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

Janet Woodcock (Greg Nash/Pool via AP Images)

'I re­al­ly don’t look back': Janet Wood­cock on her tran­si­tion away from drugs

Janet Woodcock may have one of the most historically long and drug-intense tenures in FDA history, but her new role is outside of all things pharma and the once-acting FDA commissioner isn’t looking back.

“No I really don’t look back,” Woodcock told Endpoints News via email on Monday morning. “Yes I will be transitioning. Longer discussion on infrastructure needed.”

Co­pay coupons gone wrong, again: Pfiz­er pays al­most $300K to set­tle com­plaints in four states

Pfizer has agreed to pay $290,000 to settle allegations of questionable copay coupon practices in Arizona, Colorado, Kansas, and Vermont from 2014 to 2018.

While the company has not admitted any wrongdoing as part of the settlement, Pfizer has agreed to issue restitution checks to about 5,000 consumers.

A Pfizer spokesperson said the company has “enhanced its co-pay coupons to alleviate the concerns raised by states and agreed to a $30,000 payment to each.”

Delaware court rules against Gilead and Astel­las in years-long patent case

A judge in Delaware has ruled against Astellas Pharma and Gilead in a long-running patent case over Pfizer-onwed Hospira’s generic version of Lexiscan.

The case kicked off in 2018, after Hospira submitted an Abbreviated New Drug Application (ANDA) for approval to market a generic version of Gilead’s Lexiscan. The drug is used in myocardial perfusion imaging (MPI), a type of nuclear stress test.

Taye Diggs (courtesy Idorsia)

Idor­sia inks an­oth­er celebri­ty en­dors­er deal with ac­tor and dad Taye Dig­gs as Qu­viviq in­som­nia am­bas­sador

Idorsia’s latest Quviviq insomnia campaign details the relatable dad story of a well-known celebrity — actor and Broadway star Taye Diggs.

Diggs stopped sleeping well after the birth of his son, now more than 10 years ago. Switching mom-and-dad nightly shifts to take care of a baby interrupted his sleep patterns and led to insomnia.

“When you’re lucky enough to be living out your dream and doing what you want, but because of something as simple as a lack of sleep, you’re unable to do that, it felt absolutely — it was treacherous,” he says in an interview-style video on the Quviviq website.

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Belén Garijo, Merck KGaA CEO (Kevin Wolf/AP Images for EMD Serono)

Mer­ck KGaA pumps €440M in­to ex­pand­ing and con­struct­ing Irish man­u­fac­tur­ing fa­cil­i­ties

The area of Ireland famous for Blarney Castle and its cliffsides along the Atlantic Ocean is seeing Merck KGaA expand its commitment there.

The German drug manufacturer is expanding its membrane and filtration manufacturing capabilities in Ireland. The company will invest approximately €440 million ($470 million) to increase membrane manufacturing capacity in Carrigtwohill, Ireland, and build a new manufacturing facility at Blarney Business Park, in County Cork, Ireland.

Rep. Katie Porter (D-CA) (Michael Brochstein/Sipa USA/Sipa via AP Images)

House Dems to Sen­ate lead­er­ship: Quick­ly move a rec­on­cil­i­a­tion bill with drug price ne­go­ti­a­tion re­forms

Twenty House Democrats, including Reps. Katie Porter of California and Susan Wild of Pennsylvania, are calling on Senate leaders to move quickly with a reconciliation bill (meaning they only need a simple majority for passage) with prescription drug pricing reforms, and to include adding new authority for Medicare to negotiate drug prices.

They also called on the Senate to specifically follow suit with the House passage of a $35 per month insulin cap (as Senate Majority Leader Chuck Schumer’s deadline for a vote on that provision has come and gone), and to cap Medicare Part D costs at $2,000 per year for seniors.

An NYU surgeon transplants an engineered pig kidney into the outside of a brain-dead patient (Joe Carrotta/NYU Langone Health)

'Xeno­trans­plan­ta­tion is com­ing': New NE­JM pa­per gives de­tailed look in­to 2 pig-to-hu­man kid­ney trans­plant cas­es

The thymokidney is a curious organ, if you could call it that. It’s a sort of Frankensteinian creation — a system of pig thymus embedded underneath the outer layer of a pig’s kidney, made for human transplantation.

In the first case of pig-to-human xenotransplantation of a kidney into a brain-dead patient, the thymokidney quietly featured front and center.

In that experiment, which took place in September of last year, NYU researchers led by Robert Montgomery sutured a pig thymokidney onto the leg of a brain-dead 66-year-old woman. That case was widely reported on by a horde of major media outlets, including the New York Times, the BBC, and an in-depth feature by USA Today.

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A cell ther­a­py biotech finds a de­liv­ery part­ner; FDA re­moves clin­i­cal hold on Ocu­gen's Covid can­di­date

Umoja Biopharma will pair its so-called VivoVec particles with Lupagen’s Side CAR-T delivery system to target certain cancers, the companies said Monday.

As part of the deal, Umoja’s cell therapies could be delivered via an additional route thanks to Lupagen’s extracorporeal in vivo tech. The Dallas gene therapy company’s tech is expected to provide efficiency and “highly controlled viral vector targeting of T cells,” the companies added.

Phillip Gomez, SIGA CEO

UP­DAT­ED: On the back of SIGA Tech­nolo­gies' win with the FDA, the mon­key­pox virus sees the com­pa­ny spring­ing to fur­ther ac­tion

As the cases of monkeypox now sit at well over 100 worldwide and have spread to multiple continents, the orders for any type of vaccine against monkeypox are seeing nations and medical bodies looking to get their hands on anything and everything. And now SIGA Technologies seems to be getting in on the action.

According to Euronews, SIGA Technologies, a pharmaceutical company that is focused on providing medical countermeasures to biological and chemical attacks, is now in talks with several European authorities looking to stockpile its antiviral that can counter monkeypox. The drug known as tecovirimat or Tpoxx was approved by the FDA in 2018 as a vaccine for smallpox but was approved by the European Medicines Agency to also act against monkeypox, cowpox and complications from immunization with vaccinia.