On May 10, 2022 Recursion (Nasdaq: RXRX), the clinical-stage biotechnology company industrializing drug discovery by decoding biology, reported business updates and financial results for its first quarter ending March 31, 2022 (Press release, Recursion Pharmaceuticals, MAY 10, 2022, View Source [SID1234614184]).

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"Recursion achieved several key milestones, including dosing the first participants in our clinical trial for CCM, advancing our science across multiple other programs and continuing the evolution of our Recursion OS to take on additional steps in the drug discovery process beyond target discovery and lead identification," said Recursion Co-Founder & CEO Chris Gibson, Ph.D. "It is exciting to be at this inflection point of our platform and making progress towards translating molecules into medicines with our potential treatments beginning to move through clinical development. We look forward to the additional clinical trials we plan to initiate later this year and the potential of our work and partnerships to positively impact the lives of patients and their loved ones."

Summary of Business Highlights

Clinical Programs
Cerebral cavernous malformation (CCM) (REC-994): In March 2022, we enrolled the first participant in our Phase 2 SYCAMORE clinical trial, which is a double-blind, placebo-controlled safety, tolerability and exploratory efficacy study of this drug candidate in 60 participants with CCM. At this time, multiple participants have been enrolled and dosed.
Neurofibromatosis type 2 (NF2) (REC-2282): We plan to enroll the first participant in our Phase 2/3 POPLAR-NF2 clinical trial, which is a parallel group, two stage, randomized, multicenter study of this drug candidate in participants with progressive NF2-mutated meningiomas, in the second quarter of 2022.
Familial adenomatous polyposis (FAP) (REC-4881): In April 2022, the U.S. Food and Drug Administration granted Fast Track designation for REC-4881 for the potential treatment of FAP. We plan to initiate a Phase 2, randomized, double-blind, placebo-controlled study to evaluate safety, pharmacokinetics and efficacy of this drug candidate in the third quarter of 2022.
Preclinical and Discovery Programs
Clostridium difficile colitis (REC-3964): We made progress in IND-enabling studies for REC-3964 and plan to initiate a Phase 1 study in the second half of 2022.
Oncology pipeline: We continued to make progress advancing numerous oncology programs discovered using our next generation mapping and navigating technology, including programs related to immune checkpoint resistance in STK11-mutant non-small cell lung cancer, cancer immunotherapy target ‘alpha,’ HRD-negative ovarian cancer target ‘gamma,’ hepatocellular carcinoma, small molecule MYC inhibition, ovarian cancer, and other indications. We highlighted this progress at the annual meeting of the American Association for Cancer Research (AACR) (Free AACR Whitepaper).
Roche and Genentech Collaboration: We have initiated laboratory efforts and are scaling our pilot work to create our first partnership-specific maps in an oncology indication. We have also begun the initial work for development of phenomaps in neuroscience.
Bayer AG Collaboration: We have profiled Bayer’s compound library for next generation map-based drug discovery and are actively navigating the map to seed potential programs. We have multiple first-generation brute-force programs related to the potential treatment of fibrotic diseases progressing simultaneously with our partner.
Recursion OS
Transcriptomics: We automated key processes in our transcriptomics platform, TrekSeq, to enable higher scale and robustness related to the acquisition of transcriptomics data for use as an industrialized orthogonal validation assay.
InVivomics: We completed studies to enable the simultaneous monitoring of multiple mice and their respective individual digital biomarkers within the same cage and the tracking of digital biomarkers related to group social behaviors.
First Quarter 2022 Financial Results

Cash Position: Cash, cash equivalents, and investments were $591.1 million as of March 31, 2022.
Revenue: Total revenue, consisting primarily of revenue from collaborative agreements, was $5.3 million for the first quarter of 2022, compared to $2.6 million for the first quarter of 2021. The increase was due to revenue recognized from our Roche-Genentech collaboration.
Research and Development Expenses: Research and development expenses were $32.4 million for the first quarter of 2022, compared to $24.1 million for the first quarter of 2021. The increase in research and development expenses was primarily due to an increased number of pre-clinical assets being validated and increased clinical costs as studies progressed. These increases were partially offset by a decrease in platform costs due to partnership-related materials of $9.6 million, which has been capitalized on the balance sheet.
General and Administrative Expenses: General and administrative expenses were $21.1 million for the first quarter of 2022, compared to $8.9 million for the first quarter of 2021. The increase in general and administrative expenses was due to the growth in size of the company’s operations, including an increase in salaries and wages of $6.6 million, facilities costs, information technology and security costs, and other administrative costs associated with operating a public company.
Net Loss: Net loss was $56.0 million for the first quarter of 2022, compared to a net loss of $30.7 million for the first quarter of 2021.
Additional Corporate Updates

Annual Shareholder Meeting: The Recursion Annual Meeting for shareholders will be held on Tuesday, June 14, 2022 at 12:00 pm Mountain Time.
Oncology: Marie Evangelista, Ph.D., joined Recursion as Vice President, Oncology and will be responsible for translating Recursion’s internal pipeline of oncology compounds into the clinic as well as driving aspects of the Roche-Genentech collaboration related to an indication in gastrointestinal oncology. Dr. Evangelista previously served as Senior Director, Translational Medicine at Frontier Medicines and before that spent nearly two decades at Genentech.
Communications: Ryan Kelly joined Recursion as Chief Communications Officer and will be responsible for external and internal communications. Mr. Kelly previously served as Vice President, Marketing and Communications at Virgin Hyperloop where he supported commercializing the company’s technology through global strategic communication campaigns.
Investor Relations: Jared Allenbach joined Recursion as Senior Director, Investor Relations and will engage with investors and capital markets regarding strategic financing opportunities. Mr. Allenbach previously served as an investment banker within the healthcare sector at Goldman Sachs.

On May 10, 2022 Orion Biotechnology Canada Ltd, a drug discovery and development company unlocking the therapeutic potential of G Protein-Coupled Receptors (GPCRs), reported that it will be presenting important new data at both LSX World Congress and TIDES USA 2022 conference, taking place this week – May 10-12, 2022 (Press release, Orion Biotechnology, MAY 10, 2022, View Source [SID1234614180]).

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Dr. Oliver Hartley, Orion’s Vice-President Drug Discovery, will present Orion’s novel technology for targeting undrugged peptidergic GPCRs. His presentations will describe Orion’s drug discovery platform, one of the fastest drug discovery solutions in the industry for targeting peptidergic GPCRs. Dr. Hartley will also present new data on Orion’s CCR2 antagonist, OB-004, demonstrating the ability of this powerful platform to rapidly identify best-in-class GPCR drug candidates.

"Development of a best-in-class molecule in 6 months is an extraordinary accomplishment and validates the effectiveness of our drug discovery platform." said Dr. Hartley. "I am excited to be sharing new results that illustrate how the best-in-class potency, that can be achieved using our approach, can translate into superior activity in stringent and relevant preclinical models. It also validates our decision to further advance preclinical development of OB-004 during 2022 ".

Mark Groper, CEO of Orion Biotechnology added "This exciting new data showcases our ability to rapidly and effectively target peptidergic GPCRs and is another example of how Orion’s innovative approach is able to target this valuable, undrugged group of GPCRs."

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]):

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●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

On May 10, 2022 Rallybio Corporation (Nasdaq: RLYB), a clinical-stage biotechnology company committed to identifying and accelerating the development of life-transforming therapies for patients with severe and rare diseases, reported that it has obtained worldwide exclusive rights to Sanofi’s KY1066, which will be referred to as RLYB331 going forward, a preclinical potentially first-in-class antibody (Press release, Sanofi, MAY 10, 2022, View Source [SID1234614177]). RLYB331 has the potential to address a significant unmet need for patients with severe anemia with ineffective erythropoiesis and iron overload, such as beta thalassemia (BT) and a subset of myelodysplastic syndromes (MDS), amongst others. The transaction expands Rallybio’s pipeline, is strategically consistent with Rallybio’s existing focus on hematology, and aligns with its mission to accelerate the development of life-transforming therapies for patients with severe and rare disorders.

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"With our strong focus on portfolio expansion, the in-licensing of RLYB331, our first as a public company, marks a pivotal moment for Rallybio. We believe RLYB331 is differentiated from all programs in clinical development based on its mechanism of action, with the potential to be first-in-class. We expect that RLYB331 may address a significant unmet need by correcting ineffective erythropoiesis, improving hemoglobin, reducing red blood cell transfusions and reducing iron overload in multiple hematological disorders such as beta thalassemia and myelodysplastic syndromes," said Martin Mackay, Ph.D., Chief Executive Officer of Rallybio. "This product candidate is a natural fit with our R&D expertise and our focus on hematological disorders. Along with our existing pipeline it provides an additional opportunity to leverage our deep expertise in rare diseases and to identify and accelerate the development of transformative therapies for patients with severe and rare diseases. We look forward to integrating RLYB331 into our portfolio and ultimately deliver this therapy to transform the treatment of patients with severe benign hematological disorders."

RLYB331 is a monoclonal antibody that inhibits Matriptase-2 (MTP-2). The inhibition of MTP-2 significantly increases levels of hepcidin, decreases iron load and treats ineffective erythropoiesis. The standard of care for many such hematological disorders leaves a significant unmet need in iron overload associated anemias with patients experiencing significant morbidity and consequent mortality.

Rallybio plans to prosecute preclinical activities for RLYB331 including CMC, and dose-range finding and toxicity studies, which will then support transition of the asset into clinical development.

Under the terms of the license agreement, Rallybio will make an upfront cash payment of $3 million to Sanofi, in addition to development and commercial milestones, and mid to high single digit royalties on net sales.

On May 10, 2022 Evaxion Biotech A/S (NASDAQ: EVAX), a clinical-stage biotechnology company specializing in the development of AI-driven immunotherapies to improve the lives of patients with cancer and infectious diseases, reported that it has successfully produced all batches of personalized cancer immunotherapies for all patients enrolled in the Phase 1/2a clinical trial of EVX-02 in adjuvant melanoma (Press release, Evaxion Biotech, MAY 10, 2022, View Source [SID1234614175]).

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Birgitte Rønø, Chief Scientific Officer of Evaxion, said: "I am extremely proud that the team behind EVX-02 has shown that this complex production chain is feasible and that we can provide truly unique, personalized DNA vaccines within a critical time window. And we are delighted that we mastered all steps in the production process. Every cancer is unique, as is every immune system, and this is why we create cancer therapies that are one size fits one – and only one."

She continues: "With the release of the final batch, we confirmed our manufacturing process, which we believe will allow us to progress our DNA cancer immunotherapy programs into larger global trials to explore the clinical benefits of the compounds further. We have again demonstrated our capabilities to timely deliver personalized cancer treatment tailored to the unique cancer profile of every patient in a clinical trial."

The production process of the personalized drug product consists of multiple steps. The sequencing of the tumor DNA is followed by AI-powered identification of the most promising therapeutic targets developed by Evaxion’s proprietary PIONEERTM technology. This leads to designing the personalized multi-target vaccine drug product, which is then manufactured, released to the clinical sites, and administered to the patient.

This is the second time Evaxion has conducted a clinical trial with personalized cancer immunotherapy, having previously used a peptide-based treatment.

About EVX-02

Our EVX-02 program (NCT04455503) treats adjuvant melanoma patients with our patented DNA-based immunotherapy in combination with standard of care. The patients are fully resected before the trial, meaning that their tumors have been successfully removed (surgically). In the study, the focus of the therapy is to prevent disease relapse. The EVX-02 program is a multicenter study conducted in Australia. There are currently 16 patients enrolled in the trial.