BPC May 29 update

CARA meets primary endpoint +20%; Amarin AMRN +12% on NDA acceptance under priority review

Price and Volume Movers

Cara Therapeutics, Inc. (Nasdaq:CARA) shares closed up 20% to $21.57 on news that it met the primary endpoint in its Phase 3 KALM-1 pivotal trial of Korsuva (CR845/difelikefalin) injection in hemodialysis patients with moderate-to-severe chronic kidney disease-associated pruritus (CKD-aP). The proportion of patients on Korsuva achieving a three-point or greater improvement from baseline using the Worst Itching Intensity Numeric Rating Scale (WI-NRS) score at week 12 was 51% vs. 28% for patients on placebo (p= 0.000019). Overall, the incidence of adverse events were similar across both Korsuva and placebo groups. Data from its second trial are due 2H 2019.

Amarin Corporation plc (NASDAQ:AMRN) announced that the FDA has awarded priority review for its supplemental new drug application (sNDA) for Vascepa, with a PDUFA date of September 28, 2019. Investors had anticipated a standard ten-month review instead of six months, reacted positively to the news, sending shares up 12% to $18.99. The company also noted the following in regard to an advisory committee:

the FDA did not indicate whether it plans to hold an advisory committee (AdCom) meeting to discuss this application. Amarin previously expressed that it believes an AdCom meeting organized by the FDA in conjunction with its review of the expanded label for Vascepa is likely. It is not uncommon for clarification on this topic to be provided by the FDA later in its review process.”

Fibrocell Science, Inc. (Nasdaq: FCSC) announced that the FDA has granted Regenerative Medicine Advanced Therapy (RMAT) designation to FCX-007, its gene therapy candidate for the treatment of recessive dystrophic epidermolysis bullosa (RDEB). RMAT offers eligibility for accelerated development and review of the designated product. Shares closed up 11% to $2.07.

Amicus Therapeutics (Nasdaq: FOLD) shares are trading down 7% after hours to $11.35 on news it has commenced a $150m public offering of its common stock.

Aptose Biosciences Inc. (NASDAQ: APTO), which saw its shares close up 12%, are now trading down 17% to $1.87 late in the after-hours session following news it has commenced an underwritten public offering.

Myovant Sciences (NYSE: MYOV) also announced a public offering of $100m of its common shares. Shares are trading down 8% after hours.

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Major price movers (stocks priced > $1.00, volume > 50k):

Refer to the BioPharmCatalyst Biotech Stocks page for Top 10 Gainers/Losers/Unusual Volume

ADVANCERS:

Opiant Pharmaceuticals, Inc. (OPNT): $11.50: +12%.

Syndax Pharmaceuticals, Inc. (SNDX): $7.99; +10%.

Chiasma, Inc. (CHMA): $7.48; +10%.

Sesen Bio, Inc. (SESN): $2.46; +8%.

Nymox Pharmaceutical Corporation (NYMX): $1.82; +8%.

DECLINERS:

Immunic, Inc. (IMUX): $9.38; -12%.

Selecta Biosciences, Inc. (SELB): $2.04; -11%.

Outlook Therapeutics, Inc. (OTLK): $2.5; -11%.

Sorrento Therapeutics, Inc. (SRNE): $2.95; -10%.

Zafgen, Inc. (ZFGN): $2.39; -10%.

Pipeline updates below:

Pipeline Database Updates

Drug Stage Catalyst Market Cap

AKTX – Akari Therapeutics Plc
Coversin
Atopic keratoconjunctivitis

Phase 1/2 Phase 1/2 Part B data due 4Q 2019.
$33 million

AMRN – Amarin Corporation plc
Vascepa
High Triglycerides With Mixed Dyslipidemia

PDUFA priority review PDUFA date under priority review September 28, 2019.
$6 billion

BFRA – Biofrontera AG
Ameluz
Actinic keratosis

sNDA Filing Phase 3 data met primary endpoint - March 20, 2019. sNDA filing due 3Q 2019.
$368.9 million

CANF – Can-Fite Biopharma Ltd Sponsored ADR (Israel)
Piclidenoson (CF101)
Rheumatoid arthritis

Phase 3 Phase 3 continues to enrol as of May 29, 2019.
$9.4 million

CANF – Can-Fite Biopharma Ltd Sponsored ADR (Israel)
Namodenoson (CF102)
Non-alcoholic steatohepatitis (NASH)

Phase 2 Phase 2 data due 2H 2019.
$9.4 million

CARA – Cara Therapeutics Inc.
KORSUVA (CR845/difelikefalin) injection - KALM-1
Chronic kidney disease (CKD) on hemodialysis (HD) with Moderate-to Severe Pruritus

Phase 3 Phase 3 data met primary endpoint - May 29, 2019.
$784.3 million

GLPG – Galapagos NV
Filgotinib - FINCH 1
Rheumatoid arthritis (RA)

Phase 3 Phase 3 data released March 28, 2019 - primary endpoint met.
$6.9 billion

GLPG – Galapagos NV
Filgotinib - FINCH 3
Rheumatoid arthritis (RA)

Phase 3 Phase 3 data released March 28, 2019 - primary endpoint met.
$6.9 billion

RMTI – Rockwell Medical Inc.
IV Triferic
Anemia

NDA Filing NDA filing announced May 29, 2019.
$199.2 million

TRPX – Therapix Biosciences Ltd.
THX-110
Obstructive Sleep Apnea (OSA)

Phase 2a Phase 2a top-line data due 2H 2019.
$8.8 million

TYME – Tyme Technologies Inc.
SM-88
Prostate cancer

Phase 2 Phase 2 final data to be reported late 2019 or early 2020.
$115.3 million

TYME – Tyme Technologies Inc.
SM-88
Pancreatic cancer

Phase 2 Phase 2 further data due 3Q 2019.
$115.3 million

ZYNE – Zynerba Pharmaceuticals Inc.
ZYN002
22q11.2 Deletion Syndrome

Phase 2 Phase 2 trial initiation announced May 29, 2019 with data due 1H 2020.
$264.5 million