On March 15, 2018 Novelion Therapeutics Inc. (NASDAQ: NVLN), a biopharmaceutical company dedicated to developing and commercializing therapies for individuals living with rare diseases ("Novelion" or the "Company"), reported financial results for the fourth quarter and full year ended December 31, 2017 and provided an overview of recent business activities (Press release, QLT, MAR 15, 2018, View Source [SID1234524839]).

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Chief Operating Officer Jeff Hackman said, "We are focused on executing near-term plans that we believe will position our company for sustainable future growth. These priorities include cost control and expense management, reviewing our holding and capital structure with a view toward optimizing our assets for shareholders, advancing the metreleptin development program, and continuing to provide important therapies to our patients."

Business Update

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JUXTAPID: Novelion reported net revenues of JUXTAPID of $20.1 million in the fourth quarter of 2017, $14.2 million, or 71%, of which were from prescriptions written in the U.S.

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MYALEPT: Novelion reported net revenues of MYALEPT of $18.8 million in the fourth quarter of 2017, $13.3 million, or 71%, of which were from prescriptions written in the U.S.

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Novelion reported total consolidated net revenues of $138.4 million for the year ended December 31, 2017.

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Novelion ended 2017 with $55.4 million in unrestricted cash, compared with $70.5 million at the end of the third quarter of 2017.

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As announced separately today, subsidiary Aegerion Pharmaceuticals entered into a new secured financing facility with affiliates of Sarissa Capital Management and Broadfin Capital LLC providing for a $20 million term loan to Aegerion, strengthening Aegerion’s balance sheet and liquidity, and positioning the Company for ongoing capital structure review.

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In January 2018, Novelion undertook significant cost reduction plans as it continues to manage its limited cash resources.

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With respect to its European application to register metreleptin for marketing authorization, after taking into account the results of an oral hearing of the European Medicines Agency’s Committee for Medicinal Products for Human Use ("CHMP"), which occurred in February

2018, the Company expects the opinion of the CHMP in the second quarter of 2018 and the European Commission’s approval decision in mid-2018.

Fourth Quarter 2017 Financial Results

On November 29, 2016, the Company completed its acquisition of Aegerion Pharmaceuticals, Inc. ("Aegerion"). The acquisition has been accounted for as a business combination in which Novelion was considered the acquirer of Aegerion. As such, under U.S. Generally Accepted Accounting Principles ("GAAP"), the financial statements of Novelion are treated as the historical financial statements of the consolidated companies, with the results of Aegerion being included from November 29, 2016. This release also includes pro forma adjusted non-GAAP financial information showing pro forma results of operations of Novelion as if the acquisition had occurred on January 1, 2016. Reconciliation of the financial results on a GAAP versus non-GAAP basis are provided below the financial information that follows.

GAAP total net revenues for the fourth quarter of 2017 were $38.9 million compared to the prior year’s fourth quarter net revenues of $13.6 million. GAAP net revenues for JUXTAPID in the fourth quarter of 2017 were $20.1 million compared to $8.6 million in the prior year. GAAP net revenues for MYALEPT in the fourth quarter of 2017 were $18.8 million compared to $5.0 million for the same period in 2016.

GAAP total operating expenses for the fourth quarter of 2017 were $35.9 million compared to total operating expenses of $22.0 million for the same period in 2016. GAAP SG&A expenses were $24.1 million in the fourth quarter of 2017 compared to $16.0 million for the same period in 2016. GAAP R&D expenses were $11.8 million in the fourth quarter of 2017 compared to $6.0 million for the same period in 2016.

On a pro forma basis, during the fourth quarter of 2017, SG&A expenses were $22.5 million compared to $55.9 million for the same period in 2016. The decrease in pro forma SG&A expenses in the fourth quarter of 2017 compared with the same period in 2016 was primarily related to a reduction in headcount and legal and consulting fees.

On a pro forma basis, during the fourth quarter of 2017, R&D expenses were $11.6 million compared to $14.2 million for the same period in 2016. The decrease in pro forma R&D expenses in the fourth quarter of 2017 compared with the same period in 2016 was primarily related to a reduction in headcount and the timing of vendor related activities.

GAAP net loss in the fourth quarter of 2017 was $24.6 million compared to GAAP net loss of $19.9 million during the same period in 2016.

On a pro forma basis, net loss in the fourth quarter of 2017 was $3.3 million, compared to $20.1 million for the same period in 2016.

Full Year 2017 Financial Results

GAAP total net revenues for the year ended December 31, 2017 were $138.4 million compared to $13.6 million in 2016. GAAP net revenues for JUXTAPID for the year ended December 31, 2017 were $72.1 million compared to $8.6 million in 2016. GAAP net revenues for MYALEPT for the year ended December 31, 2017 were $66.3 million compared to $5.0 million in 2016.

GAAP total operating expenses for the year ended December 31, 2017 were $148.0 million compared to total operating expenses of $44.3 million in 2016. GAAP SG&A expenses were $96.5 million for the year ended December 31, 2017 compared to $29.5 million in 2016. GAAP R&D expenses were $49.0 million for the year ended December 31, 2017 compared to $14.8 million in 2016.

Cost of product sales were $77.2 million in the year ended December 31, 2017. Cost of product sales in the current year includes $18.8 million reserves recorded for excess and obsolete inventory, which are derived from projected sales activities, respective product shelf-life and their respective fair value. Additionally, cost of product sales was also comprised of the cost of inventory sold, amortization of acquired product rights, which resulted from the acquisition of Aegerion, and estimated royalties payable related to the sales of lomitapide and metreleptin. We expect cost of product sales for metreleptin to increase in 2018 and for the next several years, due primarily to an increasing time-based royalty rate on net sales of metreleptin in the U.S.

On a pro forma basis, for the year ended December 31, 2017, SG&A expenses were $90.7 million compared to $200.1 million in 2016. For the year ended December 31, 2017, R&D expenses on a pro forma basis were $48.2 million compared to $52.1 million in 2016.

GAAP net loss for the year ended December 31, 2017 was $126.7 million compared to GAAP net loss of $52.9 million in 2016.

On a pro forma basis, net loss for the year ended December 31, 2017 was $30.0 million, compared to $136.0 million in 2016.

As of December 31, 2017, the Company’s consolidated unrestricted cash balance was $55.4 million, compared to $70.5 million at September 30, 2017 and $108.9 million at December 31, 2016. As of December 31, 2017, there were 18.7 million shares outstanding. At December 31, 2017, total debt principal was $325 million, reflecting the principal amount of convertible debt, before discount, issued by Aegerion and consolidated as a result of the acquisition.

On March 15, 2018 –AngioDynamics, Inc. (NASDAQ:ANGO), a leading provider of innovative, minimally invasive medical devices for vascular access, peripheral vascular disease, surgery and oncology, reported that Michael C. Greiner, Executive Vice President and Chief Financial Officer, will present at the Needham 17th Annual Healthcare Conference at 4:00 p.m. ET on Tuesday, March 27, 2018 in New York, NY (Press release, , 15 15, 2018, View Source [SID1234524828]).

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A live webcast of the presentation will be accessible through the "Investors" section of the Company’s website at www.angiodynamics.com and will be available for replay following the event.

On March 15, 2018 X4 Pharmaceuticals, a clinical stage biotechnology company developing novel CXCR4 inhibitor drugs to improve immune cell trafficking to treat cancer and rare disease, reported that an abstract highlighting X4P-001, the Company’s CXCR4 antagonist, has been selected for presentation at the 2018 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting, taking place April 14-18, 2018 in Chicago, IL (Press release, X4 Pharmaceuticals, MAR 15, 2018, View Source [SID1234524826])

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"These results demonstrate enhanced immune cell activation and lymphocyte infiltration with X4P-001, alone and in combination with the anti-PD-1 immunotherapy pembrolizumab (Keytruda), in the melanoma tumor microenvironment."

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Details of the poster presentation on X4P-001 are as follows:

Poster Title: X4P-001, an oral bioavailable CXCR4 antagonist, enhances immune cell infiltration and activation in the tumor microenvironment of melanoma
Author: Robert H.I. Andtbacka, Huntsman Cancer Institute, University of Utah
Session: Immune Response to Therapies 1
Abstract #: 613
Date & Time: Sunday April 15, 2018 1:00 PM – 5:00 PM CT

"We are pleased to present these clinical biomarker data from our ongoing Phase 1b clinical study with X4P-001 in patients with melanoma," said Sudha Parasuraman, MD, Chief Medical Officer of X4 Pharmaceuticals. "These results demonstrate enhanced immune cell activation and lymphocyte infiltration with X4P-001, alone and in combination with the anti-PD-1 immunotherapy pembrolizumab (Keytruda), in the melanoma tumor microenvironment."

About X4P-001-IO in Cancer

X4P-001-IO is an investigational selective, oral, small molecule inhibitor of CXCR4 (C-X-C receptor type 4) that regulates the tumor microenvironment thereby enhancing endogenous anti-tumor responses. CXCR4 is a chemokine receptor that modulates immune function and angiogenesis through the trafficking of key immune cells such as T- cells, dendritic cells, and myeloid derived suppressor cells. CXCR4 signaling is disrupted in a broad range of cancers, facilitating tumor growth by allowing cancer cells to evade immune detection and creating a pro-tumor microenvironment. X4P-001-IO is being investigated in three separate clinical studies in solid tumors.

On March 15, 2018 Obsidian Therapeutics, Inc., a biotechnology company dedicated to the development of next-generation cell and gene therapies with pharmacologic operating systems, reported that an abstract highlighting preclinical data on its technology and therapeutic applications has been selected for presentation at the 2018 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting, taking place April 14-18, 2018 in Chicago, IL (Press release, , 15 15, 2018, View Source [SID1234524825]).

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Details of the poster presentation on Tuesday, April 17, 2018, are as follows:

Poster Title: Enhancing Adoptive Cell Therapies Through Exogenous Regulation: Destabilizing Domains For Next-Generation CAR-T
Lead Author: Celeste Richardson, PhD
Session: Adoptive Cell Therapy – Poster Session 3
Abstract #: 3580 / 18
Date & Time: April 17, 2018, from 8:00am-12:00pm CT
Link to Abstract: View Source!/4562/presentation/7518

About Destabilizing Domains

Obsidian uses Destabilizing Domains (DDs) to enable pharmacologic regulation of protein activity for next-generation cell and gene therapies. Obsidian’s DDs are small, fully-human protein domains that confer conditional stability to a fused payload protein. In the absence of a specific small molecule ligand the fusion protein is rapidly degraded, whereas in the presence of the ligand, the fusion protein becomes stable and functional. Obsidian uses this approach to equip engineered cells with controllable functions that can be precisely tuned by the administration of non-immunosuppressive, small molecule medicines that are readily available and dispensed by the treating physician.

On March 15, 2018 Adamis Pharmaceuticals Corporation (NASDAQ:ADMP) reported that investors with an update concerning certain accomplishments for 2017 and accomplishments and goals for 2018 (Press release, , 15 15, 2018, view-source:View Source [SID1234524824]).

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2017 Highlights

As reflected in the company’s annual report on Form 10-K for year ending December 31, 2017, recently filed with the Securities and Exchange Commission, some of the company’s accomplishments during 2017 included the following:

New Drug Application ("NDA") for Symjepi TM (epinephrine) Injection 0.3mg product, was approved by the U.S. Food and Drug Administration ("FDA") for the treatment of allergic reactions (Type I) including anaphylaxis.
Supplemental New Drug Application ("sNDA") was submitted for the lower dose Symjepi TM (epinephrine) Injection 0.15mg product for patients weighing 33-65 pounds.
Investigational New Drug ("IND") application was submitted to the FDA for a Naloxone injection product candidate, which uses the same approved injection device, for the treatment of opioid overdose.
The number of outstanding warrants was reduced through exercise transactions, which the company believes improved its balance sheet.
U.S. Compounding, Inc. subsidiary applied for a patent and began marketing a novel equine ulcer product for which the company believes there could be a significant market.
Early 2018 Highlights

Some of the company’s accomplishments during 2018 include the following:

In January, an IND application was submitted to the FDA for a beclomethasone HFA (APC-1000) product candidate and in February, the company received approval from the FDA to proceed with Phase 3 clinical studies.
In February, Adamis received correspondence from the FDA indicating that the agency had determined that the company’s sNDA for Symjepi (epinephrine) Injection 0.15mg was sufficiently complete to permit a substantive review and indicated that no potential review issues were identified as of the date of the agency’s communication.
In March, Adamis presented human factors data for Symjepi at the American Academy of Allergy Asthma and Immunology joint congress with the World Allergy Organization, and another human factors study was published in a peer-reviewed journal.
More on APC-1000

After the IND submission to the FDA, Adamis has recently received approval by the FDA to proceed to pivotal Phase 3 studies of APC-1000 in asthmatics. APC-1000 consists of a metered dose inhaler device containing an HFA (hydrofluoroalkane) inhaled corticosteroid called Beclomethasone, intended for the treatment of asthma. An ideal inhaled corticosteroid provides adequate lung exposure with limited systemic absorption of the drug. APC-1000 is being developed with the goal of demonstrating efficacy (lung function) and lower systemic absorption, thereby differentiating it from the current market leading inhaled corticosteroids. Following commencement of the studies, each asthmatic patient will be enrolled in the study for twelve weeks.

Asthma causes recurring periods of wheezing, chest tightness, shortness of breath, and cough. Asthma affects people of all ages, but it most often starts during childhood. In the United States, more than an estimated 25 million people are known to have asthma. Based on industry sources, we estimate that the annual global sales of prescription steroid HFA and similar products are approximately $3 billion, of which we intend to target a smaller niche.

Inhaled corticosteroids play a major role in reducing the morbidity and mortality from asthma and also reduce the need for oral corticosteroids and their related systemic toxicity risks. According to the Expert Panel Report-3 on the Guidelines for the Diagnosis and Management of Asthma, inhaled corticosteroids, such as Beclomethasone, are recommended for all stages of persistent asthma in adults and children. However, inhaled corticosteroids are also known to have local and systemic adverse effects. The goal of the Phase 3 study of APC-1000 is mainly to demonstrate efficacy, but in addition, show an improved safety profile for asthmatic patients.

Dr. Carlo, commenting on the Phase 3 clinical studies of APC-1000, said, "We are pleased with the news of receiving approval to proceed to pivotal Phase 3 studies of APC-1000 by the FDA. We will continue to work with them and seek their guidance on the commercialization of what we believe is a needed product." Dr. Ronald Moss, Chief Medical Officer of Adamis, added, "Our goal is to initiate the Phase 3 studies as soon as reasonably possible, during 2018. Having a cost-effective inhaled corticosteroid with a good safety profile would, we believe, greatly benefit asthmatic patients."

Future Milestones

Some of the company’s goals for the 2018 year include the following:

Finalizing and announcing the commercialization strategy for Symjepi (epinephrine) Injection 0.3mg;
FDA approval for Symjepi TM (epinephrine) Injection 0.15mg;
Initiate pivotal Phase 3 studies of APC-1000 in asthmatics;
Complete a "proof of concept" study with dry powder inhaler platform using fluticasone;
Filing an NDA for Naloxone injection;
Increase sales of compounded medications from our U.S. Compounding, Inc. subsidiary by at least 30%.
Dr. Carlo continued, "We remain committed to bringing Symjepi TM to market and are pleased with recent developments regarding our discussions with potential commercialization partners since our last press release pertaining to this topic. Once the commercial strategy is finalized, our focus will be advancing our other product candidates with the objective of increasing shareholder value. The entire Adamis team is working hard to achieve these goals during 2018."