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FDA re­jects Kala's dry eye drug; Trou­bled In­sys finds a buy­er (of some as­sets) in Hik­ma

→ Kala Pharmaceuticals, one of the plethora of biotechs that trace their roots to the prolific lab at MIT led by Bob Langer, on Thursday disclosed that its experimental drug for short-term dry eye relief did not pass muster with the FDA. Last year, the company posted mixed data from two studies — STRIDE 1 and STRIDE 2. Kala $KALA is now banking on the ongoing STRIDE 3 study to reverse its fortunes. It expects STRIDE 3 to read out by the end of the year, and to resubmit its marketing application the first half of 2020.

Boris John­son pledges £250M to spur AI adop­tion in NHS; SQZ scoops CFO Teri Lox­am from Mer­ck

→ Last year, former UK health secretary Jeremy Hunt commissioned California-based Eric Topol, a cardiologist, author and digital medicine researcher, to carry out an independent review into the digital training needs of NHS staff. The report was released in February, detailing the promise of genomics, artificial intelligence, robotics and digital medicine in the prevention, management and treatment of disease. On Thursday, the UK health department — under new Prime Minister Boris Johnson — unveiled a £250 million investment in Al, which is already being developed in some hospitals, successfully predicting cancer survival rates and cutting the number of missed appointments.

Athenex's oral chemother­a­py for­mu­la­tion scores in piv­otal study; Gly­comine brings in $33M in Se­ries B haul

→ Athenex $ATNX, whose platform technology is engineered to convert widely-used intravenous chemotherapies into oral drugs, said its oral formulation of paclitaxel met the main goal of conferring a statistically significant improvement over IV paclitaxel in a pivotal study involving metastatic breast cancer patients. The company is now in the process of putting together a marketing application.

“Based on the results of the Phase III study, together with the preliminary results generated in the angiosarcoma study, Athenex believes that Oral Paclitaxel has the potential to represent a new class of oral anti-cancer drugs,” said chief Johnson Lau in a statement. “Adding to this potential are the favorable safety data from this study showing lower incidence of neuropathy, which is currently a major reason for discontinuing IV paclitaxel treatment.”

FDA en­dors­es No­vavax's piv­otal tri­al de­sign for flu vac­cine; Forty Sev­en bags fund­ing for myelodys­plas­tic syn­dromes pro­gram

→ Following a stream of setbacks with its RSV vaccine, Novavax is touting some regulatory progress of another candidate for a change. The FDA has apparently green-lighted the Phase III trial design for NanoFlu, its adjuvanted, recombinant quadrivalent flu vaccine targeting the elderly (65 or older). The trial is expected to begin in a couple of months and generate top-line data in the first quarter of 2020.

Guardant blood test shows promise in pre­dict­ing im­munother­a­py re­sponse; As­traZeneca's di­a­betes drug wins EU la­bel ex­pan­sion

→ The field of liquid biopsy is burgeoning, and each player (there are plenty) is doggedly pursuing consistency and accuracy — false positives induce unnecessary anxiety, and are costly. Late on Sunday, Guardant Health said its assay, Guardant360, showed in a study it can accurately detect microsatellite instability (MSI), a key biomarker used to predict response to immunotherapy regardless of tumor type. MSI is a change that occurs in the DNA of certain cells (such as tumor cells) in which the number of repeats of microsatellites (short, repeated sequences of DNA) is different than the number of repeats that was in the DNA when it was inherited.

Am­i­cus touts lat­est set of gene ther­a­py da­ta; No­vo Nordisk ex­pands dis­cov­ery deal for di­a­betes

→ The execs at Amicus Therapeutics $FOLD spared no effort in touting an early set of data for their gene therapy program to treat CLN6 Batten disease. At the interim, the snapshot results showed little change in 8 patients treated up to 24 months ago. The biotech says that they would normally have experienced a decline, given historical results for the disease. The stock, though, barely budged on the update Thursday morning.

At­lantic Health­care's drug fails late-stage test; Trans­late Bio breaks out ear­ly CF da­ta

→ Cambridge, UK-based Atlantic Healthcare disclosed that the late-stage program for its experimental drug, alicaforsen enema, has failed in patients with pouchitis, a progressive disease characterized by inflammation, ulceration and increasingly uncontrolled, frequent and urgent emptying of the bowel (up to 10-20 times a day and night). “Although we are disappointed that the Phase 3 trial did not achieve statistical significance, we believe the percentage of patients achieving remission in stool frequency and the endoscopic response observed in a number of subpopulations of patients could be noteworthy,” said chief Toby Wilson Waterworth. “Having consulted with key opinion leaders and regulatory advisors, we now plan to meet with the U.S. Food and Drug Administration and European Medicines Agency to discuss a pathway to regulatory approval.”

Found­ing NewLink CEO Charles Link is out; Chi-Med lines up two more drug fil­ings

→ Four years ago NewLink’s shares were riding high on the promise of IDO in oncology, trading at more than $50 a share. But as enthusiasm for the field plummeted on the back of some key setbacks, so did the stock. And today — with the market cap sitting at $59 million and the stock opening at $1.59 — their founding CEO, Charles Link, has hit the exit. The biotech will be headed by a committee during the transition, with CFO Carl Langren joining CMO Eugene Kennedy, general counsel Brad Powers and Lori Lawley, the VP of finance.

Gen­fit's elafi­bra­nor wins or­phan sta­tus for pri­ma­ry bil­iary cholan­gi­tis; Cidara stock soars on pos­i­tive an­ti-fun­gal mid-stage da­ta

→ NASH hopeful Genfit has been awarded orphan drug status by US and EU regulators for its experimental drug, elafibranor, to treat patients with primary biliary cholangitis (PBC), a chronic disease in which bile ducts in the liver are gradually destroyed. The PPAR dual agonist, which is in a pivotal trial involving NASH patients,  is currently in mid-stage development for PBC. The French drug developer $GNFT recently made its Nasdaq debut, and last month inked up to $228 million deal with transpacific biotech Terns Pharmaceuticals to develop the liver drug in Greater China.

EMA backs five med­i­cines; Sol­id Bio rais­es $60M in pri­vate place­ment

→ The European Medicines Agency on Friday recommended five new medicines for approval, including Bayer’s cancer drug Vitrakvi — the site-agnostic cancer drug was given a speedy approval by the FDA in late 2018 — and GW Pharmaceutical’s $GWPH cannabis-derived epilepsy drug Epidyolex, which was approved by the FDA last June under the name Epidiolex.

The EMA’s decision on GW’s drug is as an adjunctive therapy — for use in conjunction with clobazam. In the United States, it is approved as a monotherapy. “(T)he outcome is disappointing and a distinctly more conservative decision compared to that rendered by the FDA,” Stifel’s Paul Matteis wrote in a note. “What exactly this means for uptake is unclear because we don’t know of any good data source that codifies exactly how much clobazam is used in Europe; that being said, as we understand it, clobazam has been generic in Europe for a long time and is a staple of the Dravet/LGS/orphan epilepsy standard-of-care.”