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RGNX On Watch, BOLD To Report Data In Q2, ONCE Sparkles, So Long To AVXS

pharma 040518 lt

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

1. AveXis Inc. (AVXS)

Gained 81.57% to close Monday's (Apr.9) trading at $210.46.

News: The Company is all set to be acquired by Novartis (NVS) for $218 per share or a total approximate value of $8.7 billion.

AveXis' gene therapy candidate, AVXS-101, is being evaluated for the treatment of spinal muscular atrophy (SMA) Type 1 and Type 2.

We had alerted our premium subscribers to AVXS on September 15, 2016, when it was trading around $38. (Report titled "Who will win the race to treat SMA?").

2. Nemaura Medical Inc. (NMRD)

Gained 28.40% to close Monday's trading at $4.25.

Nemaura Medical is a medical technology company developing sugarBEAT as a non-invasive, affordable and flexible glucose monitoring system for adjunctive use by persons with diabetes.

News: The Company has named Bashir Timol, current Director of Strategy & Corporate Development, to the newly-created position of Chief Business Officer, effective immediately.

Recent events:

-- On January 25, 2018, the Company's shares were uplisted to Nasdaq Capital Market from the OTCQB Market.
-- On January 31, 2018, the Company announced positive summary data for its sugarBEAT European clinical trial program in January of this year.

Near-term Catalyst:

-- The Company anticipates completing its US clinical trial program for sugarBEAT by June, in readiness for Premarket Approval (PMA) submission to the FDA.

3. Abeona Therapeutics Inc. (ABEO)

Gained 18.21% to close Monday's trading at $16.55.

News: No news

Pipeline & Near-term Catalysts:

-- EB-101 for severe form of Epidermolysis Bullosa, which has successfully completed phase 1/2 clinical trial. A pivotal phase III trial of EB-101 is expected to commence this year.
-- ABO-102 for treatment of Sanfilippo syndrome type A (MPS IIIA), under phase I/II trial.
-- ABO-101 for treatment of patients with MPS IIIB (Sanfilippo syndrome Type B), also under phase I/II trial.
-- ABO-202 for Infantile Batten Disease, expected to enter clinical trials in 2018.
-- ABO-201 for Juvenile Batten Disease. The pre-IND meeting related to ABO-201 has been completed and clinical trial is expected to commence shortly.

4. Palatin Technologies Inc. (PTN)

Gained 17.07% to close Monday's trading at $1.44.

News: No news

Pipeline & Near-term Catalysts:

-- The Company's lead product candidate is Rekynda (Bremelanotide), which is proposed for Hypoactive Sexual Desire Disorder. The NDA for Rekynda was filed in 1Q, 2018.

The Company has an agreement with AMAG Pharmaceuticals (AMAG), under which AMAG has exclusive North American commercial rights to Rekynda.

-- A phase 1 single and multiple ascending dose study intended to evaluate the safety and tolerability of PL-8177 administered via subcutaneous injection was initiated in February of this year. PL-8177 is a clinical development candidate for ulcerative colitis and other inflammatory bowel diseases. Top line data from the phase I SAD/MAD study is currently expected in the third quarter of 2018.
-- A phase I pharmacokinetic & biodistribution study of oral formulation of PL-8177 is expected to commence in the fourth quarter of this year.
-- Preclinical IND enabling activities for PL-8331 for ocular inflammation are expected to be completed in the first half of this year.
-- Initiate a phase IIa trial of PL-3994 in HF-pEF patients in the first half of this year. Preliminary data from this study is expected in the first half of 2019.

5. uniQure N.V. (QURE)

Gained 16.44% to close Monday's trading at $25.57.

News: No news

Near-term Catalysts:

-- Initiate dosing of patients in the dose confirmation study of AMT-061, data from which will be available by the end of 2018.
-- Begin a pivotal study of AMT-061 for Hemophilia B in the third quarter of 2018.
-- Complete the GLP-safety and toxicology study of AMT-130 and submission of the IND in Huntington's disease in the second half of 2018.
-- Complete a preclinical therapeutic heart function study of AMT-126 in congestive heart failure in the second half of 2018.

6. REGENXBIO Inc. (RGNX)

Gained 15.62% to close Monday's trading at $31.45.

News: AveXis Inc., with which REGENXBIO has an exclusive agreement for the development and commercialization of products to treat Spinal Muscular Atrophy, has been acquired by Novartis for $218 per share.

Clinical Trials & Near-term Catalysts:

-- Completed dosing of third cohort in RGX-314 Phase I clinical trial for wet AMD.
-- Continuing dosing of second cohort in RGX-501 Phase I/II clinical trial for Homozygous Familial Hypercholesterolemia (HoFH).
-- Present topline data from RGX-314 and RGX-501 clinical trials in late 2018.
-- Initiate dosing in clinical trials for RGX-111 for MPS I and RGX-121 for MPS II in mid-2018.

7. Audentes Therapeutics Inc. (BOLD)

Gained 14.76% to close Monday's trading at $32.26.

News: No news

Clinical Trials & Near-term Catalysts:

-- Dose three additional patients in the first cohort of ASPIRO, the phase I/II clinical study of AT132 for X-Linked Myotubular Myopathy, and report additional interim data in the second quarter of 2018.
-- A phase I/II clinical study of AT342 to treat Crigler-Najjar Syndrome, dubbed VALENS, is underway. Preliminary data from this study is expected in the second quarter of 2018.
-- File IND for AT982 in Pompe Disease in mid-2018 and initiate a phase I/II clinical study in the fourth quarter of 2018.
-- Initiate a phase I/II clinical study of AT307 in CASQ2-CPVT in the fourth quarter of 2018.

8. Spark Therapeutics Inc. (ONCE)

Gained 13.37% to close Monday's trading at $70.36.

News: No news

Spark is a gene therapy platform company. The FDA approved the Company's novel gene therapy LUXTURNA last December to treat patients with a rare form of inherited vision loss.

Clinical Trials & Near-term Catalysts:

-- A phase I/II study of SPK-9001 for hemophilia B is ongoing. Spark and Pfizer have a collaboration for the development of SPK-9001, under which Spark Therapeutics is responsible for conducting all Phase 1/2 studies while Pfizer will assume responsibility for pivotal studies, any regulatory activities and potential global commercialization of any products that may result from the collaboration.
-- A phase I/II trial of SPK-8011 for hemophilia A is underway, with data expected to be available in second/third quarter of 2018.

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