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.

Op­ti­miz­ing Cell and Gene Ther­a­py De­vel­op­ment and Pro­duc­tion: How Tech­nol­o­gy Providers Like Corn­ing Life Sci­ences are Spurring In­no­va­tion

Remarkable advances in cell and gene therapy over the last decade offer unprecedented therapeutic promise and bring new hope for many patients facing diseases once thought incurable. However, for cell and gene therapies to reach their full potential, researchers, manufacturers, life science companies, and academics will need to work together to solve the significant challenges facing the industry.

David Baker working with a student on their protein design (Jason Mast)

Sci­en­tists are fi­nal­ly learn­ing how to de­sign pro­teins from scratch. Drug de­vel­op­ment may nev­er be the same

SEATTLE — It’s a cloudy Thursday afternoon in mid-July and David Baker is reclining into the futon in his corner office at the University of Washington, arms splayed out like a daytime talk show host as he coaches another one of his postdocs through the slings and arrows of scientific celebrity.

“Be jealous of your time,” he says, before plotting ways of sneaking her out of Zooms. “It’s this horrible cost to science that you’re tied up in some stupid meeting.”

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Pre­sent­ing a live End­points News event: Man­ag­ing a biotech in tur­bu­lent times

Biotech is one of the smartest, best educated industries on the planet. PhDs abound. We’ve had a long enough track record to see a new generation of savvy, experienced execs coming together to run startups.

And in these times, they are being tested as never before.

Biotech is going through quite a rough patch right now. For 2 years, practically anyone with a decent resume and some half-baked ideas on biotech could start a company and get it funded. The pandemic made it easy in many ways to pull off an IPO, with traditional road shows shut down in exchange for a series of quick Zoom meetings. Generalist investors flocked as the numbers raised soared into the stratosphere.

Patty Murray, D-WA (Graeme Sloan/Sipa USA)(Sipa via AP Images)

Sen­ate user fee reau­tho­riza­tion bill omits ac­cel­er­at­ed ap­proval re­forms, shows wide gaps with House ver­sion

The Senate health committee on Tuesday released its first version of the bill to reauthorize all the different FDA user fees. But unlike the House version, there are only a few controversial items in the Senate’s version, which does not address either accelerated approval reforms or clinical trial diversity (as the House did).

While it’s still relatively early in the process of finalizing this legislation (the ultimate statutory deadline is the end of September), the House and Senate, at least initially, appear to be starting off in different corners on what should be included.

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Warren Buffett, Berkshire Hathaway CEO

Berk­shire Hath­away pulls out of Ab­b­Vie, Bris­tol My­ers Squibb in­vest­ments

It looks like Warren Buffett is sticking to ice cream and railroads for the moment.

The billionaire CEO of Berkshire Hathaway backed out of major two holdings in the pharma industry, Forexlive first reported, including a $410 million investment in AbbVie and a $324.4 million stake in Bristol Myers Squibb.

The move comes after Berkshire abandoned its Teva shares just last quarter, Bloomberg reported.

Long-ex­pect­ed UK lay­offs im­mi­nent for No­var­tis fol­low­ing sale

Nearly a year ago, more than 200 workers at Novartis’ Grimsby, UK, facility were able to hang on to their jobs after the pharma closed a Switzerland site as a part of its workforce restructuring plan. Now, it looks like those employees’ time is up, as the site has been sold, Grimsby Telegraph reported today.

The manufacturing site has been sold to Humber Industrials, a subsidiary of International Process Plants. None of the current staff members will be working with the new owners, however.

FDA lob­bies Con­gress over rare dis­ease court rul­ing with wide im­pli­ca­tions

Usually reserved for making decisions on drug applications or enforcing what Congress stipulates, the FDA is now dipping its toe into the wild world of congressional politics as it attempts to fix a major court decision that could have a chilling effect on rare disease R&D.

The case in question from last October saw a US appeals court overturn a prior FDA court win, saying that the agency never should’ve approved a rare disease drug because a previously approved but more expensive drug with the same active ingredient has orphan drug exclusivity barring such an approval.

Peter Marks (Greg Nash/Pool via AP)

Even FDA's Pe­ter Marks is wor­ried about the com­mer­cial vi­a­bil­i­ty of gene and cell ther­a­pies

When bluebird bio’s gene therapy to treat beta thalassemia won European approval in 2019, the nearly $2 million per patient price tag for the potential cure seemed like a surmountable hurdle.

Fast forward two years later, and bluebird has withdrawn Zynteglo, the beta thal drug, along with the rest of its gene therapy portfolio from Europe, which the company said is generally unwilling to pay a fair price for the treatment.

Pri­cy in­halers re­main ex­pen­sive due to de­vice tweaks that keep com­peti­tors at bay, re­searchers find

New research published in Health Affairs today highlights the way in which the FDA’s inhaler regulations have rewarded incremental adjustments to older products, thereby enabling companies to skirt around cheaper competition.

A DC appeals court clerk and researchers from Harvard and the University of Calgary dug through all the patents and regulatory exclusivities granted to inhalers approved by the FDA between 1986 and 2020, finding that of the 62 inhalers approved, 53 (or 85%) were brand-name products, with a median of 16 years of protection from generic competition.