Mer­ck gets a PDU­FA date for its chron­ic cough con­tender; Mor­phic shares sky­rock­et on the heels of PhI da­ta roll­out in IBD

Mer­ck should know be­fore the end of this year whether or not the FDA will ap­prove its drug gefapix­ant for chron­ic cough. The reg­u­la­tor has set a PDU­FA date of Dec. 21 for the drug, giv­ing the phar­ma gi­ant a reg­u­lar re­view for a close­ly watched de­vel­op­ment pro­gram.

Roy Baynes

Gefapix­ant is the leader among a group of P2X3 re­cep­tor an­tag­o­nists that are rac­ing to com­pete for a share of this pro­gram. That’s one rea­son why the agency has in­formed Mer­ck that it plans to hold an ad­vi­so­ry com­mit­tee for the re­view.

Mer­ck’s Roy Baynes has de­scribed this one as an­oth­er “pipeline in a prod­uct,” but it’s no shoo-in. The drug hit sta­tis­ti­cal sig­nif­i­cance on risk re­duc­tion, but in­ves­ti­ga­tors al­so flagged high dropout rates due to ad­verse events in the drug arm. So even with an OK, the phar­ma gi­ant could soon face ri­vals an­gling for bet­ter ef­fi­ca­cy and a stronger safe­ty pro­file.

Bay­er and Sh­iono­gi have com­pet­i­tive pro­grams in the clin­ic, but the chief ri­val right now is Bel­lus, though BLU-5937 failed a Phase II tri­al. That drug is now in an adap­tive Phase IIb. — John Car­roll

Mor­phic shares soar af­ter Phase I read­out for nov­el in­hibitor in IBD

On Mon­day, Waltham, MA-based Mor­phic Ther­a­peu­tics un­veiled Phase I proof-of-con­cept da­ta for its oral α4β7 in­te­grin in­hibitor MORF-057 in IBD. Turns out in­vestors like that da­ta — a lot.

Mor­phic $MORF was trad­ing up about 90% ear­ly Mon­day morn­ing on the heels of the news, putting the biotech’s shares in the range of $70.

In the three high­est dose co­horts of MORF-057’s Phase I pro­gram, the drug showed a mean re­cep­tor oc­cu­pan­cy of 95%, Mor­phic said, of­fer­ing hope that the drug can suf­fi­cient­ly “sat­u­rate” the α4β7 in­te­grin.

Mor­phic is plan­ning a Phase II study based on the ear­ly-stage re­sults in ul­cer­a­tive col­i­tis. Mean­while, the biotech is run­ning a con­cur­rent food-ef­fect study. — Kyle Blanken­ship

Chi­nook hands over up­front pay­ment to launch CKD deal with Evotec

Cana­da’s Chi­nook Ther­a­peu­tics has inked a dis­cov­ery and de­vel­op­ment deal with Evotec on new drugs for chron­ic kid­ney dis­ease.

In the deal the Eu­ro­pean CRO is gain­ing an undis­closed up­front pay­ment, re­search funds and more as Evotec’s team starts the hunt for new drugs.

“CKD is a col­lec­tive term for a va­ri­ety of dis­eases that cause a grad­ual loss of kid­ney func­tion. The di­ver­si­ty in the un­der­ly­ing caus­es makes this area es­pe­cial­ly fa­vor­able for da­ta-dri­ven and sys­tem­at­i­cal­ly per­son­al­ized ap­proach­es,” said Cord Dohrmann, the CSO of Evotec. “By lever­ag­ing our com­ple­men­tary plat­forms, Evotec and Chi­nook have the best pos­si­ble start­ing po­si­tion to tack­le par­tic­u­lar rare forms of CKD with the goal to de­vel­op dis­ease-mod­i­fy­ing ther­a­peu­tics for un­der­served pa­tient pop­u­la­tions.”

Back last sum­mer Chi­nook ex­e­cut­ed a re­verse merg­er with the strug­gling Aduro to get on­to Nas­daq. The biotech raised more than $100 mil­lion to go along with the merg­er. — John Car­roll

Ox­u­lar nets $37M to ad­vance once-a-year eye dis­ease treat­ments

British biotech Ox­u­lar Lim­it­ed com­plet­ed a new $37 mil­lion fund­ing round it hopes to use to fi­nance clin­i­cal tri­als.

The cash will go to­ward Phase II stud­ies be­gin­ning lat­er this year to eval­u­ate the pro­gram OXU-001 for the treat­ment of DME, which Ox­u­lar says is the lead­ing cause of blind­ness in young adults in de­vel­oped coun­tries. Promi­nent Eu­ro­pean VC firm For­bion led the round.

Giv­en that a key chal­lenge for these pa­tients is re­peat­ed clin­i­cal vis­its, Ox­u­lar has de­vel­oped OXU-001 to be a sus­tained-re­lease for­mu­la­tion of dex­am­etha­sone that can pro­vide up to 12 months of treat­ment fol­low­ing a sin­gle dose.

Some of the funds will al­so go to­ward a Phase II study for a sep­a­rate can­di­date in retinoblas­toma. Pro­grams for uveal melanoma and age-re­lat­ed mac­u­lar de­gen­er­a­tion and di­a­bet­ic retinopa­thy will al­so be prepped for IND sub­mis­sions. — Max Gel­man

Rea­ta shoots for fast re­view of its first NDA

War­ren Huff

Af­ter a years-long clin­i­cal quest, Plano, TX-based Rea­ta has filed an NDA in search of an ap­proval for bar­dox­olone in Al­port syn­drome.

The biotech’s CEO, War­ren Huff, adds that they’re shoot­ing for pri­or­i­ty re­view sta­tus in an at­tempt to short­en the FDA’s han­dling of the NDA by 4 months. This ap­pli­ca­tion marks Rea­ta’s first for­mal shot at an FDA ap­proval.

In an up­date, Rea­ta al­so not­ed plans to re­vise an­oth­er clin­i­cal study that had been paused due to the pan­dem­ic.

We are plan­ning to amend the FAL­CON pro­to­col to in­crease the tar­get en­roll­ment from 300 pa­tients to a to­tal of 550 pa­tients.   With the planned in­crease in an­tic­i­pat­ed en­roll­ment, we ex­pect to com­plete en­roll­ment in FAL­CON by the end of 2021.

John Car­roll

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 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.

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.