INSM Breathes Easy, XOMA Rises On Analyst Upgrade, CLLS Down On Clinical Hold

(RTTNews) - The following are some of the biotech stocks that made their way onto the Day's Gainers & Losers' list of September 5, 2017.

GAINERS

1. Insmed Inc. (INSM)

Gained 119.61% to close Tuesday's (Sep.5) trading at $26.99.

News: The Company reported positive top-line results from a phase III study of Amikacin Liposome Inhalation Suspension in adult patients with treatment-refractory nontuberculous mycobacterial, a rare, progressive, destructive lung infection.

In the trial, dubbed CONVERT, the addition of Amikacin Liposome Inhalation Suspension to guideline-based therapy (GBT) eliminated evidence of nontuberculous mycobacterial lung disease caused by Mycobacterium Avium Complex in sputum by Month 6 in 29% of patients, compared to 9% of patients on GBT alone.

The Company plans to pursue accelerated approval and request priority review for Amikacin Liposome Inhalation Suspension.

2. XOMA Corporation (XOMA)

Gained 44.19% to close Tuesday's trading at $16.12.

News: Wedbush analyst has upgraded XOMA to Outperform from Neutral with a price target of $19, up from the previous price target of $9.00.

Recent events:

On August 25, 2017, the Company announced it has licensed the global commercial rights to Gevokizumab, a novel anti-IL-1 beta allosteric monoclonal antibody, to Novartis.

In a separate agreement, XOMA announced that it has granted Novartis a license to its intellectual property covering the use of IL-1 beta targeting antibodies in the treatment of cardiovascular disease.

Gevokizumab is under phase II trials in acne vulgaris; acute coronary syndromes; diabetic nephropathies; giant cell arteritis; labyrinthitis; myositis; osteoarthritis; schnitzler syndrome and scleritis.

Under the agreements, XOMA will receive $31 million in upfront payments, including a $5 million equity investment, and is eligible to receive significant pre- and post-commercialization milestone payments plus tiered high-single to mid-double-digit royalties on net sales of Gevokizumab. XOMA is also eligible to receive low-single-digit royalties on Canakinumab sales in cardiovascular indications rising to mid-single-digit royalties under certain circumstances.

A phase III study evaluating Canakinumab in combination with standard of care in people with a prior heart attack and inflammatory atherosclerosis is underway.

3. Pulmatrix Inc. (PULM)

Gained 33.51% to close Tuesday's trading at $2.47.

News: The Company has received an award from Cystic Fibrosis Foundation Therapeutics to support the development of its lead inhaled anti-fungal product candidate PUR1900 for the treatment of allergic bronchopulmonary aspergillosis (ABPA) in patients with cystic fibrosis and asthma.

The award supports advancement of PUR1900 to Phase 1/1B clinical trial in early 2018.

PUR1900 has already received an Orphan Drug designation and a Qualified Infectious Disease Product (QIDP) designation from the FDA for the treatment of fungal infections in patients with cystic fibrosis. These two designations together provide up to 12 years of market exclusivity for PUR1900 if approved for cystic fibrosis patients.

4. NantHealth Inc. (NH)

Gained 18.05% to close Tuesday's trading at $3.14.

News: No news

NantHealth is a personalized healthcare company. It derives revenue from sales of licensed software and maintenance, software-as-a-service, hardware, services, and its universal diagnostics solution GPS Cancer to healthcare providers, payors and self-insured employers. The Company made its debut on the NASDAQ Global Select Market on June 2, 2016, offering its shares at a price of $14.00 each.

Recent event:

On August 30, 2017, the Company announced that it has entered into an agreement for GPS Cancer with Asia Genomics, a provider and distributor of molecular testing services and key player in the field of personalized medicine in Southeast Asia.

5. Akcea Therapeutics Inc. (AKCA)

Gained 17.58% to close Tuesday's trading at $22.74.

News: The Company announced the appointment of Michael MacLean, chief financial officer; Mustafa Noor, chief development officer; Samuel Yonren, vice president and head of pharmacovigilance and drug safety and Kyle Jenne, U.S. commercial head.

Recent event:

On August 31, 2017, the Company 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.

6. Voyager Therapeutics Inc. (VYGR)

Gained 9.74% to close Tuesday's trading at $10.93.

News: No news

Near-term catalysts:

-- Initiation of pivotal phase 2-3 program of VY-AADC for advanced Parkinson's disease expected during late 2017. -- Filing of an investigational new drug application for VY-SOD101 for the treatment of amyotrophic lateral sclerosis caused by mutations in the superoxide dismutase 1 gene during late 2017 or early 2018.

7. Regenxbio Inc. (RGNX)

Gained 9.49% to close Tuesday's trading at $24.80.

News: No news

Recent event:

On August 25, 2017, the Company announced that it will acquire Dimension Therapeutics Inc. (DMTX) in an all-stock transaction for an implied value of approximately $3.41 per share.

The transaction is expected to close by year-end 2017.

Near-term catalysts:

-- A phase I clinical trial evaluating RGX-314 for the treatment of wet age-related macular degeneration (wet AMD) is underway and an interim update from the trial is expected to be provided by the end of 2017. -- A phase I/II clinical trial evaluating RGX-501 for the treatment of homozygous familial hypercholesterolemia (HoFH) is ongoing - with an interim update expected by the end of 2017.

8. MannKind Corporation (MNKD)

Gained 9.09% to close Tuesday's trading at $2.04.

News: The Company has fully relocated its corporate headquarters to a 12,596-square-foot location in the Westlake Landmark office park in Westlake Village, California. The move places MannKind, which is currently commercializing inhaled insulin Afrezza inhalation powder, in an area rich with biopharmaceutical talent.

LOSERS

1. Cellectis S.A. (CLLS)

Lost 20.26% to close Tuesday's trading at $25.66.

News: The FDA has placed a clinical hold on the Company's phase I studies of UCART123 in acute myeloid leukemia and in blastic plasmacytoid dendritic cell neoplasm (BPDCN).

The clinical hold was initiated after Cellectis reported one fatality in the BPDCN clinical trial.

Cellectis is focused on developing CAR T-cell therapies to cure cancer.

2. aTyr Pharma Inc. (LIFE)

Lost 9.84% to close Tuesday's trading at $2.75.

News: No news

Pipeline:

The Company's most advanced drug candidate is Resolaris which is being developed for adult Limb girdle muscular dystrophy 2B; adult Facioscapulohumeral muscular dystrophy (FSHD) and early onset FSHD; iMod.Fc for interstitial lung diseases and ORCA programs.

aTyr recently completed two extension studies of Resolaris in patients with limb-girdle muscular dystrophy 2B (LGMD2B), adult facioscapulohumeral muscular dystrophy (FSHD), and early onset FSHD. Top-line results from Phase 1b/2 trial of Resolaris in patients with early onset FSHD were announced in April of this year.

Another drug candidate iMod.Fc for interstitial lung diseases is expected to move into phase I testing later this year.

3. Adverum Biotechnologies Inc. (ADVM)

Lost 8.93% to close Tuesday's trading at $2.55.

News: No news

Near-term catalysts:

Begin enrollment in a Phase 1/2 trial of ADVM-043 for treating alpha-1 antitrypsin (A1AT) deficiency in the fourth quarter of 2017.

File IND applications for ADVM-053 for treating hereditary angioedema and ADVM-022 for wet AMD this year.

4. MyoKardia Inc. (MYOK)

Lost 7.77% to close Tuesday's trading at $40.35.

News: No news

Recent events:

-- On August 14, 2017, the Company announced the closing of its previously announced underwritten public offering of 4.02 million shares of common stock, at the public offering price of $35.50 per share, including full exercise by the underwriters of their option to purchase additional shares.

-- On August 7, 2017, the Company reported positive topline data from the first patient cohort of its phase II study of Mavacamten in symptomatic, obstructive hypertrophic cardiomyopathy patients, dubbed PIONEER-HCM. The news sent the stock soaring over 83% to $31.45 that day.