APLS Sets Out Its Vision, APEN Gains Weight, Packed Year Ahead For DRNA

(RTTNews) - The following are some of today's top gainers in the pharma/biotech sector.

1. Apellis Pharmaceuticals Inc. (APLS)

Gained 16.19% to close Thursday's (Dec.21) trading at $16.58.

News: The Company announced that it has finalized the trial design for its planned phase III program evaluating its lead drug candidate APL-2 for the treatment of patients with geographic atrophy (GA) associated with age-related macular degeneration (AMD).

The phase III program will consist of 2 identical studies, and is planned to begin in the second half of 2018.

Recent event:

The Company debuted on the NASDAQ Global Select Market on November 9, 2017, offering its shares at a price of $14.00 each.

Clinical Trials:

APL-2 has been successfully studied in a phase II clinical trial (FILLY) in patients with geographic atrophy associated with age-related macular degeneration. APL-2 is also under two phase Ib trials in paroxysmal nocturnal hemoglobinuria - one as monotherapy and the other as add-on to Soliris.

2. Fate Therapeutics Inc. (FATE)

Gained 12.42% to close Thursday's trading at $5.07.

News: No news

Recent event:

-- On December 15, 2017, the Company closed an underwritten public offering of 10.95 million shares of its common stock and full exercise of the underwriters' option to purchase additional shares at a public offering price of $4.20 per share. Net proceeds from this offering were approximately $42.9 million.

Clinical Trials:

-- The phase II efficacy stage of PROTECT study was initiated earlier this month.

PROTECT is a phase 1/2 clinical trial of ProTmune in adult subjects with hematologic malignancies undergoing matched unrelated donor mobilized peripheral blood HCT. The PROTECT study is intended to evaluate ProTmune to prevent acute graft-versus-host disease (GvHD).

All subjects receiving ProTmune in the PROTECT Phase 1 stage are being followed for a period of two years following HCT. As of a November 29, 2017 data cut-off, all subjects remained relapse-free, and there were no events of graft failure and no serious adverse events related to ProTmune reported by investigators.

-- A phase I study of FATE-NK100, the Company's first-in-class adaptive memory natural killer (NK) cell product candidate for advanced acute myelogenous leukemia, known as VOYAGE, is underway. -- A phase I trial of FATE-NK100 for the treatment of women with ovarian cancer resistant to, or recurrent on, platinum-based treatment, dubbed APOLLO, was initiated as recently as this month.

3. MannKind Corporation (MNKD)

Gained 10.53% to close Thursday's trading at $2.52, reversing yesterday's loss of 11%.

Looks like the positive label update on inhaled insulin Afrezza, the marketed product of MannKind, in late September, and Afrezza T.V. commercial market expansion have not done much to accelerate its growth, according to reports.

4. Apollo Endosurgery Inc. (APEN)

Gained 9.13% to close Thursday's trading at $5.02.

The Company deals with less invasive medical devices for bariatric and gastrointestinal procedures.

Recent event:

-- On November 27, 2017, the Company received FDA Clearance for OverStitch Sx endoscopic suturing system. It will be introduced in the US and Europe in the first half of 2018.

Commenting on the news, Christopher Gostout, MD, Chief Medical Officer at Apollo Endosurgery said, "The launch of OverStitch Sx will allow suturing technology to be available to nearly any physician with access to an endoscope. Broader physician access to endoscopic suturing allows for greater patient access to a variety of minimally invasive endoscopic treatments."

5. Acceleron Pharma Inc. (XLRN)

Gained 9.00% to close Thursday's trading at $41.68.

News: No news

Clinical Trials & Near-term Catalysts:

-- A phase III trial of Luspatercept in patients with myelodysplastic syndromes, dubbed MEDALIST, and a phase III trial of Luspatercept in patients with beta-thalassemia, known as BELIEVE are underway. -- The Part 1 of a phase II trial of ACE-083 in patients with facioscapulohumeral muscular dystrophy (FSHD), one of the most prevalent forms of muscular dystrophy in adults is ongoing. -- The Part 1 of a phase II trial of ACE-083 in patients with Charcot-Marie-Tooth (CMT) disease, one of the most common inherited neurological diseases leading to focal muscle weakness, is also ongoing.

-- Report top-line results from MEDALIST and BELIEVE phase III trials in mid-2018. -- Initiate a phase III trial of Luspatercept in first-line, lower-risk MDS, dubbed COMMANDS, in the first half of 2018. -- Report FSHD phase II results for cohort 1 in Part 1 in January 2018. -- Report FSHD phase II results for all dose-escalation cohorts in Part 1 in 2018. -- Report CMT phase II results from all dose-escalation cohorts in Part 1 by year-end 2018. -- Initiate a phase II trial of Sotatercept in pulmonary arterial hypertension in the first half of 2018.

6. Dicerna Pharmaceuticals Inc. (DRNA)

Gained 8.15% to close Thursday's trading at $8.63. This is the second straight day of gain for the stock.

News: No news

The Company is involved in developing subcutaneously delivered ribonucleic acid interference (RNAi)-based pharmaceuticals to treat chronic liver diseases, cardiovascular diseases and viral infectious diseases.

Recent event:

-- On December 14, 2017, the Company priced its follow-on public offering of common stock at $7.00 per share.

Near-term catalysts:

-- Upon approval by the MHRA in the United Kingdom, a phase I study of DCR-PHXC for primary hyperoxaluria is expected to be conducted in the first quarter of 2018. -- File an IND application in the U.S. and/or CTA in Europe for a second candidate for an undisclosed rare disease involving the liver in the second quarter of 2018. -- File an IND application in the U.S. or CTA in Europe for DCR-HBVS program, which targets HBV directly, at approximately the end of 2018.