Alex­ion has scored an ad­di­tion­al FDA OK for its key­stone rare ther­a­py Soliris, un­lock­ing what a key an­a­lyst calls a sig­nif­i­cant part of its C5 fran­chise and el­e­vat­ing that port­fo­lio fur­ther up the block­buster ranks.

The drug is now ap­proved to treat neu­romyelitis op­ti­ca spec­trum dis­or­der, a rare au­toim­mune con­di­tion char­ac­ter­ized by sud­den at­tacks on the cen­tral ner­vous sys­tem. The dis­ease of­ten strikes women in their 30s and 40s, and could be con­fused with mul­ti­ple scle­ro­sis. While off-la­bel Rit­ux­an helps most pa­tients — ap­prox­i­mate­ly 70% — keep the dis­ease at bay, re­laps­es still threat­en to do ir­re­versible harm and cause se­vere dis­abil­i­ty to those for whom the stan­dard of care doesn’t work.

Ge­of­frey Porges

In a Phase III test­ing the drug in pa­tients who had mul­ti­ple re­laps­es de­spite pri­or im­muno-sup­pres­sive ther­a­pies, Soliris re­duced the risk of NMOSD re­lapse by 94.2% com­pared to place­bo (p<0.0001) — a stel­lar re­sult. And in the lat­est da­ta cut at 144 weeks, 96% of the Soliris group didn’t ex­pe­ri­ence an at­tack, com­pared to 45% of pa­tients in the place­bo arm who re­mained re­lapse-free.

No­tably, reg­u­la­tors did not lim­it Soliris’ use to non-re­spon­ders, of­fer­ing a blan­ket la­bel that cov­ers any pa­tient with an­ti-aqua­por­in-4 an­ti­bod­ies — which is be­lieved to ac­ti­vate the com­ple­ment sys­tem that Soliris in­hibits.

That sur­prised SVB Leerink’s George Porges, a big cheer­leader of the drug, but he is care­ful to note that a broad­er la­bel does not guar­an­tee a broad­er pa­tient pop­u­la­tion.

De­spite the la­bel, we don’t think Soliris will cap­ture sig­nif­i­cant use in front line treat­ment giv­en Rit­ux­an’s fa­vor­able cost-ben­e­fit pro­file (~70% dis­ease con­trol un­der Rit­ux­an and ~$60K first year cost com­pared ~$700K first year cost for Soliris). We ex­pect most in­sur­ance pay­ers to re­quire ev­i­dence of Rit­ux­an fail­ure be­fore re­im­burs­ing for Soliris in this dis­ease, but do ex­pect rel­a­tive­ly open ac­cess to Soliris for re­lapsed pa­tients in late line treat­ment.

He notes that Alex­ion is soon start­ing a Phase III tri­al in the same in­di­ca­tion for Ul­tomiris, its fol­lowup to Soliris that’s eas­i­er to use. By his es­ti­mates, be­tween the two drugs the NMOSD sales alone will bring in around $1 bil­lion in 2028, rep­re­sent­ing one-fifth of the to­tal C5 fran­chise that year.

As far as com­pe­ti­tion goes, Roche and its Chugai sub­sidiary has some Phase III da­ta to show for its IL-6 in­hibitor satral­izum­ab. As­traZeneca spin­out Viela Bio is fast on its heels with the CD-19 in­hibitor inebi­lizum­ab. But with no di­rect ri­val in the C5 class, Porges sees Soliris cap­tur­ing the dom­i­nant share in re­lapse cas­es.

For Alex­ion’s part, R&D chief John Orloff is hap­py to high­light the fact that Soliris is the first drug ap­proved to re­duce the risk of NMOSD re­lapse. Re­views in Eu­rope and Japan are on­go­ing.

Biohaven is at risk of making a habit of disappointing its investors. 

Late Friday the biotech $BHVN reported that the FDA had rejected its application for riluzole, an old drug that they had made over into a sublingual formulation that dissolves under the tongue. According to Biohaven, the FDA had a problem with the active ingredient used in a bioequivalence study back in 2017, which they got from the Canadian drugmaker Apotex.

Let the long battle for biosimilars in the cancer space begin.

Amgen has launched its Avastin and Herceptin copycats — licensed from the predecessors of Allergan — almost two years after the FDA had stamped its approval on Mvasi (bevacizumab-awwb) and three months after the Kanjinti OK (trastuzumab-anns). While the biotech had been fielding biosimilars in Europe, this marks their first foray in the US — and the first oncology biosimilars in the country.

→ On the same day it announced a $17.5 million Series C, life sciences and health data company Seer unveiled that it had lured former FDA commissioner and ex-CMS administrator Mark McClellan on to its board. “Mark’s deep understanding of the health care ecosystem and visionary insights on policy reform will be crucial in informing our thinking as we work to bring our liquid biopsy and life sciences products to market,” said Seer chief and founder Omid Farokhzad in a statement.

Gilead CEO Daniel O’Day’s new task hunting up a CSO for the company isn’t stopping the industry’s dominant antiviral player from doing pipeline deals.

The big biotech today snapped up 3 preclinical antiviral programs from pharma giant Novartis, with drugs promising to treat human rhinovirus, influenza and herpes viruses. We don’t know what the upfront is, but the back end has $291 million in milestones baked in.