Durable, safe and ef­fec­tive long-term kid­ney drug da­ta from Tri­ci­da fu­el block­buster po­ten­tial

Kid­ney drug de­vel­op­er Tri­ci­da’s shares shot up on Thurs­day, af­ter long-term da­ta high­light­ed its lead drug’s durable ef­fi­ca­cy and safe­ty pro­file as a treat­ment for meta­bol­ic aci­do­sis, in which faulty kid­neys are not able to ex­pel the acid caus­ing a buildup in the body.

The drug — TRC101 — is de­signed to bind to hy­drochlo­ric acid in the GI tract, trig­ger­ing the ejec­tion of acid via ex­cre­tion — there­by di­min­ish­ing acid lev­els and stim­u­lat­ing blood bi­car­bon­ate. The com­pa­ny ex­pects to sub­mit an ap­pli­ca­tion to mar­ket the drug by the sec­ond half of this year for the con­di­tion com­mon­ly caused by chron­ic kid­ney dis­ease (and be­lieved to ac­cel­er­ate the pro­gres­sion of CKD), in­crease the risk of mus­cle wast­ing and cause the loss of bone den­si­ty. If ap­proved, it will be the first drug to win the FDA nod specif­i­cal­ly for meta­bol­ic aci­do­sis.

Ger­rit Klaern­er

Da­ta post­ed last June showed the drug in­duced a sta­tis­ti­cal­ly sig­nif­i­cant in­crease in blood bi­car­bon­ate in 208 pa­tients af­ter 12 weeks in a Phase III study, hours af­ter the com­pa­ny ad­ver­tised plans to go pub­lic in a $150 mil­lion IPO. On Thurs­day, Tri­ci­da re­vealed da­ta from a 40-week ex­ten­sion to that study in the 185 pa­tients that com­plet­ed one year as part of the tri­al.

The drug met all the pri­ma­ry and sec­ondary goals of the ex­ten­sion por­tion of the tri­al. The main goal of the ex­ten­sion study was the as­sess­ment of the long-term safe­ty pro­file of TRC101 ver­sus place­bo — and da­ta showed 2.6% of the pa­tients on TRC101 ver­sus 9.8% on place­bo dis­con­tin­ued the 40-week treat­ment pe­ri­od pre­ma­ture­ly.

Sig­nif­i­cant­ly, da­ta on the drug’s dura­bil­i­ty were al­so pos­i­tive. Over 52 weeks, 63% of the 111 TRC101-treat­ed pa­tients ex­hib­it­ed an in­crease in blood bi­car­bon­ate lev­el of at least 4 mil­liequiv­a­lents per liter, or achieved a blood bi­car­bon­ate lev­el in the nor­mal range — com­pared to 38% of the 74 place­bo sub­jects (p=0.0015).

The sta­tis­ti­cal plan al­so called for the eval­u­a­tion of TRC101 ver­sus place­bo for the com­pos­ite end­point of: all-cause mor­tal­i­ty, dial­y­sis/kid­ney trans­plant or a ≥50% de­cline in es­ti­mat­ed glomeru­lar fil­tra­tion rate (a test used to check how well the kid­neys are work­ing) — DD50 when tak­en to­geth­er — over the 52 week pe­ri­od. Of the 124 sub­jects giv­en TRC101, 4% (5) had a DD50 event, in­clud­ing one pa­tient who ini­ti­at­ed dial­y­sis. In con­trast, of the 93 sub­jects ran­dom­ized to the place­bo group, 10.8% (10) sub­jects had a DD50 event, in­clud­ing four deaths and one who ini­ti­at­ed dial­y­sis.

While the tri­al was not pow­ered to as­sess all-cause mor­tal­i­ty and/or the pro­gres­sion of CKD out­comes, Tri­ci­da said it ob­served a 65% re­duc­tion in the an­nu­al­ized event rate of the com­pos­ite end­point of all-cause mor­tal­i­ty and/or the pro­gres­sion of CKD in TRC101-treat­ed sub­jects ver­sus the place­bo group.

“The 52-week…re­sults far ex­ceed­ed our ex­pec­ta­tions,” said com­pa­ny chief Ger­rit Klaern­er said in a state­ment. “We did not an­tic­i­pate that we would ob­serve ev­i­dence of clin­i­cal ben­e­fit be­yond the in­crease in blood bi­car­bon­ate in pa­tients treat­ed with TRC101 un­til the read out of the re­sults of our post­mar­ket­ing tri­al…in the 2022 to 2023 time­frame.”

Shares of the South San Fran­cis­co-based drug de­vel­op­er $TC­DA leapt more than 57% on Thurs­day, clos­ing at $37.80.

Cowen’s Phil Nadeau, who deemed the da­ta ‘im­pres­sive,’ ex­pects TRC101 will achieve $1 bil­lion in rev­enue by 2025.

(T)here is a ma­jor need to con­trol meta­bol­ic aci­do­sis and slow the pro­gres­sion of CKD. Though sodi­um bi­car­bon­ate is ef­fec­tive, its high sodi­um con­cen­tra­tion makes most CKD pa­tients in­el­i­gi­ble for it…With 25 mil­lion peo­ple in the U.S. hav­ing stage 3, 4, or 5 CKD, TRC101 al­so ad­dress­es a large pa­tient pop­u­la­tion, and even mod­est pen­e­tra­tion could yield bil­lions in sales.

(T)he com­pos­ite end­point da­ta com­bined with the phys­i­cal func­tion­ing scores show clear, clin­i­cal­ly mean­ing­ful ben­e­fits to pa­tients. In fact, the dra­mat­ic re­duc­tion in the com­pos­ite end­point im­plies that physi­cians need to treat at most 3 pa­tients in or­der for 1 to de­rive a clin­i­cal­ly mean­ing­ful re­duc­tion in CKD pro­gres­sion.

Sep­a­rate­ly, Tri­ci­da on Thurs­day said it had amend­ed its debt fa­cil­i­ty with Her­cules Cap­i­tal, rais­ing the to­tal amount avail­able to up to $200 mil­lion from the $100 mil­lion agreed in Feb­ru­ary 2018.

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.”

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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Vlad Coric, Biohaven CEO

UP­DAT­ED: Fresh off $11.6B sale to Pfiz­er, New Bio­haven hits Phase III set­back just weeks af­ter Vlad Coric chalked up promise

When Pfizer bought up Biohaven’s migraine portfolio in the largest M&A deal of the year earlier this month, Biohaven CEO Vlad Coric promised the rest of the pipeline, which will live on under the umbrella of New Biohaven, still has a lot to offer. But that vision took a dent Monday as the drugmaker revealed it’s once again flopped on troriluzole.

The glutamate regulator failed to meet the primary endpoint on a Phase III study in patients with spinocerebellar ataxia, an inherited disorder that impairs a person’s ability to walk, speak and swallow. SCA can also lead to premature death.

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Bay­er sounds re­treat from a $670 mil­lion CAR-T pact in the wake of a pa­tient death

Two months after Atara Biotherapeutics hit the hold button on its lead CAR-T 2.0 therapy following a patient death, putting the company under the watchful eye of the FDA, its Big Pharma partners at Bayer are bowing out of a $670 million global alliance. And the move is forcing a revamp of Atara’s pipeline plans, even as research execs vow to continue work on the two drugs allied with Bayer 18 months ago, which delivered a $60 million cash upfront.

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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.