On May 9, 2016 Flamel Technologies (NASDAQ: FLML) reported its financial results for the first quarter of 2016 (Filing, Q1, Flamel Technologies, 2016, MAY 9, 2016, View Source [SID:1234512418]).

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First Quarter Highlights Include:

· Total revenue for first quarter 2016 was $36.2 million, compared to $32.7 million during the same period last year.

· GAAP net loss for the first quarter was ($6.4) million, or ($0.15) per diluted share, compared to GAAP net income of $11.6 million, or $0.27 per diluted share during the same period last year.

· Adjusted EBITDA was $11.8 million, compared to $12.9 million in the prior year.*

· Adjusted net income for the first quarter was $1.6 million, or $0.04 per diluted share, compared to an adjusted net income of $4.7 million, or $0.11 per diluted share, during the same period last year. *

· Cash and marketable securities at March 31, 2016 were $160.0 million, compared to $144.8 million at December 31, 2015 and $113.2 million at March 31, 2015.

· Special Protocol Assessment (SPA) submitted to the U.S. Food and Drug Administration (FDA) for the once nightly version of Micropump sodium oxybate.

* Non-GAAP financial measure. Descriptions of Flamel’s non-GAAP financial measures are included under the caption "Non-GAAP Disclosures and Adjustments" included within this document and reconciliations of such non-GAAP financial measures to their most closely applicable GAAP financial measures are found in the "Supplemental Information" section within this document.

Michael Anderson, Flamel’s Chief Executive Officer, commented, "We were pleased with our strong revenues of $36.2 million during the first quarter. The entrance of a third competitor to the neostigmine market in December 2015 was less impactful to pricing and share for Bloxiverz during the first quarter than initially anticipated; however, we still expect a decline in market share throughout the year to approximately 30% to 35%. As expected on April 29th, we received FDA approval for our third unapproved marketed drug, Akovaz, which is our formulation of ephedrine sulfate injection. Following launch in the third quarter of this year, Akovaz will provide yet another stream of cash flow to help us continue executing against our strategic plan of advancing our pipeline products and growth through acquisitions, the first of which we made early in the first quarter. We continue to focus on integrating the FSC business and revenues are meeting our expectations. We are particularly excited by physicians’ reception of Karbinal ER, which accounted for the majority of revenues from the FSC product portfolio."

Mr. Anderson continued, "At the end of the first quarter, we submitted our SPA to the FDA for our pivotal trial of Micropump sodium oxybate, which will provide the FDA an opportunity to review our trial protocol and provide feedback. We expect to begin patient registration for our pivotal trial in mid-year 2016. We view sodium oxybate as our most valuable pipeline asset, and will continue to take the necessary steps to ensure the Company returns maximum value to our shareholders."

First Quarter 2016 Results

The Company achieved revenues during the first quarter of 2016 of $36.2 million, compared to $32.7 million during the same period last year. On a GAAP basis, Flamel recorded a net loss of ($6.4) million during the first quarter, or ($0.15) per diluted share, compared to a net income of $11.6 million, or $0.27 per diluted share, for the same period last year. Adjusted net income for the first quarter was $1.6 million, or $0.04 per diluted share, compared to an adjusted net income of $4.7 million, or $0.11 per diluted share, during the same period last year. The decline in adjusted diluted EPS from the previous year was primarily due to higher SG&A resulting from investments made in infrastructure and people in order to execute the Company’s strategic plan. Included in GAAP net loss in the first quarter of 2016 was a $7.9 million charge to its contingent consideration liability resulting from the Company’s reassessment of its long term Éclat revenue forecast. In addition, the Company incurred a foreign currency exchange loss of ($2.9) million, compared to a foreign currency exchange gain of $11.5 million in the prior year quarter. Please see the Supplemental Information section within this document for a reconciliation of adjusted EBITDA, adjusted net income and adjusted diluted EPS to the respective GAAP amounts.

Cash flow from operations was $22.5 million, compared to $25.3 million in the same period last year. Cash and marketable securities at March 31, 2016 were $160.0 million, compared to $144.8 million at December 31, 2015, an increase of $15.2 million.

2016 Revenue and R&D Spending Guidance

The Company is maintaining its full year 2016 revenue guidance of $110 – $130 million and expects the recently acquired FSC products, AcipHex Sprinkle, Karbinal ER, Cefaclor for Oral Suspension and Flexichamber, to contribute revenues in the range of $10 – $15 million. As a result of the multiple clinical trials expected to run throughout 2016, the Company expects research & development expenses to be in the range of $35 – $50 million, up from $25.6 million in 2015.

On May 9, 2016 GTx, Inc. (Nasdaq: GTXI) reported the first public presentation of preclinical data from the Company’s selective androgen receptor degrader (SARD) program (Press release, GTx, MAY 9, 2016, View Source;p=RssLanding&cat=news&id=2166265 [SID:1234512115]). The results demonstrate that the Company’s highly potent SARDs selectively bind to the ligand binding domain (LBD) and interact with the N-terminal domain (NTD) of the androgen receptor (AR) and inhibit and degrade the AR at very low concentrations. These preclinical results suggest that the Company’s SARDs may be the first-in-class dual-interacting AR antagonists and degraders.

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The preclinical data are being presented at the 2016 American Urological Association (AUA) Annual Meeting taking place May 6-10, 2016, in San Diego, California.

Poster: Novel Dual-Binding Selective Degraders of Full Length and Splice Variant Androgen Receptors for the Treatment of Castration-Resistant Prostate Cancer

Presenter: Ramesh Narayanan, Ph.D., Director, Center for Cancer Drug Discovery and Associate Professor, Department of Medicine, University of Tennessee and Consultant for GTx, Inc.

Date:
Saturday, May 7 at 10:30 am until Tuesday, May 10 at 1:00 pm

According to Dr. Narayanan, "One of the limitations of current prostate cancer therapy is that some men with castration-resistant prostate cancer do not respond or eventually develop resistance to the therapy. These preclinical results suggest that novel SARD compounds may degrade and inhibit multiple forms of the androgen receptor, including AR splice variants, and may therefore potentially treat CRPC in men who are non-responsive to androgen therapy."

The Company’s lead SARD compounds are currently being evaluated in preclinical studies in order to select the best SARD compounds for continued development, with a goal of initiating first human clinical trials in 2017.

About SARDs

Selective Androgen Receptor Degrader (SARD) technology is being evaluated as a potentially novel treatment for men with castration-resistant prostate cancer (CRPC), including those who do not respond or are resistant to currently approved therapies. GTx believes that its novel SARD compounds will degrade multiple forms of the androgen receptor, including AR splice variants, such as AR-V7. GTx licensed the SARD technology from the University of Tennessee Research Foundation in 2015 with the goal of expanding its portfolio of product candidates targeting hormonal receptors.

About Prostate Cancer

Prostate cancer that is localized to the prostate can be effectively treated with surgery, radiation, brachytherapy and other modalities in an effort to eradicate all of the disease and cure the patient. In some cases, the tumor advances locally or metastasizes; these are examples of advanced prostate cancer. The goal of treatment for advanced prostate cancer is to control the tumor and keep the patient alive for as long as possible.

In advanced prostate cancer, a number of treatments with hormone blocking therapies or chemotherapy are used to slow the spread of metastases, shrink existing tumors, reduce symptoms and improve quality of life. Although most men with advanced prostate cancer are not cured of their disease, they can live a normal life for many years.

CPI-444 is an orally administered antagonist of the adenosine A2A receptor (Company Pipeline, Corvus Pharmaceuticals, MAY 9, 2016, View Source [SID:1234512139]). It is designed to block the action of adenosine that is produced by tumors. CPI-444 will enter a Phase 1b study in early 2016. In collaboration with Genentech, this study will evaluate CPI-444 as a single agent and in combination with the investigational agent, atezolizumab, an anti-PDL-1 antibody.

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On May 10, 2016 Fortress Biotech, Inc. (NASDAQ: FBIO) ("Fortress"), a biopharmaceutical company dedicated to acquiring, developing and commercializing novel pharmaceutical and biotechnology products, reported its financial results and recent corporate highlights for the quarter ended March 31, 2016 (Press release, Fortress Biotech, MAY 9, 2016, View Source;FID=1500085377 [SID:1234512312]).

Dr. Lindsay A. Rosenwald, Chairman, President and CEO of Fortress, said, "This year, we have continued to build our portfolio of products under development and Journey Medical Corporation’s roster of marketed products. We have also made significant progress advancing the pipeline of many of our other Fortress Companies, including Mustang Bio, which presented positive initial Phase I data on its CAR‐T therapy MB‐101 for the treatment of glioblastoma at the American Society of Gene and Cell Therapy 19th Annual Meeting. In addition, we are excited to possibly bring National Holdings Corporation under the Fortress umbrella with the goal of building a world‐class biotech and life sciences investment banking operations franchise. In 2016, we plan to continue to seek business development opportunities for Fortress and our Fortress Companies, as we expand our therapeutic focus and advance multiple milestones in our robust pipeline. We look forward to another transformative year in support of our mission of rapidly advancing meaningful treatments to people in need."

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Financial Results:
At March 31, 2016, Fortress’ consolidated cash and cash equivalents totaled $81.4 million compared to $98.2 million at December 31, 2015, a decrease of $16.8 million for the quarter. These totals exclude restricted cash of $14.6 million. The majority of the cash payments were related to Fortress and Fortress Companies previously accrued liabilities and upfront fees.

Revenue totaled $0.7 million for the first quarter of 2016.

Research and development expenses were $7.7 million for the first quarter of 2016, compared to $1.6 million for the first quarter of 2015.  

General and administrative expenses were $7.9 million for the first quarter of 2016, compared to $3.5 million for the first quarter of 2015.   

Net loss was $12.2 million, or $0.31 per share, for the first quarter of 2016, compared to a net loss of $12.1 million, or $0.31 per share, for the first quarter of 2015.   

Noncash stock‐based compensation expense included in net loss for the first quarter of 2016 was $2.9 million, compared to $1.5 million for the first quarter of 2015.

Recent Corporate Highlights:

Avenue Therapeutics, Inc.
During the three months ended March 31, 2016, Avenue completed a pharmacokinetics (PK) study for intravenous (IV) Tramadol.

Checkpoint Therapeutics, Inc.
In February 2016, Checkpoint repaid its National Securities Corporation (NSC) Debt of $2.8 million.

FBIO Acquisition, Inc.
In April 2016, Fortress, FBIO Acquisition, Inc. and National Holdings Corporation ("NHLD") entered into an agreement and plan of merger for the acquisition of NHLD by FBIO Acquisition, Inc.

Helocyte, Inc.
In February 2016, Helocyte entered into an Investigator‐Initiated Clinical Research Support Agreement with the City of Hope National Medical Center, to support a Phase 2 clinical study of its Triplex immunotherapy for CMV control in allogeneic stem cell transplant recipients. The Phase 2 study is additionally supported by grants from the National Cancer Institute.   

In February 2016, Helocyte entered into an option agreement with The University of Texas Health Science Center at Houston, for the exclusive rights to license certain intellectual property and clinical data relating to the use of bone marrow derived mononuclear cells for the treatment of severe Traumatic Brain Injury.   

In March 2016, Helocyte entered into an Investigator‐Initiated Clinical Research Support Agreement with the City of Hope National Medical Center, to support a Phase 2 clinical study of its PepVax immunotherapy for CMV control in allogeneic stem cell transplant recipients. The Phase 2 study is additionally supported by grants from the National Cancer Institute.  

Journey Medical Corporation (JMC)
In January 2016, JMC entered into a licensing agreement with a third party to distribute a prescription wound cream. JMC intends to commercialize this product in the second quarter of 2016.  

In January 2016, JMC entered into a licensing agreement with a third party to distribute an emollient for the treatment of various types of dermatitis. JMC intends to commercialize this product in the second quarter of 2016. Both products will be marketed under the JMC brand.

Mustang Bio, Inc.
In April 2016, Mustang announced that two abstracts pertaining to MB‐101 (IL13Rα2‐specific CAR T cells) for the treatment of glioblastoma were selected for presentation at the American Society of Gene and Cell Therapy 19thAnnual Meeting (ASGCT) (Free ASGCT Whitepaper). Pre‐clinical and preliminary Phase I data were presented at ASGCT (Free ASGCT Whitepaper) on Thursday, May 5.

On May 9, 2016 Halozyme Therapeutics, Inc. (NASDAQ: HALO) reported financial results for the first quarter ended March 31, which included an increase in revenue of 128 percent from the prior-year period and a net loss of $19.8 million, or $0.16 per share, compared to a net loss in the first quarter of 2015 of $15.1 million, or $0.12 per share (Press release, Halozyme, MAY 9, 2016, View Source [SID:1234512116]).

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"During the first quarter, we continued to execute against our two-pillar strategy with ongoing clinical studies of PEGPH20 and through growing the value of our ENHANZE platform," said Dr. Helen Torley, president and chief executive officer. "We made good progress toward our goal of initiating greater than 90 percent of HALO-301 sites by the end of the year and in evaluating the recommended dose to take into the expansion phase of our lung and gastric cancer studies.

"With our ENHANZE platform, we continued to see strong growth in royalty revenue combined with progress from our partners’ programs. During the quarter, Lilly nominated its third target triggering an $8 million milestone and Pfizer nominated an additional target triggering a $1.5 million milestone. These developments highlight the great potential associated with our ENHANZE technology franchise."

First Quarter 2016 and Recent Highlights include:

Dosing of first patient in HALO-301 | Pancreatic study in March, a phase 3 study to explore PEGPH20 with gemcitabine and ABRAXANE (nab-paclitaxel) in metastatic pancreatic cancer patients. The company plans to initiate sites outside the United States beginning in the second quarter and to reach its target of greater than 90 percent of centers ready to start screening patients by the end of the year.
Approval by the Food and Drug Administration (FDA) of an investigational device exemption for the companion diagnostic test developed with Ventana to prospectively identify patients with high levels of hyaluronan, or HA, in the company’s phase 3 study.
Progressing towards dose expansion in its phase 1b/2 PRIMAL study of PEGPH20 plus docetaxel in non-small cell lung cancer patients. The company is now evaluating patients at a dose of 2.2 µg/kg and remains on track to move into the dose expansion phase of the study in the second half of 2016.
Advancing into the second dosing cohort and recently submitting a protocol amendment in its phase 1b study of PEGPH20 plus KEYTRUDA (pembrolizumab) in lung and gastric cancer patients. The company submitted the protocol amendment to the FDA based on bleeding events observed in heavily pretreated relapsed gastric cancer patients. These events were not classified as dose limiting toxicities or determined by investigators to be related to PEGPH20. Halozyme is awaiting feedback from the FDA and plans to resume enrollment in the second dosing cohort following approval of the amendment.
Eli Lilly nominating their third target to be studied with Halozyme’s ENHANZE platform, triggering an $8 million milestone payment to Halozyme which will be received in the second quarter.
Pfizer nominating an additional target to be studied with Halozyme’s ENHANZE platform, triggering a $1.5 million milestone to Halozyme.
Baxalta receiving a positive opinion for HYQVIA from the Committee for Medicinal Products for Human Use for a pediatric indication in Europe. In addition, Baxalta initiated a phase 3 trial in patients with chronic inflammatory demyelinating polyneuropathy.
Expansion of oncology pipeline and demonstration of expertise in the tumor microenvironment with two new preclinical programs, an immune checkpoint inhibitor targeting adenosine and a novel antibody-drug conjugate targeting epidermal growth factor receptor. Preclinical data for the discovery and early development of these potential drug candidates were shared during the 2016 American Association for Cancer Research (AACR) (Free AACR Whitepaper) annual conference.
First Quarter 2016 Financial Highlights

Revenue for the first quarter was $42.5 million, compared to $18.7 million for the first quarter of 2015, driven primarily by milestone payments from Lilly and AbbVie, as well as royalties from partner sales of Herceptin SC, MabThera SC and HYQVIA. Revenue for the quarter included $11.4 million in royalties, $9.0 million in sales of bulk rHuPH20 primarily for use in manufacturing collaboration products and $3.9 million in HYLENEX recombinant (hyaluronidase human injection) product sales.
Research and development expenses for the first quarter were $40.1 million, compared to $16.7 million for the first quarter of 2015. The planned increases were primarily due to expenses for preclinical and clinical support of PEGPH20 and clinical API supply to ENHANZE partners.
Selling, general and administrative expenses for the first quarter were $10.8 million, compared to $9.4 million for the first quarter of 2015. The increase was primarily due to an increase in personnel expenses, including stock compensation, for the period.
Net loss for the first quarter was $19.8 million, or $0.16 per share, compared to a net loss in the first quarter of 2015 of $15.1 million, or $0.12 per share.
Cash, cash equivalents and marketable securities were $238.6 million at Mar. 31, 2016 compared to $108.3 million at Dec. 31, 2015.
Financial Outlook for 2016

For the full year 2016, the company is updating its previously announced guidance. Halozyme now expects:

Net revenues to be in the range of $130 million to $145 million, an increase from the prior range of $110 million to $125 million, driven by unplanned ENHANZE milestones and an increase in bulk product sales to ENHANZE partners;
Operating expenses to be in the range of $245 million to $260 million, a narrowing of the bottom end of the prior range of $240 million to $260 million as a result of the increase in product sales to ENHANZE partners;
Cash flow to be in the range of $45 million to $65 million, an increase from the prior range of $35 million to $55 million; and
Year-end cash balance to be in the range of $150 million to $170 million, an increase from the prior range of $140 million to $160 million.