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 and Big Phar­ma: A blue­print for a suc­cess­ful part­ner­ship

Strategic partnerships have long been an important contributor to how drugs are discovered and developed. For decades, big pharma companies have been forming alliances with biotech innovators to increase R&D productivity, expand geographical reach and better manage late-stage commercialization costs.

Noël Brown, Managing Director and Head of Biotechnology Investment Banking, and Greg Wiederrecht, Ph.D., Managing Director in the Global Healthcare Investment Banking Group at RBC Capital Markets, are no strangers to the importance of these tie-ups. Noël has over 20 years of investment banking experience in the industry. Before moving to the banking world in 2015, Greg was the Vice President and Head of External Scientific Affairs (ESA) at Merck, where he was responsible for the scientific assessment of strategic partnership opportunities worldwide.

No­var­tis' sec­ond at­tempt to repli­cate a stun­ning can­cer re­sult falls flat

Novartis’ hopes of turning one of the most surprising trial data points of the last decade into a lung cancer drug has taken another setback.

The Swiss pharma announced Monday that its IL-1 inhibitor canakinumab did not significantly extend the lives or slow the disease progression of patients with previously untreated locally advanced or metastatic non-small cell lung cancer when compared to standard of-care alone.

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How Chi­na turned the ta­bles on bio­phar­ma's glob­al deal­mak­ing

Fenlai Tan still gets chills thinking about the darkest day of his life.

Three out of eight lung cancer patients who received a tyrosine kinase inhibitor developed by his company, Betta Pharma, died in the span of a month. Tan, the chief medical officer, was summoned to Peking Union Medical College Hospital, where the head of the clinical trial department told him that the trial investigators would be conducting an autopsy to see if the patients had died of the disease — they were all very sick by the time they enrolled — or of interstitial lung disease, a deadly side effect tied to the TKI class that’s been reported in Japan.

ARCH-backed SciNeu­ro kicks off search for CNS au­toan­ti­bod­ies with new deal; Mer­ck + Gilead an­nounce PhII tri­al for HIV com­bo

From the very beginning at SciNeuro, CEO Min Li has envisioned a mix of licensing deals and scientific efforts to replicate the breakout success of China’s oncology companies in neuroscience.

The GlaxoSmithKline vet has now inked a deal that somewhat straddles the line between the two strategies.

Teaming up with Mabylon out of Zurich, SciNeuro is now looking to test the hypothesis that the human immune system can play a role in fighting neurodegenerative diseases by discovering and developing human autoantibodies against neurological “targets of mutual interests.” The new partners offered TAR DNA binding protein-43 (TDP-43) and apolipoprotein E (APOE), which are linked to ALS and Alzheimer’s, as examples.

Ugur Sahin, AP Images

As pres­sure to share tech­nol­o­gy mounts, BioN­Tech se­lects Rwan­da for lat­est vac­cine site

BioNTech’s first mRNA-based vaccine site in Africa will call Rwanda home, and construction is set to start in mid-2022, the company announced Tuesday at a public health forum.

The German company signed a memorandum of understanding, after a meeting between Rwanda’s Minister of Health, Daniel Ngamije, Senegal’s Minister of Foreign Affairs Aïssata Tall Sall, and senior BioNTech officials. Construction plans have been finalized, and assets have been ordered. The agreement will help bring end-to-end manufacturing to Africa, and as many as several hundred million doses of vaccines per year, though initial production will be more modest.

No­var­tis dumps AveX­is pro­gram for Rett syn­drome af­ter fail­ing re­peat round of pre­clin­i­cal test­ing

Say goodbye to AVXS-201.

The Rett syndrome gene therapy drug made by AveXis — the biotech that was bought, kept separate, then renamed and finally absorbed by Novartis into its R&D division — has been dropped by the biopharma.

In Novartis’ third quarter financial report, the pharma had found that preclinical data did not support development of the gene therapy into IND-enabling trials and beyond. The announcement comes a year after Novartis told the Rett Society how excited it was by the drug — and its potential benefits and uses.

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Vas Narasimhan, Novartis CEO (Simon Dawson/Bloomberg via Getty Images)

With San­doz con­tin­u­ing to drag on No­var­tis, Vas Narasimhan says he may fi­nal­ly be ready for a sale or spin­off

After years of rehab work aimed at getting Sandoz in fighting trim to compete in a market overshadowed by declining prices, CEO Vas Narasimhan took a big step toward possibly selling or spinning off the giant generic drug player.

The pharma giant flagged plans to launch a strategic review of the business in its Q3 update, noting that “options range from retaining the business to separation.”

Analysts have been poking and prodding Novartis execs for years now as Narasimhan attempted to remodel a business that has been a drag on its performance during most of his reign in the CEO suite. The former R&D chief has made it well known that he’s devoted to the innovative meds side of the business, where they see the greatest potential for growth.

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FDA is much worse than its reg­u­la­to­ry peers at proac­tive­ly dis­clos­ing da­ta, re­searchers find

The European Medicines Agency and Health Canada continue to outpace the FDA when it comes to proactively releasing data on drugs and biologics the agency has reviewed, leading to further questions of why the American agency can’t be more transparent.

In a study published recently in the Journal of Law, Medicine, & Ethics, Yale and other academic lawyers and researchers found that between 2016 and April 2021, the EMA proactively released data for 123 unique medical products, while Health Canada proactively released data for 73 unique medical products between 2019 and April 2021. What’s more, the EMA and Health Canada didn’t proactively release the same data on the same drugs. In stark contrast, the FDA in 2018 only proactively disclosed data supporting one drug that was approved that year.

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James Peyer, Cambrian CEO

Brent Saun­ders joins $100M Se­ries C for a com­pa­ny out to be the Bridge­Bio of ag­ing

About a year ago, James Peyer, a CEO and co-founder of the little known longevity biotech Cambrian Biopharma, was trying to find some R&D talent last year when he met with more than a bit of experience in that department: David Nicholson, the former R&D chief of the erstwhile pharma giant Allergan.

It turned out Nicholson already had an interest in Peyer’s field. In their Allergan days, he and COO Brent Saunders held weekly meetups where they tried to figure out how to take the company’s dominance in aesthetics — which, until recently, was often what people meant by anti-aging science — and expertise with more traditional drug development, and use it to make drugs that extend people’s lifespan.

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