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ALNY In Top Form, LENS Catches Investors' Eyes, PRQR, AXON Inch Closer To D-Day

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The following are some of the biotech stocks that made their way onto the Day's Gainers & Losers' list of September 20, 2017.

GAINERS

1. Alnylam Pharmaceuticals Inc. (ALNY)

Gained 51.71% to close Wednesday's (Sep.20) trading at $113.84.

News: The Company's phase III study of Patisiran, an investigational RNAi therapeutic being developed for patients with hereditary ATTR amyloidosis with polyneuropathy, dubbed APOLLO, met its primary efficacy endpoint and all secondary endpoints.

Alnylam expects to file its first New Drug Application in late 2017 and first Marketing Authorisation Application shortly thereafter.

Alnylam 's partner Sanofi Genzyme is currently preparing for regulatory filings for Patisiran in Japan, Brazil and other countries, to begin in the first half of 2018. If approved, Alnylam will commercialize Patisiran in the U.S., Canada and Western Europe, with Sanofi Genzyme commercializing the product in the rest of the world.

2. Presbia PLC (LENS)

Gained 22.70% to close Wednesday's trading at $4.00.

News: No news

Pipeline:

The Company's lead product candidate is Presbia Flexivue Microlens, a revolutionary optical lens implant for treating presbyopia, the age-related loss of near vision.

Near-term catalyst:

The Company expects to seek approval of Presbia Flexivue Microlens in the fourth quarter of this year.

3. Arbutus Biopharma Corporation (ABUS)

Gained 20.72% to close Wednesday's trading at $6.70.

News: The Company's lipid nanoparticle (LNP) licensee Alnylam Pharma announced positive data from APOLLO trial.

Patisiran is enabled by Arbutus' lipid nanoparticle (LNP) technology. Per the terms of the LNP license agreement for Patisiran, Arbutus will be owed single digit royalties on sales of Patisiran.

To know more about ABUS, please visit our Company Spotlight column.

4. Dicerna Pharmaceuticals Inc. (DRNA)

Gained 18.21% to close Wednesday's trading at $4.48.

News: No news

Near-term catalysts:

-- The Company plans to file a clinical trial application (CTA) in Europe for DCR-PHXC for primary hyperoxaluria in late 2017 and commence human clinical trials 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.

5. SCYNEXIS Inc. (SCYX)

Gained 11.93% to close Wednesday's trading at $2.44.

News: No news

Near-term catalysts:

-- Initiation of a phase II study of IV Formulation of SCY-078 for the treatment of patients with invasive Candida infections in 2018.

On March 2, 2017, the FDA asked the Company to hold the initiation of any new clinical studies with the intravenous (IV) formulation of SCY-078 until it completed a review of all available pre-clinical and clinical data of the IV formulation of SCY-078.

The clinical hold decision was issued by the FDA following a review of three mild-to-moderate thrombotic events in healthy volunteers receiving the IV formulation of SCY-078 at the highest doses and highest concentrations in a Phase 1 study.

6. Versartis Inc. (VSAR)

Gained 11.48% to close Wednesday's trading at $21.85.

News: No news

Near-term catalyst:

-- The topline data from the Company's phase III trial of Somavaratan in pediatric growth hormone deficiency patients are expected around the end of September.

The trial, dubbed VELOCITY, has enrolled 137 patients, and is designed to demonstrate non-inferiority of Somavaratan compared to daily recombinant human growth hormone (rhGH). The height velocity at 12 months is the primary efficacy measure of the study.

7. ProQR Therapeutics N.V. (PRQR)

Gained 10% to close Wednesday's trading at $5.50.

News: No news

Near-term catalyst:

-- The top-line data from phase 1b clinical trial of QR-010 in cystic fibrosis patients with F508del mutation post-market close on September 25, 2017.

The study consisted of 8 cohorts, each of which had 8 patients, making up the total to 64 patients. In each cohort, 6 patients received QR-010 and 2 patients received placebo.

The study primarily assess safety, tolerability and pharmacokinetics of QR-010, and a number of exploratory efficacy endpoints including sweat chloride, weight gain, change in CFQ-R Respiratory Symptom Score and FEV1. The study is not powered for statistical significance on the exploratory efficacy endpoints.

8. Ophthotech Corp. (OPHT)

Gained 9.84% to close Wednesday's trading at $2.79.

News: The Company announced the initiation of an open-label phase IIa clinical trial of product candidate Zimura in patients with wet age-related macular degeneration (AMD).

Near-term catalysts:

-- Clinical trials of Zimura in Stargardt Disease and Idiopathic Polypoidal Choroidal Vasculopathy are expected to be initiated before the end of this year.

9. Mersana Therapeutics Inc. (MRSN)

Gained 9.39% to close Wednesday's trading at $16.78.

News: No news

Recent event:

The Company went public on the NASDAQ Global Select Market on June 28, 2017 at an offering price of 15.00 per share.

Near-term catalysts:

-- Interim results from phase I trial of lead oncology drug candidate XMT-1522 in patients with advanced tumors expressing HER2, including breast cancer, non-small-cell-lung-cancer (NSCLC) and gastric cancer are expected around the end of this year.
-- File the second IND shortly and begin clinical studies in early 2018 for XMT-1536, a potential first-in-class Dolaflexin ADC targeting NaPi2b-expressing tumors.

10. Axovant Sciences Ltd. (AXON)

Gained 7.95% to close Wednesday's trading at $26.20.

News: No news

Near-term catalyst:

-- Results from phase III study of Intepirdine in subjects with mild-to-moderate Alzheimer's disease on a stable background of donepezil therapy, dubbed MINDSET, are expected to be reported in late September 2017.

LOSERS

1. Iovance Biotherapeutics Inc. (IOVA)

Lost 16.37% to close Wednesday's trading at $7.15.

News: The Company has offered to sell 7.69 million shares of its common stock at a public offering price of $6.50 per share.

The gross proceeds from the offering are expected to be $50 million. The underwriters have a 30-day option to purchase up to 1.15 million additional shares of common stock at the public offering price, less the underwriting discounts and commissions. The offering is expected to close on or about September 25, 2017, subject to customary closing conditions.

2. Nabriva Therapeutics plc (NBRV)

Lost 10.12% to close Wednesday's trading at $8.26.

News: No news

Recent events:

-- On September 18, 2017, the Company reported positive top-line results from its phase III trial of Lefamulin (IV/oral) in patients with moderate to severe community acquired bacterial pneumonia, dubbed LEAP-1.
-- On September 19, 2017, the Company offered to sell 9.41 million ordinary shares at a public offering price of $8.50 each. The underwriters have an option for a period of 30 days to purchase up to an additional 1.41 million ordinary shares at the public offering price, less the underwriting discounts and commissions. The offering is expected to close on or about September 22, 2017.

3. Vascular Biogenics Ltd. (VBLT)

Lost 9.03% to close Wednesday's trading at $6.55.

New: No news

Near-term catalysts:

-- A final DSMC review of the Company's phase III study of VB-111 in patients with recurrent glioblastoma (rGBM), dubbed GLOBE, is expected in late September.
-- Top-line data from the GLOBE trial anticipated in Q1 2018.
-- Initiation of a phase III study of VB-111 in ovarian cancer by year-end 2017.
-- Initiation of exploratory clinical study of VB-111 in combination with a checkpoint inhibitor in lung cancer in Q1 2018.

by RTT Staff Writer

For comments and feedback: editorial@rttnews.com

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