Bio­Marin isn’t go­ing to let a ri­val’s suc­cess over the week­end get in the way of mak­ing a bee-line to the FDA with an im­per­fect, but pre­sentable, gene ther­a­py for he­mo­phil­ia A.

On Mon­day the biotech $BM­RN re­port­ed that its talks with the FDA and the EMA con­vinced ex­ecs to make a leap for an ac­cel­er­at­ed ap­proval with da­ta on just a hand­ful of pa­tients. And they’re hand­ing over Phase I/II and in­ter­im Phase III re­sults in Q4 to make their case.

That de­ci­sion was made de­spite a con­sid­er­able back­lash against the lat­est snap­shot of the da­ta, with wors­en­ing ef­fi­ca­cy over time and a low­er ini­tial re­sponse in their piv­otal tri­al — but ex­cel­lent bleed rates, so far. Bio­Marin says that with a break­through drug des­ig­na­tion in the USA and Eu­rope’s prime sta­tus, they have good rea­son to be­lieve they are on track to field the world’s first gene ther­a­py.

In­vestors ap­pear some­what skep­ti­cal though, with Bio­Marin shares down 2.4% in Mon­day af­ter­noon trad­ing.

Here’s the bet they’re mak­ing, out­lined in their lat­est state­ment:

Fac­tor VI­II lev­els sus­tained over a three-year pe­ri­od were suf­fi­cient to achieve strik­ing he­mo­sta­t­ic ef­fi­ca­cy. Fac­tor VI­II ex­pres­sion has en­tered a plateau phase where the rate of de­cline has sub­stan­tial­ly slowed, which could be in­dica­tive of durable, long-term ex­pres­sion.

Leerink’s Joseph Schwartz of­fers a bull­ish as­sess­ment of Bio­Marin’s gam­ble:

Based on BM­RN’s Ph.3 da­ta, the com­pa­ny met their pre-spec­i­fied cri­te­ria for ac­cel­er­at­ed ap­proval – 8 pts achiev­ing mean fac­tor VI­II/FVI­II lev­els above 40 IU/dL. With on­ly 17 pts (out of 20 pts) eval­u­at­ed so far, it is pos­si­ble that the re­main­ing 3 could pro­vide more con­fi­dence in Val­rox al­though it is not re­quired for ac­cel­er­at­ed ap­proval since BM­RN has al­ready met the bar. The com­pa­ny is fo­cused on get­ting Val­rox to mar­ket as soon as pos­si­ble by sub­mit­ting their fil­ing in 4Q19 based on their Ph.3 in­ter­im analy­sis and aims to com­plete GEN­Er8-1 (6E13 vg/kg) en­roll­ment in ear­ly fall 2019. As­sum­ing BM­RN could in­crease and op­ti­mize ef­fi­ca­cy — re­call, chro­mogenic mea­sures were high­er in Ph.1/2 at weeks 23-26 than Ph.3 (68 IU/dL mean/57 IU/dL me­di­an in Ph.1/2 vs 36 IU/dL mean/33 IU/dL me­di­an Ph.3) — we be­lieve that po­ten­tial stud­ies BM­RN may con­duct lat­er could fur­ther boost sales and in­crease Val­rox’s at­trac­tive­ness among he­mo­phil­ia A pts, es­pe­cial­ly as com­pet­ing pro­grams (i.e., SG­MO/PFE) ap­pear to be de­liv­er­ing com­pet­i­tive ef­fi­ca­cy.

Spark Ther­a­peu­tics $ONCE is wide­ly viewed as the run­ner up for now, while Roche strug­gles to com­plete its $4.3 bil­lion buy­out as soon as pos­si­ble. But Sang­amo/Pfiz­er gar­nered some care­ful sec­ond looks a few days ago as they post­ed bet­ter ef­fi­ca­cy and a fast re­sponse among 4 evalu­able pa­tients. That team al­so out­lined plans to make a leap for­ward on an ac­cel­er­at­ed path­way as they sought to catch up with the two fron­trun­ners.

Any stum­ble at this point could prove a set­back, but if they stay on track Sang­amo and Pfiz­er may set up a sit­u­a­tion where pa­tients can choose an im­per­fect gene ther­a­py or wait for a bet­ter one. And Bio­Marin has out­lined plans to charge in the range of $2 mil­lion to $3 mil­lion per treat­ment — with the ca­pa­bil­i­ty of earn­ing $10 bil­lion to $15 bil­lion a year — adding stick­er shock for pay­ers to the list of chal­lenges.

The FDA hasn’t detected any potential safety signals, including for stroke, in people aged 65 years and older who have received Pfizer’s bivalent Covid booster, one senior official told members of the agency’s Vaccines and Related Biological Products Advisory Committee (VRBPAC) on Thursday.

The update comes as the FDA and CDC investigate a “preliminary signal” that may indicate an increased risk of ischemic stroke in older Americans who received Pfizer’s updated shot.

The FDA has stopped use of another drug as a result of the new coronavirus variants. On Thursday, the agency announced that AstraZeneca’s antibody combo Evusheld, which was an important prevention option for many immunocompromised people and others, is no longer authorized.

The FDA said it made its decision based on the fact that Evusheld works on fewer than 10% of circulating variants.

Evusheld was initially given emergency authorization at the end of 2021. However, as Omicron emerged, so did studies that showed Evusheld might not work against the dominant Omicron strain. In October, the FDA warned healthcare providers that Evusheld was useless against the Omicron subvariant BA.4.6. It followed that up with another announcement earlier this month that it did not think Evusheld would work against the latest Omicron subvariant XBB.1.5.

XyloCor Therapeutics says patients with heart disease who got its gene therapy could exercise for longer and had fewer chest pain attacks. The biotech announced it completed a Phase I/II trial of the gene therapy Thursday morning, and plans to move forward with a pivotal trial.

In the Phase II portion of the trial, 28 patients with angina (or chest pain) caused by coronary artery disease and who had no other treatment options were enrolled and were given the highest tested dose from the first part of the trial. Patients were followed for six months.

Magenta Therapeutics is pausing an early-stage clinical trial after a patient died. The death was deemed to be possibly related to its drug, MGTA-117.

The biotech said the pause of the Phase I/II trial is voluntary and gives it time to review all available data before deciding what to do next. It’s also reported the known information to the FDA.

The dose-escalation trial was designed to test whether MGTA-117, an antibody-drug conjugate, could serve as a more targeted alternative to high-intensity chemotherapy as a conditioning agent for cancer patients who are set to receive a stem cell transplant. It recruited patients with relapsed/refractory acute myeloid leukemia and myelodysplastic syndrome.

Regeneron has won a patent case against Swiss pharma giant Novartis over the delivery system for its eye drug Eylea.

The US Patent Trial and Appeal Board ruled that Novartis’ pre-filled syringe for injecting its eye medication Lucentis was “unpatentable” and handed the victory to Regeneron and its AMD drug Eylea.

In the initial complaint in 2020, Novartis alleged to the US International Trade Commission that certain pre-filled syringes for the intravitreal injection, and ultimately Regeneron’s delivery system for Eylea, were infringing on Novartis’ patent. Regeneron filed a petition to review Novartis’ claims in 2021.