The ver­dict is in from Ver­tex’s clin­i­cal team.

The biotech was pre­sent­ed with a pleas­ant dilem­ma in March when a cock­tail for cys­tic fi­bro­sis mix­ing VX-​445, teza­caftor and iva­caftor passed Phase III with fly­ing col­ors, match­ing the re­sults they got for VX-659 with the oth­er two drugs, al­so known as Symdeko and Ka­ly­de­co. That left ob­servers hang­ing as to which com­bo will head to reg­u­la­tors.

Ver­tex ul­ti­mate­ly de­cid­ed to go with VX-445 — af­ter re­view­ing some more da­ta it’s dis­clos­ing to­day.

Resh­ma Ke­wal­ra­mani Ver­tex

At week 24 of treat­ment, pa­tients with one F508del mu­ta­tion and one min­i­mal func­tion mu­ta­tion on the drug main­tained ab­solute im­prove­ment in per­cent pre­dict­ed forced ex­pi­ra­to­ry vol­ume in one sec­ond of 14.3% (p<0.0001). For a key sec­ondary end­point, the same group of pa­tients saw a 63% re­duc­tion in the an­nu­al­ized rate of pul­monary ex­ac­er­ba­tions com­pared to place­bo, with the same stel­lar p-val­ue.

As pre­vi­ous­ly re­port­ed, pa­tients on the VX-445 triple had their ppFEV1 im­prove by an av­er­age of 13.8% at week 4. In a sep­a­rate tri­al for VX-​659, the num­ber was 14%.

“Peo­ple with CF who have one F508del mu­ta­tion and one min­i­mal func­tion mu­ta­tion are the largest re­main­ing group of CF pa­tients with­out a treat­ment op­tion for the un­der­ly­ing cause of their dis­ease,” CMO Resh­ma Ke­wal­ra­mani said in a state­ment.

The biotech fur­ther not­ed that the de­ci­sion “was based on a de­tailed as­sess­ment of mul­ti­ple fac­tors, in­clud­ing fa­vor­able pro­files for safe­ty, tol­er­a­bil­i­ty and drug-drug in­ter­ac­tions, the abil­i­ty for co-ad­min­is­tra­tion with hor­mon­al con­tra­cep­tives, and the lack of pho­to­sen­si­tiv­i­ty.”

Ver­tex will now take these da­ta, along­side an­oth­er 4-week Phase III study CF pa­tients with two F508del mu­ta­tions, to the FDA in Q3 and then the EMA in Q4.

Cred­it Su­isse an­a­lysts are ful­ly on board with the plan, say­ing it sig­nals a “clear path for­ward for the triple com­bo, with longer-term da­ta sup­port­ing con­tin­ued ef­fi­ca­cy. We ex­pect ap­proval in 1H20, al­low­ing the co. to treat 90% of CF pts WW. OUS re­im­burse­ment ques­tions re­main.”

Im­age: Ver­tex Phar­ma­ceu­ti­cals

Hepatitis delta, also known as hepatitis D, is a liver infection caused by the hepatitis delta virus (HDV) that results in the most severe form of human viral hepatitis for which there is no approved therapy.

HDV is a single-stranded, circular RNA virus that requires the envelope protein (HBsAg) of the hepatitis B virus (HBV) for its own assembly. As a result, hepatitis delta virus (HDV) infection occurs only as a co-infection in individuals infected with HBV. However, HDV/HBV co-infections lead to more serious liver disease than HBV infection alone. HDV is associated with faster progression to liver fibrosis (progressing to cirrhosis in about 80% of individuals in 5-10 years), increased risk of liver cancer, and early decompensated cirrhosis and liver failure.
HDV is the most severe form of viral hepatitis with no approved treatment.
Approved nucleos(t)ide treatments for HBV only suppress HBV DNA, do not appreciably impact HBsAg and have no impact on HDV. Investigational agents in development for HBV target multiple new mechanisms. Aspirations are high, but a functional cure for HBV has not been achieved nor is one anticipated in the forseeable future. Without clearance of HBsAg, anti-HBV investigational treatments are not expected to impact the deadly course of HDV infection anytime soon.

Another executive is migrating from the echelons of Big Pharma to the corridors of small biotech.

In April 2018, Badrul Chowdhury took his more than two decades of experience at the FDA to AstraZeneca, where he took on the role of senior vice president and chief physician-scientist for respiratory, inflammation and autoimmunity late-stage development in biopharmaceuticals R&D.

After about a year and a half in this role, Chowdhury is moving to a small Texas biotech called Savara, where he will serve as chief medical officer.

When Amazon developed a machine-learning tool to make its recruitment process more efficient — the man-made system absorbed the gender-bias of its human makers, and the project was aborted. In the field of biopharmaceuticals, the way researchers train their machine learning algorithms can skew the outcome of predictions. But before those predictions can be made, the engine must learn to read to make sense of explosive volume of knowledge out there.

As a former academic and a seasoned drug developer, Burt Adelman knew when he was recruited as a senior advisor to Novo Ventures in 2017 that one of his key priorities needs to be introducing the fund to the network he was so deeply embedded in.

“I was thinking long and hard on how can I, as a Boston insider, help Novo really get inside the ecosystem of Boston biotech?” he recalled in an interview with Endpoints News.

Wellington Management made some uncustomary waves at the beginning of the year when it threw its considerable weight against Bristol-Myers Squibb’s $74 billion Celgene buyout. But after Bristol-Myers’ biggest investor conceded that game to the influential proxy firms involved, they’re now going to end the year by rolling out a big new investment fund for a new stable of fledgling biotechs on the private side of the industry.

Myovant has long had a secret weapon in its uterine rivalry with AbbVie: Men.

While the small Swiss biotech has jockeyed with the Illinois-based giant for a foothold in the endometriosis and uterine fibroid therapy market, the company has been developing the same lead compound, relugolix, for use in one of the most common cancers for the uterus-less: prostate cancer. Today, Myovant is out with positive topline results from its big Phase III trial on the gonadotropin-releasing hormone (GnRH) antagonist. They say they’ve reached every primary and secondary endpoint with p values less than .0001.