On May 9, 2019 Heron Therapeutics, Inc. (Nasdaq: HRTX), a commercial-stage biotechnology company focused on improving the lives of patients by developing best-in-class treatments to address some of the most important unmet patient needs, reported financial results for the three months ended March 31, 2019 and highlighted recent corporate progress (Press release, Heron Therapeutics, MAY 9, 2019, View Source [SID1234536048]).

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Recent Corporate Progress

Pain Management Franchise

Complete Response Letter Received from the FDA Regarding the NDA for HTX-011: A Complete Response Letter (CRL) was received from the U.S. Food and Drug Administration (FDA) on April 30, 2019 regarding the Company’s New Drug Application (NDA) for HTX-011 for postoperative pain management. The CRL stated that the FDA is unable to approve the NDA in its present form based on the need for additional Chemistry, Manufacturing and Controls (CMC) and non-clinical information. Based on the complete review of the NDA, the FDA did not identify any clinical safety or efficacy issues, and there is no requirement for further clinical studies or data analyses.

European Medicines Agency Validation of Marketing Authorisation Application for HTX-011 for Postoperative Pain Management: In April of 2019, Heron announced that the Marketing Authorisation Application (MAA) for its investigational agent, HTX-011, for postoperative pain management, was validated by the European Medicines Agency (EMA). The EMA granted eligibility to the Centralised Procedure for HTX-011 based on it meeting the criteria of a medicinal product constituting a significant scientific innovation. The Centralised Procedure allows applicants to receive a marketing authorisation that is valid throughout the European Union (EU). With the validation of the MAA, an opinion from the EMA Committee for Medicinal Products for Human Use (CHMP) is anticipated in the first half of 2020.

77% of Patients Treated with HTX-011 Remain Opioid-Free 72 Hours Post-Surgery in Multi-center Clinical Study in Bunionectomy: In March of 2019, we reported positive topline results of a multi-center postoperative pain management study in which 31 patients undergoing bunionectomy surgery received HTX-011 together with a regimen of generic, over-the-counter, oral analgesics (acetaminophen and ibuprofen). Seventy-seven percent (77%) of patients were opioid-free 72 hours post-surgery, and 100% of these patients remained opioid-free 28 days post-surgery. Patients mean pain scores stayed in the mild range through 72 hours.

90% of Patients Treated with HTX-011 Remain Opioid-Free 72 Hours Post-Surgery in Multi-center Clinical Study in Hernia Repair: In January of 2019, we reported positive topline results of a multi-center postoperative pain management study in which 63 patients undergoing hernia repair surgery received HTX-011 together with a regimen of generic, over-the-counter, oral analgesics (acetaminophen and ibuprofen). Ninety percent (90%) of patients were opioid-free 72 hours post-surgery, and 81% were still opioid-free 28 days post-surgery.
CINV Franchise

FDA Approval of sNDA to Expand CINVANTI Label for IV Push: In February of 2019, the FDA approved Heron’s supplemental New Drug Application (sNDA) for CINVANTI (aprepitant) injectable emulsion, for intravenous (IV) use. The sNDA requested FDA approval to expand the administration of CINVANTI beyond the already approved administration method (a 30-minute IV infusion) to include a 2-minute IV injection.

First Quarter 2019 Net Product Sales: First-quarter 2019 net product sales for the chemotherapy-induced nausea and vomiting (CINV) franchise were $31.6 million, up 173% and 10% from the first and fourth quarters of 2018, respectively. This included net product sales of $28.0 million for CINVANTI injectable emulsion and $3.6 million for SUSTOL (granisetron) extended-release injection. Heron reaffirms full-year 2019 net product sales guidance for the CINV franchise of $115 million to $120 million.
"We are focused on resubmitting the NDA for HTX-011 as soon as possible to bring this important medicine to market to help patients manage their postoperative pain without the need for opioids," said Barry Quart, Pharm.D., President and Chief Executive Officer of Heron. "Our CINV franchise remains strong, highlighted by our strong net product sales in the first quarter and our label expansion for CINVANTI for IV push."

Financial Results

Net product sales for the three months ended March 31, 2019 were $31.6 million compared to $11.6 million for the same period in 2018.

Heron’s net loss for the three months ended March 31, 2019 was $63.0 million, or $0.80 per share, compared to $52.3 million, or $0.81 per share for the same period in 2018. Net loss for the three months ended March 31, 2019 included non-cash, stock-based compensation expense of $17.9 million compared to $7.7 million for the same period in 2018.

As of March 31, 2019, Heron had cash, cash equivalents and short-term investments of $289.2 million, compared to $332.4 million as of December 31, 2018. Net cash used for operating activities for the three months ended March 31, 2019 was $49.0 million compared to $61.7 million for the same period in 2018. Heron expects to end the year with more than $190 million in cash, cash equivalents and short-term investments.

About HTX-011 for Postoperative Pain

HTX-011, which utilizes Heron’s proprietary Biochronomer drug delivery technology, is an investigational, long-acting, extended-release formulation of the local anesthetic bupivacaine in a fixed-dose combination with the anti-inflammatory meloxicam for the management of postoperative pain. By delivering sustained levels of both a potent anesthetic and a local anti-inflammatory agent directly to the site of tissue injury, HTX-011 was designed to deliver superior pain relief while reducing the need for systemically administered pain medications such as opioids, which carry the risk of harmful side effects, abuse and addiction. HTX-011 has been shown to reduce pain significantly better than placebo or bupivacaine solution in five diverse surgical models: hernia repair, abdominoplasty, bunionectomy, total knee arthroplasty and breast augmentation. HTX-011 was granted Fast Track designation from the FDA in the fourth quarter of 2017 and Breakthrough Therapy designation in the second quarter of 2018. Heron submitted an NDA to the FDA for HTX-011 in October of 2018 and received Priority Review designation in December of 2018. A CRL was received from the FDA regarding the NDA for HTX-011 on April 30, 2019 relating to CMC and non-clinical information. No issues related to clinical efficacy or safety were noted. An MAA for HTX-011 was validated by the EMA in March 2019 for review under the Centralised Procedure.

About CINVANTI (aprepitant) injectable emulsion

CINVANTI, in combination with other antiemetic agents, is indicated in adults for the prevention of acute and delayed nausea and vomiting associated with initial and repeat courses of highly emetogenic cancer chemotherapy (HEC), including high-dose cisplatin, and nausea and vomiting associated with initial and repeat courses of moderately emetogenic cancer chemotherapy (MEC). CINVANTI is an IV formulation of aprepitant, a substance P/neurokinin-1 (NK1) receptor antagonist (RA). CINVANTI is the first IV formulation to directly deliver aprepitant, the active ingredient in EMEND capsules. Aprepitant (including its prodrug, fosaprepitant) is the only single-agent NK1 RA to significantly reduce nausea and vomiting in both the acute phase (0 – 24 hours after chemotherapy) and the delayed phase (24 – 120 hours after chemotherapy). CINVANTI is the only IV formulation of an NK1 RA indicated for the prevention of acute and delayed nausea and vomiting associated with HEC and nausea and vomiting associated with MEC that is free of polysorbate 80 or any other synthetic surfactant. The FDA-approved dosing administration included in the United States prescribing information for CINVANTI is a 30-minute infusion or a 2-minute injection.

Please see full prescribing information at www.CINVANTI.com.

About SUSTOL (granisetron) extended-release injection

SUSTOL is indicated in combination with other antiemetics in adults for the prevention of acute and delayed nausea and vomiting associated with initial and repeat courses of moderately emetogenic chemotherapy (MEC) or anthracycline and cyclophosphamide (AC) combination chemotherapy regimens. SUSTOL is an extended-release, injectable 5-HT3 receptor antagonist that utilizes Heron’s Biochronomer drug delivery technology to maintain therapeutic levels of granisetron for ≥5 days. The SUSTOL global Phase 3 development program was comprised of two, large, guideline-based clinical studies that evaluated SUSTOL’s efficacy and safety in more than 2,000 patients with cancer. SUSTOL’s efficacy in preventing nausea and vomiting was evaluated in both the acute phase (0 – 24 hours after chemotherapy) and delayed phase (24 – 120 hours after chemotherapy).

On May 9, 2019 Intrexon Corporation (NASDAQ: XON), a leader in the engineering and industrialization of biology to improve the quality of life and health of the planet, reported its first quarter financial results for 2019 (Press release, Intrexon, MAY 9, 2019, View Source [SID1234536047]).

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Recent Business Highlights:

Intrexon announced alignment of its operations into two units, Intrexon Health and Intrexon Bioengineering, to better deploy resources, realize inherent synergies and position the company for growth with a core focus on healthcare. Intrexon Health, will be led by Randal J. Kirk, Chairman and Chief Executive Officer of Intrexon while Intrexon Bioengineering will be led by LTG (Ret.) Thomas Bostick, PhD, PE, Chief Operating Officer of Intrexon, who will also assume the title of President, Intrexon Bioengineering; and

As Intrexon announced previously, the Company believes it will end the year with approximately the same net cash and short-term investment position that it held on April 3, 2019. The Company is making progress toward this goal through a combination of partnering, asset sales and operating cost reductions. The Company has initiated plans to reduce its original 2019 operating budget by approximately $70 million and commenced actions toward achieving this goal.

Intrexon Health

Precigen, Inc., a wholly-owned subsidiary of Intrexon, opened its new nearly 5,000 square foot facility in Germantown, Maryland to support gene therapy manufacturing. The U.S. Food and Drug Administration (FDA) good manufacturing practices (GMP) compliant facility was designed with agility and control in mind, focusing on rapid manufacturing and the ability to scale production appropriately to meet early stage clinical trial needs;

Intrexon and its majority-owned subsidiary Triple-Gene LLC (formerly Xogenex LLC) completed enrollment in the first cohort of a Phase 1 clinical trial of INXN-4001, an investigational new drug which is the world’s first triple effector gene drug candidate being evaluated for the treatment of heart failure; and

Intrexon continues advancing its proprietary yeast-based platform for robust production of cannabinoids with consistent yield and purity and with production of CBGA at 700 mg/L in stirred tank fermenter. The platform is on track to realize a target cost of goods of <$1000 USD/kg.

Intrexon Bioengineering

Intrexon entered into a strategic licensing agreement with Surterra Wellness to utilize Intrexon’s Botticelli next generation plant propagation platform to enable rapid production of Surterra’s proprietary cannabis cultivars for the Florida market;

Oxitec, Ltd., a wholly-owned subsidiary of Intrexon, signed two, multi-year development agreements with a collaborator to apply its 2nd generation, self-limiting technology to develop solutions for the soybean looper and fall armyworm, two insect pests that cause significant damage to agricultural crops globally; and

AquaBounty Technologies, Inc. (NASDAQ: AQB), an investment of Intrexon, announced the FDA lifted the Import Alert on AquAdvantage Salmon (AAS) in March. Environment and Climate Change Canada has also approved the company’s Rollo Bay production facility for the commercial manufacture and grow-out of AAS.

First Quarter 2019 Financial Highlights:

Total revenues of $23.3 million;

Net loss of $60.7 million attributable to Intrexon, or $(0.40) per basic share, including non-cash charges of $17.8 million;

Adjusted EBITDA of $(39.2) million, or $(0.26) per basic share; and

Cash, cash equivalents, and short-term investments totaled $181.6 million and the value of common equity securities totaled $1.8 million at March 31, 2019.

"Our realignment into Intrexon Health and Intrexon Bioengineering is well underway," commented Mr. Kirk. "On the healthcare side it is satisfying and exciting for all of us that we currently are dosing in clinical trials several novel therapeutic candidates of our design that incorporate our unique technology. Moreover, as we prepare to get underway with patient dosing of two Ultra-CAR-T candidates, we see the potential realization of a set of aspirations that we have held — and declared — since before our IPO and believe that these should constitute major advances in immuno-oncology. We await data with great anticipation."

Mr. Kirk concluded, "Our share price carries implications as well, so we are determined to prosecute all that we have set ourselves to accomplish in healthcare while realizing maximum value for our shareholders from our non-healthcare assets, the portfolio of Intrexon Bioengineering. These realizations may be participating or transactional in nature, but we are determined to achieve them and indeed are currently advancing several such prospects. Combined with the cost-cutting program that we have implemented, we expect our company will be a leaner, more-focused enterprise but not one lacking ambition or impact."

"Intrexon Bioengineering’s highly skilled teams are contributing their breadth of expertise across diverse fields to advance programs in food, agriculture, environmental, and industrial fields. We believe these programs offer a unique opportunity to realize the potential of engineered solutions in generating more efficient and sustainable approaches than current industry practices. Intrexon Bioengineering is focused on delivering these technologies and products with the goal of increasing shareholder value," added Dr. Bostick.

First Quarter 2019 Financial Results Compared to Prior Year Period

Total revenues decreased $16.3 million from the quarter ended March 31, 2018. Collaboration and licensing revenues decreased $13.9 million from the quarter ended March 31, 2018 primarily due to the reacquisition of rights previously licensed to certain significant collaborators, including ZIOPHARM Oncology, Inc., and ARES Trading S.A., the result of which eliminated or substantially reduced revenues generated from those collaborations. The decline was also attributable to the mutual termination of the Company’s ECC with OvaScience, Inc., in March 2018. Product revenues decreased $2.3 million, or 32%, primarily due to lower customer demand for pregnant cows and cloned products. Gross margin on products declined in the current period as a result of fewer products sold and increased costs associated with new product offerings. Service revenues decreased $0.9 million, or 7%. The decrease in service revenues and the gross margin thereon relates to fewer embryos produced per bovine in vitro fertilization cycle performed as a result of unfavorable production results.

Research and development expenses decreased $4.2 million, or 11%. Research and development depreciation and amortization expense decreased $2.0 million primarily due to intangible assets that were impaired or abandoned in 2018. Research and development salaries, benefits and other personnel costs decreased $1.3 million primarily due to the closing of one of the Company’s research

and development facilities in Brazil in 2018. Selling, general and administrative (SG&A) expenses decreased $6.1 million, or 16%. SG&A salaries, benefits and other personnel costs decreased $4.8 million primarily due to decreased compensation expenses related to performance and retention incentives for SG&A employees as well as decreased share-based compensation expense as a result of certain 2014 stock option grants becoming fully vested in March 2018.

The Company will not host a conference call associated with this financial results release.

On May 9, 2019 Adamis Pharmaceuticals Corporation (NASDAQ: ADMP) reported financial results for the first quarter ended March 31, 2019 and provided a business update (Press release, Adamis Pharmaceuticals, MAY 9, 2019, View Source [SID1234536046]).

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Dr. Dennis J. Carlo, President and Chief Executive Officer of Adamis Pharmaceuticals, stated, "With the launch of our SYMJEPITM product this year, I believe Adamis is transitioning from a development-stage company with product candidates in the pipeline, into a commercial-stage company with multiple sources of revenue. Currently, I anticipate revenues from three sources for our company. First, SYMJEPI sales, mainly coming from the Sandoz retail launch, which we believe will occur shortly; second, sales from US Compounding, which have been steadily increasing; and finally, upfront payment and sales from anticipated commercialization arrangements relating to our naloxone product candidate. With all of the possible developments and potential revenue streams, I believe 2019 will be a very good year for Adamis."

Product Updates

SYMJEPI (epinephrine) Injection (0.3mg and 0.15mg)

On January 16, 2019, we announced that Sandoz had launched SYMJEPI (epinephrine) 0.3 mg Injection in the U.S. market. SYMJEPI will be rolled out via a phased launch and will initially be available in the institutional setting, an established channel where Sandoz has a significant experience and knowledge, followed by anticipated introduction into the retail market. We also anticipate that Sandoz will launch the lower dose SYMJEPI (epinephrine) 0.15 mg Injection product in the U.S. markets. With the continued rollout of SYMJEPI into the retail market, the company anticipates that the revenue stream to Adamis will increase in future quarters.

APC-6000 (naloxone)

On March 14, 2019, Adamis announced that the FDA had accepted the company’s New Drug Application (NDA) for review and provided a target agency action date of October 31, 2019. Naloxone is an opioid antagonist used to treat narcotic overdoses. The company believes that its higher dose naloxone product candidate, if approved, could be an important part of the solution to this growing health crisis. The company is in discussions with several potential commercial partners for the naloxone injection product candidate.

Other Pipeline Products

In order to focus resources on the naloxone product candidate, and in an effort to reduce operating expenses, the company has slowed development of its other pipeline product candidates for the near term. This includes a delay in the continuation of the start of patient enrollment for the company’s Phase 3 study for beclomethasone HFA (APC-1000) and a hold on the company’s sublingual tadalafil (APC-8000) product candidate. The company will prioritize future development for these products based on the availability of capital to support them and its ongoing evaluation of commercial potential.

Drug Outsourcing Facility

During the first quarter of 2019, the company continued to make changes in its wholly-owned subsidiary, US Compounding (USC), including the elimination of many lower margin products, reductions in operating cost and overhead, changes to senior leadership, and investments in and improvements to manufacturing processes, with the goals of improving overall efficiency, reducing operating expenses, and improving margins.

First Quarter 2019 Financial Results

Revenues were approximately $4.9 million and $3.2 million for the three months ended March 31, 2019 and 2018, respectively. Revenues increased by approximately $1.7 million in the first quarter of 2019 compared to the comparable period of 2018. This represents an approximate 18% increase over the last quarter of 2018 and an approximate 53% increase over the first quarter of 2018. The increase in revenues reflected the continued growth in sales of the USC division and, in part, revenues from the initial launch of SYMJEPI.

Selling, general and administrative expenses ("SG&A") for the first quarter increased approximately 23% over the first quarter of 2018, which were approximately $8.0 million and $6.5 million respectively. The single largest contributor to this increase was the initial annual maintenance fee paid to the FDA for SYMJEPI and the balance was due to an increase in direct materials, supplies and obsolete inventory caused by an increase in production of SYMJEPI and at US Compounding.

Research and development expenses for the first quarter of 2019 decreased $5.6 million from the fourth quarter of 2018. The bulk of this reduction was due to the completion of two late stage development projects for the company’s naloxone injection (APC-6000) and the sublingual tadalafil (APC-8000) product candidates.

Cash and equivalents at the end of the first quarter was $9.2 million. The company’s goal for the last three quarters of 2019 is to keep net cash used in operating and investing activities in the range of approximately $9-12 million. The company currently believes that the combination of reduced spending, cash on hand, an increase in cash flows from Sandoz and USC, and anticipated licensing fees or payments if we enter into a commercialization agreement relating to the naloxone product, would provide sufficient funding for the company through the end of 2019.

Targeted Future Milestones

●Launch by Sandoz of the SYMJEPI 0.3mg and 0.15mg products for the U.S. retail market;
●FDA approval for the higher dose naloxone product candidate – target agency action date of October 31, 2019;
●Commercial agreements for the naloxone product candidate and for the SYMJEPI products outside of the U.S.; and
●US Compounding becoming net positive for Adamis in 2019.

Conference Call

Adamis will host a conference call and live webcast on Thursday, May 9, 2019 at 2:00 pm Pacific Time to discuss its financial and operating results for the first quarter 2019 as well as provide an update on business developments and activities.

US Dial-in (Toll Free): 1-800-458-4148

TOLL/International Dial-in: 1-323-794-2597

Conference ID: 1311319

Webcast: View Source

In addition, a telephone playback of the call will be available after approximately 5:00 pm PT on May 9, 2019. To listen to the replay, call toll free 1-844-512-2921 within the United States or 1-412-317-6671 when calling internationally (toll). Please use the replay PIN number 1311319.

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.