The following are some of the biotech stocks that made their way onto the Day's Gainers & Losers' list of August 22, 2017.
GAINERS
1. Ovid Therapeutics Inc. (OVID)
Gained 24.43% to close Tuesday's (Aug.22) trading at $8.25.
News: The Company has advanced its second drug candidate into phase II testing.
The Company has dosed the first patient in its phase 1b/2a clinical trial of TAK-935/OV935 in adults with rare epilepsies. Data from this trial are expected next year.
Ovid has a global collaboration with Takeda Pharmaceutical Company Ltd., focused on the clinical development and commercialization of TAK-935/OV935 that was formed in January of this year.
The Company made its debut on the NASDAQ Global Select Market on May 5, 2017, offering its shares at a price of $15.00 each.
Pipeline:
The Company's pipeline also includes OV101, a potential treatment for Angelman syndrome and Fragile X syndrome. A phase II clinical trial of OV101 in adults with Angelman syndrome, dubbed STARS, and a phase I trial of OV101 in adolescents diagnosed with Angelman syndrome or Fragile X syndrome are underway.
Near-term catalyst:
-- The top line data from the phase I trial evaluating OV101 in adolescents diagnosed with Angelman syndrome or Fragile X syndrome are expected to be available in the second half of 2017.
2. Akcea Therapeutics Inc. (AKCA)
Gained 14.92% to close Tuesday's trading at $17.33.
News: No news
Pipeline:
The most advanced product candidate in the pipeline is Volanesorsen, co-developed by Ionis Pharmaceuticals Inc. (IONS). The Company reported positive results from a phase III study in patients with familial chylomicronemia syndrome in March of this year.
Volanesorsen is also under a phase III trial for the treatment of familial partial lipodystrophy.
AKCEA-APO(a)-LRx, AKCEA-ANGPTL3-LRx and AKCEA-APOCIII-LRx are the other three drug candidates in the pipeline, all of which were discovered and are being co-developed by Ionis.
Recent events:
-- The Company announced the closing of its IPO on July 19, 2017. Akcea shares made their debut on the NASDAQ Global Select Market on July 14, 2017, at an opening price of $8.13 each.
The gross proceeds from the offering were $143.8 million. In addition, Novartis Pharma AG purchased $50.0 million of Akcea's common stock in a separate private placement concurrent with the completion of Akcea's initial public offering at a price per share equal to the initial public offering price of $8.00 per share.
-- On July 27, 2017, the Company submitted a marketing authorization application for Volanesorsen for the treatment of patients with familial chylomicronemia syndrome to the European Medicines Agency.
Near-term catalysts:
--File for regulatory approval of Volanesorsen in the U.S. and Canada in September.
--Present results from the Phase 1/2 study evaluating AKCEA-APOCIII-LRx in healthy volunteers and patients with elevated triglycerides in the second half of 2017.
--Initiate Phase 2 studies with AKCEA-ANGPTL3-LRx in the second half of 2017, including a study in patients with non-alcoholic fatty liver disease (NAFLD) and metabolic complications and studies in patients with rare hyperlipidemias.
--Initiate a Phase 2b dose-ranging study evaluating AKCEA-APOCIII-LRx in patients with hypertriglyceridemia and established cardiovascular disease in the second half of 2017.
3. Blueprint Medicines Corp. (BPMC)
Gained 12.61% to close Tuesday's trading at $47.77.
News: The Company will be presenting updated data from its ongoing phase 1 clinical trial evaluating BLU-554 in patients with advanced hepatocellular carcinoma on September 10, 2017 in Madrid, Spain and at the 11th International Liver Cancer Association (ILCA) Annual Conference on September 17, 2017 in Seoul, South Korea.
Pipeline:
Apart from BLU-554, the Company's clinical pipeline includes BLU-285, being evaluated in the indications of Gastrointestinal Stromal Tumors and Systemic Mastocytosis, and BLU-667 for patients with non-small cell lung cancer, medullary thyroid cancer and other advanced solid tumors that harbor a RET alteration, all of which are under phase I testing.
Near-term catalysts:
-- Initiate a global, pivotal Phase 3 clinical trial of BLU-285 in third line patients with KIT-driven Gastrointestinal Stromal Tumors in the first half of 2018.
-- Provide updated data from phase I clinical trial of BLU-285 in patients with advanced systemic mastocytosis by the end of 2017.
4. Sienna Biopharmaceuticals Inc. (SNNA)
Gained 9.76% to close Tuesday's trading at $22.50.
News: No news
Recent event:
The Company made its debut on the NASDAQ on July 27, 2017 at an opening price of $19.05, a little higher than the offering price of $15 per share.
Pipeline:
The Company's lead candidate is SNA-120 for the treatment of pruritus, or itch, associated with psoriasis, as well as for psoriasis itself; SNA-125 for the treatment of atopic dermatitis, psoriasis and pruritus, and SNA-001 for both acne vulgaris and the reduction of unwanted light-pigmented hair.
Near-term catalysts:
-- Initiate a phase IIb study of SNA-120 for the treatment of pruritus and psoriasis by the end of 2017.
-- Initiate two phase I/II Proof of Concept studies of SNA-125 atopic dermatitis, psoriasis and pruritus in the first half of 2018.
-- Report top-line data from pivotal trials of SNA-001 for both acne vulgaris and the reduction of unwanted light-pigmented hair in the second half of 2018.
5. Altimmune Inc. (ALT)
Gained 7.89% to close Tuesday's trading at $2.17.
News: The Company announced that it has closed its offering of roughly 15,656 shares of Series B convertible preferred stock and warrants.
The Company received approximately $13.0 million in net proceeds from the offering.
The Company has also submitted an Investigational New Drug application to the FDA in preparation for phase II trial of its recombinant flu vaccine for NasoVAX. Data from the trial is expected sometime in the first quarter of 2018.
6. Five Prime Therapeutics Inc. (FPRX)
Gained 7.43% to close Tuesday's trading at $30.09.
News: No news
Near-term catalysts:
-- The Company is advancing its large Phase 1a/1b immuno-oncology trial studying its Cabiralizumab with Bristol-Myers Squibb's (BMY) OPDIVO in multiple tumor settings. Enrollment in some of the cohorts has been completed. Enrollment in all the cohorts of the study is expected to be completed by the end of the year.
-- Initial clinical trial data from the Phase 1a/1b immuno-oncology is anticipated to be presented at the SITC annual meeting in November.
7. PTC Therapeutics Inc. (PTCT)
Gained 7.39% to close Tuesday's trading at $19.03.
News: No news
Near-term catalysts:
-- The NDA for Translarna, a treatment for nonsense mutation Duchenne muscular dystrophy, is under FDA review with a decision due by October 24, 2017.
Before the regulatory agency makes a final decision, Translarna is slated to be reviewed by a panel of outside experts on September 28, 2017.
Translarna is approved within the European Union (EU) Member States, Iceland, Liechtenstein, Norway, Israel and South Korea. Ever since its launch in December 2014, Translarna has had substantial year-over-year net sales growth.
To know more about PTCT, please visit our Company Spotlight column.
8. Insmed Inc. (INSM)
Gained 7.10% to close Tuesday's trading at $12.83.
News: No news
Near-term catalysts:
-- Report data from a phase III study of Amikacin liposome inhalation suspension in treatment of refractory Nontuberculous Mycobacterial lung disease caused by Mycobacterium avium complex, dubbed INS-212 CONVERT, in September/October.
-- Initiate enrollment of phase 2 dose-ranging study of INS1007 in non-cystic fibrosis bronchiectasis in the second half of 2017.
9. Versartis Inc. (VSAR)
Gained 7.01% to close Tuesday's trading at $17.55.
News: No news
Near-term catalysts:
-- Report topline data from phase III trial of Somavaratan in pediatric growth hormone deficiency, dubbed VELOCITY, around the end of September.
-- Complete enrollment of phase III trial of Somavaratan in pre-pubertal Japanese children with growth hormone deficiency, dubbed J14VR5, in the second half of this year.
LOSERS
1. Cardiome Pharma Corp. (CRME)
Lost 18.85% to close Tuesday's trading at $2.11. This is the second straight day of loss for the stock.
Recent event:
On August 21, 2017, Cardiome announced that the FDA is not satisfied with its proposed data package to support the resubmission of its Brinavess New Drug Application, sending the stock down more than 32% that day.
Brinavess is used to rapidly restore normal heart rhythm in adult patients (aged 18 years or over) who have recently started having atrial fibrillation. The drug was approved in Europe in 2010 but is not approved in the U.S. yet. It was turned down by the U.S. regulatory agency in 2008.
2. ARCA biopharma Inc. (ABIO)
Lost 8.70% to close Tuesday's trading at $1.05.
News: No news
Near-term catalyst:
-- The Company expects to report top-line data from GENETIC-AF trial late in the first quarter of 2018.
GENETIC-AF is a phase 2B/3 double-blind, clinical superiority trial comparing the safety and efficacy of ARCA bio's investigational drug Gencaro to an approved drug TOPROL-XL for the treatment and prevention of recurrent atrial fibrillation or flutter (AF/AFL) in heart failure patients with reduced left ventricular ejection fraction (HFrEF).
3. BioLine Rx Ltd (BLRX)
Lost 6.80% to close Tuesday's trading at $1.02.
News: The Company has sought regulatory approval to commence a phase III registrational trial of BL-8040 for the mobilization of hematopoietic stem cells for autologous transplantation in patients with multiple myeloma.
The trial, named GENESIS, is expected to commence by the end of 2017, following receipt of regulatory approvals.
Other anticipated events:
-- Three phase 1b/2 trials for BL-8040 in combination with atezolizumab, Genentech's anti-PDL1 cancer immunotherapy agent, in pancreatic, gastric and non-small cell lung cancers are expected to commence in the second half of 2017.
-- A Phase 1b/2 trial for BL-8040 in combination with Genentech's atezolizumab in acute myeloid leukemia is expected to commence in the second half of this year.
-- Partial results from immuno-oncology Phase 2a study in pancreatic cancer for BL-8040 in combination with Merck's KEYTRUDA are expected in H2 2017; top line results expected in H2 2018.
4. Tocagen Inc. (TOCA)
Lost 5.90% to close Tuesday's trading at $9.94.
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.
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