IPXL Reveals Oriental Stakeholder, LENS Has A Vision, STDY Is Very Shaky

(RTTNews) - The following are some of the biotech stocks that made their way onto the Day's Gainers & Losers' list of August 31, 2017.

GAINERS

1. Impax Laboratories Inc. (IPXL)

Gained 20.61% to close Thursday's (Aug. 31) trading at $21.65.

News: A SEC filing shows that China-based Shanghai Fosun Pharmaceutical (Group) Co. Ltd. owns 5.19% stake in the Company.

2. NewLink Genetics Corp. (NLNK)

Gained 14.37% to close Thursday trading at $8.12.

News: No news

Upcoming events:

The company will be participating in:

-- Baird Global Healthcare Conference on September 7 at 1:25pm ET in New York City. -- Cantor Fitzgerald Global Healthcare Conference on September 25 at 4:10pm ET in New York City.

Near-term catalysts:

-- Updated data from phase II trial of Indoximod plus Gemcitabine/Nab-paclitaxel for patients with metastatic pancreatic cancer to be presented at an oncology meeting in late 2017 or early 2018. -- Initiation of a pivotal trial of Indoximod in combination with PD-1 checkpoint blockade for patients with advanced melanoma, with the goal of full enrollment by end of 2018.

3. Presbia PLC (LENS)

Gained 11.99% to close Thursday trading at $2.99.

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.

4. Adaptimmune Therapeutics plc (ADAP)

Gained 12.36% to close Thursday trading at $7.00.

The Company is focused on developing enhanced T-cell therapies that work with the immune system to improve detection and targeting of cancer cells, resulting in destruction of those cancer cells.

News: No news

Pipeline:

The advanced drug candidate in the pipeline is NY-ESO TCR therapeutic candidate, under multiple Phase 1/2 clinical trials in patients with solid tumors and hematological malignancies including synovial sarcoma, multiple myeloma, melanoma, NSCLC and ovarian cancer. NY-ESO TCR therapeutic candidate in being developed in partnership with GSK.

The Company's wholly-owned product candidates are MAGE-A10 T-cell therapy, under phase I/II studies in lung cancer, bladder, melanoma and head and neck cancers; AFP TCR, under phase I study in patients with hepatocellular carcinoma, and MAGE-A4 TCR, under phase I trial in multiple solid tumors.

5. Cara Therapeutics Inc. (CARA)

Gained 11.43% to close Thursday trading at $14.33.

News: The Company will be presenting data from its phase IIb chronic osteoarthritis pain study of Oral CR845 in Medical meetings in September.

The data from the phase IIb chronic osteoarthritis pain study of Oral CR845 was reported on June 29, 2017 after market close.

The study tested three tablet strengths of CR845, 1.0 mg, 2.5 mg and 5.0 mg, dosed twice a day (BID) over an eight-week treatment period in 476 patients with osteoarthritis of the hip or knee experiencing moderate-to-severe pain. Patients with osteoarthritis of the hip maintained on the 5.0 mg dose to the end of the eight-week treatment period exhibited a statistically significant 39 percent reduction in mean joint pain score while those on 1.0 mg and 2.5 mg tablet strengths did not exhibit significant reductions in mean joint pain scores compared to placebo. The news sent the stock plunging nearly 40% on June 30, 2017.

Near-term catalysts:

-- End of Phase 2 meeting with the FDA in the third quarter of 2017 to finalize pivotal program for I.V CR845 for the treatment of chronic kidney disease-associated pruritus (CKD-aP) in hemodialysis patients; initiation of a Phase 3 pivotal trial expected in the fourth quarter of 2017. -- Initiation of a Phase 1 trial of Oral CR845 in CKD-aP non-hemodialysis patients expected in fourth quarter of 2017. -- Investigational New Drug application submission for Oral CR845 in patients with chronic liver disease-associated pruritus expected in the fourth quarter of 2017. -- Completion of enrollment for CLIN-3001, the Company's ongoing adaptive pivotal Phase 3 trial of I.V. CR845 for the treatment of acute postoperative pain, expected in the fourth quarter of 2017.

6. bluebird bio Inc. (BLUE)

Gained 10.98% to close Thursday trading at $124.85.

bluebird is also a CAR-T player.

CAR T-cells are a type of immunotherapy in which a patient's T cells are extracted from their blood and modified in a lab to produce receptors on their surface called chimeric antigen receptors, or CARs. These special receptors allow the T cells to recognize and attach to a specific protein, or antigen, on tumor cells.

The engineered T cells are infused back into the patient where they multiply and with guidance from their engineered receptor, recognize and kill cancer cells. (Source: National cancer Institute).

On August 28, 2017, Gilead Sciences announced that it is going to acquire Kite Pharma, a clinical-stage developer of chimeric antigen receptor (CAR) T-cell therapies, for $180.00 per share in cash, equivalent to a total value of approximately $11.9 billion.

On August 31, 2017, the FDA approved Novartis AG'S (NVS) Kymriah, a CAR-T therapy for certain pediatric and young adult patients with a form of acute lymphoblastic leukemia.

The stocks of the CAR-T players have been boosted by the encouraging news since then.

News: BMO Capital analyst Matthew Luchini has raised his price target on bluebird bio stock to $134 from $108.

7. Bellicum Pharmaceuticals Inc. (BLCM)

Gained 10.40% to close Thursday trading at $10.83.

The Company is developing novel stem cell transplant, TCR and CAR T cell therapies.

Pipeline:

The company's lead product candidate is BPX-501, which is being evaluated in multiple phase 1/2 clinical trials in adults and pediatric patients with leukemias, lymphomas, and genetic blood diseases in the U.S. and Europe.

Near-term catalysts:

-- Enrollment in a Phase 1/2 clinical trial of BPX-501 in children with leukemias, lymphomas, or orphan inherited blood disorders, for which HSCT (hematopoietic stem cell transplantation) is curative, dubbed EU BP-004, remains on track for completion by the end of 2017. -- An observational trial in pediatric patients receiving transplants from matched unrelated donors (MUD) without BPX-501 is expected to be initiated in the third quarter of 2017. -- The top-line results from the above two studies are expected in the second half of 2018, with MAA filing planned for 2019.

8. Urogen Pharma Ltd. (URGN)

Gained 10.11% to close Thursday trading at $23.85.

News: No news

The Company made its debut on the NASDAQ as recently as May 4, 2017, offering its shares at a price of $13.00 each.

The lead drug candidate of Urogen is MitoGel, a novel sustained release formulation of Mitomycin C, for the treatment of non-muscle invasive, low-grade upper tract urothelial carcinoma (UTUC).

A phase III trial of MitoGel for the treatment of low-grade upper tract urothelial carcinoma, dubbed OLYMPUS, is underway.

Recent event:

On August 29, 2017, the FDA granted Fast Track designation for MitoGel.

LOSERS

1. SteadyMed Ltd. (STDY)

Lost 33.90% to close Thursday trading at $3.90.

News: The FDA has refused to even accept the Company's New Drug Application for Trevyent for the treatment of Pulmonary Arterial Hypertension.

Trevyent is a PatchPump wearable injector prefilled with a therapeutic drug, Treprostinil. The active pharmaceutical ingredient used in United Therapeutics' approved PAH drug Remodulin is also Treprostinil.

In its Refusal to File Letter, the FDA has concluded that the application is not sufficiently complete to permit a substantive review.

The Company has been asked to provide further information on certain device specifications and performance testing as well as additional design verification and validation testing on the final, to-be-marketed Trevyent product.

2. Akcea Therapeutics Inc. (AKCA)

Lost 10.12% to close Thursday trading at $18.65.

News: The Company has submitted a New Drug Application to the FDA for volanesorsen, an investigational medicine for the treatment of familial chylomicronemia syndrome (FCS).

Volanesorsen is co-developed by Akcea and Ionis Pharmaceuticals Inc. (IONS).

Near-term catalysts:

--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. Sienna Biopharmaceuticals Inc. (SNNA)

Lost 5.51% to close Thursday trading at $25.02.

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.

4. Mesoblast Limited (MESO)

Lost 5.29% to close Thursday trading at $5.91.

News: No news

Near-term catalysts:

-- The Phase 2b trial using MPC-150-IM in 159 end-stage CHF patients with a LVAD is expected to complete enrollment in Q3 CY17, with top-line results expected in Q1 CY18. -- The Phase 3 trial using MPC-150-IM in patients with Class II/III CHF is continuing to enrol through FY18, with full enrollment expected to occur in 2H CY18. -- The Phase 3 trial using MSC-100-IV in children with steroid refractory acute GVHD is expected to complete enrollment with top-line data readout expected in 2H CY17. -- The Phase 3 trial using MPC-06-ID in patients with CLBP is expected to complete enrollment in Q4 CY17. -- 12-month results for the Phase 2 trial using MPC-300-IV in patients with biologic-refractory RA are expected in Q3 CY17.