IPXL Mulls Merger, QURE Attracts Attention, No Let Up In ICPT Ocaliva Concerns

(RTTNews) - The following are some of the biotech stocks that made their way onto the Day's Gainers & Losers' list of September 21, 2017.

GAINERS

1. uniQure N.V. (QURE)

Gained 12.01% to close Thursday's (Sep.21) trading at $9.51.

News: No news

Near-term catalysts:

-- An investigational new drug application for AMT-130 in Huntington's disease is expected to be filed in 2018. -- The Company is planning to meet with U.S. and European regulators in the early fall to further discuss its plans to begin a pivotal program of AMT-060 in hemophilia B in 2018.

2. Impax Laboratories Inc. (IPXL)

Gained 10.44% to close Thursday's trading at $23.80.

News: The Company is mulling merger with Amneal Pharmaceuticals LLC, a privately held generic pharmaceutical manufacturer, reports the Wall Street Journal.

3. Intellia Therapeutics Inc. (NTLA)

Intellia is a genome editing company, focused on the development of proprietary, potentially curative therapeutics using the CRISPR/Cas9 system.

Gained 8.33% to close Thursday's trading at $25.75.

News: No news

4. Supernus Pharmaceuticals Inc. (SUPN)

Gained 7.21% to close Thursday's trading at $39.40.

News: No news

Recent event:

-- On September 18, 2017, the Company announced that it has decided to eliminate the lower dose of 18 mg of SPN-810 in its first phase III clinical trial of SPN-810 for impulsive aggression in patients aged 6 to 12 years who have attention deficit hyperactivity disorder.

Moving forward, all patients will be randomized to either the 36 mg dose arm or placebo, and enrollment is expected to continue through mid-2018.

5. Collegium Pharmaceutical Inc. (COLL)

Gained 6.57% to close Thursday's trading at $10.06.

News: No news

Recent event:

-- On August 9, 2017, the Company reported financial results for the second quarter of 2017.

Net loss for the quarter ended June 30, 2017 shrunk to $21.1 million or $0.72 per share on net product revenue of $3.6 million. This compared with a net loss of $24.5 million or $1.05 per share on nil product revenue for the quarter ended June 30, 2016.

LOSERS

1. Intercept Pharmaceuticals Inc. (ICPT)

Lost 24.89% to close Thursday's trading at $73.70.

News: The FDA has warned about serious liver injury with Ocaliva, the Company's drug for rare chronic liver disease due to incorrect dosing.

Ocaliva was granted accelerated approval by the FDA last May for the treatment of primary biliary cholangitis, and was commercially launched in the United States in June 2016.

According to the FDA, there have been 19 cases of death associated with Ocaliva. For seven of these patients, the cause of death was reported to be worsening of PBC disease, and they had received Ocaliva 5 mg daily, instead of a dose no greater than 10 mg twice weekly as recommended in the drug's label.

On September 12, 2017, the Company announced that it issued a letter warning physicians against overdosing patients with Ocaliva.

The letter dated September 8, 2017, sent to health care providers, cautions that liver injury, liver decompensation, liver failure, and death have been reported in patients with moderate to severe hepatic impairment when Ocaliva was dosed more frequently than recommended in labeling for such patients.

Intercept shares have lost 35% of their value since the Ocaliva issue came into the limelight on September 12, 2017.

2. Juno Therapeutics Inc. (JUNO)

Lost 7.94% to close Thursday's trading at $41.62.

News: The Company launched a follow-on public offering of $225 million of shares of its common stock.

In addition, Juno expects to grant the underwriters a 30-day option to purchase up to an additional $33.75 million of shares of its common stock.

3. Acceleron Pharma Inc. (XLRN)

Lost 7.10% to close Thursday's trading at $35.05.

News: The Company has priced an underwritten public offering of 5.40 million shares of common stock at a price of $37.00 each. The gross proceeds from the offering are estimated to be about $200 million.

In connection with this offering, Acceleron has granted the underwriters a 30-day option to purchase up to an additional 810,810 shares of common stock.

Pipeline:

The Company's lead product candidate is Luspatercept, which is being developed for Myelodysplastic Syndromes (MDS) Beta-Thalassemia, and Myelofibrosis.

A phase III study of Luspatercept in patients with anemia due to lower-risk MDS with ring sideroblasts who require regular RBC transfusions, dubbed MEDALIST, and a phase III study of Luspatercept in patients with anemia due to beta-thalassemia who require regular RBC transfusions, dubbed BELIEVE, are underway.

Near-term catalysts:

-- The top-line results from the MEDALIST and BELIEVE phase III trials are expected in mid-2018.

4. Tocagen Inc. (TOCA)

Lost 6.32% to close Thursday's trading at $11.86.

News: No news

Recent event:

On April 19, 2017, the Company announced the closing of its IPO and exercise of underwriters' option to purchase additional shares, which were sold at a price of $10.00 each.

Pipeline:

Tocagen's lead product candidate is a combination of an investigational biologic, Toca 511, and an investigational small molecule, Toca FC, designed to be used together.

Near-term catalysts:

-- A phase 2/3 clinical trial of Toca 511 & Toca FC, for the treatment of patients with recurrent brain cancer, dubbed Toca 5, is underway. Top-line results from the phase 2 portion of the Toca 5 study are anticipated in the first half of 2018. -- Initiation of Phase 1b trial, called Toca 7, evaluating Toca 511 & Toca FC in patients with newly diagnosed high grade glioma expected in the first half of 2018.