Ae­tion to col­lab­o­rate with UK's NICE on RWE; 4D's cys­tic fi­bro­sis gene ther­a­py cleared for clin­i­cal tri­als

New York-based re­al world ev­i­dence, or RWE, firm Ae­tion is team­ing up with the Na­tion­al In­sti­tute for Health and Care Ex­cel­lence in the Unit­ed King­dom to an­a­lyze da­ta such as hos­pi­tal records, pre­scrip­tion records and more to study clin­i­cal ef­fec­tive­ness and fill gaps in ev­i­dence.

Car­olyn Mag­ill

As Ae­tion CEO Car­olyn Mag­ill told End­points News, the com­pa­ny’s goal is to col­lect ev­i­dence that would be hard­er to get out­side of a con­trolled set­ting, such as a clin­i­cal tri­al.

“So [in a clin­i­cal tri­al] you have a nurse tap­ping you on the shoul­der say­ing, ‘Did you take your med­ica­tion to­day? Did you al­ways take it at the same point in time?’ Where­as once the drug is on the mar­ket, our reg­u­lar lives get in the way,” Mag­ill said.

Mag­ill said that RWE helps cap­ture what hap­pens to pop­u­la­tions who may be un­der­rep­re­sent­ed in clin­i­cal tri­als — such as women of child­bear­ing age, chil­dren, and those who have dif­fer­ent com­bi­na­tions of chron­ic ill­ness­es.

In April, af­ter talk­ing for sev­er­al years, rep­re­sen­ta­tives from NICE and Ae­tion worked along­side rep­re­sen­ta­tives from the EMA and co-pub­lished a pa­per on a pos­si­ble struc­ture for or­ga­niz­ing frag­ment­ed RWE rec­om­men­da­tions world­wide to achieve com­pre­hen­sive guid­ance. That col­lab­o­ra­tion marked Ae­tion’s first part­ner­ship with an in­ter­na­tion­al HTA body, ac­cord­ing to a com­pa­ny state­ment an­nounc­ing the col­lab­o­ra­tion.

“As NICE con­tin­ues to de­liv­er on our five-year strate­gic vi­sion, in­clud­ing rapid, ro­bust, and re­spon­sive tech­nol­o­gy eval­u­a­tion, un­der­stand­ing how to make the best use of RWE is vi­tal in help­ing us re­solve gaps in knowl­edge and dri­ving for­ward ac­cess to in­no­va­tions for pa­tients,” Páll Jóns­son, NICE’s pro­gramme di­rec­tor for da­ta, said in a state­ment.

4D’s aerosolized cys­tic fi­bro­sis gene ther­a­py cleared for hu­man tri­als

Biotech 4D Mol­e­c­u­lar Ther­a­peu­tics said to­day that their in­ves­ti­ga­tion­al, cys­tic fi­bro­sis gene ther­a­py 4D-710 has been cleared by the FDA for clin­i­cal tri­als with an ap­proved IND.

4D ex­pects to be able to start a Phase I/II clin­i­cal tri­al with ap­prox­i­mate­ly 18 pa­tients be­fore the end of the year.

David Kirn

“Ul­ti­mate­ly, we be­lieve 4D-710 has the po­ten­tial to treat a broad cys­tic fi­bro­sis pa­tient pop­u­la­tion, in­clud­ing those pa­tients treat­ed with cur­rent CFTR mod­u­la­tors, all of which re­quire dai­ly dos­ing over the pa­tient’s life­time and gen­er­al­ly re­sult in on­ly par­tial cor­rec­tion of lung func­tion,” said 4DMT CEO and co-founder David Kirn in a state­ment.

This an­nounce­ment comes just a few months af­ter Roche sev­ered their part­ner­ship with 4D, re­sult­ing in shares of $FDMT tank­ing 10%.

The non-prof­it Cys­tic Fi­bro­sis Foun­da­tion will pur­chase 125,715 shares of 4D’s com­mon stock for fur­ther de­vel­op­ment of the drug for ap­prox­i­mate­ly $4 mil­lion, ac­cord­ing to a com­pa­ny state­ment.

CRO In­o­tiv buys out Pla­to Bio­Phar­ma in $15 mil­lion deal

In­di­ana-based CRO In­o­tiv is ac­quir­ing in vi­vo re­search firm Pla­to Bio­phar­ma as part of an in vi­vo phar­ma­col­o­gy ex­pan­sion, ac­cord­ing to a com­pa­ny an­nounce­ment to­day.

In a $15 mil­lion deal with $10 mil­lion in cash, along with com­mon shares and promis­so­ry notes mak­ing up the oth­er $5 mil­lion, In­o­tiv ex­pects to re­tain all 34 of Pla­to’s em­ploy­ees af­ter the trans­ac­tion is com­plete.

“The ac­qui­si­tion of PBI marks an­oth­er mile­stone for In­o­tiv as we con­tin­ue to ex­e­cute on our strat­e­gy to build a com­plete range of con­tract re­search ser­vices,” said In­o­tiv chief strat­e­gy of­fi­cer John Sagartz. “This ad­di­tion al­lows us to sup­port and ac­cel­er­ate PBI’s growth plans, while fur­ther ex­pand­ing in­to more phys­i­o­log­ic and phar­ma­co­log­ic of­fer­ings.”

“We look for­ward to join­ing the team and broad­en­ing In­o­tiv’s ser­vice of­fer­ing,” Pla­to founder and CEO Craig Pla­to said in a state­ment. “Our core val­ues and col­lab­o­ra­tive ap­proach align well, and our fo­cus on rig­or­ous sci­en­tif­ic ex­e­cu­tion and ded­i­ca­tion to de­liv­er­ing ex­cep­tion­al client ex­pe­ri­ence will con­tin­ue to be para­mount.”

Xi Jin­ping calls for im­proved and in­creased biose­cu­ri­ty mea­sures against zoonot­ic dis­ease trans­mis­sion

Chi­nese Pres­i­dent Xi Jin­ping called for “ac­tive de­fens­es” last week to pre­vent an­i­mal-to-hu­man trans­mis­sion of dis­eases dur­ing his ad­dress to the Po­lit­i­cal Bu­reau of the CPC Cen­tral Com­mit­tee.

In a group ses­sion on biose­cu­ri­ty, Xi, who is al­so the gen­er­al sec­re­tary of the Com­mu­nist Par­ty of Chi­na (CPC) Cen­tral Com­mit­tee, called for a man­age­ment net­work to re­spond to any po­ten­tial dis­eases that might arise in the fu­ture.

He al­so stressed more su­per­vi­sion over the safe­ty of bi­o­log­i­cal re­sources. Specif­i­cal­ly, Xi point­ed out a need to strength­en ex­am­i­na­tion of im­port­ed goods, and urged pun­ish­ment for all il­le­gal acts.

Call­ing the buildup of biose­cu­ri­ty “a long-term and ar­du­ous task,” Xi urged con­tin­u­al ef­forts and promised to take part in world biose­cu­ri­ty gov­er­nance.

EQRx to col­lab­o­rate with new­ly-pub­lic Ab­sci on pro­tein-based ther­a­peu­tics

EQRx an­nounced a dis­cov­ery col­lab­o­ra­tion with Ab­sci to­day to ad­vance pro­tein-based ther­a­peu­tics.

Ab­sci went pub­lic just a few months ago in Ju­ly, where it raised $200 mil­lion at $16 a share.

The two com­pa­nies will col­lab­o­rate to joint­ly en­gi­neer and de­vel­op sev­er­al clin­i­cal can­di­dates across mul­ti­ple ther­a­peu­tic ar­eas, in­clud­ing on­col­o­gy and im­munol­o­gy. Ab­sci has the op­tion to make ad­di­tion­al in­vest­ments at dif­fer­ent stages of de­vel­op­ment in ex­change for an in­creased share of prod­uct sales.

“We are ex­cit­ed to work with Ab­sci to­wards our goal of pro­vid­ing in­no­v­a­tive, cost-ef­fec­tive treat­ment op­tions for pa­tients,” said EQRx chief of Rx cre­ation Car­los Gar­cia-Echev­er­ria in a state­ment.

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.

So — that pig-to-hu­man trans­plant; Po­ten­tial di­a­betes cure reach­es pa­tient; Ac­cused MIT sci­en­tist lash­es back; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

We’re incredibly excited to welcome Beth Bulik, seasoned pharma marketing reporter, to the team. You can find much of her work in our new Marketing channel — and in her weekly newsletter, Endpoints PharmaRx, which will launch in early November. Add it to your subscriptions here.

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

No, sci­en­tists are not any clos­er to pig-to-hu­man trans­plants than they were last week

Steve Holtzman was awoken by a 1 a.m. call from a doctor at Duke University asking if he could put some pigs on a plane and fly them from Ohio to North Carolina that day. A motorcyclist had gotten into a horrific crash, the doctor explained. He believed the pigs’ livers, sutured onto the patient’s skin like an external filter, might be able to tide the young man over until a donor liver became available.

UP­DAT­ED: Agenus calls out FDA for play­ing fa­vorites with Mer­ck, pulls cer­vi­cal can­cer BLA at agen­cy's re­quest

While criticizing the FDA for what may be some favoritism towards Merck, Agenus on Friday officially pulled its accelerated BLA for its anti-PD-1 inhibitor balstilimab as a potential second-line treatment for cervical cancer because of the recent full approval for Merck’s Keytruda in the same indication.

The company said the BLA, which was due for an FDA decision by Dec. 16, was withdrawn “when the window for accelerated approval of balstilimab closed,” thanks to the conversion of Keytruda’s accelerated approval to a full approval four months prior to its PDUFA date.

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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 are 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 are 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.

No­vo CEO Lars Fruer­gaard Jør­gensen on R&D risk, the deal strat­e­gy and tar­gets for gen­der di­ver­si­ty

 

I kicked off our European R&D summit last week with a conversation involving Novo Nordisk CEO Lars Fruergaard Jørgensen. Novo is aiming to launch a new era of obesity management with a new approval for semaglutide. And Jørgensen had a lot to say about what comes next in R&D, how they manage risk and gender diversity targets at the trendsetting European pharma giant.

John Carroll: I’m here with Lars Jørgensen, the CEO of Novo Nordisk. Lars, it’s been a really interesting year so far with Novo Nordisk, right? You’ve projected a new era of growing sales. You’ve been able to expand on the GLP-1 franchise that was already well established in diabetes now going into obesity. And I think a tremendous number of people are really interested in how that’s working out. You have forecast a growing amount of sales. We don’t know specifically how that might play out. I know a lot of the analysts have different ideas, how those numbers might play out, but that we are in fact embarking on a new era for Novo Nordisk in terms of what the company’s capable of doing and what it’s able to do and what it wants to do. And I wanted to start off by asking you about obesity in particular. Semaglutide has been approved in the United States for obesity. It’s an area of R&D that’s been very troubled for decades. There have been weight loss drugs that have come along. They’ve attracted a lot of attention, but they haven’t actually ever gained traction in the market. My first question is what’s different this time about obesity? What is different about this drug and why do you expect it to work now whereas previous drugs haven’t?

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Marty Duvall, Oncopeptides CEO

On­copep­tides stock craters as it pulls can­cer drug Pepax­to from the mar­ket

Shares of Oncopeptides crashed more than 70% in early Friday trading after the company said it’s pulling its multiple myeloma drug Pepaxto (melphalan flufenamide) from the US market after failing a confirmatory trial. The move will force the company to close its US and EU business units and enact significant layoffs.

The FDA had scheduled an adcomm meeting next Thursday to discuss Pepaxto, which first won accelerated approval in February and costs about $19,000 per course of treatment. The committee was to weigh in on whether the confirmatory trial demonstrated a worse overall survival in the treatment arm compared to the control arm.

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Pfiz­er pitch­es its Covid-19 vac­cine for younger chil­dren ahead of ad­comm next week

Pfizer will present its case to the FDA’s vaccine adcomm next week, seeking authorization for a lower-dose version of its Covid-19 vaccine for kids ages 5 through 12, which the Biden administration said will likely begin rolling out early next month.

Two primary doses of the 10 µg vaccine (the dose for those ages 12 and up is 30 μg) given 3 weeks apart in this group of children “have shown a favorable safety and tolerability profile, robust immune responses against all variants of concern including Delta, and vaccine efficacy of 90.7% against laboratory-confirmed symptomatic COVID-19,” the company said in briefing documents ahead of next Tuesday’s meeting of the FDA’s Vaccines and Related Biological Products Advisory Committee.