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.

Fewer Approvals, but Neurology Rivals Oncology and Sees Major Innovations

This report, the fourth in our Trinity Drug Index series, outlines key themes and emerging trends in the industry as we progress towards a new world of targeted and innovative products. It provides a comprehensive evaluation of the performance of novel drugs approved by the FDA in 2016, scoring each on its commercial performance, therapeutic value, and R&D investment (Table 1: Drug ranking – Ratings on a 1-5 scale).

For start-up biotechnology companies and resource stretched pharmaceutical organisations, launching a novel product can be challenging. Lean teams can make setting a launch strategy and achieving your commercial goals seem like a colossal undertaking, but can these barriers be transformed into opportunities that work to your brand’s advantage?
We spoke to Managing Consultant Frances Hendry to find out how Blue Latitude Health partnered with a fledgling subsidiary of a pharmaceutical organisation to launch an innovative product in a
complex market.
What does the launch environment look like for this product?
FH: We started working on the product at Phase II and now we’re going into Phase III trials. There is a significant unmet need in this disease area, and everyone is excited about the launch. However, the organisation is still evolving and the team is quite small – naturally this causes a little turbulence.

Back in November, the Department of Health and Human Services took the rare step of filing a complaint against Gilead for infringing on government-owned patents related to the HIV drug Truvada (emtricitabine/tenofovir disoproxil fumarate) for pre-exposure prophylaxis (PrEP).

But on Thursday, Gilead filed its own retort, making clear that it does not believe it has infringed on the Centers for Disease Control and Prevention’s (CDC) Truvada patents because they are invalid.

→ Less than a year ago Gilead’s antiviral remdesivir failed to make the cut as investigators considered a raft of potential drugs that could be used against an Ebola outbreak. But it may gain a new mission with the outbreak of the coronavirus in China, which is popping up now around the world.

Gilead put out a statement saying that they’re now in discussions with health officials in the US and China about testing their NUC against the virus. It’s the latest in a growing lineup of biopharma companies that are marshaling R&D forces to see if they can come up with a vaccine or therapy to blunt the spread of the virus, which has now sickened hundreds, killed at least 17 people and led the Chinese government to start quarantining cities.

Back at the beginning of 2015, Deerfield Management co-led a $10 million Series C for a private gene therapy startup, reshaping the company and bringing in new leaders to pave way for an IPO just a year later.

Fast forward four more years and the startup, AveXis, is now a subsidiary of Novartis marketing the second-ever gene therapy to be approved in the US.

For its part, Deerfield has also grown more comfortable and ambitious about the nascent field. And the investment firm is now putting down its biggest bet yet: a $1.1 billion contract development and manufacturing facility to produce everything one needs for cell and gene therapy — faster and better than how it’s currently done.

Different therapeutic traits brandished by the three approved therapies for sickle cell disease all extend life expectancy, but their impact on quality of life is uncertain and their long-term cost-effectiveness is not up to scratch according to the thresholds considered reasonable by ICER, the non-profit concluded in a draft guidance report on Thursday.

Sickle cell disease (SCD), which encompasses a group of inherited red blood cell disorders that typically afflict those of African ancestry, impacts hemoglobin — and is characterized by episodes of searing pain as well as organ damage.