No­var­tis sets the tone for the ra­dio­phar­ma push with a prized ac­qui­si­tion win­ning break­through ther­a­py des­ig­na­tion

The crown jew­el of a multi­bil­lion-dol­lar No­var­tis ac­qui­si­tion has won a new en­dorse­ment from the FDA, and it’s a pro­gram at the fore­front of the in­dus­try’s ra­dio­phar­ma push.

Reg­u­la­tors grant­ed Lu-PS­MA-617 break­through ther­a­py des­ig­na­tion Wednes­day, No­var­tis an­nounced, rough­ly three months af­ter the can­di­date read out pos­i­tive Phase III re­sults in metasta­t­ic cas­tra­tion-re­sis­tant prostate can­cer. The move marks not on­ly a win for No­var­tis but for the up-and-com­ing ra­dio­phar­ma­ceu­ti­cals field, which has at­tract­ed heavy in­ter­est in the last cou­ple years.

Sim­i­lar to an­ti­body-drug con­ju­gates, ra­dio­phar­ma pro­grams de­liv­er pay­loads to spe­cif­ic tar­gets. Where­as AD­Cs drop off drug pay­loads at their in­tend­ed sites, ra­di­oli­gands di­rect ra­dioac­tive mol­e­cules or iso­topes to bind to tu­mor sur­faces.

For the No­var­tis pro­gram, the ra­dioiso­tope tar­gets the prostate-spe­cif­ic mem­brane anti­gen, or PS­MA, which is com­mon­ly found in metasta­t­ic prostate can­cer. The Swiss bio­phar­ma got their hands on the can­di­date with a $2.1 bil­lion buy­out of En­do­cyte af­ter the biotech hus­tled it in­to a Phase III study.

It’s now that tri­al that’s proved the im­pe­tus for No­var­tis’ newest BTD. Back in March, No­var­tis an­nounced Lu-PS­MA-617 met both pri­ma­ry end­points in over­all sur­vival and ra­di­ograph­ic pro­gres­sion-free sur­vival.

Then, ear­li­er this month, No­var­tis fol­lowed up with more de­tailed da­ta at AS­CO. Re­searchers showed Lu-PS­MA-617 on top of stan­dard of care re­duced death by 38% and post­ed a 60% re­duc­tion in ra­di­ograph­ic PFS or death com­pared to the stan­dard on its own. Both end­points notched one-sided p-val­ues of p<0.001.

Though there was a much high­er rate of side ef­fects re­lat­ed to the ther­a­py — 85.3% in the drug arm against 28.8% in the con­trol — No­var­tis said the safe­ty was con­sis­tent with pre­vi­ous clin­i­cal tri­als.

And the high rate of ad­verse events did not re­sult in a large nu­mer­i­cal dif­fer­ence in dis­con­tin­u­a­tions. In the drug arm, 11.9% of pa­tients stopped tak­ing Lu-PS­MA-617 and 8.5% halt­ed stan­dard of care treat­ment. Among those tak­ing on­ly stan­dard of care, 7.8% dis­con­tin­ued treat­ment.

No­var­tis hasn’t said when it plans to sub­mit its fi­nal pitch to the FDA, on­ly that they were tak­ing these da­ta to reg­u­la­to­ry agen­cies “as soon as pos­si­ble.” The com­pa­ny re­cent­ly launched two oth­er Phase III stud­ies for Lu-PS­MA-617 in ear­li­er lines of treat­ment, in­clud­ing in the mCR­PC pre-tax­ane set­ting and in the metasta­t­ic hor­mone-sen­si­tive set­ting.

With the BTD now in hand, No­var­tis is stak­ing it­self as one of the lead­ers in the in­dus­try’s ra­dio­phar­ma push. They’ve made oth­er in­vest­ments in the field, in­clud­ing an ear­li­er ac­qui­si­tion of Ad­vanced Ac­cel­er­a­tor Ap­pli­ca­tions from France back in 2017. That deal gave the phar­ma gi­ant a ra­dio­phar­ma plat­form and the drug Lu­tathera for $3.9 bil­lion.

But oth­er play­ers are mak­ing in­roads in ra­dio­phar­ma, with Ger­many’s ITM rais­ing $109 mil­lion in loan fi­nanc­ing back in April and the biotech Ak­tis se­cur­ing a $72 mil­lion round. Rayze­Bio has been the biggest re­cent win­ner, how­ev­er, land­ing a $258 mil­lion Se­ries C just this week.

Oth­er play­ers like Fu­sion Phar­ma­ceu­ti­cals and Clo­vis are al­so an­gling for mar­ket en­try, with the for­mer so­lid­i­fy­ing an IPO last June for $212.5 mil­lion.

At the In­flec­tion Point for the Next Gen­er­a­tion of Can­cer Im­munother­a­py

While oncology researchers have long pursued the potential of cellular immunotherapies for the treatment of cancer, it was unclear whether these therapies would ever reach patients due to the complexity of manufacturing and costs of development. Fortunately, the recent successful development and regulatory approval of chimeric antigen receptor-engineered T (CAR-T) cells have demonstrated the significant benefit of these therapies to patients.

Stéphane Bancel, Moderna CEO

'This is not go­ing to be good': Mod­er­na CEO Ban­cel warns of a 'ma­te­r­i­al drop' in vac­cine ef­fi­ca­cy as Omi­cron spreads

Even as public health officials remain guarded about their comments on the likelihood Omicron will escape the reach of the currently approved Covid-19 vaccines, there’s growing scientific consensus that we’re facing a variant that threatens to overwhelm the vaccine barricades that have been erected.

Stéphane Bancel, the CEO of Moderna, one of the leading mRNA players whose quick vault into the markets with a highly effective vaccine created an instant multibillion-dollar market, added his voice to the rising chorus early Tuesday. According to Bancel, there will be a significant drop in efficacy when the average immune system is confronted by Omicron. The only question now is: How much?

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 124,400+ biopharma pros reading Endpoints daily — and it's free.

Philip Dormitzer, new GSK global head of vaccines R&D

Glax­o­SmithK­line poach­es Pfiz­er's vi­ral vac­cines lead in rush to cap­i­tal­ize on fu­ture of mR­NA

GlaxoSmithKline has appointed Philip Dormitzer, formerly chief scientific officer of Pfizer’s viral vaccines unit, as its newest global head of vaccines R&D, looking to leverage one of the leading minds behind Pfizer and BioNTech’s RNA collaboration that led to Covid-19 jab Comirnaty, the British drug giant said Tuesday.

Dormitzer had been with Pfizer for a little more than six years, joining up after a seven-year stint with Novartis, where he reached the role of US head of research and head of global virology for the company’s vaccines and diagnostics unit.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 124,400+ biopharma pros reading Endpoints daily — and it's free.

In­tro­duc­ing End­points Stu­dio, a new way to ad­ver­tise with End­points-craft­ed brand­ing cam­paigns

Since our start in 2016, Endpoints has grown fast while executing our mission to cover biopharma’s most critical developments for industry pros worldwide. As readership has grown, our advertising business has too. Endpoints advertising partners support the mission and engage their desired audiences through announcements on our email and web platforms, brand recognition in our event coverage and sponsorships of Endpoints daily and weekly reports.

Lan Huang, BeyondSpring CEO

Months af­ter shock­ing in­vestors with lung can­cer win, Be­yond­Spring's lead drug hits road­block at the FDA

BeyondSpring shocked investors in early August after its once-marginal lead drug suddenly showed a lot of promise in a common form of lung cancer. With hopes high, the FDA has now slammed the door on that drug in another indication — does that spell bad news for BeyondSpring’s Cinderella story?

The FDA issued BeyondSpring a complete response letter for its plinabulin in combination with granulocyte colony-stimulating factor (G-CSF) for the prevention of chemotherapy-induced neutropenia, effectively shutting down the drug’s immediate chances at a marketing approval, the biotech said Wednesday.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 124,400+ biopharma pros reading Endpoints daily — and it's free.

With on­ly burns to show in gene ther­a­py, Astel­las inks deal with AAV spe­cial­ist Dyno in push for a bet­ter cap­sid

On the hunt for a better AAV capsid for gene therapy, Eric Kelsic’s Dyno Therapeutics has set itself apart with its focus on machine learning to help speed discovery. Now, Japanese drugmaker Astellas — fresh off a slate of gene therapy burns — is taking a bet on Dyno as it looks to the future.

Astellas and Dyno will work together as part of an R&D pact to develop next-gen AAV vectors for gene therapy using Dyno’s CapsidMap platform directed at skeletal and cardiac muscle, the companies said Wednesday. Under the terms of the deal, Dyno will design AAV capsids for gene therapy, while Astellas will be responsible for conducting preclinical, clinical and commercialization activities for gene therapy product candidates using the capsids.

J&J and Sanofi's mul­ti­ple myelo­ma tit-for-tat con­tin­ues, as sub­cu­ta­neous Darza­lex wins com­bo ap­proval

J&J and Sanofi have gone back and forth in their multiple myeloma tug-of-war. Earlier this year, Sanofi notched an approval of Sarclisa in combination with Amgen’s Kyprolis to try to outflank the big conglomerate, but J&J is clapping back.

Wednesday afternoon, Amgen announced that the subcutaneous version of J&J’s blockbuster Darzalex is also now approved as a combo with Kyprolis and dexamethasone. The green light came through for adults with relapsed or refractory multiple myeloma who had progressed on one to three earlier lines of therapy.

As first Omi­cron case in US crops up, re­searchers won­der: which an­ti­bod­ies, vac­cines will hold up?

As Covid-19 drug and vaccine developers race to figure out which of their products might be hampered by the new variant, the CDC on Wednesday afternoon announced the first confirmed case of the Omicron variant (B.1.1.529) in the US, found in San Francisco.

The unidentified individual was a traveler who returned from South Africa on Nov. 22, 2021, was fully vaccinated, and had mild symptoms that the CDC described as improving. All close contacts have been contacted and have tested negative, the centers said.

FDA ad­comm nar­row­ly votes in fa­vor of Mer­ck­'s an­tivi­ral for out­pa­tient Covid-19

With little explanation for why Merck’s potential Covid-19 antiviral was less effective in reducing Covid hospitalizations and deaths in a full analysis of a Phase III trial versus an interim look, the FDA’s antimicrobial drugs advisory committee on Tuesday voted 13-10 in favor of the pill’s benefits outweighing the risks for adults within 5 days of developing Covid symptoms.

Molnupiravir will likely be authorized by FDA in the coming days for adults with mild or moderate Covid-19. While Pfizer’s antiviral may prove to be more effective, Merck’s pill will be another weapon in the armamentarium of Covid-19 treatments for countries around the world, adding to the mAb treatments already in use in the outpatient space from Regeneron, Eli Lilly and Vir/GlaxoSmithKline.