HIV pa­tients will have to wait a while longer for a long-act­ing vi­ral sup­pres­sion reg­i­men as the FDA has re­ject­ed Vi­iV Health­care’s NDA for the month­ly in­jec­tion of cabote­gravir and rilpivirine.

Reg­u­la­tors hand­ed the GSK sub­sidiary a com­plete re­sponse let­ter days be­fore the orig­i­nal PDU­FA date on De­cem­ber 29. While Vi­iV had been ex­pect­ing a smooth sail to the fin­ish line based on glow­ing clin­i­cal re­sults, the CRL cit­ed con­cerns with chem­istry man­u­fac­tur­ing and con­trols.

“There have been no re­port­ed safe­ty is­sues re­lat­ed to CMC and there is no change to the safe­ty pro­file of the prod­ucts used in clin­i­cal tri­als to date,” Vi­iV added in a short state­ment.

Kim Smith

A de­lay here with the month­ly in­jec­tion of cabote­gravir and rilpivirine al­so means that Vi­iV will have to push back on the planned sN­DA fil­ing for the 2-month­ly reg­i­men, which was slat­ed for the first quar­ter of 2020.

Gilead’s block­buster cock­tails, both dou­blets and triplets, have been im­mense­ly suc­cess­ful at keep­ing HIV to un­de­tectable lev­els, but they have still left pa­tients sad­dled with the bur­den of tak­ing dai­ly pills — which Vi­iV said is not on­ly in­con­ve­nient but al­so car­ries an emo­tion­al bur­den. With their new for­mu­la­tion of their own ex­per­i­men­tal cabote­gravir (in­te­grase in­hibitor) and J&J’s rilpivirine (non-nu­cle­o­side re­verse tran­scrip­tase in­hibitor), Vi­iV was look­ing to shake up the treat­ment land­scape and carve out a big­ger share from the Gilead-dom­i­nat­ed mar­ket.

The long-act­ing reg­i­men is de­signed for pa­tients who are al­ready vi­ro­log­i­cal­ly sup­pressed, have nev­er ex­pe­ri­enced vi­ro­log­i­cal fail­ures, and have not de­vel­oped re­sis­tance to ei­ther of the in­gre­di­ents. Vi­iV head of R&D Kim Smith has es­ti­mat­ed that around 10% to 15% of all pa­tients in the clin­ic would be can­di­dates.

Two late-stage stud­ies in­volv­ing 1180 pa­tients sug­gest­ed that their in­jec­tion was just as ef­fec­tive as stan­dard an­ti­retro­vi­ral ther­a­py in vi­ro­log­ic sup­pres­sion, with sim­i­lar rates of re­sis­tance at 1%. And as they fol­low up on a Phase IIb tri­al, re­searchers found that 83% of pa­tients re­main vi­ral­ly sup­pressed at the three-year mark.

That, along­side sim­i­lar­ly pos­i­tive da­ta for the 2-month­ly reg­i­men, would have made a ground­break­ing treat­ment op­tion. The com­pa­ny said it’s work­ing close­ly with the FDA to fig­ure out the next steps.

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.

Every few years, a public health crisis (think Ebola, Zika) spurred by a rogue pathogen triggers a small-biotech rally, as drugmakers emerge from the woodwork with ambitious plans to treat the mounting outbreak. In most cases, that enthusiasm never quite delivers.

Things are no different, as the coronavirus outbreak in Wuhan, China takes hold. There have been close to 300 confirmed human infections in China, and at least four deaths. Coronaviruses are a large family of viruses, which include MERS and SARS. On Tuesday, the CDC reported the virus was detected in a US traveler returning from Wuhan.

The venture tide might have subsided, the IPO window may be closing and certain listed biotechs may be having a tough time amid Neil Woodford’s well-publicized demised, but there’s still plenty to celebrate in the UK BioIndustry Association’s eyes.

Overall investment in UK biotech last year fell from the record-breaking £2.2 billion levels of 2018 to £1.3 billion — including £679 million in venture capital, a meager £64 million in IPOs plus £596 million when you add up all public financings, according to a new report from the BIA.

→ Blueprint Medicines filed an amendment to its application to get the gastrointestinal stromal tumor (GIST) drug Ayvakit approved in fourth-line GIST, the company disclosed in the prospectus for a new $325 million public offering.  Blueprint got a big accelerated OK on the drug this month in a particular mutation, but because the FDA decided to split their review in two, they didn’t hear on fourth-line GIST. They were supposed to hear before February 14, but this amendment could push that date back by 3 months. Blueprint wrote that the amendment is designed to allow the company to comply with the FDA’s request for data from the Phase III Voyage trial before they give a judgment.

Late last year Novartis abandoned a cardio drug from Ionis’ spinoff Akcea just after the pharma giant snapped up inclisiran, going the RNAi way in guarding against heart disease in the $9.7 billion Medco buyout.

Now the pharma goliath — which is headed down the PCSK9 road with a drug it believes can be used in a mass population — can get a clearer picture of just what they gave up.

Akcea $AKCA and the mother company $IONS put out a statement early Wednesday saying that their Phase II study of AKCEA-APOCIII-LR delivered solid efficacy data, with the high dose clearly outperforming placebo in significantly reducing triglycerides as a means to cutting the risk of cardiovascular disease. In addition, investigators concluded that the drug slashed apoC-III, very low-density lipoprotein and remnant cholesterol while boosting “good” HDL levels.

Update: Nominations open through end of day, Monday, January 27

Two years ago, when we did our first Endpoints 20-under-40, we profiled a set of up-and-comers who promised to help reshape the industry as we know it. Now we’re back and once again looking for the top 20 biopharma professionals under the age of 40. We’ll be profiling folks who have accomplished a lot at a young age but seem on the verge of accomplishing so much more.