An­ti­fun­gal drug de­vel­op­er Scynex­is, which burst in­to the zeit­geist as one of the sole US-based com­pa­nies with a Can­di­da au­ris ther­a­py in hu­man tri­als, on Thurs­day dis­closed that the same ex­per­i­men­tal drug had hit the main goal in a late-stage tri­al in pa­tients with vagi­nal yeast in­fec­tions.

The oral drug, ibrex­a­fungerp, was be­ing test­ed in the 376-pa­tient, place­bo-con­trolled VAN­ISH-303 tri­al. Pa­tients en­rolled had ex­pe­ri­enced an acute episode of vul­vo­vagi­nal can­didi­a­sis (VVC), with signs and symp­toms score of four or greater on a scale of ze­ro to 18.

The drug in­duced a ‘clin­i­cal cure’ (de­fined as com­plete res­o­lu­tion — or score of 0 — at the test-of-cure vis­it on day 10), rate of 50.5%, mark­ing a sta­tis­ti­cal­ly sig­nif­i­cant per­for­mance over the place­bo arm (p=0.001), Scynex­is said.

The sec­ondary end­point of my­co­log­i­cal erad­i­ca­tion was al­so met. On the safe­ty side, se­vere ad­verse events were rare, but there were more cas­es re­port­ed in the place­bo group than the ibrex­a­fungerp group, the com­pa­ny added.

An­oth­er twin late-stage study test­ing ibrex­a­fungerp, called VAN­ISH-306, is be­ing con­duct­ed — and da­ta is ex­pect­ed next year. As­sum­ing it is pos­i­tive, re­sults from both tri­als will be used to sub­mit a mar­ket­ing ap­pli­ca­tion in the sec­ond half of 2020. (The drug is al­so be­ing eval­u­at­ing for use in the CAN­DLE study in pa­tients with re­cur­rent VVC — the re­sults are ex­pect­ed in 2021. )

Mar­co Tagli­et­ti Scynex­is

“We be­lieve that ibrex­a­fungerp, as a nov­el, non-azole, oral ther­a­py, can ad­dress large un­met med­ical needs for women with VVC who may not re­spond to flu­cona­zole, the on­ly oral op­tion cur­rent­ly avail­able, which is fungista­t­ic against Can­di­da and al­so has well-doc­u­ment­ed con­cerns around drug-drug in­ter­ac­tions and em­bryo-fe­tal tox­i­c­i­ty for women of child­bear­ing age,” Scynex­is chief Mar­co Tagli­et­ti said in a state­ment on Thurs­day.

Ibrex­a­fungerp, if ap­proved, could end up as the on­ly oral al­ter­na­tive to flu­cona­zole ap­proved for acute VVC in­fec­tions, al­though My­covia is test­ing an­oth­er ther­a­py, VT-1161, for pa­tients with re­cur­rent VVC, Need­ham’s Alan Carr wrote in a note, adding that he ex­pects ibrex­a­fungerp to launch in 2021.

“Our da­ta sug­gest 15% of pts re­turn to their physi­cian be­cause ex­ist­ing treat­ments are in­ad­e­quate…physi­cian in­ter­est in a new drug is high and we es­ti­mate even mod­est (25%) pen­e­tra­tion of the sec­ond-line set­ting gen­er­ates peak sales (for ibrex­a­fungerp) of over $450M.”

It has been decades since a fresh fam­i­ly of an­ti­fun­gals has come on to the mar­ket — at the mo­ment there are three class­es of an­ti­fun­gals in clin­i­cal use, in­clud­ing azoles and echinocan­dins. The most pop­u­lar an­ti­fun­gals in use to­day were in­tro­duced in the 1980s and this class is in­creas­ing­ly los­ing its po­ten­cy, as fun­gal pathogens be­come more re­sis­tant.

Scynex­is’s ibrex­a­fungerp be­longs to a new class of an­ti­fun­gals called glu­can syn­thase in­hibitors, and has shown to be ef­fec­tive against a broad range of fun­gal in­fec­tions, in­clud­ing re­sis­tant strains, Tagli­et­ti told End­points News in a pri­or in­ter­view.

One of the biggest chal­lenges fac­ing the field of an­ti­fun­gal drug de­vel­op­ment is the lack of in­cen­tives, such as those af­ford­ed to com­pa­nies in the an­tibac­te­r­i­al and an­tivi­ral space, he said, adding that when Scynex­is ap­proached BAR­DA for fund­ing, they were told an­ti­fun­gals were not a pri­or­i­ty in Wash­ing­ton.

“I hope this aware­ness will now reach Wash­ing­ton.”

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.

Roche’s Kadcyla has become the first antibody-drug conjugate to enter the Chinese market, marking a dramatic advance for both the Swiss pharma giant and the therapeutic class.

The local arm of Roche announced the approval late Tuesday, which covers the therapy’s use in the adjuvant setting in patients with early HER-2 positive breast cancer who still have residual invasive disease after receiving paclitaxel and Herceptin as neoadjuvant treatment.

Pascal Soriot has spent more than 20 years at the top of an industry recently found to emit more carbon than the automotive industry.

He called himself a “global citizen,” and traveled often across three-plus continents. While CEO of AstraZeneca, he commissioned a flight service — media-dubbed AstraZeneca airlines — from Cambridge to the company’s other European hub in Gothenburg. He made few, if any, public statements on the environment or his companies’ impact on it.

With an FDA decision date looming, Roche on Wednesday unveiled positive pivotal data on its blockbuster-bound oral spinal muscular atrophy (SMA) drug in patients with the most severe form of the muscle-wasting disease.

The FDA is set to make its decision on the therapy, risdiplam, by May 24. It is expected to compete with Biogen’s Spinraza and Novartis’ Zolgensma.

Partnered with PTC Therapeutics, the Roche drug was tested in 41 patients aged 1-7 months with type 1 SMA, a rare genetic muscle-wasting disease. The trial, dubbed FIREFISH, measured efficacy via the proportion of infants sitting without support after 12 months of treatment, and longer.

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.