CNCE Hits Right Notes, BXRX Faces FDA In Feb., All Eyes On AGTC

pharma 071417 22jan20

Today's Daily Dose brings you news about Akcea's promising trial results of antisense drug AKCEA-APOCIII-LRx, Applied Genetic's upcoming event, the near-term catalyst of Baudax Bio and Sierra Oncology's reverse stock split.

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Shares of Akcea Therapeutics Inc. (AKCA), a majority-owned affiliate of Ionis Pharmaceuticals Inc., and Ionis Pharmaceuticals Inc. (IONS) rose more than 10% on Wednesday, following positive topline results from the phase II study of the antisense drug AKCEA-APOCIII-LRx in the treatment of patients with hypertriglyceridemia who are at risk for or have established cardiovascular disease (CVD).

The study met the primary endpoint of significant triglyceride lowering and multiple secondary endpoints with a favorable safety and tolerability profile.

According to the Company, at the highest, once-monthly dose of 50 mg, more than 90% of patients achieved serum triglycerides of less than or equal to 150 mg/dL, compared to less than 5% of patients in the placebo group. The mean triglyceride level of patients at baseline was 285 mg/dL.

AKCA closed Wednesday's trading at $19.24, up 10.96%.

Applied Genetic Technologies Corp. (AGTC) is all set to announce interim six-month data from the dose-escalation groups, and an update of enrollment for its ongoing two parallel phase I/II studies of its gene therapy candidate for Achromatopsia, dubbed CNGB3 and CNGA3, on January 23, 2020.

Achromatopsia is a condition characterized by partial or total absence of color vision. People with complete achromatopsia cannot perceive any colors; they see only black, white, and shades of gray. Incomplete achromatopsia is a milder form of the condition that allows some color discrimination (Source: Genetics Home Reference, NIH).

On January 9, 2020, the Company had reported positive interim six-month data from its ongoing phase I/II clinical trial of AAV gene therapy product candidate expressing the RPGR protein for the treatment of X-linked retinitis pigmentosa. The news sent the stock up as much as over 130% to $9.50 that day.

AGTC touched a new 52-week high of $10.42 in intraday trading on Wednesday, before closing at $9.86, up 19.52%.

The FDA decision on Baudax Bio Inc.'s (BXRX) New Drug Application for intravenous (IV) meloxicam for the management of moderate to severe pain is set for February 20, 2020.

The intravenous Meloxicam has been twice rejected by the FDA - in May 2018 and in March 2019. The Company had appealed the 2019 Complete Response Letter.

The assignment of the decision date of February 20, 2020, follows the FDA's acceptance of Baudax's latest complete response package relating to its 2019 complete response letter (CRL) and appeal filing, seeking approval for IV meloxicam.

Baudax Bio is a spinoff from Recro Pharma Inc (REPH) and began trading on the NASDAQ Capital Market on November 22, 2019.

BXRX closed Wednesday's trading at $8.53, up 21.16%.

Concert Pharmaceuticals Inc. (CNCE) has received a Notice of Allowance from the U.S. Patent and Trademark Office for patent related to CTP-543, the Company's investigational treatment for alopecia areata.

The patent, which is expected to expire in 2037, covers pharmaceutical compositions of CTP-543 and methods of treating alopecia areata with CTP-543.

Last month, the Company successfully completed a phase II trial of CTP-543 in alopecia areata. Alopecia areata is an autoimmune disease in which the immune system attacks hair follicles, resulting in partial or complete loss of hair on the scalp and body.

Concert Pharma intends to conduct an end of phase II meeting with the FDA and advance CTP-543 into phase III testing in 2020.

Concert has been locked in a patent battle with Incyte Corp. (INCY) related to CTP-543. Concert's CTP-543 is deuterated Ruxolitinib.

Ruxolitinib, a drug that selectively inhibits Janus kinases 1 and 2 (JAK1 and JAK2), is marketed by Incyte in the U.S. as Jakafi and by Novartis as Jakavi outside the U.S for the treatment of patients with myelofibrosis and polycythemia vera.

In April 2017, Incyte filed an inter parties review, or IPR, petition with the PTAB, of the U.S. PTO, challenging the validity of U.S. Patent No. 9,249,149, which claims deuterium-modified versions of Ruxolitinib, including CTP-543, are a novel composition of matter. Last April, the Patent Trial and Appeal Board (PTAB) ruled that the claims of the '149 patent are not patentable. Concert appealed the ruling of PTAB last June.

Amid all these appeals and counter appeals, CTP-543 being issued a new patent is good news for Concert Pharma.

CNCE closed Wednesday's trading at $13.09, up 33.03%.

Sierra Oncology Inc. (SRRA) has implemented a 1-for-40 reverse stock split that became effective Wednesday at 4:30 p.m. Eastern Time.

The Company's common stock will trade on a split-adjusted basis at the market open on January 23, 2020.

SRRA closed Wednesday's trading at $0.34, down 11.14%.

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