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.

It’s official. Memorial Sloan Kettering has picked a brain cancer expert as its new physician-in-chief and CMO, replacing José Baselga, who left under a cloud after being singled out by The New York Times and ProPublica for failing to properly air his lucrative industry ties.

His replacement, who now will be in charge of MSK’s cutting-edge research work as well as the cancer care delivered by hundreds of practitioners, is Lisa M. DeAngelis. DeAngelis had been chair of the neurology department and co-founder of MSK’s brain tumor center and was moved in to the acting CMO role in the wake of Baselga’s departure.

More than two years ago Ritter Pharmaceuticals managed to find enough silver lining in its Phase IIb/III study — after missing the top-line mark — to propel its lactose intolerance toward a confirmatory trial. But as it turned out, the enthusiasm only set the biotech and its investors up to be sorely disappointed.

This time around there’s little left to salvage. Not only did RP-G28 fail to beat placebo in reducing lactose intolerance symptoms, patients in the treatment group actually averaged a smaller improvement. On a composite score measuring symptoms like abdominal pain, cramping, bloating and gas, patients given the drug had a mean reduction of 3.159 while the placebo cohort saw a 3.420 drop on average (one-sided p-value = 0.0106).

An early-stage update on Adverum Biotechnologies’ intravitreal gene therapy has triggered investor concern, after patients with wet age-related macular degeneration (AMD) saw their vision deteriorate, despite signs that the treatment is improving retinal anatomy.

Adverum, on Wednesday, unveiled 24-week data from the OPTIC trial of its experimental therapy, ADVM-022, in six patients who have been administered with one dose of the therapy. On average, patients in the trial had severe disease with an average of 6.2 anti-VEGF injections in the eight months prior to screening and an average annualized injection frequency of 9.3 injections.

Too often, Alex Arfaei arrived too late. 

An analyst at BMO Capital Markets, he’d meet with biotech or pharmaceutical heads for their IPO or secondary funding and his brain, trained on a biology degree and six years at Merck and Endo, would spring with questions: Why this biomarker? Why this design? Why not this endpoint? Not that he could do anything about it. These execs were coming for clinical money; their decisions had been made and finalized long ago.

In the first of what it hopes will be a couple of major regulatory milestones for its new drug, Ardelyx has bagged an FDA approval to market Ibsrela (tenapanor) for irritable bowel syndrome.

The drug’s first application will be for IBS with constipation (IBS-C), inhibiting sodium-hydrogen exchanger NHE3 in the GI tract in such a way as to increase bowel movements and decrease abdominal pain. This comes on the heels of two successful Phase III trials.