Re­gen­eron and Sanofi land the big one, win­ning FDA ap­proval for their megablock­buster hope­ful Dupix­ent

George Yan­copou­los, Pres­i­dent and CSO, Re­gen­eron

The FDA has ap­proved the new eczema drug dupilum­ab from Re­gen­eron and Sanofi, of­fi­cial­ly launch­ing a new drug that is like­ly on course to cre­at­ing a megablock­buster fran­chise for these two part­ners. Wide­ly tapped as the biggest pro­gram in the in­dus­try’s pipeline this year, dupilum­ab now will be brand­ed as Dupix­ent. And the mar­ket here could be worth up­wards of $5 bil­lion a year.

“Peo­ple with mod­er­ate-to se­vere atopic der­mati­tis cope with in­tense, some­times un­bear­able symp­toms that can im­pact them for most of their lives,” said Julie Block, the CEO of the Na­tion­al Eczema As­so­ci­a­tion. “To date, there have been few op­tions avail­able to treat peo­ple with mod­er­ate-to-se­vere atopic der­mati­tis who have un­con­trolled dis­ease. That’s why to­day’s ap­proval of Dupix­ent is so im­por­tant for our com­mu­ni­ty. Now we have a treat­ment that is ex­pect­ed to help ad­dress pa­tients suf­fer­ing from this dev­as­tat­ing dis­ease.”

The drug will be priced at $37,000 a year, a shade high­er than many an­a­lysts had ex­pect­ed, but not sig­nif­i­cant­ly out of line.

“We have set a fair and re­spon­si­ble price for Dupix­ent,” says a Sanofi spokesper­son. “We are en­cour­aged by the on­go­ing con­ver­sa­tions we’ve had with health plans and phar­ma­cy ben­e­fit man­agers about cov­er­age for Dupix­ent for pa­tients, and we have a shared com­mit­ment with them to en­sure pa­tients who should re­ceive it can re­ceive it.”

Ge­of­frey Porges, Leerink

Some top an­a­lysts wouldn’t dis­agree with that re­mark. Leerink’s Ge­of­frey Porges not­ed that the dis­count price should fall right around $30,000, which should be ac­cept­able to pay­ers. He not­ed:

While pay­ers may still be­lieve Dupix­ent is an ex­pen­sive ther­a­py, the drug is cost-ef­fec­tive for the in­di­cat­ed pa­tients, ac­cord­ing to the in­flu­en­tial ICER re­port. In­deed, in to­day’s press Ex­press Scripts’ (ES­RX, MP) CMO Steven Miller com­ment­ed that the man­u­fac­tur­ers had set a “re­spon­si­ble price,” and pay­ers ap­pear to be tak­ing cred­it for in­flu­enc­ing the pric­ing de­ci­sion. Re­cent drug launch­es have been harmed by un­clear ef­fi­ca­cy, broad­ly-la­beled pa­tient pop­u­la­tions that do not match avail­able clin­i­cal tri­al da­ta, and high list prices. By con­trast, Dupix­ent’s well-de­fined la­belled in­di­ca­tion and clear out­comes ben­e­fits, as well as the com­plete lack of FDA-ap­proved med­i­cines for post-top­i­cals mod­er­ate-to-se­vere AD, should lim­it the im­pact of step-ed­its and pri­or au­tho­riza­tion re­quire­ments.

Baird’s Bri­an Sko­r­ney ran a physi­cian sur­vey and found a large num­bers of doc­tors are ea­ger to start pre­scrib­ing this drug.

Bri­an Sko­r­ney, Baird an­a­lyst

“Im­por­tant­ly, physi­cian sen­ti­ment points to a mar­ket for Dupix­ent of about 300,000-350,000 pa­tients, in line with man­age­ment’s ex­pec­ta­tions,” he not­ed Tues­day morn­ing. “We wouldn’t be sur­prised to see an ex­cep­tion­al­ly strong launch here, but we con­tin­ue to point out that con­sen­sus ex­pec­ta­tions are al­ready build­ing that in.

The two bio­phar­ma part­ners — with Re­gen­eron whip­ping up the IL-4/IL-13 an­ti­body and Sanofi weigh­ing in with its late-stage mus­cle — have divvied up the mar­ket 50/50. And they’ve al­ready fired off a pre­emp­tive le­gal blast against a liti­gious Am­gen, their arch neme­sis which is sit­ting on IP for both tar­gets.

Am­gen has ag­gres­sive­ly pur­sued a law­suit against the Re­gen­eron/Sanofi’s team for their PC­SK9 drug Pralu­ent, which com­petes against Am­gen’s Repatha. At one point, Am­gen scored a stun­ning up­set, win­ning a court rul­ing in fa­vor of shelv­ing the ri­val drug, which has since been set aside.

In the part­ners’ suit against Am­gen, they say that they’ve learned that Am­gen has hired coun­sel to pur­sue a claim in­volv­ing patent ‘487, which in­volves IL-4 and IL-13 drugs. Am­gen tried, and failed, to de­vel­op their own IL4/IL-13 drug — AMG317 — and failed in Phase II. And Re­gen­eron and Sanofi are seek­ing a pre­emp­tive rul­ing against Am­gen, which loves to field its ag­gres­sive le­gal team when­ev­er it suits their strat­e­gy or the lawyers be­lieve there’s some rev­enue to grab off the ta­ble.

Re­gen­eron and Sanofi have much to pro­tect here, if the an­a­lysts are right. Pfiz­er scored an ap­proval for its atopic der­mati­tis drug Eu­crisa (crisabo­role), a PDE-4 in­hibitor, which has some block­buster am­bi­tions of its own. But Dupix­ent is ex­pect­ed to be game chang­er for pa­tients.

Just a few weeks ago in­ves­ti­ga­tors spelled out a new batch of stel­lar da­ta, com­plet­ing a ma­jor Phase III ef­fort that care­ful­ly re­viewed safe­ty and ef­fi­ca­cy da­ta for a wide range of pa­tients.

In the new CHRONOS da­ta re­viewed at the an­nu­al meet­ing of the Amer­i­can Acad­e­my of Der­ma­tol­ogy in Or­lan­do ear­li­er this month, in­ves­ti­ga­tors not­ed that the se­vere itch­ing pa­tients are af­flict­ed by was re­duced by 55% and 58% in the two drug arms af­ter 16 weeks, com­pared to 29% of pa­tients on TCS alone. And the dis­ease score rat­ing for pa­tients dropped by 4 or more points among 77% of the pa­tients in the drug arms com­pared to 37% of the place­bo/top­i­cal cor­ti­cos­teroid group.

Re­gen­eron and Sanofi need to make a quick suc­cess of this drug, and they’ll like­ly roll out an ag­gres­sive mar­ket­ing ef­fort now that they have re­ceived a long-await­ed green light from the FDA. They still be­lieve that they can make a big suc­cess of Repatha, but pay­ers have kept it care­ful­ly bot­tled up, wait­ing for car­dio out­comes. Rev­enue is mar­gin­al.

Then there’s sar­ilum­ab, held up by the FDA months ago so that Sanofi could clear up some em­bar­rass­ing man­u­fac­tur­ing is­sues.

Dupix­ent is a go. And the Re­gen­eron/Sanofi team is now tak­ing the mar­ket­ing field with a care­ful­ly planned blitz. They can’t af­ford an­oth­er set­back.

Sanofi in par­tic­u­lar, which has been sad­dled with a large­ly in­ef­fec­tive French R&D group, needs to show it can de­vel­op and field a ma­jor new drug. Now they have an­oth­er shot at glo­ry.

“Dupix­ent is the re­sult of years of tire­less re­search by our sci­en­tists in­to the un­der­ly­ing caus­es of al­ler­gic and atopic dis­eases. In atopic der­mati­tis, Dupix­ent was shown to help clear the skin and man­age the in­tense itch caused by the dis­ease,” said George D. Yan­copou­los, the found­ing sci­en­tist and CSO at Re­gen­eron. “To­day’s ap­proval would not be pos­si­ble with­out the ded­i­ca­tion of the clin­i­cal in­ves­ti­ga­tors and the par­tic­i­pa­tion of the pa­tients who took part in the glob­al LIB­ER­TY AD clin­i­cal pro­gram.”

5AM Ven­tures: Fu­el­ing the Next Gen­er­a­tion of In­no­va­tors

By RBC Capital Markets
With Andy Schwab, Co-Founder and Managing Partner at 5AM Ventures

Key Points

Prescription Digital Therapeutics, cell therapy technologies, and in silico medicines will be a vital part of future treatment modalities.
Unlocking the potential of the microbiome could be the missing link to better disease diagnosis.
Growing links between academia, industry, and venture capital are spinning out more innovative biotech companies.
Biotech is now seen by investors as a growth space as well as a safe haven, fuelling the recent IPO boom.

Biohaven CEO Vlad Coric (Photo Credit: Andrew Venditti)

Pssst: That big Bio­haven Alzheimer's study? It was a bust. Even the sub­group analy­sis ex­ecs tout­ed was a flop

You know it’s bad when a biopharma player plucks out a subgroup analysis for a positive take — even though it was way off the statistical mark for success, like everything else.

So it was for Biohaven $BHVN on MLK Monday, as the biotech reported on the holiday that their Phase II/III Alzheimer’s study for troriluzole flunked both co-primary endpoints as well as a key biomarker analysis.

The drug — a revised version of the ALS drug riluzole designed to regulate glutamate — did not “statistically differentiate” from placebo on the Alzheimer’s Disease Assessment Scale-Cognitive Subscale 11 (ADAS-cog) and the Clinical Dementia Rating Scale Sum of Boxes (CDR-SB).  The “hippocampal volume” assessment by MRI also failed to distinguish itself from placebo for all patients fitting the mild-to-moderate disease profile they had established for the study.

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CEO Stephen Yoder (Pieris)

Pieris fi­nal­ly vaults FDA hold on next-gen sol­id tu­mor hunter, clear­ing the path for mid-stage tri­al

Finally freed from the restraints of a partial FDA clinical hold on its lead HER2-positive solid tumor candidate, Pieris Pharmaceuticals is now racing toward Phase II.

The FDA slapped a partial hold on Pieris’ PRS-343 back in July, restricting the biotech from enrolling new patients in a Phase I trial. While Pieris was allowed to continue dosing patients who were already enrolled, the agency requested they conduct an additional “in-use and compatibility study” before recruiting any more.

News brief­ing: Ve­rastem CMO ex­its two weeks af­ter join­ing com­pa­ny; Ther­mo Fish­er inks $550M M&A deal

Two weeks after joining Verastem Oncology as chief medical officer, Frank Neumann is leaving the company for another job.

Neumann had joined Verastem after leaving bluebird bio, which surprisingly split into two companies last week, one in oncology and one in rare diseases. It’s not yet clear to where Neumann is headed next, but he noted in a statement that Verastem’s data and strategy were “truly exciting.”

FDA hits the brakes on His­to­gen's knee car­ti­lage ther­a­py, ask­ing for more in­fo on man­u­fac­tur­ing process

A month after filing the IND application for its human extracellular matrix designed to regenerate knee cartilage, Histogen has hit a roadblock.

The FDA on Tuesday verbally notified the San Diego-based biotech that it was placing a clinical hold on the planned Phase I/II clinical trial of HST-003 due to pending CMC information and additional questions needed to complete their review.

Histogen had planned to test the safety and efficacy of implanting hECM within microfracture interstices and related cartilage defects to regenerate that cartilage in conjunction with a microfracture procedure. The company said in a press release that it expects to receive written notice of the clinical hold from the FDA by Feb 12.

Andrew Allen, Gritstone CEO (Gritstone via website)

Grit­stone con­tin­ues Covid-19 push with deal to de­vel­op 'self-am­pli­fy­ing RNA' vac­cines, as shares con­tin­ue bal­loon­ing

Gritstone Oncology has had a big week, and it’s only Wednesday.

On Tuesday, the biotech revealed plans to start clinical testing of an experimental Covid-19 vaccine — in tandem with NIAID — that can also target other coronaviruses, with the goal of preventing future pandemics should SARS-CoV-2 prove difficult to cure with current vaccines. Then, on Wednesday morning, Gritstone licensed lipid nanoparticle technology from Genevant Sciences to develop what it’s calling “self-amplifying RNA vaccines” against Covid-19.

Artist rendering of the Assembly Square site in Somerville, MA (BioMed Realty)

Bio­Med Re­al­ty snaps up in­no­va­tion cam­pus site with­in earshot of pricey and bustling Boston biotech hub

On the short list of the premier biotech hubs in the world, the Boston area has transformed into a home for innovation — and ridiculously high rent. Now, a real estate firm is seeking tenants for a major site in neighboring Somerville with more than enough elbow room.

Snapped up by BioMed Realty, the land — which consists of an existing 162,000 square-foot office building and a 7.5 acre site — will serve as an “innovation space” for a variety of research, technology and life science tenants, the real estate company said in a press release. Financial terms weren’t disclosed.

Hal Barron, GSK R&D chief (GSK via YouTube)

Glax­o­SmithK­line's $4B bis­pe­cif­ic can­cer drug al­liance with Mer­ck KGaA hit by big set­back with a PhI­II fail­ure on NSCLC

Close to 2 years ago, GSK’s R&D team eagerly agreed to pay up to $4 billion-plus to ally itself with Merck KGaA on a mid-stage bispecific called bintrafusp alfa, which intrigued them with the combination of a TGF-β trap with the anti-PD-L1 mechanism in one fusion protein.

But today the German pharma company says that their lead study on lung cancer was a bust, as independent monitors said there was no reason to believe that the experimental drug — targeting PD-L1/TGF-Beta — could beat Keytruda.

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Union members strike in France, AP Images

Paul Hud­son faces down French unions in fight to re­struc­ture Sanofi

Sanofi CEO Paul Hudson is facing a familiar adversary in his efforts to cut up to 1,680 jobs from the French pharma giant: French unions.

Around 200 union members staged a one-day strike Tuesday at Sanofi’s main Covid-19 vaccine plant in Marcy-l’Étoile to protest the cuts, The Associated Press reported, with other members joining at other facilities across the country.

France’s finance minister Bruno Le Maire, meanwhile, went on French radio twice this week to talk about the company. On Monday, per Reuters, he told RTL that Sanofi would not close any plants or lay off any employees in the restructuring. But on Wednesday morning, he re-emerged on BFM and said he would like three things from the drugmaker, including confirmation that there will be no site closures and layoffs.