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.

In a bewildering twist to the long-suffering market for antibiotics — there has actually been a bidding war for an antibiotic company: Tetraphase.

It all started back in March, when the maker of Xerava (an FDA approved therapy for complicated intra-abdominal infections) said it had received an offer from AcelRx for an all-stock deal valued at $14.4 million.

The offer was well-timed. Xerava was approved in 2018, four years after Tetraphase posted its first batch of pivotal trial data, and sales were nowhere near where they needed to be in order for the company to keep its head above water.

Lonza chairman Albert Baehny took his time headhunting a new CEO for the company, making it absolutely clear he wanted a Big Pharma or biotech CEO with a good long track record in the business for the top spot. In the end, he went with the gold standard, turning to Roche’s ranks to recruit Pierre-Alain Ruffieux for the job.

Ruffieux, a member of the pharma leadership team at Roche, spent close to 5 years at the company. But like a small army of manufacturing execs, he gained much of his experience at the other Big Pharma in Basel, remaining at Novartis for 12 years before expanding his horizons.

Another Big Pharma is entering the Covid-19 antibody hunt.

AbbVie has announced a collaboration with the Netherlands’ Utrecht University and Erasmus Medical Center and the Chinese-Dutch biotech Harbour Biomed to develop a neutralizing antibody that can treat Covid-19. The antibody, called 47D11, was discovered by AbbVie’s three partners, and AbbVie will support early preclinical work, while preparing for later preclinical and clinical development. Researchers described the antibody in Nature Communications last month.

In 2011, GlaxoSmithKline’s Benlysta became the first biologic to win approval for lupus patients. Nine years on, the British drugmaker has unveiled detailed positive results from a study testing the drug in lupus patients with associated kidney disease — a post-marketing requirement from the initial FDA approval.

Lupus is a drug developer’s nightmare. In the last six decades, there has been just one FDA approval (Benlysta), with the field resembling a graveyard in recent years with a string of failures including UCB and Biogen’s late-stage flop, as well as defeats in Xencor and Sanofi’s programs. One of the main reasons the success has eluded researchers is because lupus, akin to cancer, is not just one disease — it really is a disease of many diseases, noted Al Roy, executive director of Lupus Clinical Investigators Network, an initiative of New York-based Lupus Research Alliance that claims it is the world’s leading private funder of lupus research, in an interview.

A month after word first broke of the Trump Administration’s plan to rapidly accelerate the development and production of a Covid-19 vaccine, the White House has selected the five vaccine candidates they consider most likely to succeed, The New York Times reported.

Most of the names in the plan, known as Operation Warp Speed, will come as little surprise to those who have watched the last four months of vaccine developments: Moderna, which was the first vaccine to reach humans and is now the furthest along of any US effort; J&J, which has not gone into trials but received around $500 million in funding from BARDA earlier this year; the joint AstraZeneca-Oxford venture which was granted $1.2 billion from BARDA two weeks ago; Pfizer, which has been working with the mRNA biotech BioNTech; and Merck, which just entered the race and expects to put their two vaccine candidates into humans later this year.

→ Merck — one of the last big pharma bastions in the beleaguered field of antibiotic drug development — on Friday said the FDA had signed off on using its combination drug, Recarbrio, with hospital-acquired bacterial pneumonia and ventilator-associated bacterial pneumonia. The drug could come handy for use in hospitalized patients who are afflicted with Covid-19, who carry a higher risk of contracting secondary bacterial infections. Once SARS-CoV-2, the virus behind Covid-19, infects the airways, it engages the immune system, giving other pathogens free rein to pillage and plunder as they please — the issue is particularly pertinent in patients on ventilators, which in any case are breeding grounds for infectious bacteria.