year, how­ev­er, the com­pa­ny did raise a $7.4M Se­ries A.

→  Shares of Verona Phar­ma shot up 25% $VR­NA on Fri­day af­ter the Lon­don-based biotech tout­ed Phase IIa da­ta for its cys­tic fi­bro­sis ther­a­py RPL554. Pa­tients in the small study achieved a 6% sus­tained im­prove­ment in FEV1, ac­cord­ing to re­searchers, af­ter a sin­gle dose. The com­pa­ny de­scribes the drug as a “dual in­hibitor of the en­zymes phos­pho­di­esterase 3 and 4 that acts as both a bron­chodila­tor and an an­ti-in­flam­ma­to­ry agent in a sin­gle com­pound.”

→ The FDA has ac­cept­ed for re­view Re­gen­eron $REGN and its part­ner Sanofi’s $SNY mar­ket­ing ap­pli­ca­tion for Dupix­ent (dupilum­ab). The duo is ask­ing for the OK to sell the drug as an add-on main­te­nance treat­ment in cer­tain adults and ado­les­cents at least 12 years old with mod­er­ate-to-se­vere asth­ma. The ap­pli­ca­tion is backed by clin­i­cal da­ta from 2,888 adults and ado­les­cents from three piv­otal tri­als, the com­pa­nies said in a state­ment. The ac­tion date is set for Oc­to­ber 20. Dupix­ent, a ful­ly hu­man mon­o­clon­al an­ti­body, is al­ready ap­proved in the US for atopic der­mati­tis.

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.