On August 9, 2021 TRACON Pharmaceuticals (NASDAQ: TCON), a clinical stage biopharmaceutical company focused on the development and commercialization of novel targeted cancer therapeutics and utilizing a cost efficient, CRO-independent product development platform to partner with ex-U.S. companies to develop and commercialize innovative products in the U.S., reported that the Independent Data Monitoring Committee for the ENVASARC pivotal trial has recommended that the trial proceed as planned following the review of 12 week safety data from more than 20 patients enrolled into the trial as of May 2021 (Press release, Tracon Pharmaceuticals, AUG 9, 2021, View Source [SID1234586145]). The safety data reviewed included data from more than 10 patients enrolled into cohort A of treatment with single agent envafolimab and more than 10 patients enrolled into cohort B of treatment with envafolimab and Yervoy (ipilimumab).

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"Envafolimab continues to be well tolerated as a single agent and when combined with Yervoy in refractory sarcoma patients who are enrolled in the ENVASARC trial. We remain on track for the Independent Data Monitoring Committee to review interim efficacy data in the fourth quarter of this year," said James Freddo, M.D., TRACON’s Chief Medical Officer.

About Envafolimab

Envafolimab (KN035), a novel, single-domain antibody against PD-L1, is the first subcutaneously injected PD-(L)1 inhibitor to be studied in pivotal trials. Envafolimab is currently being studied in the ENVASARC Phase 2 pivotal trial in the U.S. sponsored by TRACON, has been studied in a completed Phase 2 pivotal trial as a single agent in MSI-H/dMMR advanced solid tumor patients in China and is being studied in an ongoing Phase 3 pivotal trial in combination with gemcitabine and oxaliplatin in advanced biliary tract cancer patients in China, with both Chinese trials sponsored by 3D Medicines. TRACON’s partners Alphamab Oncology and 3D Medicines submitted an NDA to the NMPA in China for envafolimab in MSI-H/dMMR cancer that was accepted for review in December 2020 and granted priority review in January 2021. In the Phase 2 MSI-H/dMMR advanced solid tumor trial, the confirmed objective response rate (ORR) by blinded independent central review in MSI-H/dMMR colorectal cancer (CRC) patients treated with envafolimab who failed a fluoropyrimidine, oxaliplatin and irinotecan was 32%, which was similar to the 28% confirmed ORR reported in the Opdivo package insert in MSI-H/dMMR CRC patients who failed a fluoropyrimidine, oxaliplatin, and irinotecan and the 33% confirmed ORR reported for Keytruda in MSI-H/dMMR CRC patients who failed a fluoropyrimidine, oxaliplatin and irinotecan in cohort A of the KEYNOTE-164 clinical trial.

About ENVASARC (NCT04480502)

The ENVASARC pivotal trial is a multicenter, open label, randomized, non-comparative, parallel cohort study at approximately 25 top cancer centers in the United States that began dosing in December 2020. TRACON expects the trial to enroll 160 patients with UPS or MFS who have progressed following one or two lines of prior treatment and have not received an immune checkpoint inhibitor, with 80 patients enrolled into cohort A of treatment with single agent envafolimab and 80 patients enrolled into cohort B of treatment with envafolimab and Yervoy. The primary endpoint is ORR by blinded independent central review with duration of response a key secondary endpoint.

On August 9, 2021 City of Hope, a world-renowned cancer research and treatment center, reported that acclaimed Pacific Shores Medical Group has joined City of Hope (Press release, City of Hope, AUG 9, 2021, View Source [SID1234586163]). This agreement significantly expands City of Hope’s clinical network and provides thousands of patients in Southern California with increased access to breakthrough discoveries and lifesaving treatments.

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City of Hope adds seven locations – Glendale, Huntington Beach, Irvine, Newport Beach, Torrance and two in Long Beach – in one of the largest clinical network expansions in its history. With locations across five counties, City of Hope is delivering advanced cancer care closer to where people live — alleviating the burden on patients who have had to travel far from home for world-class and highly specialized cancer treatment.

"Together with the highly esteemed providers joining us from Pacific Shores, we continue to advance the delivery of cancer care and expand patient access to City of Hope’s unparalleled research, treatment and care," said Robert W. Stone, president and chief executive officer, City of Hope, and the Helen and Morgan Chu Chief Executive Officer Distinguished Chair. "Pacific Shores allows us to continue expanding our mission to serve more patients, families and communities across Southern California and beyond."

The 14 Pacific Shores physicians, 11 nurse practitioners and physician assistants and more than 200 staff members became part of City of Hope on Aug. 9, 2021. Pacific Shores patients will be able to continue to receive excellent care with their current physicians, while gaining access to the expertise of more than 1,000 City of Hope researchers and highly specialized physicians, hundreds of additional clinical trials, pioneering treatments, expanded cancer education and prevention resources, and new innovations that are making care more convenient and effective.

"Pacific Shores physicians chose to join City of Hope because of a shared interest in advancing cancer medicine through research, clinical trials and breakthrough treatments. We are delighted that our patients will have unprecedented access to City of Hope’s leading-edge treatments, extensive clinical trials and highly specialized cancer expertise. Additionally, our physicians and staff welcome the opportunity to count on many world-renowned scientists and clinicians as colleagues and collaborators," said N. Simon Tchekmedyian, M.D., founder and CEO of Pacific Shores Medical Group.

"As a Pacific Shores patient for 23 years, I am grateful that I will continue to have my trusted physician’s care now at City of Hope — known for saving the lives of people with cancer," said Elizabeth Lucas, 84, of Long Beach. "This is care that is the best of all possible combinations. I’m getting world-renowned care close to my home."

Established in 1986, Pacific Shores Medical Group has locations across Los Angeles and Orange counties. A cancer care pioneer, Pacific Shores is recognized for excellence in cancer therapy practices, receiving certification from the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Quality Oncology Practice Initiative. It is also one of the most experienced cancer groups in the country, with more than 80,000 patient visits a year.

"This is exactly what is needed in the pursuit to treat and cure cancer – a coming together of a best-in-class physician group and a world-renowned academic cancer research and treatment institution," said Edward S. Kim, M.D., M.B.A., physician-in-chief, City of Hope Orange County, and vice physician-in-chief, City of Hope National Medical Center. "City of Hope is leading the way in re-defining how cancer breakthroughs are delivered – equitably and efficiently – to communities."

Pacific Shores joining City of Hope adds to City of Hope’s expanding footprint in Orange County, where it is developing a cancer campus of the future and a network of advanced cancer care. Lennar Foundation Cancer Center is scheduled to open in the summer of 2022, followed by the county’s only specialty hospital exclusively focused on treating and curing cancer scheduled to open in 2025. City of Hope Newport Beach, the first phase of City of Hope Orange County’s expansion, has been serving patients since January 2020.

"By welcoming Pacific Shores patients, physicians and staff to City of Hope, we are fulfilling our promise of bringing our advanced cancer care into the heart of our communities. City of Hope is honored to work with the nation’s leading researchers and clinicians, and we are privileged to welcome these like-minded experts from Pacific Shores to our team. Hope is indeed growing across the Southern California region," said Annette M. Walker, president, City of Hope Orange County.

On August 9, 2021 Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company, reported that management will participate in a fireside chat at the 2021 Wedbush PacGrow Healthcare Conference on Wednesday, August 11th at 2:55pm Eastern Time (Press release, Sangamo Therapeutics, AUG 9, 2021, View Source [SID1234586252]).

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The virtual session will be webcast live and may be accessed on the Sangamo Therapeutics website in the Investors and Media section under Events and Presentations. The presentation will be available on the Sangamo Therapeutics website after the event.

On August 9, 2021 bluebird bio, Inc. (NASDAQ: BLUE) reported financial results and business highlights for the second quarter ended June 30, 2021 and provided operational updates, including the announcement that the U.S. Food and Drug Administration (FDA) placed a clinical hold on clinical studies of elivaldogene autotemcel (eli-cel, Lenti-D) gene therapy (licensed as SKYSONA in Europe) for cerebral adrenoleukodystrophy (CALD) (Press release, bluebird bio, AUG 9, 2021, View Source [SID1234586078]).

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"I’m tremendously proud of what bluebird has accomplished this quarter both operationally and strategically to ready ourselves to launch both bluebird bio and 2seventy bio," said Nick Leschly, chief bluebird. "Seven months after announcing our intent to split, and thanks to the incredibly hard work by our teams, we have created a solid foundation for both organizations. The ABECMA launch is exceeding expectations, the oncology INDs and severe genetic disease (SGD) biologics license application (BLA) filings are tracking for later this year, and we have established clear visions and leadership teams for each business. Importantly, we have made tough strategic decisions to reshape the overall cost structure to allow both companies to launch in a strong position to execute through important value-creating milestones."

BUSINESS SEPARATION

In January 2021, bluebird announced its intent to separate into two independent, publicly traded companies (bluebird bio and 2seventy bio). The company expects the separation to be completed by the end of 2021 and to be tax-free to bluebird shareholders.

Key members of the executive teams of both companies have been announced, effective upon completion of the planned separation. This includes Andrew Obenshain as CEO of bluebird and Nick Leschly as CEO of 2seventy.
The full board of directors for both companies will be announced closer to the separation date.
2seventy has confidentially filed its Form 10 Registration Statement with the U.S. Securities and Exchange Commission (SEC), in which it describes the planned tax-free spin-off of 2seventy as a publicly traded company.
bluebird plans to distribute 100% of the outstanding shares of 2seventy common stock to bluebird’s stockholders on a pro-rata basis.
Based on current cash position and the expected $110M upfront payment upon closing of the National Resilience, Inc. strategic collaboration, the Company anticipates having a cash balance of approximately $900M at the time of separation. Together with existing and emerging sources of revenue, we expect our cash balance will be sufficient to fund approximately 24 months of operations for bluebird and 2seventy under current business plans.
ELI-CEL SAFETY UPDATE

The company received a reported Suspected Unexpected Serious Adverse Reaction (SUSAR) of myelodysplastic syndrome (MDS), that is likely mediated by Lenti-D lentiviral vector (LVV) insertion, in a patient who was treated with eli-cel, or Lenti-D drug product for CALD over one year ago in the Phase 3 ALD-104 study. Evidence currently available suggests that specific design features of Lenti-D LVV likely contributed to this event. The company has shared this information with the independent data monitoring committee of the study and the FDA has placed the eli-cel program on a clinical hold. The company does not anticipate the clinical hold to impact its programs in sickle cell disease (SCD), β-thalassemia or oncology. Subject to resolution of the clinical hold, the company anticipates completing the submission of the rolling BLA for eli-cel in 2021.

"Our hearts go out to this patient and his family, who are dealing with a challenging diagnosis," said Nick Leschly, chief bluebird. "Given what we know, we remain confident that eli-cel can offer hope for patients and families impacted by this devastating disease who have very few treatment options. We are committed to working with regulators and physicians in order to resolve this hold as soon as possible and bring this important therapy to patients in need."

BLUEBIRD BIO BUSINESS UPDATE

Today, bluebird bio is announcing that the company intends to focus its SGD business on the U.S. market and on further investments in research and development to optimize its core three programs in SCD, β-thalassemia and CALD, as well as on the development of a pipeline exploring new disease indications using in vivo LVV technology. The company remains focused and is on track to complete the rolling submissions of the U.S. BLAs in β-thalassemia in 3Q 2021 and CALD in 2021, pending resolution of the eli-cel clinical hold.

In connection with the planned completion of the business separation in the fourth quarter of 2021 and pivot to U.S.-centric efforts for SGD, bluebird plans an orderly wind down of its operations in Europe and to explore how to give patients in Europe access to its gene therapies, including potentially out-licensing the ex-U.S. rights to its three lead products to a company with European experience and capabilities.

"bluebird’s decision to focus on the U.S. market is driven by the challenges of achieving appropriate value recognition and market access for ZYNTEGLO in Europe, which makes bringing its transformative gene therapies like ZYNTEGLO and SKYSONA to patients and physicians in Europe untenable for a small innovative company at this time," said Andrew Obenshain, president, severe genetic diseases, bluebird bio. "While European regulators have been innovative partners in supporting accelerated regulatory paths for these therapies, European payers have not yet evolved their approach to gene therapy in a way that can recognize the innovation and the expected life-long benefit of these products. We are committed to and hope to find a potential partner who can help us carry forward our therapies in Europe."

BLUEBIRD BIO RECENT HIGHLIGHTS

BB1111 AND ZYNTEGLO CLINICAL HOLD LIFT – On June 7, 2021, bluebird announced that the FDA has lifted the clinical holds on the Phase 1/2 HGB-206 and Phase 3 HGB-210 studies of LentiGlobin for SCD gene therapy (bb1111) for adult and pediatric patients with SCD, and the Phase 3 Northstar-2 (HGB-207) and Northstar-3 (HGB-212) studies of betibeglogene autotemcel gene therapy (beti-cel; licensed as ZYNTEGLO in the EU and the UK) for adult, adolescent and pediatric patients with transfusion-dependent β-thalassemia (TDT). The company is working closely with study investigators and clinical trial sites to resume all study activities as soon as possible.
β-THALASSEMIA

EHA DATA – On June 11, 2021, bluebird presented data from several studies of beti-cel in adult, adolescent and pediatric patients with TDT. These data were presented during EHA (Free EHA Whitepaper)2021 Virtual, the 26th Annual Congress of the European Hematology Association (EHA) (Free EHA Whitepaper). With 51 patients enrolled, data from the long-term follow-up study (LTF-303) show that all patients treated with beti-cel who achieve transfusion independence (TI) remain free from transfusions, with the longest follow-up of seven years. Across Phase 3 studies, 89% (32/36) of evaluable patients across ages and genotypes achieved TI and remain transfusion free, including 91% (20/22) of evaluable pediatric patients under the age of 18. Data from bluebird bio’s Phase 1/2 and Phase 3 clinical studies represent more than 220 patient-years of experience with beti-cel.
EU MARKETING AUTHORIZATION – On July 9, 2021, bluebird announced that the European Medicines Agency’s (EMA) Pharmacovigilance Risk Assessment Committee (PRAC) has concluded based on the review of all available data that the benefit-risk balance of ZYNTEGLO (beti-cel) remains favorable. bluebird bio informed the EMA that the company has lifted the voluntary marketing suspension. Today, bluebird is announcing that on the basis of the PRAC decision, the EMA CHMP (Committee for Medicinal Products for Human Use) has endorsed the positive recommendation for ZYNTEGLO by the PRAC. A confirmatory decision by the European Commission (EC) is expected in 3Q 2021.
CEREBRAL ADRENOLEUKODYSTROPHY

SKYSONA EC DECISION – On July 21, 2021, bluebird announced that the EC granted marketing authorization of SKYSONA, a one-time gene therapy for the treatment of early CALD in patients less than 18 years of age with an ABCD1 genetic mutation, and for whom a human leukocyte antigen (HLA)-matched sibling hematopoietic (blood) stem cell (HSC) donor is not available.
2SEVENTY BIO RECENT HIGHLIGHTS

ABECMA LAUNCH – This quarter, bluebird and Bristol-Myers Squibb (BMS) launched ABECMA (idecabtagene vicleucel; ide-cel) in the U.S. ABECMA generated total U.S. revenues of $24 million in 2Q 2021 which bluebird shares equally with BMS. As of June 30, 2021, over 65 sites in the U.S. had been qualified to treat patients. bluebird and BMS have reported robust demand for ABECMA and are working to continue to increase manufacturing capacity over time.
STRATEGIC MANUFACTURING ALLIANCE – On July 28, 2021, bluebird and National Resilience, Inc. (Resilience) announced a strategic alliance aimed to accelerate the early research, development and delivery of cell therapies. As part of the agreement, Resilience will acquire bluebird’s Research Triangle (bRT) manufacturing facility located in North Carolina for $110 million and will retain all of the more than 100 highly skilled technical staff and administrators currently employed at the site. Resilience will continue to support vector supply for both bluebird and 2seventy. The two companies are also finalizing a definitive agreement to establish partner programs that will share expense and revenue for successful commercialized oncology products and in parallel establish a next-generation manufacturing R&D collaboration. Additionally, 2seventy plans to invest in internal drug product manufacturing capability and capacity to support its future clinical studies.
KARMMA ASCO (Free ASCO Whitepaper) DATA – On May 19, 2021, bluebird and BMS announced new data and analyses from the pivotal KarMMa study evaluating ABECMA. These data showed a 24.8-month median overall survival in triple-class exposed relapsed/refractory multiple myeloma. With more than 24-month median follow-up, results represent longest follow-up to date from a global clinical trial of a CAR T cell therapy in multiple myeloma with 73% overall response rate and responses ongoing with a median duration of response in patients achieving a ≥CR of 21.5 months. Analysis of characteristics of neurotoxicity (NT) observed in KarMMa study reinforce well-understood safety profile of ABECMA with mostly Grade 1/2 occurrences of NT having early onset and resolution.
UPCOMING ANTICIPATED MILESTONES

BLUEBIRD BIO

bluebird anticipates the separation of its SGD and oncology businesses into two independent, publicly traded companies (bluebird bio and 2seventy bio) to be completed by the end of 2021.
TDT: The company is on track to complete its rolling BLA submission to the FDA for beti-cel in 3Q 2021. This submission is anticipated to include adult, adolescent and pediatric patients with transfusion dependent β-thalassemia across all genotypes (including non-β0/β0 genotypes and β0/β0 genotypes).
CALD: Subject to resolution of the clinical hold, the company plans to complete its rolling BLA submission to the FDA for eli-cel in 2021.
SCD: The company is continuing to evaluate the impact of the recently-lifted clinical hold on bb1111 and plans to continue to work closely with the FDA in their review of these events to provide an update on the company’s development plan and timeline for submission for regulatory approval of bb1111 by year end.
SCD: The company plans to present clinical data from its ongoing HGB-206 clinical study of bb1111 by the end of 2021.
2SEVENTY BIO

Continued commercial launch of ABECMA in the U.S.
Submission of 1-2 investigational new drug (IND) applications by the end of 2021.
Presentation of clinical data from the ongoing CRB-402 study of bb21217 by the end of 2021.
SECOND QUARTER 2021 FINANCIAL RESULTS

Cash Position: Cash, cash equivalents and marketable securities as of June 30, 2021 and December 31, 2020 were $941.6 million and $1.27 billion, respectively. The decrease in cash, cash equivalents and marketable securities is primarily related to cash used in support of ordinary course operating activities.
Revenues: Total revenues were $7.5 million for the three months ended June 30, 2021 compared to $198.9 million for the three months ended June 30, 2020. Total revenues were $20.3 million for the six months ended June 30, 2021 compared to $220.8 million for the six months ended June 30, 2020. The decrease for both periods was primarily driven by a cumulative catch-up adjustment to revenue recorded in connection with the May 2020 BMS contract modification in the second quarter of 2020.
R&D Expenses: Research and development expenses were $144.3 million for the three months ended June 30, 2021 compared to $156.3 million for the three months ended June 30, 2020. Research and development expenses were $298.8 million for the six months ended June 30, 2021 compared to $310.4 million for the six months ended June 30, 2020. The decrease for both periods was primarily driven by decreased manufacturing expenses, a decrease in license and milestone fees, and a decrease in clinical costs in light of safety events in the HGB-206 study of LentiGlobin for SCD gene therapy.
SG&A Expenses: Selling, general and administrative expenses were $78.6 million for the three months ended June 30, 2021 compared to $68.6 million for the three months ended June 30, 2020. Selling, general and administrative expenses were $165.5 million for the six months ended June 30, 2021 compared to $141.9 million for the six months ended June 30, 2020. The increase for both periods was primarily driven by an increase in consulting fees associated with the ongoing project to separate the company’s severe genetic disease and oncology businesses into two independently traded companies as well as an increased employee compensation, benefit, and other headcount related expenses.
Net Loss: Net loss was $241.7 million for the three months ended June 30, 2021 compared to $21.5 million for the three months ended June 30, 2020. Net loss was $447.5 million for the six months ended June 30, 2021 compared to $224.1 million for the six months ended June 30, 2020.
Investor Conference Call Information

bluebird will hold a conference call to discuss this update on Monday, August 9 at 8:00 a.m. ET. Investors may listen to the call by dialing (844) 825-4408 from locations in the United States or +1 (315) 625-3227 from outside the United States. Please refer to conference ID number 9698691.

To access the live webcast of bluebird’s presentation, please visit the "Events & Presentations" page within the Investors & Media section of the bluebird website at View Source A replay of the webcast will be available on the bluebird website for 90 days following the event.

On August 9, 2021 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, immunologic diseases (e.g., complement-mediated diseases and cancers) and central nervous system disorders, reported recent highlights and developments as well as financial results for the second quarter ended June 30, 2021, which include (Press release, Omeros, AUG 9, 2021, View Source [SID1234586114]):

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OMIDRIA revenues for the second quarter of 2021 were $28.8 million compared to $21.1 million in the first quarter. The 37 percent increase over the prior quarter primarily reflects growth in sales of OMIDRIA (phenylephrine and ketorolac intraocular solution) 1%/0.3% in ambulatory surgery centers (ASCs).
Net loss in the second quarter of 2021 was $28.6 million, or $0.46 per share, including non-cash expenses of $3.9 million, or $0.06 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, for the previous quarter.
At June 30, 2021, Omeros had cash, cash equivalents and short-term investments available for operations of $73.7 million.
Omeros’ Biologics License Application (BLA) for narsoplimab in the treatment of hematopoietic stem cell transplant-associated thrombotic microangipathy (HSCT-TMA or TA-TMA) is under priority review by the U.S. FDA with an action date of October 17, 2021 under the Prescription Drug User Fee Act (PDUFA).
Omeros announced preliminary results from the Phase 1 clinical trial of OMS906, the company’s MASP-3 inhibitor, which showed that (i) OMS906 was well tolerated at all doses tested and (ii) human pharmacokinetic and pharmacodynamic data are consistent with once-monthly or less frequent subcutaneous dosing.
"In the second quarter of 2021, Omeros achieved a number of important milestones," said Gregory A. Demopulos, M.D., Omeros’ chairman and chief executive officer. "As OMIDRIA sales continue to grow, CMS in its recent OPPS Proposed Rule reaffirmed its determination that OMIDRIA receive separate payment when used in ASCs, and our MASP-3 inhibitor OMS906 is successfully advancing through its Phase 1 clinical trial. Several other important milestones should reach resolution in the near term – FDA’s decision on our pending BLA for narsoplimab in the treatment of patients with TA-TMA, results from narsoplimab in the COVID-19 I-SPY platform clinical trial, and the outcome of the NOPAIN Act, which, if enacted by Congress, would mandate Medicare separate payment for non-opioid surgical pain management drugs like OMDRIA not only in ASCs but also in hospital outpatient departments. The remainder of our pipeline also pressed ahead, including the GPR174 program, the core of our immuno-oncology platform – a platform that has expanded beyond GPR174 with new CAR-T and adoptive cellular therapy programs. Omeros’ momentum is building, and we look forward to seeing what the rest of the year brings."

Second Quarter and Recent Developments

Recent developments regarding OMIDRIA include the following:
In July 2021, the Centers for Medicare and Medicaid Services (CMS) released its Outpatient Prospective Payment System (OPPS) and ASC Payment System proposed rule for calendar year 2022, which reaffirmed its earlier decision that OMIDRIA, when used in the ASC setting, qualifies for separate payment under CMS’ policy regarding non-opioid pain management surgical drugs. This policy has been in effect since 2019.
Recent developments regarding narsoplimab, Omeros’ lead human 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:
Omeros announced data from the second cohort of 10 critically ill COVID-19 patients treated with narsoplimab under compassionate use in Bergamo, Italy.
All of the patients had comorbidities and/or risk factors for poor outcomes, were mechanically ventilated, and had failed other therapies, including steroids.
90 percent of the patients were intubated prior to initiation of narsoplimab treatment.
80 percent of the patients recovered, survived and were discharged. The two deaths involved a 76-year-old who died of complications related to his pre-existing cardiomyopathy and a 68-year-old who began narsoplimab treatment after 13 days of intubation.
A manuscript from the Omeros-Cambridge Center for Complement and Inflammation Research (OC3IR) on the inhibition of the lectin pathway and MASP-2 as a potential treatment for severe COVID-19 was published in the peer-reviewed journal Frontiers in Immunology.
An abstract on the pharmacokinetic and pharmacodynamic modeling of lectin pathway inhibition by narsoplimab was accepted for presentation at the 16th International Symposium on IgA nephropathy.
Updates regarding Omeros’ other development programs and platforms include the following:
Omeros announced preliminary results from the Phase 1 clinical trial evaluating the pharmacokinetics/pharmacodynamics (PK/PD) and safety of OMS906 in healthy subjects. OMS906 inhibits MASP-3, the key activator of the alternative pathway of complement. MASP-3 is responsible for the conversion of pro-factor D to mature factor D. Data from the first five cohorts of the trial’s single-ascending dose stage show that (i) OMS906 was well tolerated at all doses tested (up to 5 mg/kg) and (ii) a single 3 mg/kg intravenous dose of OMS906 and a single dose of the lowest subcutaneous concentration tested each suppressed mature complement factor D below minimum detectable levels. The human PK/PD data were consistent with once-monthly or less frequent subcutaneous dosing. Following completion of the single- and multiple-ascending dose stages of the Phase 1 trial, Omeros plans to initiate a Phase 2 clinical trial.
A paper detailing the mechanism of action of PDE7 inhibition in nicotine addiction was published in the peer-reviewed Journal of Neuroscience and was selected for inclusion in the journal’s Featured Research page. Omeros has completed a successful Phase 1 trial with the lead compound in its PDE7 inhibitor program.
Financial Results

For the second quarter of 2021, OMIDRIA revenues were $28.8 million compared to $21.1 million for the first quarter.

Total costs and expenses for the second quarter of 2021 were $52.8 million compared to $51.7 million for the first quarter. The increase was primarily due to increased selling, general and administrative expenses in preparation for the anticipated U.S. launch of narsoplimab and additional employee-related costs. Research and development costs decreased quarter over quarter due to the timing of narsoplimab manufacturing-related costs, which are expensed rather than included as inventory until the initial marketing approval for narsoplimab is certain.

For the three months ended June 30, 2021, Omeros reported a net loss of $28.6 million, or $0.46 per share, which included non-cash expenses of $3.9 million, or $0.06 per share. This compares to a net loss in the previous quarter 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 June 30, 2021, the company had $73.7 million of cash, cash equivalents and short-term investments. The company also has a line of credit, which permits borrowing up to the lesser of $50.0 million and 85 percent of eligible accounts receivable less certain reserves. Omeros also has an "at the market" program in place that allows the company to sell, from time to time, up to $150.0 million of its common stock.

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 4195376. 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 4195376.

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