On May 10, 2022 Epizyme (Nasdaq: EPZM), a fully integrated, commercial-stage biopharmaceutical company developing and delivering transformative therapies for cancer patients against novel epigenetic targets, reported first quarter 2022 financial results and provided a business update (Press release, Epizyme, MAY 10, 2022, View Source [SID1234614098]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"The relapsed or refractory follicular lymphoma treatment landscape is rapidly changing, and we believe TAZVERIK is poised to grow at an accelerated rate as the year progresses. We saw encouraging progress in important commercial metrics during the first quarter despite some seasonal impact related in part to the Medicare Part D drug benefit design and year-end prescription variability, which we continue to evaluate. TAZVERIK demand experienced a strong rebound in March, and we entered the second quarter with positive momentum. We believe that TAZVERIK has the potential to reach many more patients based on changes in the current treatment options for patients in the R/R FL market and the updated NCCN Guidelines for FL," said Grant Bogle, President and Chief Executive Officer. "We are also continuing to advance our key clinical programs and look forward to sharing updated SYMPHONY-1 data from the Phase 1b cohort at ASCO (Free ASCO Whitepaper)."

Recent Progress

TAZVERIK (tazemetostat) commercial progress:

TAZVERIK generated net product revenue of $8.7 million for the first quarter of 2022, including $0.5 million related to the sale of TAZVERIK commercial product for third-party pharmaceutical company use in clinical trials. TAZVERIK commercial net sales in the first quarter of 2022 were $8.1 million, representing an increase of approximately 10% when compared to $7.4 million in the fourth quarter of 2021.

Commercial demand increased 16% in the first quarter of 2022 versus the fourth quarter of 2021 levels while total demand (commercial demand and free goods supplied through the patient assistance program) in the first quarter of 2022 was similar to fourth quarter 2021 levels. The Company believes the difference in total demand as compared with commercial demand was related, in part, to limitations of the Medicare Part D drug benefit design and year-end prescription variability, which followed a similar pattern in 2021. While total demand was soft in the beginning of the quarter, it rebounded in March to its highest monthly level since launch. Additional time is needed to fully evaluate and understand seasonality and fluctuations.

Recent market research suggests that TAZVERIK market share continues to grow in the third-line setting for both EZH2 mutation-positive and wild-type populations, consistent with the Company’s commercial focus and messaging. The amount of free goods supplied to patients through Epizyme’s patient assistance program represented approximately 15% of total demand for the first quarter of 2022. This rate was consistent with the first quarter of 2021.

National Comprehensive Cancer Network (NCCN) released updated NCCN Guidelines for B-Cell Lymphomas: The recently updated NCCN Guidelines for grade 1-2 follicular lymphoma (FL) now include tazemetostat as a suggested treatment regimen in the second line for elderly or infirm patients with EZH2 wild type or unknown relapsed/refractory (R/R) disease in patients who have no satisfactory alternative treatment options. For third-line and subsequent therapy, tazemetostat is a suggested treatment regimen for patients with EZH2 mutation-positive disease or patients with EZH2 wild-type or unknown R/R disease who have no satisfactory alternative treatment options.

Global enrollment open and actively screening in the randomized Phase 3 portion of SYMPHONY-1 (EZH-302): Dosing of the first patient was recently completed in the Phase 3 portion of the SYMPHONY-1 study, and the study is open globally and is actively screening and enrolling patients. SYMPHONY-1 is the confirmatory study assessing tazemetostat in combination with rituximab + lenalidomide (R2) compared with R2 plus placebo in patients with R/R FL previously treated with at least one systemic therapy, including those who are rituximab-refractory and/or have experienced progression of disease within two years (POD24). Updated data from the Phase 1b portion of SYMPHONY-1 was accepted for a poster presentation, which will be shared at the upcoming American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting in Chicago from June 3-7. Data to be presented include updated overall response rate and complete response rate data as well as a subgroup analysis of rituximab-refractory and POD24 patients. The Company continues to follow this Phase 1b cohort of patients and plans to present additional updated data later this year.

LYSA Phase 2 study enrollment nearly complete; top-line results expected in second half of 2022: Enrollment in the FL arm is complete for the Phase 2 portion of the Lymphoma Study Association (LYSA) study, a Phase 1b/2 combination study of tazemetostat with R-CHOP in high-risk, front-line FL and diffuse large B-cell lymphoma (DLBCL) patients. Epizyme, in collaboration with LYSA, anticipates presenting top-line results from the Phase 2 portion of the study in the second half of 2022.

CELLO-1 Phase 2 study 85% enrolled; updated safety run-in data and interim data from the Phase 2 portion of the study expected in second half of 2022: The Phase 2 portion of the CELLO-1 study (EZH-1101), which is evaluating tazemetostat plus enzalutamide compared to enzalutamide monotherapy in metastatic castration-resistant prostate cancer patients, is approximately 85% enrolled toward a target of 80 patients. In 2022, Epizyme expects to complete enrollment in the randomized Phase 2 portion of the study and present updated data from the safety run-in portion as well as interim safety and efficacy data from the Phase 2 portion of the study in the second half of the year.

We continue to screen patients in ARIA (EZH-1501), the Phase 1b/2 tazemetostat hematological basket study, and SET-101, the Phase 1/1b study of EZM0414. Epizyme plans to provide updates on these programs in the second half of 2022.

First Quarter 2022 Financial Results

Cash Position: Cash, cash equivalents and marketable securities were $199.7 million as of March 31, 2022, compared to $176.8 million as of December 31, 2021.

Revenue: Total revenue was $8.7 million for the first quarter of 2022, an increase of 14% vs. $7.6 million for the first quarter of 2021. Net product revenue of TAZVERIK in the U.S was $8.7 million for the first quarter of 2022, an increase of 40% vs. $6.2 million for the first quarter of 2021.

Operating Expenses: Total GAAP operating expenses were $59.6 million for the first quarter of 2022, a decrease of 17% vs. $72.0 million for the first quarter of 2021, reflecting focused efforts on streamlining operations. Total non-GAAP adjusted operating expenses were $53.0 million for the first quarter of 2022, compared to $63.7 million for the first quarter of 2021.

R&D expenses: GAAP R&D expenses were $29.8 million for the first quarter of 2022, compared to $32.7 million for the first quarter of 2021. Non-GAAP adjusted R&D expenses were $27.8 million for the first quarter of 2022, compared to $30.3 million for the first quarter of 2021.

SG&A expenses: GAAP SG&A expenses were $27.2 million for the first quarter of 2022, compared to $36.4 million for the first quarter of 2021, representing a 25% decrease following the previously announced operating expense and workforce reductions. Non-GAAP adjusted SG&A expenses were $23.6 million for the first quarter of 2022, compared to $31.5 million for the first quarter of 2021.

Net Loss (GAAP): Net loss attributable to common stockholders was $55.5 million, or $0.38 per share, for the first quarter of 2022, compared to $70.3 million, or $0.69 per share, for the first quarter of 2021.

A reconciliation of non-GAAP adjusted financial measures directly comparable to GAAP financial measures is presented in the table attached to this press release.

Conference Call Information

Epizyme will host a conference call today, May 10, at 8:30 a.m. ET. To participate, please dial (877) 844-6886 (domestic) or (970) 315-0315 (international) and refer to conference ID 5369344. A webcast, as well as supplemental slides to support the webcast, will be available in the investor section of the Company’s website at www.epizyme.com, and will be archived for 60 days following the call.

About Non-GAAP Financial Measures

In addition to financial information prepared in accordance with the U.S. generally accepted accounting principles (GAAP), this press release includes the following non-GAAP financial measures: total non-GAAP adjusted operating expenses on a historical basis, non-GAAP adjusted R&D expenses on a historical basis and non-GAAP adjusted SG&A expenses on a historical basis. Epizyme derives these non-GAAP financial measures by excluding certain expenses and other items from the respective GAAP financial measure, that is most directly comparable to each non-GAAP financial measure. Specifically, the non-GAAP financial measures exclude stock-based compensation expense and depreciation and amortization of intangibles. The Company’s management believes that these non-GAAP financial measures are useful to both management and investors in analyzing its ongoing business and operating performance. Management does not intend the presentation of these non-GAAP financial measures to be considered in isolation or as a substitute for results prepared in accordance with GAAP, but as a complement to provide greater transparency. In addition, these non-GAAP financial measures may differ from similarly named measures used by other companies.

About TAZVERIK (tazemetostat)

TAZVERIK is a methyltransferase inhibitor indicated for the treatment of:

Adults and pediatric patients aged 16 years and older with metastatic or locally advanced epithelioid sarcoma not eligible for complete resection.

Adult patients with relapsed or refractory follicular lymphoma whose tumors are positive for an EZH2 mutation as detected by an FDA-approved test and who have received at least two prior systemic therapies.

Adult patients with relapsed or refractory follicular lymphoma who have no satisfactory alternative treatment options.

These indications are approved under accelerated approval based on overall response rate and duration of response. Continued approval for these indications is contingent upon verification and description of clinical benefit in confirmatory studies.

The most common (≥20%) adverse reactions in patients with epithelioid sarcoma are pain, fatigue, nausea, decreased appetite, vomiting and constipation. The most common (≥20%) adverse reactions in patients with follicular lymphoma are fatigue, upper respiratory tract infection, musculoskeletal pain, nausea and abdominal pain.

View the U.S. Full Prescribing Information here: Epizyme.com.

About EZM0414

EZM0414 is a potent selective, oral, small molecule, investigational drug agent that inhibits the histone methyltransferase, SETD2, which plays a role in oncogenesis. SETD2 methylates histone as well as non-histone proteins, and this activity is involved in several key biological processes including transcriptional regulation, RNA splicing, and DNA damage repair. Based on the preclinical data on SETD2 inhibition by EZM0414 in multiple settings, including high risk t(4;14) multiple myeloma (MM) and in other B-cell malignancies such as diffuse large B-cell lymphoma (DLBCL), the Company is conducting SET-101, a Phase 1/1b study of EZM0414, for the treatment of adult patients with relapsed or refractory MM and DLBCL.

On May 9, 2022 Horizon Therapeutics (Nasdaq: HZNP) and the Chicago Sky reported a multi-year partnership, which includes robust community programming as well as marketing and branding assets (Press release, Horizon Therapeutics, MAY 10, 2022, View Source [SID1234614114]). As part of the partnership, Horizon will be the presenting sponsor of the newly unveiled Chicago Sky Basketball Academy, as well as one of twelve presenting sponsors of the Sky’s social justice program, Beyond Basketball, which focuses on economic development, youth financial literacy, voting rights and food deserts. The partnership will also include rehabbing a basketball court in an underserved community each season.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"We are proud to partner with the Chicago Sky, an organization that shares our commitment to empowering people in communities throughout the Chicagoland area, home to our U.S. headquarters," said Tim Walbert, chairman, president and chief executive officer, Horizon. "I look forward to building meaningful community initiatives together that advance the understanding and dialogue around pressing societal issues, including education equity and social justice."

"Horizon Therapeutics has changed lives through purposeful and transformative work in science and we’re thrilled to promote the company’s work through this groundbreaking partnership," said Sky CEO and President Adam Fox. "The Sky organization is fortunate to have a committed partner to help us broaden interest in the WNBA, to advance social justice campaigns, and create meaningful change in our communities."

This represents the most recent sports marketing partnership in the Chicagoland area for Horizon, which also includes partnerships with the Chicago Cubs, Chicago Bulls and Chicago Bears. Horizon was also recently named the title partner of the Irish Open in a six-year deal, which will begin with the 2022 Horizon Irish Open at Mount Juliet Estate in County Kilkenny, Ireland, in late June.

On May 10, 2022 Innate Pharma SA (Euronext Paris: IPH; Nasdaq: IPHA) ("Innate" or the "Company") reported its consolidated financial results for the quarter ending March 31, 2022 (Press release, Innate Pharma, MAY 10, 2022, View Source [SID1234614130]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"Again this quarter we made significant progress in our pipeline in particular with the presentation of positive data and clinical progress with our anti-NKG2A, monalizumab. We also saw a $50 million milestone from AstraZeneca triggered due to the first patient dosed in April in the Phase 3 lung cancer trial. This means our cash position is considerably strengthened to fund our pipeline ambitions into 2024," said Mondher Mahjoubi, Chief Executive Officer of Innate Pharma. "We look forward to additional clinical milestones this year from our broad antibody pipeline, specifically readouts for lacutamab in the second half and further progress on ANKETTM, as we leverage scientific expertise and strong partnerships to deliver innovative treatments for people with cancer."

Webcast and conference call will be held today at 2:00pm CEST (8:00am EDT)

The live webcast will be available at the following link:
View Source

Participants may also join via telephone using the dial-in details below:
France: 0805 620 704
United States: 1 844 200 6205 / 1 646 904 5544
United Kingdom: 44 208 0682 558
All other locations: +1 929 526 1599
Access code: 051477

This information can also be found on the Investors section of the Innate Pharma website, www.innate-pharma.com.
A replay of the webcast will be available on the Company website for 90 days following the event.

Pipeline highlights:

Lacutamab (IPH4102, anti-KIR3DL2 antibody):

The Phase 2 TELLOMAK study in Sézary syndrome and mycosis fungoides (MF) continues to progress and the Company expects to report preliminary data from both cohorts in the second half of 2022. In March 2022, Innate announced the opening of a new MF all-comers cohort in the TELLOMAK study. The all-comers cohort will be recruiting both KIR3DL2 expressors and non-expressors to explore the correlation between the level of KIR3DL2 expression and treatment outcomes utilizing a formalin-fixed paraffin embedded (FFPE) assay as a potential companion diagnostic.
Two clinical trials are underway evaluating lacutamab in patients with KIR3DL2-expressing, relapsed/refractory peripheral T-cell lymphoma (PTCL):
Phase 1b trial: a Company-sponsored Phase 1b clinical trial to evaluate lacutamab as a monotherapy in patients with KIR3DL2-expressing relapsed PTCL.
Phase 2 KILT (anti-KIR in T Cell Lymphoma) trial: The Lymphoma Study Association (LYSA) investigator-sponsored, randomized trial to evaluate lacutamab in combination with chemotherapy GEMOX (gemcitabine in combination with oxaliplatin) versus GEMOX alone in patients with KIR3DL2-expressing relapsed/refractory PTCL.
ANKETTM (Antibody-based NK cell Engager Therapeutics):

Recruitment continues in the Phase 1/2 clinical trial by Sanofi evaluating IPH6101/SAR443579, the first NKp46/CD16-based NK cell engager, in patients with relapsed or refractory acute myeloid leukemia (R/R AML), B-cell acute lymphoblastic leukemia (B-ALL) or high risk- myelodysplastic syndrome (HR-MDS).
IPH64, the second ANKETTM drug candidate of the research collaboration with Sanofi, is progressing and the Company looks forward to updates on this asset.
Innate will provide updates on IPH65, the tetra-specific ANKETTM, throughout the year as progress is made toward an IND-enabling study in 2023.
Monalizumab (anti-NKG2A antibody), partnered with AstraZeneca:

On April 29, 2022, Innate announced a $50 million milestone payment from AstraZeneca was triggered for dosing the first patient in the Phase 3 clinical trial, PACIFIC-9, evaluating durvalumab (anti-PD-L1) in combination with monalizumab or AstraZeneca’s oleclumab (anti-CD73) in patients with unresectable, Stage III non-small cell lung cancer (NSCLC) who have not progressed following definitive platinum-based concurrent chemoradiation therapy (CRT). This is a post-period event.
Detailed results from the randomized AstraZeneca-sponsored Phase 2 COAST clinical trial, including monalizumab data in combination with durvalumab, were published in the Journal of Clinical Oncology on April 22, 2022. The results were initially presented during the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress 2021. The results of the interim analysis showed monalizumab in combination with durvalumab improved progression-free survival (PFS) and objective response rate (ORR) compared to durvalumab alone in patients with unresectable, Stage III non-small cell lung cancer (NSCLC) who had not progressed after concurrent chemoradiation therapy (CRT). The Journal of Clinical Oncology publication now includes exploratory subgroup analysis.
An oral presentation on April 11, 2022 at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting from the AstraZeneca-sponsored Phase 2 NeoCOAST randomized trial in resectable, early-stage NSCLC highlighted improved disease responses with durvalumab in combination withmonalizumab, oleclumab or danvatirsen, when compared to durvalumab alone. The follow-up randomized clinical trial, NeoCOAST-2, is enrolling patients with resectable, stage IIA-IIIA NSCLC to receive neoadjuvant durvalumab combined with chemotherapy and either oleclumab or monalizumab, followed by surgery and adjuvant durvalumab plus oleclumab or monalizumab.
The AstraZeneca-sponsored Phase 3 INTERLINK-1 trial of monalizumab plus cetuximab in immuno-oncology-pretreated head and neck cancer is ongoing with final data expected in 2024.
IPH5201 (anti-CD39), partnered with AstraZeneca:

Data for the Phase 1 trial in solid tumors with IPH5201 alone or in combination with durvalumab (PD-L1) are expected to be presented in 2023.
IPH5301 (anti-CD73):

In March 2022, The Institut Paoli-Calmettes announced that the first patient had been dosed in the investigator-sponsored Phase 1 trial of IPH5301 (CHANCES). The trial will be conducted in two parts, Part 1, the dose escalation, followed by a Part 2 safety expansion study cohort. Part 2 will evaluate IPH5301 in combination with chemotherapy and trastuzumab in HER2+ cancer patients.
ATM program:

On May 05, 2022, Innate announced the commencement of an At-The-Market (ATM) program, pursuant to which it may, from time to time, offer and sell to eligible investors a total gross amount of up to $75 million American Depositary Shares ("ADS"). Each ADS representing one ordinary share of Innate.
Financial Results:

Cash, cash equivalents and financial assets of the Company amounted to €131.7 million as of March 31, 2022. At the same date, financial liabilities amounted to €43.8 million. Cash, cash equivalents and financial assets as of March 31, 2022 do not include the $50.0 million payment to be received from AstraZeneca.

Revenues for the first three months of 2022 amounted to €2.6 million (€4.5 million for the same period in 2021). For the three-month period, ended March 31, 2022, revenue from collaboration and licensing agreements mainly results from the spreading of the payments received under our agreements with AstraZeneca.

On May 10, 2022 Bridge Biotherapeutics Inc. (KQ288330), a South Korean clinical-stage biotechnology company focused on developing novel drugs for cancer, fibrosis and inflammation, reported it will deliver an oral presentation highlighting the analysis data from the first-in-patient study of BBT-176[1] at the International Association for the Study of Lung Cancer 2022 World Conference on Lung Cancer (IASLC 2022 WCLC) (Press release, Bridge Biotherapeutics, MAY 10, 2022, View Source [SID1234614147]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

At its latest presentation to investors, Bridge Biotherapeutics shared interim Phase 1 study data of BBT-176 in advanced non-small cell lung cancer (NSCLC) showing that the drug candidate was well-tolerated and demonstrated efficacy against EGFR-mutation positive NSCLC, including C797S containing EGFR triple mutations. According to the company’s presentation, BBT-176 has shown anti-tumor efficacy with two partial response cases, one in the 160 mg QD cohort (51 percent tumor shrinkage) and the other in the 320 mg QD cohort (30 percent tumor shrinkage).

Pharmacokinetics data show that BBT-176 exhibits dose-dependent exposure. In addition, safety data have shown that adverse events (AEs) typically associated with EGFR inhibitors have been found but were limited to Grade 1-3 adverse events. The Phase 1 dose escalation study will be continued through an exploratory cohort to determine the maximum tolerable dose (MTD) and recommended Phase 2 dose (RP2D).

Sang-Yoon Lee, M.D., Bridge Biotherapeutics’ chief medical officer, said, "We are encouraged by these interim results from our Phase 1 trial of BBT-176, a fourth-generation EGFR TKI. In addition to continuing the trial, Bridge Biotherapeutics is promoting innovation by exploring the potential clinical value of liquid biopsies for both diagnosis and evaluation of therapeutic responses in NSCLC. We believe this will be another innovative aspect of our clinical development of BBT-176. Bridge Biotherapeutics remains focused on delivering novel treatment options to address the high unmet medical needs of advanced NSCLC patients."

The company’s oral presentation on BBT-176 is expected to be held on August 8, during IASLC 2022 WCLC, in Vienna, Austria.

On May 10, 2022 Omeros Corporation (Nasdaq: OMER), a clinical-stage biopharmaceutical company committed to discovering, developing and commercializing small-molecule and protein therapeutics for large-market as well as orphan indications targeting inflammation and immunologic diseases, including complement-mediated diseases and cancers, reported recent highlights and developments as well as financial results for the first quarter ended March 31, 2022, which include (Press release, Omeros, MAY 10, 2022, View Source [SID1234614179]):

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

●On December 23, 2021, Omeros completed the sale of its commercial ophthalmic product OMIDRIA (phenylephrine and ketorolac intraocular solution) 1%/0.3% and certain related assets and liabilities to Rayner Surgical Inc. ("Rayner"). As a result of the transaction, the company reclassified all revenues and expenses related to OMIDRIA to discontinued operations for fiscal year 2021 in its financial statements. Omeros is entitled to royalties on Rayner’s worldwide net sales of OMIDRIA at rates that vary based on geography and certain regulatory contingencies. The royalty rate for U.S. net sales of OMIDRIA is currently 50 percent.

●For the quarter ended March 31, 2022, Omeros earned royalties of $13.8 million based on Rayner’s net sales of $27.7 million, all of which were in the U.S. This is a $6.6 million increase from the $21.1 million of OMIDRIA net sales reported by Omeros in the prior year quarter.

●Net loss was $33.0 million in the current quarter, or $0.53 per share, which included $4.2 million of non-cash expenses, or $0.07 per share. This compares to a net loss of $35.1 million, or $0.57 per share for the prior year quarter, which included $4.1 million of non-cash expenses, or $0.07 per share.

●At March 31, 2022, Omeros had $142.2 million of cash, cash equivalents and short-term investments available for operations, which is a reduction of $15.0 million from December 31, 2021. In addition, at March 31, 2022 Omeros had $16.3 million in receivables, consisting primarily of OMIDRIA royalties related to the first quarter, which are due for payment this month.

●In February 2022, Omeros had a Type A post-action meeting with the United States Food and Drug Administration (FDA) to discuss the Complete Response Letter (CRL) issued by FDA last year regarding the Company’s biologics license application (BLA) for narsoplimab in the treatment of hematopoietic stem cell transplant-associated thrombotic microangiopathy (TA-TMA). FDA was delayed in providing official minutes of the meeting, in which the review division repeated a number of critiques that the Company felt had been adequately addressed or were inaccurate. After close consultation with outside legal and regulatory advisors, Omeros has prepared and expects soon to submit a request for formal dispute resolution. Formal dispute resolution is an official pathway that enables a sponsor to appeal a decision by an FDA division to a higher authority within FDA, in this case the Office of New Drugs. Omeros’ request is for regular approval based on the data in the existing BLA.

"Following our Type A post-action meeting with FDA and preparing our draft request for formal dispute resolution, we remain highly confident in the strength of our data and of the entirety of our BLA," said Gregory A. Demopulos, M.D., Omeros’ chairman and chief executive officer. "We believe that the BLA warranted approval last year and, given the immediate patient need for narsoplimab, that formal dispute resolution represents the most expeditious path to approval. We now are finalizing the request with our team of regulatory and legal advisors and expect to submit it within a couple of weeks. Physician support is broad, our case for appeal is strong, and we expect to be successful. In addition to our

focus on regulatory approval, we believe that there are a series of value-creating events throughout the remainder of 2022, including OMS906 data in patients with paroxysmal nocturnal hemoglobinuria, data from our efforts in COVID-19, as well as updates on our Phase 1 study evaluating our long-acting MASP-2 inhibitor OMS1029, completion of enrollment for the proteinuria endpoint in our narsoplimab ARTEMIS-IgAN trial, and the potential to earn an OMIDRIA-related $200-million commercial milestone payment."

First Quarter and Recent Developments

●Recent developments regarding narsoplimab, Omeros’ lead monoclonal antibody targeting mannan-binding lectin-associated serine protease-2 (MASP-2) in advanced clinical programs for the treatment of TA-TMA, immunoglobulin A (IgA) nephropathy, atypical hemolytic uremic syndrome (aHUS) and severely ill COVID-19 patients, include the following:

oIn April 2022, a manuscript detailing the results of Omeros’ pivotal study assessing efficacy and safety of narsoplimab for the treatment of TA-TMA was published in the Journal of Clinical Oncology (JCO), the flagship publication of the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper). The manuscript, entitled "Narsoplimab, a Mannan-Binding Lectin-Associated Serine Protease-2 Inhibitor, for the Treatment of Adult Hematopoietic Stem-Cell Transplantation–Associated Thrombotic Microangiopathy" is available online and will be included in an upcoming print volume of JCO.

oOmeros engaged with key stakeholders in the transplant community through its presence at TANDEM 2022, the joint annual meetings of the American Society for Transplantation and Cellular Therapy (ASTCT) and the Center for International Blood & Marrow Transplant Research (CIBMTR), which was held last month in Salt Lake City. As part of the conference, Omeros received an award from the President of ASTCT in recognition of Omeros’ work in raising awareness of TA-TMA and advancing the medical and scientific understanding in the field.

oNarsoplimab is also being evaluated for the treatment of hospitalized COVID-19 patients in the I-SPY COVID-19 platform trial sponsored by Quantum Leap Healthcare Collaborative. To date, no drug investigated in the trial has been reported to show a benefit relative to the background therapy in the trial. Quantum’s analysis of the narsoplimab data is being finalized, and we all look forward to sharing the outcome of the trial.

oEnrollment in Omeros’ Phase 3 Artemis IgAN trial continues to progress towards an anticipated read out of 9-month follow-up data on proteinuria in the first half of next year. Our investigational new drug application for narsoplimab in IgAN has now been approved by the Chinese regulatory authority. We look forward to completing the remaining regulatory requirements and initiating enrollment there as soon as possible.

Recent developments regarding OMS906, Omeros’ lead clinical monoclonal antibody targeting MASP-3, the key activator of the alternative pathway, and OMS1029, the company’s long-acting MASP-2 inhibitor, include the following:

oOmeros continues its preparations to initiate a Phase 1b trial of OMS906 in patients with paroxysmal nocturnal hemoglobinuria (PNH). Enrollment is expected to begin this summer. As previously disclosed, dosing in the single-ascending-dose study of OMS906 in healthy subjects is completed. There were no safety signals of concern, and pharmacokinetic/pharmacodynamic (PK/PD) data support once-monthly to once-quarterly subcutaneous or intravenous dosing.

oPreparations are also underway for a Phase 1 trial assessing safety and tolerability and PK/PD of OMS1029 in healthy human subjects. First-in-human-enabling toxicology studies are complete and there was no safety signal of concern. Dosing in humans is expected to be once-monthly to once-quarterly by subcutaneous or intravenous administration based on animal PK/PD data to date. Enrollment is targeted to begin this summer.
Financial Results

On December 23, 2021, Rayner acquired OMIDRIA and the associated business operations. The completion of the sale required Omeros to reclassify all revenues and expenses related to OMIDRIA to discontinued operations for fiscal year 2021 in its financial statements.

Upon closing of the sale of OMIDRIA, Omeros recorded an OMIDRIA contract royalty asset of $184.6 million representing the minimum expected net present value of future U.S. royalty payments. During the first quarter of 2022, we earned royalties of $13.8 million on sales of OMIDRIA which we recorded as a reduction to the OMIDRIA contract royalty asset. We also recorded $7.0 million of income in discontinued operations representing interest income and remeasurement adjustments to the OMIDRIA contract royalty asset.

Total costs and expenses for the first quarter of 2022 were $35.0 million compared to $45.3 million for the first quarter of 2021. The decrease was primarily due to reduced narsoplimab manufacturing activities and reduced narsoplimab pre-launch marketing activities.

Net loss was $33.0 million in the first quarter of 2022, or $0.53 per share, which included $4.2 million of non-cash expenses, or $0.07 per share. This compares to a net loss of $35.1 million, or $0.57 per share, which included non-cash expenses of $4.1 million, or $0.07 per share.

As of March 31, 2022, the company had $142.2 million of cash, cash equivalents and short-term investments, a reduction of $15.0 million from December 31, 2021, and $16.3 million in receivables, net.

Conference Call Details

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 1198541. 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 1198541.

To access the live or subsequently archived webcast of the conference call on the internet, go to the company’s website at View Source