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Athenex, Inc. Announces First Quarter 2018 Results

On May 14, 2018 Athenex, Inc. (NASDAQ:ATNX), a global biopharmaceutical company dedicated to the discovery, development and commercialization of novel therapies for the treatment of cancer and related conditions, reported its financial results and business highlights for the first quarter 2018 (Press release, Athenex, MAY 14, 2018, View Source;p=RssLanding&cat=news&id=2348822 [SID1234526557]).

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Dr. Johnson Lau, Athenex’s Chief Executive Officer, stated, "Our strong performance in the first quarter of 2018 reflects the continued positive momentum generated by our clinical programs and commercial efforts in our first year as a public company. In addition to the positive feedback from the FDA on the Phase III Clinical Study design for Oraxol, our rapid patient recruitment rate for our KX2-391 Ointment Phase III clinical trials is encouraging and puts us on track to announce topline data for these studies in the third quarter of 2018. Additionally, our landmark strategic partnership with Almirall and our commercial platform allowed us to generate record revenues during the first quarter, thus reinforcing our confidence in achieving our revenue guidance for 2018."

Dr. Lau continued, "In addition to these first quarter highlights, we recently announced the FDA has granted us Orphan Drug Designation for Oraxol for the treatment of angiosarcoma, a rare form of malignant blood vessel cancer. This further validates the strategy for the development of Oraxol and is a testament to the quality of our global team. As we move in to the balance of 2018, we are very excited by the multitude of growth opportunities across our platform and look forward to achieving the milestones in our business that will create value for all of our stakeholders."

First Quarter 2018 and Recent Business Highlights:

Clinical Platforms:

Orascovery:
Received Orphan Drug Designation from the US FDA for Oraxol in angiosarcoma, a rare form of malignant blood vessel cancer
Met and exceeded enrollment target for Oraxol Phase III Clinical Trial in metastatic breast cancer and announced positive feedback from the FDA regarding the trial design;
Obtained IND approval for Oraxol from the Chinese FDA to begin clinical trials in China;
Completed the first cohort of patients in its Phase 1b clinical trial of Oraxol (oral paclitaxel) plus CYRAMZA (ramucirumab) in gastric cancer patients who failed previous chemotherapies;

Src Kinase Inhibition
Completed patient enrollment for both Phase III clinical studies of KX2-391 Ointment for actinic keratosis indications months ahead of schedule; and
Phase II clinical study data for KX2-391 Ointment for the treatment of actinic keratosis was presented at the American Academy of Dermatology Annual Meeting (abstract ID 6134).
Commercial Business:

Launched 8 new products during the first quarter;
Athenex Pharmaceutical Division ("APD") currently markets 19 products in the U.S. with 32 SKUs; and
Athenex Pharma Solutions ("APS"), our 503(b) outsourced facility, currently markets 5 products with 23 SKUs.
First Quarter 2018 Financial Results:

Revenue for the three months ended March 31, 2018 was $37.8 million, an increase of $33.2 million, as compared to $4.6 million for the three months ended March 31, 2017. The increase was primarily attributable to $25.0 million in upfront license fees related to the collaboration agreement with Almirall, S.A., $8.6 million specialty products sold through our Commercial Platform, and $0.4 million in the sales of our 503B products. This was offset by decreases in other out-licensing fees of $0.5 million, contract manufacturing revenue of $0.2 million and API and medical device sales of $0.1 million.

Cost of product sales for the three months ended March 31, 2018 totaled $11.3 million, an increase of $8.5 million, as compared to $2.8 million for the three months ended March 31, 2017. This was primarily due to the increase of $7.2 million cost of product sales from the recently launched specialty products and $1.3 million cost of product sales from 503B and API products. The decrease in gross profit was primarily due to the impact of the costs incurred for the scale-up of production for new products in our 503B outsourcing facility.

Research and development expenses for the three months ended March 31, 2018 totaled $21.3 million, a decrease of $5.1 million, as compared to $26.4 million for the three months ended March 31, 2017. This was primarily due to $14.4 million decreased of drug licensing fees to Hanmi and Gland and offset by $6.9 million increase of clinical study costs for Phase III trials of Oraxol and KX-01 Ointment, $1.0 million increase of compensation expenses, $0.9 million increase of product development and supplies related to 503B products and $0.5 million increase in preclinical studies and API R&D expenses.

Selling, general and administrative expenses for the three months ended March 31, 2018, totaled $13.1 million, an increase of $3.3 million, as compared to $9.8 million for the three months ended March 31, 2017. The increase was primarily due to a $1.4 million increase of compensation expenses, $1.4 million increase of office expenses and professional fees and a $0.5 million increase of sales and marketing costs related to the launch of our proprietary products.

For the three months ended March 31, 2018, the Company incurred a net loss of $7.3 million compared to a net loss of $41.0 million a year ago.

Cash and cash equivalents and short-term investments totaled $106.5 million as of March 31, 2018, compared to $51.0 million as of December 31, 2017. This increase was due to an underwritten public offering of 4,765,000 shares of common stock in the first quarter of 2018, pursuant to which the Company raised net proceeds of $68.1 million, net of underwriting discounts, commissions and offering expenses and an upfront license fee payment of $30 million from Almirall. The Company believes that its existing cash and cash equivalents and short-term investments will be sufficient to fund current operating plans through approximately early-2019.

Outlook and Upcoming Milestones:

Clinical Platforms:

The enrollment of patients for Oraxol Phase III Clinical Trial is on target for the Company to be able to conduct a second interim analysis in the Oraxol KX-ORAX-001 Phase III clinical trial in the third quarter of 2018.
Expect topline data for Phase III KX2-391 Ointment studies to be available in the third quarter of 2018.

Commercial Business:

Full-year 2018 revenue in the range of $100 million to $125 million, inclusive of licensing-fee revenue associated with the partnership agreement with Almirall.
Corporate Updates:

Expect Dunkirk facility construction to be completed by the first quarter of 2019.
Conference Call and Webcast Information:

The Company will host a conference call and audio webcast on Monday, May 14, 2018 at 9:00 a.m. Eastern Time. To participate in the call, dial (855) 227-0567 (domestic) or (612) 979-9912 (international) fifteen minutes before the conference call begins and reference the conference passcode 9093904. A replay will be available approximately one hour after the recording through Monday, May 21, 2018 and can be accessed by dialing (855) 859-2056. The live conference call and replay can also be accessed via audio webcast at the Investor Relations section of the Company’s website, located at www.athenex.com. An archive will be available at this website until June 14, 2018.

Abivax Completes Enrolment of Phase IIa Clinical Trial of ABX464 to Treat Patients with Ulcerative Colitis

On May 14, 2018 ABIVAX (Euronext Paris: FR0012333284 – ABVX), a biotechnology company harnessing the immune system to develop a functional cure for HIV as well as treatments for inflammatory/autoimmune diseases and cancer, reported the completion of enrollment of its Phase IIA clinical trial ABX464-101 in 30 patients with moderate-to-severe ulcerative colitis (Press release, ABIVAX, MAY 13, 2018, View Source [SID1234526552]).

"ABIVAX is very pleased to announce the completion of enrolment in this first clinical trial exploring ABX464 for treatment of inflammatory indications," said Prof. Dr. Hartmut Ehrlich, M.D., chief executive officer at ABIVAX. "I would like to highlight the outstanding enthusiasm and dedication of Prof. Dr. Severine Vermeire, M.D., the Principal Investigator of this multinational trial and past president of ECCO (European Crohn’d and Colitis Organization), and thank the clinical trial sites, which kept recruitment on target, allowing us to report the top-line results from this important clinical trial in the autumn of this year," added Dr. Ehrlich.

ABX464-101 is a randomized, double-blind, placebo-controlled clinical trial (Phase 2a proof-ofconcept study) aimed at evaluating the safety and efficacy of ABX464 50 mg given once daily versus placebo for two months in subjects with moderate-to-severe active ulcerative colitis who have failed or are intolerant to immunomodulators, anti-TNFα, vedolizumab and/or corticosteroids. This clinical study is being conducted in 17 centers in seven European countries: Belgium, France, Germany, Austria, Hungary, Poland and Czech Republic. As of today, 30 out of the planned 30 patients have been randomized 2:1 to receive ABX464 or placebo, respectively. The study employs state-of-the art
technologies for monitoring potential treatment effects, including numerical recording of the colonoscopies with centralized reading.

ABX464-102 is a 12 months open label follow-up study for patients who completed ABX464-101, and 10 patients are already recruited into this clinical trial as of today. The rationale for the ABX464-101 study was derived from encouraging preclinical data, which demonstrated ABX464 had a strong anti-inflammatory effect. In macrophages, this effect was shown to be mediated by a 50-fold increase of the expression of IL-22, a cytokine known as a potent
suppressor of inflammatory processes, and a ten-fold increase of miR124 in peripheral blood mononuclear cells(PBMCs). mIR124 is a micro-RNA with potent anti-inflammatory properties and has recently been described as a tumor suppressor gene. Inflammation is a cornerstone of IBD, including ulcerative colitis and Crohn’s disease. When evaluated in a mouse model of IBD, ABX464 demonstrated a long-lasting effect in preventing the typical
symptoms of inflammatory colitis, including histological changes1
.
Prof. Dr. Severine Vermeire, M.D., Head of the IBD center at the University Hospitals Leuven, Belgium and Principal Investigator of the study, said: "reaching our recruitment goal of 30 patients marks an important step as there is a strong need to develop additional drugs in this indication; too many patients still do not respond or stop responding to current treatments. We are looking forward to the top-line data from this study and also to transferring as many patients as possible onto the one year open-label extension study with ABX464, which will provide us with very important long-term safety and efficacy data."

Ulcerative colitis is a debilitating inflammatory bowel disease in adults and children, with limited therapeutic management options for many patients. There is an estimated number of close to 1 million patients with ulcerative colitis in the United States, and global pharmaceutical sales for this disease are estimated to be around 5.7 billion US$ in 2017.

Radius Health to Present at the Bank of America Merrill Lynch 2018 Annual Healthcare Conference

On May 11, 2018 Radius Health, Inc. (Nasdaq:RDUS) reported that Jesper Høiland, President and Chief Executive officer and Pepe Carmona, Senior Vice President and Chief Financial Officer of the Company, will present a corporate update at the Bank of America Merrill Lynch 2018 Annual Healthcare Conference on Tuesday, May 15, 2018 (Press release, Radius, MAY 11, 2018, View Source [SID1234526549]).

Information on the presentation is as follows:

Event: Bank of America Merrill Lynch 2018 Annual Healthcare Conference
Date: Tuesday, May 15, 2018
Time: 1:40 p.m. PDT
Location: Encore Hotel, Las Vegas, NV
A live webcast of the presentation will be available by visiting the Investors section of Radius’ website at View Source A replay of the webcast will be archived on Radius’ website for 30 days following the presentation

TRILLIUM THERAPEUTICS REPORTS FIRST QUARTER 2018 FINANCIAL AND OPERATING RESULTS

On May 11, 2018 Trillium Therapeutics Inc. (NASDAQ/TSX: TRIL), a clinical stage immuno-oncology company developing innovative therapies for the treatment of cancer, reported financial and operating results for the three months ended March 31, 2018 (Press release, Trillium Therapeutics, MAY 11, 2018, View Source [SID1234526543]).

"Following the initial signals of monotherapy responses reported at ASH (Free ASH Whitepaper) last year, we have increasingly focused both our TTI-621 clinical trials on patients with T-cell malignancies throughout the first quarter of 2018" said Dr. Niclas Stiernholm, president and CEO of Trillium Therapeutics. "We have also been preparing to launch our second clinical CD47 program, TTI-622. Having both an IgG1 and an IgG4 SIRPaFc fusion protein in clinical testing should allow us to address important scientific questions related to the impact of the Fc region in various clinical scenarios, including combination therapy."

2018 First Quarter Highlights:

Reported refinements to both our phase 1 trials of TTI-621 to focus near-term efforts on patients with cutaneous T-cell lymphoma (CTCL) and peripheral T-cell lymphoma (PTCL). This action builds on the monotherapy results of TTI-621 presented at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in December 2017 where weekly infusions of TTI-621 were shown to be well tolerated and intratumoral injection was observed to reduce local lesions in 9 out of 10 patients with mycosis fungoides, a common type of CTCL.
The U.S. Food and Drug Administration granted an Orphan Drug Designation to TTI-621 for the treatment of cutaneous T-cell lymphoma. Orphan Drug Designation qualifies the sponsor of the drug candidate for various development incentives, which include an exemption from fees under the Prescription Drug User Fee Act and a seven-year marketing exclusivity period following approval.
Presented preclinical TTI-622 data at the 2018 AACR (Free AACR Whitepaper) Annual Meeting demonstrating that TTI-622 induces the phagocytosis of a broad panel of tumor cells derived from patients with both hematological and solid tumors. As a monotherapy, TTI-622 treatment resulted in decreased tumor growth and improved survival in a B cell lymphoma xenograft model, as well as enhanced the efficacy of cetuximab (anti-EGFR) and daratumumab (anti-CD38) antibodies in solid and hematological xenograft models, respectively. We expect to enroll the first patient in a Phase 1 clinical trial of TTI-622 in Q2 2018.
First Quarter 2018 Financial Results

As of March 31, 2018, Trillium had cash and cash equivalents and marketable securities, and working capital of $73.9 million and $61.7 million, respectively, compared to $81.8 million and $68.9 million, respectively at December 31, 2017. The decrease in cash and cash equivalents and marketable securities, and working capital was due mainly to cash used in operations of approximately $9.5 million.

Net loss for the three months ended March 31, 2018 of $8.6 million was lower than the loss of $11.5 million for the three months ended March 31, 2017. The net loss was lower due mainly to a net foreign currency gain of $1.6 million for the three months ended March 31, 2018, compared to a net foreign currency loss of $0.4 million in the prior year period. Research and development expenses decreased by $0.9 million in 2018 as a result of lower manufacturing activity for TTI-621 and TTI-622. These decreases were partially offset by higher clinical trial expenses.

Rocket Pharmaceuticals Reports First Quarter 2018 Financial Results and Operational Highlights

On May 11, 2018 Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT) ("Rocket"), a leading U.S.-based multi-platform gene therapy company, reported financial results for the quarter ended March 31, 2018, and provided an update on the Company’s recent achievements, as well as upcoming milestones (Press release, Rocket Pharmaceuticals, MAY 11, 2018, View Source;p=irol-newsArticle&ID=2348660 [SID1234526542]).

"2018 is off to a great start for Rocket. During the first quarter, we continued to make excellent progress advancing our pipeline of lentiviral vector (LVV) and adeno-associated viral vector (AAV) gene therapy programs towards key value inflection points. We look forward to presenting updated patient data from the Fanconi Anemia program later this month, and to disclosing our AAV-based gene therapy program by the end of the year," said Gaurav Shah, M.D., Chief Executive Officer and President of Rocket. "We remain on track for multiple regulatory filings across our pipeline over the next 12 months. As a result, there is the potential for four programs to be in the clinic in 2019, with clinical data for up to two programs."

Dr. Shah continued, "While near-term milestones are certainly important, Rocket’s long-term strategy is based on a sustainable and integrated multi-platform approach. We remain focused on our mission of continued innovation to deliver best-in-class curative cell and gene therapies to patients living with devastating diseases."

Anticipated Milestones Over the Next 12 -18 Months

Fanconi Anemia (FA): Updated patient data from the ongoing Phase 1/2 study of RP-L102 conducted with CIEMAT is expected over the next 12 months. The next update will be provided at the American Society of Gene and Cell Therapy’s (ASGCT) (Free ASGCT Whitepaper) 21st Annual Meeting in Chicago. The oral presentation is scheduled during the Presidential Symposium on Friday, May 18, 2018, at 2:45 p.m. Central Time.
Leukocyte Adhesion Deficiency-I (LAD-I): An Investigational Medicinal Product Dossier (IMPD) is expected to be filed in the fourth quarter of 2018. Clinical data is expected over the next 12-18 months.
Pyruvate Kinase Deficiency (PKD): An IMPD application is expected to be filed in early 2019.
Infantile Malignant Osteopetrosis (IMO): Preclinical studies continue to advance in support of filing a first-in-human clinical study.
AAV: Disclosure of the disease indication and preclinical data is planned for the second half of 2018.
Upcoming Conferences

Bank of America Merrill Lynch Health Care Conference 2018. Rocket is scheduled to present on Tuesday, May 15, at 9:20 a.m. Pacific Time.
UBS Global Healthcare Conference. Rocket is scheduled to present on Wednesday, May 23, at 9:30 a.m. Eastern Time.
Jefferies Global Healthcare Conference. Rocket is scheduled to present on Thursday, June 7, at 2:00 p.m. Eastern Time.
First Quarter 2018 Financial Results

Cash position. Cash, cash equivalents and short-term investments as of March 31, 2018, were $182.7 million, which includes a $52.0 million fully convertible debenture which expires in 2021.
R&D expenses. Research and development expenses were $5.7 million for the quarter ended March 31, 2018, compared to $2.3 million for the quarter ended March 31, 2017.
G&A expenses. General and administrative expenses were $8.7 million for the quarter ended March 31, 2018, compared to $0.6 million for the quarter ended March 31, 2017. The increase in G&A expenses was primarily due to one-time merger related expenses of $5.3 million.
Net loss. Net loss was $15.3 million or $(0.42) per share (basic and diluted) for the quarter ended March 31, 2018, compared to $2.7 million or $(0.39) per share (basic and diluted) for the quarter ended March 31, 2017.
Shares outstanding. Approximately 39.4 million shares of common stock were outstanding as of March 31, 2018.
Financial Guidance

Cash position. Based on its current operating plan, Rocket expects its cash, cash equivalents and short-term investments as of March 31, 2018, will be sufficient to run its operations into 2020.