On May 9, 2019 Omeros Corporation (NASDAQ: OMER), a commercial-stage biopharmaceutical company committed to discovering, developing and commercializing small-molecule and protein therapeutics for large-market as well as orphan indications targeting inflammation, complement-mediated diseases, disorders of the central nervous system and immune-related diseases, including cancers, reported recent highlights and developments as well as financial results for the first quarter ended March 31, 2019, which include (Press release, Omeros, MAY 9, 2019, View Source [SID1234536045]):

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1Q 2019 total and OMIDRIA revenues were $21.8 million compared to $1.6 million and $22.0 million in the first and fourth quarters of 2018, respectively. 1Q 2019 revenues reflect the seasonally lower volume of cataract surgery performed in the first quarter and the timing of normal wholesaler purchases shifting from the end of March to the first week of April.

"Sell-through" – the number of units sold by wholesalers to ASCs and to hospitals – for 1Q 2019 was a record high, increasing 14 percent from the previous high-water mark set in 4Q 2018. Sell-through in the current quarter has continued to grow at a double-digit rate over the same period in the first quarter.

Net loss in 1Q 2019 was $24.3 million, or $0.50 per share, which includes non-cash expenses of $6.0 million, or $0.12 per share. Overall decrease in cash, cash equivalents and short-term investments for the quarter was $13.3 million.

At March 31, 2019, the company had cash, cash equivalents and short-term investments available for operations of $47.2 million.

Since the previously reported FDA meeting held in the first quarter of 2019, which resulted in a streamlined path to submission of a Biologics License Application (BLA) for narsoplimab in HSCT-TMA, the company has had two additional meetings with FDA. The first covered chemistry, manufacturing and controls (CMC), and the company’s CMC commercialization plan remains on track. In the second, a clinical meeting, Omeros reached agreement with FDA on the large majority of the criteria for the primary endpoint. The company expects to complete agreement on the few remaining details in the very near future and is confident that the efficacy and safety data for narsoplimab will support BLA approval in HSCT-TMA.

CMS recently issued a preliminary decision to establish a unique permanent HCPCS J-code for OMIDRIA. The decision is expected to be finalized no later than November with the J-code becoming effective on the first day of the following quarter. A J-code provides a uniform, simpler and widely accepted process for providers to bill for OMIDRIA across both Medicare and commercial insurance plans.

"We are pleased with the company’s performance to date in 2019, having made substantial strides on all fronts," said Gregory A. Demopulos, M.D., Omeros’ chairman and chief executive officer. Once again we

saw OMIDRIA posting record quarterly sell-through numbers, up 14 percent over the record set in 4Q 2018, and the product’s growth trajectory has continued this quarter. Narsoplimab is advancing across three Phase 3 programs and, based on recent interactions with FDA, we expect very soon to wrap up the remaining criteria for the primary endpoint in stem cell TMA, are confident that our data will support a BLA approval and are moving forward with preparations for a commercial launch. Our PDE7 inhibitor, OMS527, is also faring well in its Phase 1 clinical trial, which is slated to finish later this quarter or next. Further expanding our complement franchise, both OMS906, our antibody against MASP-3, and our small-molecule MASP-2 inhibitor are planned to enter the clinic next year. We are also continuing to drive the development of our small-molecule compounds targeting GPR174, a receptor that increasingly appears to control a major cancer pathway. Across all of these efforts, our primary focus remains the patient. In over 600,000 procedures, cataract surgery patients have experienced the benefits of OMIDRIA, and we look forward to making narsoplimab commercially available worldwide in the near future."

First Quarter and Recent Developments

Recent developments regarding OMIDRIA include the following:

The Centers for Medicare & Medicaid Services (CMS) issued a preliminary decision to establish a unique permanent Healthcare Common Procedure Coding System (HCPCS) J-code for OMIDRIA. The preliminary decision is included in the publicly available agenda for the agency’s upcoming HCPCS Workgroup meeting on May 15, 2019. The decision is expected to be finalized no later than November 2019, with the code becoming effective on the first day of the following quarter. If finalized, assignment of a J-code for OMIDRIA would provide a uniform and widely accepted process that is expected to simplify billing for the drug across Medicare as well as commercial insurance plans.

In May 2019, the results of a "real-world" clinical study were presented at the annual meeting of the American Society of Cataract and Refractive Surgery and American Society of Ophthalmic Administrators held in San Diego. The study compared the incidence of cystoid macular edema (CME), a sight-threatening complication of cataract surgery, in patients undergoing cataract surgery using OMIDRIA with postoperative NSAIDs alone versus those using postoperative corticosteroids, with and without NSAIDs, in the absence of OMIDRIA. The retrospective analysis of cataract surgery performed on 504 eyes (357 patients) showed that use of OMIDRIA reduced the incidence of CME by 3- to 12-fold compared to published data on cataract procedures performed without OMIDRIA. This study, together with the 17 peer-reviewed articles already published on the benefits of OMIDRIA, supports Omeros’ ongoing efforts to secure permanent separate payment for the drug.

Recent developments regarding narsoplimab, Omeros’ lead human monoclonal antibody targeting mannan-binding lectin-associated serine protease-2 (MASP-2) in Phase 3 clinical programs for the treatment of hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA), Immunoglobulin A (IgA) nephropathy, and atypical hemolytic uremic syndrome (aHUS), include the following:

Omeros has had two additional meetings with the U.S. Food and Drug Administration (FDA) – one to cover chemistry, manufacturing and controls-related topics in preparation for commercialization and, most recently, to finalize the criteria for the HSCT-TMA trial’s primary endpoint on which the clinical data will be assessed. The company’s CMC commercialization plan remains on track. In the clinical meeting, Omeros reached agreement with FDA on the large majority of the criteria for the primary endpoint, which will include both laboratory and organ function components. The meeting included a detailed discussion of the primary endpoint and the company believes that it has a good understanding – and is comfortable with – FDA’s position on the final few remaining details. Omeros expects to complete agreement on these last few details in the very near future. In light of discussions with FDA and the available data on narsoplimab treatment of HSCT-TMA patients, the company is confident that its efficacy and safety data will support approval.

In March 2019, Omeros launched a disease education initiative at the annual meeting of the European Society for Blood and Marrow Transplantation (EBMT) with a well-attended educational session sponsored by Omeros and entitled "How do I…diagnose HSCT-TMA." A focus of the session was the relationship between HSCT-TMA and the broader syndrome of disorders caused by endothelial injury, which is important across the company’s development of MASP-2 inhibitors, including narsoplimab.

Omeros also announced the presentation at EBMT of a case report of resolution of gastrointestinal HSCT-TMA following narsoplimab treatment. The case was presented by Rafael Duarte M.D., Ph.D., F.R.C.P., Associate Professor, Head of Hematopoietic Transplantation and Hemato-oncology Section, University Hospital Puerta de Hierro Majadahonda, Madrid and Secretary of the EBMT. Dr. Duarte described an 18-year-old patient with biopsy-proven HSCT-TMA of the gastrointestinal tract causing severe gastrointestinal bleeding requiring transfusions. Upon receiving narsoplimab, her TMA resolved and all transfusions were discontinued. The patient continues to do well after cessation of narsoplimab treatment.

Omeros’ Phase 3 trial evaluating narsoplimab for IgA nephropathy, referred to as ARTEMIS-IGAN, continues to enroll. Results from the Phase 2 study of narsoplimab in IgA nephropathy are expected to be presented at the annual Congress of the European Renal Association – European Dialysis and Transplant Association in Budapest in June. In addition, together with its Academic Leadership Committee of international experts on IgA nephropathy, the company is preparing a series of manuscripts directed to narsoplimab and its IgA nephropathy program with the first manuscript planned for submission soon.

Updates regarding Omeros’ other development programs and platforms include the following:

The development of small-molecule MASP-2 inhibitors continues, and lead compounds are being optimized for potency, oral bioavailability and target selectivity. Omeros expects to enter the clinic with an orally administered MASP-2 inhibitor next year.

The company’s MASP-3 inhibitor, OMS906, is expected to enter clinical trials in the first half of 2020.

In the company’s Phase 1 trial for OMS527, which targets treatment of addiction and compulsive disorders, the company has completed dosing all six cohorts in the single-

ascending-dose portion of the trial, and three multiple-ascending-dose cohorts. The trial is expected to be completed during the second or third quarter of this year. The drug has been well tolerated and pharmacokinetic data are consistent with once-daily dosing with or without food. A Phase 2a study targeting nicotine addiction is planned assuming successful completion of Phase 1.

In Omeros’ proprietary G protein coupled receptor (GPCR) platform, development efforts are focused on several targets, including GPR174. Based on its data, the company believes that GPR174 controls a major pathway in cancer, and modulation of the receptor could provide a seminal advance in immuno-oncologic treatments for a wide range of solid and liquid tumors. Development continues on small molecule compounds targeting GPR174 with the objective of entering the clinic as soon as possible.

In May 2019, Omeros launched a new corporate website at www.omeros.com.

Omeros has approval for and is finalizing an accounts receivable-based line of credit that, if the company chooses to implement it, would provide for borrowing availability of up to $50 million depending on the company’s available borrowing base.

Financial Results

For the quarter ended March 31, 2018, revenues were $21.8 million, all relating to sales of OMIDRIA. This compares to OMIDRIA revenues of $1.6 million and of $22.0 million in the first and fourth quarters of 2018, respectively. 1Q 2019 revenues reflect the seasonally lower volume of cataract surgery performed in the first quarter and the timing of normal wholesaler purchases shifting from the end of March to the first two days of April, when net sales to wholesalers were approximately $2.4 million. Inventory units on hand at wholesalers at December 31, 2018 and March 31, 2019 were effectively the same. Gross-to-net deductions decreased from 28.3 percent in 4Q 2018 to 27.0 percent in 1Q 2019.

Sell-through for 1Q 2019 was a record high, increasing 14 percent from the previous high-water mark set in the 4Q 2018. Sell-through in the current quarter has continued to grow at a double-digit rate over the same period in the first quarter.

Total costs and expenses for the three months ended March 31, 2019 were $41.0 million compared to $29.3 million for the same period in 2018. The increase in the current year quarter was due primarily to higher third-party manufacturing scale-up costs for our narsoplimab program as we continue to increase our production capacity to meet anticipated clinical and commercial requirements, as well as increased expenses associated with pre-commercialization activities for narsoplimab and sales and marketing costs related to the re-introduction of OMIDRIA.

For the three months ended March 31, 2019, Omeros reported a net loss of $24.3 million, or $0.50 per share, which included non-cash expenses of $6.0 million, or $0.12 per share. This compares to the prior year’s first quarter for which Omeros reported a net loss of $30.1 million, or $0.62 per share, which included non-cash expenses of $4.3 million, or $0.09 per share.

Overall decrease in cash, cash equivalents and short-term investments for 1Q 2019 was $13.3 million. As of March 31, 2018, the company had $47.2 million of cash, cash equivalents and short-term investments available for operations.

Conference Call Details

Omeros’ management will host a conference call to discuss the financial results and to provide an update on business activities. The call will be held today at 1:30 p.m. Pacific Time; 4:30 p.m. Eastern Time. To

access the live conference call via phone, please dial (844) 831-4029 from the United States and Canada or (920) 663-6278 internationally. The participant passcode is 4095776. Please dial in approximately 10 minutes prior to the start of the call. A telephone replay will be available for one week following the call and may be accessed by dialing (855) 859-2056 from the United States and Canada or (404) 537-3406 internationally. The replay passcode is 4095776.

To access the live or subsequently archived webcast of the conference call on the internet, go to the company’s website at www.omeros.com and select "Events" under the Investors section of the website. To access the live webcast, please connect to the website at least 15 minutes prior to the call to allow for any software download that may be necessary.

On May 9, 2019 Marker Therapeutics, Inc. (NASDAQ:MRKR), a clinical-stage immuno-oncology company specializing in the development of next-generation T cell-based immunotherapies for the treatment of hematological malignancies and solid tumor indications, reported financial results for the first quarter ended March 31, 2019 (Press release, TapImmune, MAY 9, 2019, View Source [SID1234536044]).

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"We continued to make significant progress this quarter. Based on our early interactions with the U.S. FDA, we are confident in our submission package for our IND for post-transplant acute myeloid leukemia (AML). In light of the feedback from the FDA, we plan to submit an IND for a Phase 2 clinical trial in the third quarter, which should enable us to enroll the first patient by the end of the year," said Peter L. Hoang, President & CEO of Marker Therapeutics. "Depending on the results and upon further discussions with the FDA, we believe that if results are positive, this has the potential to serve as a pivotal trial—particularly if results are consistent with data generated to date from our MultiTAA therapies in investigator-sponsored trials. We look forward to initiating our first company-sponsored trial in such an important disease area, for which there are limited treatment options. Additionally, we anticipate reporting an update from our solid tumor program in pancreatic cancer in the third quarter."

PROGRAM HIGHLIGHTS AND CURRENT UPDATES

Multi-Antigen Targeted (MultiTAA) T Cell Therapies

·Company Prepares IND for Potentially Pivotal Trial in Post-Transplant AML
Based on interactions with the U.S. Food and Drug Administration (FDA) on the clinical trial design of the planned Marker-sponsored Phase 2 clinical trial in post-transplant AML, the Company remains on track to submit an Investigational New Drug (IND) application in the third quarter, and anticipates first patient enrolled by the end of 2019. The multicenter trial will evaluate clinical efficacy of Marker’s MultiTAA-specific T cells in patients with AML in both the adjuvant and active disease setting, following an allogeneic hematopoietic stem cell transplant (HSCT). The dose to be administered in the trial is expected to be the maximum tolerated dose currently determined in the Baylor College of Medicine-sponsored Phase 1 trial. In the adjuvant setting, patients will be randomized to either MultiTAA therapy at approximately 90 days post-transplant or standard of care observation, while the active disease patients will receive MultiTAA T cells as part of a single-arm group at approximately 90 days post-transplant.

·MultiTAA T Cell Therapies Continue to Generate Positive Clinical Data Across Various Indications
In several ongoing investigator-sponsored Phase 1 clinical trials led by Baylor College of Medicine (BCM), Marker’s MultiTAA therapies have demonstrated the potential to mediate a meaningful anti-tumor effect, as well as significant in vivo expansion of T cells. Across all hematological indications studied in these trials—including AML, lymphoma, acute lymphoblastic leukemia (ALL), and multiple myeloma—MultiTAA therapy has appeared to be well-tolerated, with no incidence of cytokine release syndrome, neurotoxicity or any other serious adverse events related to the therapy.

·Company to Report Update from Phase 1/2 Trial in Pancreatic Cancer in Q3 2019
Marker plans to report additional interim data from BCM’s ongoing Phase 1/2 clinical trial in pancreatic cancer in the third quarter of 2019. The Phase 1/2 trial is a three-arm trial which includes chemo-responsive patients (Arm A), chemo-refractory patients (Arm B) and an exploratory arm for patients who have surgically-resectable disease (Arm C). Patients in the chemo-responsive arm have completed at least three months of standard-of-care chemotherapy, and are receiving up to six administrations of MultiTAA T cells in alternation with chemotherapy. Patients in the chemo-refractory arm are either ineligible for chemotherapy or have progressed on chemotherapy, and are receiving up to six doses of MultiTAA T cells as a monotherapy. The surgically-resectable patients are receiving a dose of T cells prior to surgical resection which allows Marker to assess the tumor samples for T-cell infiltration, epitope spreading and other important characteristics. These patients are eligible to receive five additional doses of T cells after surgical resection.

T Cell Based Vaccines

·Ovarian Cancer Data
As of January 2019, the Company has completed enrollment in its Phase 2 clinical trial in ovarian cancer using TPIV200 as a maintenance therapy for patients in their first remission after surgery and platinum-based chemotherapy, with a total of 120 patients enrolled, randomized, and treated at 17 clinical sites. The trial completed enrollment six months faster than anticipated and the Company expects to reach its planned interim analysis trigger of 55 patients who have progressed before the end of 2019 and to report the results of this interim analysis in the fourth quarter of 2019.

·Triple Negative Breast Cancer Data
The Company reported initial findings from its dose-finding, four-arm Phase 2 clinical trial in triple negative breast cancer, including low- and high-dose TPIV200 with or without cyclophosphamide. Of 27 patients evaluated for immunogenicity, 26 showed significant immune response to the vaccine treatment. Of 80 patients treated at 11 clinical sites, 14 have shown disease progression, as of April 30, 2019, following treatment with TPIV200.

FIRST QUARTER 2019 FINANCIAL RESULTS

Net loss for the quarter ended March 31, 2019 was $5.3 million compared to a net loss of $3.2 million for the quarter ended March 31, 2018.

Research and development expenses were $2.8 million for the quarter ended March 31, 2019, an increase of $1.2 million, compared to $1.6 million for the quarter ended March 31, 2018. The increase was primarily attributable to increased headcount-related expenses, stock-based compensation expenses and consulting expenses resulting from the expansion of our internal infrastructure as we advance the clinical development of our MultiTAA T cell product candidates.

General and administrative expenses were $2.8 million for the quarter ended March 31, 2019, an increase of $1.2 million, compared to $1.6 million for the quarter ended March 31, 2018. The increase was primarily attributable to an increase in general and administrative headcount and stock-based compensation expenses.

CASH POSITION AND GUIDANCE

At March 31, 2019, Marker had cash and cash equivalents of $57.7 million. The Company believes that its existing cash and cash equivalents will fund the Company’s current operations into late 2020.

UPCOMING NEAR-TERM POTENTIAL MILESTONES

·First update in solid tumor program planned for third quarter of 2019;
·IND submission for Company-sponsored Phase 2 AML clinical trial in the third quarter of 2019, with first patient enrolled by end of 2019;
·Interim analysis readout in the TPIV200 ovarian trial expected in fourth quarter of 2019; and
·Overall update on ongoing clinical trials in cell therapy to be provided by the end of 2019.

On May 9, 2019 Palatin Technologies, Inc. (NYSE American: PTN), a biopharmaceutical company developing targeted, receptor-specific peptide therapeutics for the treatment of diseases with significant unmet medical need and commercial potential, reported results for its third quarter ended March 31, 2019 (Press release, Palatin Technologies, MAY 9, 2019, View Source [SID1234536043]).

Recent Highlights and Program Updates

Female Sexual Dysfunction / Vyleesi(bremelanotide)

●Vyleesi, the trade name for bremelanotide – Under development for Hypoactive Sexual Desire Disorder ("HSDD"):

●The Prescription Drug User Fee Act ("PDUFA") date for completion of FDA review of the Vyleesi New Drug Application ("NDA") is June 23, 2019

●The U.S. Food and Drug Administration ("FDA") requested a Phase 1 study in premenopausal volunteers assessing short term daily use of Vyleesi. This study, conducted by Palatin and our exclusive licensee for North America, AMAG Pharmaceuticals, was completed and data has been submitted to the FDA

●Palatin is in discussions with potential collaboration partners for certain regions outside of the licensed territories of North America, China and South Korea

Anti-Inflammatory / Autoimmune Programs

●Melanocortin Agonists under development for the treatment of inflammatory and autoimmune diseases such as dry eye, uveitis, diabetic retinopathy and inflammatory bowel diseases:

●PL-8177, a selective MC1r peptide agonist:

●Announced positive top line results of an oral clinical study for ulcerative colitis and other inflammatory bowel diseases

●Phase 2 proof-of-concept clinical study with the oral formulation in ulcerative colitis patients anticipated to commence in the fourth quarter of calendar year 2019

●Phase 2 proof-of-concept clinical study with a systemic formulation in non-infectious uveitis (NIU) patients anticipated to commence in the fourth quarter of calendar year 2019

●Continuing investigation of other possible indications for systemic administration

●Program is under internal evaluation for orphan designations

●PL-9643, a melanocortin peptide agonist:

●Continuing with preclinical Investigational New Drug ("IND") enabling activities for ocular diseases

●Program is under internal evaluation for orphan designations

Natriuretic Peptide Receptor ("NPR") System Program

●We have designed and are developing potential NPR candidate drugs that are selective for one or more different natriuretic peptide receptors, including natriuretic peptide receptor-A ("NPR-A"), natriuretic peptide receptor B ("NPR-B"), and natriuretic peptide receptor C ("NPR-C"):

●PL-3994, an NPR-A agonist that has potential utility in treatment of a number of cardiovascular diseases, including genetic and orphan diseases resulting from a deficiency of endogenous active NPR-A:

● Active collaborations with several institutions ongoing

●PL-5028, a dual NPR-A and NPR-C agonist in development for cardiovascular diseases, including reducing cardiac hypertrophy and fibrosis:

●Active collaborations with several institutions ongoing

Genetic Obesity Program

●Melanocortin receptor 4 ("MC4r") peptide PL-8905 and orally-active small molecule PL-9610 under investigation for the treatment of rare genetic metabolic and obesity disorders:

●Program is under internal evaluation for orphan designation

Corporate

●Decreased debt and related liabilities from $7.2 million at June 30, 2018 to $1.8 million at March 31, 2019.

Third Quarter Fiscal 2019 Financial Results
Palatin reported a net loss of $(5.7) million, or $(0.03) per basic and diluted share, for the quarter ended March 31, 2019, compared to a net loss of $(0.7) million, or $(0.00) per basic and diluted share, for the same period in 2018.

The difference in financial results between the three months ended March 31, 2019 and 2018 was mainly attributable to the recognition of $9.0 million in license and contract revenue during the 2018 period pursuant to our license agreement with AMAG.

Revenue
There were no revenues recorded in the quarter ended March 31, 2019.

For the quarter ended March 31, 2018, all the revenue Palatin recognized was related to our license agreement with AMAG.

Operating Expenses
Total operating expenses for the quarter ended March 31, 2019 were $5.8 million compared to $9.5 million for the comparable quarter in 2018. The decrease in operating expenses was mainly attributable to the completion of the Vyleesi Phase 3 clinical trial program and ancillary studies necessary to file the NDA for Vyleesi in March 2018.

Other Income/Expense
Total other income, net was $35,648 for the quarter ended March 31, 2019 compared to total other expense, net of $(0.2) million for the same period in 2018. The difference consisted primarily of the decrease in interest expense related to Palatin’s venture debt.

Income Tax
There was no income tax expense, or benefit, recorded in the quarter ended March 31, 2019.

Pursuant to the license agreements with our Chinese and South Korean licensees, $500,000 and $82,500, respectively, was withheld in accordance with tax withholding requirements in China and the Republic of Korea, respectively, and was recorded as an expense during the fiscal year ended June 30, 2018. For the quarter ended March 31, 2018, Palatin recorded an income tax benefit of $18,746 related to those withholding amounts utilizing an estimated effective annual income tax rate applied to the loss for the quarter and the remaining balance as of March 31, 2018 of $275,111 was included in prepaid expenses and other current assets. Any potential credit to be received by Palatin on its United States tax returns is currently offset by Palatin’s valuation allowance.

Cash Position
Palatin’s cash and cash equivalents were $19.8 million as of March 31, 2019, compared to cash and cash equivalents of $38.0 million at June 30, 2018. Current liabilities were $4.9 million as of March 31, 2019, compared to $10.8 million as of June 30, 2018.

Palatin believes that existing capital resources will be sufficient to fund our planned operations through at least May 31, 2020.

CONFERENCE CALL / WEBCAST
Palatin will host a conference call and webcast on May 9, 2019 at 11:00 a.m. Eastern Time to discuss the results of operations in greater detail and provide an update on corporate developments. Individuals interested in listening to the conference call live can dial 1-800-667-5617 (domestic) or 1-334-323-0509 (international), conference ID 7024541. The webcast and replay can be accessed by logging on to the "Investor/Webcasts" section of Palatin’s website at View Source A telephone and webcast replay will be available approximately one hour after the completion of the call. To access the telephone replay, dial 1-888-203-1112 (domestic) or 1-719-457-0820 (international), passcode 7024541. The webcast and telephone replay will be available through May 16, 2019.

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On May 9, 2019 Sophiris Bio Inc. (NASDAQ: SPHS) (the "Company," "We" or "Sophiris"), a biopharmaceutical company developing topsalysin (PRX302), a first-in-class, pore-forming protein, in late-stage clinical trials for the treatment of patients with urological diseases, reported financial results for the first quarter of 2019 (Press release, Sophiris Bio, MAY 9, 2019, View Source [SID1234536042]).

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"We continue to have dialog with the European Medicines Agency (EMA) regarding the design for a potential Phase 3 clinical trial, which we developed with input from our scientific advisory board for the treatment of localized prostate cancer," said Randall E. Woods, president and CEO of Sophiris. "We anticipate obtaining formal scientific advice from the EMA before the end of the second quarter and look forward to taking the next step in the clinical development of topsalysin for the focal treatment of localized prostate cancer before the end of the year. We have cash runway to continue operations into September and believe that the ideal funding option for a potential registration Phase 3 clinical trial will either be a potential development partnership or other strategic transaction."

Financial Results:

At March 31, 2019, the Company had cash, cash equivalents and securities available-for-sale of $9.0 million and working capital of $4.7 million. The Company expects that its cash and cash equivalents and securities available-for-sale will be sufficient to fund its operations through September 2019, assuming no new clinical trials are initiated and the Company continues operating as a going concern. The Company will require significant funding to advance topsalysin in clinical development. As of March 31, 2019, the outstanding principal balance of the Company’s term loan was $7.0 million. The Company began making principal payments on its term loan in April 2019.

The Company reported a net loss of $2.4 million or ($0.08) per share for the three months ended March 31, 2019, compared to net loss of $3.3 million or ($0.11) per share for the three months ended March 31, 2018.

Research and development expenses

Research and development expenses were $1.6 million for the three months ended March 31, 2019, compared to $3.3 million for the three months ended March 31, 2018. The decrease in research and development costs is primarily attributable to decreases in the costs associated with manufacturing activities for topsalysin, and to a lesser extent, a decrease in clinical costs associated with our Phase 2b clinical trial of topsalysin for localized prostate cancer. Included in the research and development costs for the three months ended March 31, 2019, were costs associated with the completion of a fill finish campaign at commercial scale which produced drug product for future clinical trials. Analysis for release of this recently filled drug product is underway.

General and administrative expenses

General and administrative expenses were $1.3 million for the three months ended March 31, 2019, compared to $1.2 million for the three months ended March 31, 2018.

Gain on revaluation of the warrant liability

Gain on revaluation of the warrant liability was $0.6 million for the three months ended March 31, 2019, compared to $1.4 million for the three months ended March 31, 2018. As the Company’s warrants may require the Company to pay the warrant holder cash under certain provisions of the warrant, the Company accounts for the warrants as a liability, and the Company is required to calculate the fair value of these warrants each reporting date. Certain inputs utilized in the Company’s Black-Scholes fair value calculation may fluctuate in future periods based upon factors which are outside of the Company’s control. A significant change in one or more of these inputs used in the calculation of the fair value may cause a significant change to the fair value of the Company’s warrant liability, which could also result in a material non-cash gain or loss being reported in the Company’s consolidated statement of operations and comprehensive loss.

On May 9, 2019 Progenics Pharmaceuticals, Inc. (Nasdaq:PGNX) reported financial results for the first quarter 2019 and provided a business update (Press release, Progenics Pharmaceuticals, MAY 9, 2019, View Source [SID1234536041]).

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"We are excited to report that the U.S. commercial launch of AZEDRA for the treatment of advanced or metastatic pheochromocytoma and paraganglioma is proceeding well and as expected. We will record our initial AZEDRA revenues in the second quarter," said Mark Baker, Chief Executive Officer of Progenics.

Mr. Baker continued, "We have also made excellent progress across our PSMA-targeted prostate cancer pipeline designed to find, fight and follow cancer. Today we announced that we have initiated our Phase 2 trial for 1095, our small molecule radiotherapeutic, and will begin actively enrolling patients in this important study this quarter. Our PyL program has been attracting increased interest from treating physicians based on the data highlighting PyL’s clinical utility and potential to improve treatment decision making. Our Phase 3 CONDOR study is enrolling ahead of schedule, with more than 50% of patients now enrolled, and the data presented earlier this week at American Urological Association’s Annual Meeting showcased PyL’s significant diagnostic advantages compared to traditional imaging modalities. We continue to partner with leading organizations worldwide, including Curium, in order to maximize the reach of our prostate cancer imaging agents and address unmet needs in the detection and therapeutic management of prostate cancer. We look forward to building on our positive momentum as we diligently execute our strategy to improve the lives of patients we serve and deliver value for our shareholders."

First Quarter and Recent Key Business Highlights

AZEDRA (iobenguane I 131) 555 MBq/mL injection for intravenous use, Ultra-orphan Radiotherapeutic

U.S. Launch of AZEDRA Progressing with 22 Treatment Requests from Patients Received and 12 Centers Throughout the U.S. are Ready to Treat Patients

AZEDRA is the first and only approved therapy in the U.S. for the treatment of adult and pediatric patients 12 years and older with iobenguane scan positive, unresectable, locally advanced or metastatic pheochromocytoma or paraganglioma who require systemic anticancer therapy. As of today, there are 12 multidisciplinary treatment centers across the U.S. activated for patient treatment and 22 treatment requests have been received and patients have been scheduled for treatment.

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Pursuing Regulatory Path for Additional Indications for AZEDRA

The Company plans to meet with the U.S. Food and Drug Administration (FDA) in a life cycle management meeting to discuss a trial to support an expanded label for AZEDRA in multiple MIBG-avid tumors. Feedback from an advisory board meeting with leading physicians in February 2019 indicated support for a clinical study to research the use of AZEDRA in multiple MIBG-avid tumor indications, including gastroenteropancreatic neuroendocrine tumors ("GEP-NETS") and other neuroendocrine tumors ("NETS") given the high unmet medical need.

Upcoming AZEDRA Presentation at ASCO (Free ASCO Whitepaper)

At the upcoming American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2019 Annual Meeting in Chicago, Illinois, the abstract entitled, "Long-term Survival and Safety from a Multi-center, Open-label, Pivotal Phase 2 Study of Iobenguane I 131 in Patients (Pts) with Unresectable, Locally Advanced or Metastatic Pheochromocytoma or Paraganglioma (PPGL)" will be presented in a poster session on June 3, 2019.

Continued Progress Across Entire PSMA-Targeted Prostate Cancer Pipeline

Initiated Phase 2 Trial of 1095

Progenics has initiated a Phase 2 trial of 1095 in combination with enzalutamide in chemotherapy-naïve patients with metastatic castration-resistant prostate cancer (mCRPC) and will begin enrolling patients within the quarter. Progenics’1095 is a small molecule radiotherapeutic designed to selectively bind to the extracellular domain of prostate specific membrane antigen (PSMA), a protein that is highly expressed on prostate cancer cells. Based on the early data in this open label study, the Company will evaluate initiating a pivotal trial of 1095 in 2020.

Leveraging Investigator-Sponsored Studies to Expand and Advance PyL Development

As part of its PyL development strategy, Progenics is leveraging investigator-sponsored studies to expand clinical experience with the imaging agent and further demonstrate the candidate’s broad potential. Recent data from investigator-sponsored studies presented at the American Urological Association meeting and published in The Journal of Nuclear Medicine reinforce the potential of PyL to detect and monitor prostate cancer and inform treatment decisions. Progenics provides PyL to investigators for clinical research purposes via its PyL Access Program and through other initiatives.

Enrollment Ahead of Schedule in Ongoing Phase 3 Trial of PyL

The Company announced in December 2018 that the first patient was dosed in the Phase 3 CONDOR trial evaluating the diagnostic performance and clinical impact of PyL. The Phase 3 CONDOR trial is a multi-center, open label trial that will enroll approximately 200 male patients with biochemical recurrence of prostate cancer in 14 sites in the United States and Canada. Patient enrollment for the trial is ahead of schedule and is expected to be completed in the fourth quarter of 2019. The Company plans to report data in early 2020. An abstract entitled "A Phase 3, Multicenter Study to Assess the Diagnostic Performance and Clinical Impact of 18F-DCFPyL PET/CT in Men with Suspected Recurrence of Prostate Cancer (CONDOR)" will be presented in a poster session at ASCO (Free ASCO Whitepaper) on June 1, 2019.

Curium to Meet with EMA to Advance PyL in Europe

Curium, Progenics’ European PyL partner, has requested a scientific advice meeting with the European Medicines Agency (EMA) to discuss the regulatory path forward for PyL in Europe. Progenics entered into an exclusive license agreement in December 2018 with Curium for the development and commercialization of PyL in Europe in which Progenics would be entitled to royalties on net sales.

RELISTOR, Treatment for Opioid-Induced Constipation (partnered with Bausch Health Companies, Inc.)

First Quarter 2019 RELISTOR Worldwide Net Sales of $27.7 Million

The first quarter 2019 worldwide net sales of RELISTOR, as reported by its partner Bausch Health Companies, Inc. (formerly known as Valeant Pharmaceuticals, Inc.), translated to $4.2 million in royalty revenue for Progenics for the quarter, up 36% over the first quarter of 2018.

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Leronlimab (PRO 140), Monoclonal Antibody for HIV (owned and developed by CytoDyn, Inc.)

CytoDyn Files First of Three Sections of BLA for Leronlimab (PRO 140), an Anti-CCR5 Monoclonal Antibody for HIV Infection; Progenics is Entitled to Royalties and Milestone Payments

CytoDyn announced in March 2019 that it filed its first of three sections of its Biologics License Application (BLA) to the FDA for leronlimab for the treatment of HIV under the Rolling Review process. Leronlimab is a fully humanized, anti-CCR5 monoclonal antibody that Progenics sold to CytoDyn in 2012. Under the terms of the agreement, Progenics is eligible to receive an additional $5.0 million milestone payment upon U.S. or E.U. approval of the sale of the drug, as well as 5% royalty on net sales of the approved product.

First Quarter 2019 Financial Results

First quarter revenue totaled $4.3 million, up from $3.2 million in the first quarter of 2018, reflecting RELISTOR royalty income of $4.2 million compared to $3.1 million in the corresponding period of 2018.

First quarter research and development expenses increased by $4.3 million compared to the corresponding prior year period, resulting primarily from one-time transition costs for the AZEDRA manufacturing site and higher clinical and contract manufacturing costs for PyL. First quarter selling, general and administrative expenses increased by $2.5 million compared to the corresponding prior year period, primarily attributable to higher costs associated with the buildout of the commercial infrastructure to support the launch and distribution of AZEDRA and higher legal expenses. Progenics also recorded non-cash adjustments of $0.9 million in the first quarter 2019, related to changes in the fair value estimate of the contingent consideration liability. For the three months ended March 31, 2019, Progenics recognized interest expense of $1.1 million related to the RELISTOR royalty-backed loan.

Net loss for the first quarter was $18.7 million, or $0.22 per diluted share, compared to net loss of $13.4 million, or $0.19 per diluted share, in the corresponding 2018 period.

Progenics ended the first quarter with cash and cash equivalents of $109.6 million, a decrease of $28.1 million compared to cash and cash equivalents as of December 31, 2018, which includes approximately $10.8 million related to the acquisition, transition and start-up costs of the Somerset manufacturing site for the AZEDRA launch.

Conference Call and Webcast

Progenics will review first quarter 2019 results in a conference call today at 8:30 a.m. EST. To participate, please dial (877) 250-8889 (domestic) or (720) 545-0001 (international) and reference conference ID 5167901. A live webcast will be available in the Media Center of the Progenics website, www.progenics.com, and a replay will be available there for two weeks.

Indication

AZEDRA (iobenguane I 131) is indicated for the treatment of adult and pediatric patients 12 years and older with iobenguane scan positive, unresectable, locally advanced or metastatic pheochromocytoma or paraganglioma who require systemic anticancer therapy.

Important Safety Information

Warnings and Precautions:

Risk from Radiation Exposure: AZEDRA contributes to a patient’s overall long-term radiation exposure. Long-term cumulative radiation exposure is associated with an increased risk for cancer. These risks of radiation associated with the use of AZEDRA are greater in pediatric patients than in adults. Minimize radiation exposure to patients, medical personnel, and household contacts during and after treatment with AZEDRA consistent with institutional good radiation safety practices and patient management procedures.

Myelosuppression: Among the 88 patients who received a therapeutic dose of AZEDRA, 33% experienced Grade 4 thrombocytopenia, 16% experienced Grade 4 neutropenia, and 7% experienced Grade 4 anemia. Five percent of patients experienced febrile neutropenia. Monitor blood cell counts weekly for up to 12 weeks or until levels return to baseline or the normal range. Withhold and dose reduce AZEDRA as recommended in the prescribing information based on severity of the cytopenia.

Secondary myelodysplastic syndrome, leukemia, and other malignancies: Myelodysplastic syndrome (MDS) and acute leukemias were reported in 6.8% of the 88 patients who received a therapeutic dose of AZEDRA. The time to development of MDS or acute leukemia ranged from 12 months to 7 years. Two of the 88 patients developed a non-hematological malignancy.

Hypothyroidism: Hypothyroidism was reported in 3.4% of the 88 patients who received a therapeutic dose of AZEDRA. Initiate thyroid-blocking medications starting at least 1 day before and continuing for 10 days after each AZEDRA dose to reduce the risk of hypothyroidism or thyroid neoplasia. Evaluate for clinical evidence of hypothyroidism and measure thyroid-stimulating hormone (TSH) levels prior to initiating AZEDRA and annually thereafter.

Elevations in blood pressure: Eleven percent of the 88 patients who received a therapeutic dose of AZEDRA experienced a worsening of pre-existing hypertension defined as an increase in systolic blood pressure to ≥160 mmHg with an increase of 20 mmHg or an increase in diastolic blood pressure to ≥ 100 mmHg with an increase of 10 mmHg. All changes in blood pressure occurred within the first 24 hours post infusion. Monitor blood pressure frequently during the first 24 hours after each therapeutic dose of AZEDRA.

Renal toxicity: Of the 88 patients who received a therapeutic dose of AZEDRA, 9% developed renal failure or acute kidney injury and 22% demonstrated a clinically significant decrease in glomerular filtration rate (GFR) measured at 6 or 12 months. Monitor renal function during and after treatment with AZEDRA. Patients with baseline renal impairment may be at greater risk of toxicity; perform more frequent assessments of renal function in patients with mild or moderate impairment. AZEDRA has not been studied in patients with severe renal impairment.

Pneumonitis: Fatal pneumonitis occurred 9 weeks after a single dose in one patient in the expanded access program. Monitor patients for signs and symptoms of pneumonitis and treat appropriately.

Embryo-fetal toxicity: Based on its mechanism of action, AZEDRA can cause fetal harm. Verify pregnancy status in females of reproductive potential prior to initiating AZEDRA. Advise females and males of reproductive potential of the potential risk to a fetus and to use effective contraception during treatment with AZEDRA and for 7 months after the final dose. Advise males with female partners of reproductive potential to use effective contraception during treatment and for 4 months after the final dose.

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Risk of infertility: Radiation exposure associated with AZEDRA may cause infertility in males and females. Radiation absorbed by testes and ovaries from the recommended cumulative dose of AZEDRA is within the range where temporary or permanent infertility can be expected following external beam radiotherapy.

Adverse Reactions:

The most common severe (Grade 3–4) adverse reactions observed in AZEDRA clinical trials (≥ 10%) were lymphopenia (78%), neutropenia (59%), thrombocytopenia (50%), fatigue (26%), anemia (24%), increased international normalized ratio (18%), nausea (16%), dizziness (13%), hypertension (11%), and vomiting (10%). Twelve percent of patients discontinued treatment due to adverse reactions (thrombocytopenia, anemia, lymphopenia, nausea and vomiting, multiple hematologic adverse reactions).

Drug Interactions:

Based on the mechanism of action of iobenguane, drugs that reduce catecholamine uptake or that deplete catecholamine stores may interfere with iobenguane uptake into cells and therefore interfere with dosimetry calculations or the efficacy of AZEDRA. These drugs were not permitted in clinical trials that assessed the safety and efficacy of AZEDRA. Discontinue the drugs listed in the prescribing information for at least 5 half-lives before administration of either the dosimetry dose or a therapeutic dose of AZEDRA. Do not administer these drugs until at least 7 days after each AZEDRA dose.

For important risk and use information about AZEDRA, please see Full Prescribing Information.

To report suspected adverse reactions, contact Progenics Pharmaceuticals, Inc. at 844-668-3950 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

Reference:

AZEDRA prescribing information. New York, NY: Progenics Pharmaceuticals, Inc.; 08 2018 and 07 2018.

About RELISTOR

Progenics has exclusively licensed development and commercialization rights for its first commercial product, RELISTOR, to Bausch Health Companies, Inc. RELISTOR Tablets (450 mg once daily) are approved in the United States for the treatment of opioid-induced constipation (OIC) in patients with chronic non-cancer pain. RELISTOR Subcutaneous Injection (12 mg and 8 mg) is a treatment for OIC approved in the United States and worldwide for patients with advanced illness and chronic non-cancer pain.

IMPORTANT SAFETY INFORMATION – RELISTOR (methylnaltrexone bromide) tablets, for oral use and RELISTOR (methylnaltrexone bromide) injection, for subcutaneous use

RELISTOR tablets and injection are contraindicated in patients with known or suspected gastrointestinal obstruction and patients at increased risk of recurrent obstruction, due to the potential for gastrointestinal perforation.

Cases of gastrointestinal perforation have been reported in adult patients with opioid-induced constipation and advanced illness with conditions that may be associated with localized or diffuse reduction of structural integrity in the wall of the gastrointestinal tract (e.g., peptic ulcer disease, Ogilvie’s syndrome, diverticular disease, infiltrative gastrointestinal tract malignancies or peritoneal metastases). Take into account the overall risk-benefit profile when using RELISTOR in patients with these conditions or other conditions which might result in impaired integrity of the gastrointestinal tract wall (e.g., Crohn’s disease). Monitor for the development of severe, persistent, or worsening abdominal pain; discontinue RELISTOR in patients who develop this symptom.

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If severe or persistent diarrhea occurs during treatment, advise patients to discontinue therapy with RELISTOR and consult their healthcare provider.

Symptoms consistent with opioid withdrawal, including hyperhidrosis, chills, diarrhea, abdominal pain, anxiety, and yawning have occurred in patients treated with RELISTOR. Patients having disruptions to the blood-brain barrier may be at increased risk for opioid withdrawal and/or reduced analgesia and should be monitored for adequacy of analgesia and symptoms of opioid withdrawal.

Avoid concomitant use of RELISTOR with other opioid antagonists because of the potential for additive effects of opioid receptor antagonism and increased risk of opioid withdrawal.

The use of RELISTOR during pregnancy may precipitate opioid withdrawal in a fetus due to the immature fetal blood brain barrier and should be used during pregnancy only if the potential benefit justifies the potential risk to the fetus. Because of the potential for serious adverse reactions, including opioid withdrawal, in breastfed infants, advise women that breastfeeding is not recommended during treatment with RELISTOR. In nursing mothers, a decision should be made to discontinue nursing or discontinue the drug, taking into account the importance of the drug to the mother.

A dosage reduction of RELISTOR tablets and RELISTOR injection is recommended in patients with moderate and severe renal impairment (creatinine clearance less than 60 mL/minute as estimated by Cockcroft-Gault). No dosage adjustment of RELISTOR tablets or RELISTOR injection is needed in patients with mild renal impairment.

A dosage reduction of RELISTOR tablets is recommended in patients with moderate (Child-Pugh Class B) or severe (Child-Pugh Class C) hepatic impairment. No dosage adjustment of RELISTOR tablets is needed in patients with mild hepatic impairment (Child-Pugh Class A). No dosage adjustment of RELISTOR injection is needed for patients with mild or moderate hepatic impairment. In patients with severe hepatic impairment, monitor for methylnaltrexone-related adverse reactions.

In the clinical studies, the most common adverse reactions were:

OIC in adult patients with chronic non-cancer pain

RELISTOR tablets (≥ 2% of RELISTOR patients and at a greater incidence than placebo): abdominal pain (14%), diarrhea (5%), headache (4%), abdominal distention (4%), vomiting (3%), hyperhidrosis (3%), anxiety (2%), muscle spasms (2%), rhinorrhea (2%), and chills (2%).

RELISTOR injection (≥ 1% of RELISTOR patients and at a greater incidence than placebo): abdominal pain (21%), nausea (9%), diarrhea (6%), hyperhidrosis (6%), hot flush (3%), tremor (1%), and chills (1%).

OIC in adult patients with advanced illness

RELISTOR injection (≥ 5% of RELISTOR patients and at a greater incidence than placebo): abdominal pain (29%) flatulence (13%), nausea (12%), dizziness (7%), and diarrhea (6%).