On May 9, 2023 Seres Therapeutics, Inc. (Nasdaq: MCRB), a leading microbiome therapeutics company, reported its first quarter 2023 financial results and provided business updates (Press release, Seres Therapeutics, MAY 9, 2023, View Source [SID1234631294]).

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"We were thrilled to recently announce the FDA approval of VOWST, the first and only FDA-approved orally administered microbiota-based therapeutic, and with the favorable label indication received from the FDA. Adult recurrent C. difficile infection patients who could benefit from using VOWST, per the label, can access it, including those with first recurrence. The approval of VOWST marks Seres’ transformation to a commercial organization and provides definitive validation of the promise of our microbiome technology platform," said Eric Shaff, President and Chief Executive Officer at Seres. "We are looking forward to launching VOWST in the United States in June alongside our collaborator, Nestlé Health Science.

"We also continue to advance our earlier-stage pipeline, including SER-155, designed to prevent infections and/or graft-versus-host disease in patients undergoing allogeneic hematopoietic stem cell transplant, or allo-HSCT. We are also very pleased to report today new Phase 1b data from study Cohort 1 that support our therapeutic objective of reducing serious enteric infections, resulting bloodstream infections and GvHD. These encouraging initial data support the continued development in the ongoing placebo-controlled study Cohort 2.

"Finally, we have substantially strengthened our balance sheet and expect to further enhance our cash position with the receipt of a $125 million milestone payment from Nestlé based on the FDA approval."

First Quarter and Recent Program and Corporate Updates

FDA Approval of VOWST: In April 2023, Seres and Nestlé Health Science announced the Food and Drug Administration (FDA) approval of VOWST (fecal microbiota spores, live-brpk), formerly called SER-109, an orally administered microbiota-based therapeutic to prevent recurrence of C. difficile Infection (CDI) in adults following antibacterial treatment for recurrent CDI (rCDI). VOWST is thought to facilitate restoration of the gut microbiome. The Company anticipates VOWST product availability and launch in June.

The FDA approval of VOWST was supported by a robust Phase 3 development program that included the ECOSPOR III and ECOSPOR IV studies. VOWST was previously granted Breakthrough Therapy and Orphan Drug Designations by the FDA.

ECOSPOR III was a multicenter, randomized, placebo-controlled study in individuals with rCDI, the results of which were published in the New England Journal of Medicine. The study’s primary objective was to demonstrate the reduction of CDI recurrence with VOWST. In ECOSPOR III, VOWST was shown to reduce CDI recurrence at eight weeks, with approximately 88% of individuals recurrence-free at eight weeks post-treatment, compared to 60% in participants who received placebo. In addition, at six months post-treatment, 79% of the VOWST group were demonstrated to be recurrence-free, compared to 53% in the placebo group. No treatment-related serious adverse events were observed in the active arm and the frequency of treatment-related adverse events was similar between the VOWST and placebo arms. The most common adverse reactions through eight weeks in VOWST treated participants versus placebo were solicited events of abdominal distention (31.1% VOWST versus 29.3% placebo), fatigue (22.2% VOWST versus 21.7% placebo), constipation (14.4% VOWST versus 10.9% placebo), chills (11.1% versus 7.6% placebo), and unsolicited event of diarrhea (10.0% versus 4.3% placebo).

ECOSPOR IV was an open-label, single arm study evaluating VOWST in 263 adult participants with rCDI. Study results were published in the JAMA Network Open. The ECOSPOR IV study results contributed to the VOWST safety database and supported product approval.

Seres and Nestlé Health Science are committed to helping appropriate patients who have been prescribed VOWST obtain access. Additional details about VOWST access programs will be available at launch. Please see vowst.com for further information.

In July 2021, Seres and Nestlé Health Science entered into an agreement to jointly commercialize VOWST in the U.S. and Canada. Nestlé Health Science is leveraging its global pharmaceutical business and assuming the role of lead commercialization party, including the utilization of its existing infrastructure, 150-person gastrointestinal sales force, payer access team and a recently hired 20-person hospital salesforce.

New SER-155 Phase 1b Cohort 1 Study Results: In a separate press release issued today, Seres announced new safety and pharmacology study data including:

•
favorable tolerability profile observed, with no serious adverse events attributed to SER-155 administration;

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bacteria in the SER-155 consortia engrafted into the gastrointestinal (GI) microbiome, with a magnitude and kinetics consistent with expectation from prior clinical results from other Seres microbiome therapeutics; and

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cumulative incidence of domination with ESKAPE pathogen families was rare and observed at substantially lower incidence rates than observed in a reference population of allo-HSCT patients.1

SER-155 is an investigational, oral, 16 strain, cultivated microbiome therapeutic designed to prevent colonization and reduce the abundance of ESKAPE pathogens (e.g., from families such as Enterococcaceae, Enterobacteriaceae, Streptococcaceae, Staphylococcaceae) in the GI tract to reduce the risk of enteric driven bloodstream infections and other downstream consequences such as GvHD in patients receiving allo-HSCT. SER-155 has the potential to also impact antimicrobial resistance (AMR), including infections caused by carbapenem-resistant Enterobacteriaceae (CRE) and vancomycin-resistant Enterococci (VRE). The development of SER-155 is supported by SER-109 Phase 3 ECOSPOR III study exploratory results showing the decolonization of gut pathogens, including bacterial carrying antibiotic resistance genes, in the GI microbiome following SER-109 administration.

The ongoing SER-155 Phase 1b study includes two cohorts with Cohort 1 designed to assess safety and drug pharmacology, including the engraftment of drug bacteria in the gastrointestinal tract.

Enrollment of the Cohort 2 study is ongoing, incorporating a randomized, double-blinded placebo-controlled design to further evaluate safety and engraftment, as well as clinical outcomes, and will enroll approximately 60 subjects administered either SER-155 or placebo at a 1:1 ratio. The Company anticipates obtaining Cohort 2 study data in mid-2024.

Infection Protection research: The Company continues to conduct research to bring forward new microbiome therapeutics as a novel approach for Infection Protection for medically compromised individuals, including those with cancer neutropenia, cirrhosis or solid organ transplant. Preclinical studies are evaluating the potential to reduce the abundance of targeted pathogens to decrease the potential for pathogen transmission, strengthen epithelial barriers to further reduce translocation and the frequency of bloodstream infections, and to modulate immune responses to tackle medical complications such as graft-versus-host disease (GvHD). The Company plans to announce an additional Infection Protection clinical development program in 2023.

Ulcerative Colitis (UC) research: The Company previously reported clinical, microbiome and metabolomic data from the SER-287 Phase 2b study and the first cohort of its SER-301 Phase 1b study. Available data for these investigational microbiome therapeutics suggest that there may be an opportunity to utilize biomarker-based patient selection and stratification for future studies. Research activities remain ongoing to inform potential further development activities.

Financial Results

Seres reported a net loss of $71.2 million for the first quarter of 2023, as compared with a net loss of $56.6 million for the same period in 2022.

Research and development expenses for the first quarter of 2023 were $44.0 million, compared with $39.6 million for the same period in 2022. The research and development expenses were primarily related to Seres’ VOWST clinical development program and manufacturing costs, as well as personnel expenses. Included in the first quarter 2023 R&D expenses of $44 million is approximately $16 million of commercial manufacturing costs for VOWST. Following the approval of VOWST, R&D expenses in the P&L will no longer include VOWST commercial manufacturing costs, as these costs will be capitalized and recognized on Seres’ balance sheet.

General and administrative expenses for the first quarter of 2023 were $22.5 million, compared with $18.6 million for the same period in 2022. General and administrative expenses were primarily related to personnel expenses, professional fees, including VOWST commercial readiness and pre-launch expenses, and facility costs.

The Company has expanded its capabilities across the organization in support of VOWST approval and launch and is focused on driving operational efficiencies and pursuing opportunities to optimize its cost structure.

Seres ended the first quarter of 2023 with $106.5 million in cash, cash equivalents and investments as compared with $181.3 million at the end of 2022.

In April 2023, Seres announced that it had entered into a new $250 million senior secured debt facility provided by funds managed by Oaktree Capital Management, L.P. The Company drew the first tranche of $110 million at closing, with three additional tranches available. These additional tranches include $90 million that will be available in two tranches of $45 million each based upon the achievement of certain applicable VOWST sales targets, and an additional $50 million will be available to the Company at Oaktree’s discretion to support potential future business development activities. Of the $110 million advanced by Oaktree at closing, approximately $53 million retires outstanding debt, and after deducting fees and expenses, the net proceeds to the Company were approximately $50 million.

Seres is also due to receive a $125 million milestone payment from Nestlé Health Science associated with the FDA approval of VOWST. The Company also anticipates the receipt of proceeds from the supply of VOWST initial inventory to Nestlé.

Seres pro-forma cash balance as of March 31, 2023, is approximately $282 million, including the VOWST approval milestone and the net proceeds from its debt financing with Oaktree.

Conference Call Information

Seres’ management will host a conference call today, May 9, 2023, at 8:30 a.m. ET. To access the conference call, please dial 800-715-9871 (domestic) or 646-307-1963 (international) and reference Conference ID 5098595. To join the live webcast, please visit the "Investors and News" section of the Seres website at www.serestherapeutics.com.

A webcast replay will be available on the Seres website beginning approximately two hours after the event and will be archived for at least 21 days.

INDICATION AND IMPORTANT SAFETY INFORMATION FOR VOWST

INDICATION

VOWST is indicated to prevent the recurrence of Clostridioides difficile infection (CDI) in individuals 18 years of age and older following antibacterial treatment for recurrent CDI (rCDI).

Limitation of Use: VOWST is not indicated for treatment of CDI.

IMPORTANT SAFETY INFORMATION

WARNINGS AND PRECAUTIONS

Transmissible infectious agents: Because VOWST is manufactured from human fecal matter, it may carry a risk of transmitting infectious agents. Report any infection that is suspected to have been transmitted by VOWST to Aimmune Therapeutics, Inc. at 1-833-246-2566.

Potential presence of food allergens: VOWST may contain food allergens. The potential to cause adverse reactions due to food allergens is unknown.

ADVERSE REACTIONS

The most common adverse reactions (reported in ≥5% of participants) were abdominal distension (31.1%), fatigue (22.2%), constipation (14.4%), chills (11.1%), and diarrhea (10.0%).

To report SUSPECTED ADVERSE REACTIONS, contact Aimmune Therapeutics at 1-833-AIM-2KNO (1-833-246-2566), or the FDA at 1-800-FDA-1088, or visit www.fda.gov/MedWatch.

DRUG INTERACTIONS

Do not administer antibacterials concurrently with VOWST.

Please see Full Prescribing Information and Patient Information

On May 9, 2023 Sensei Biotherapeutics, Inc. (Nasdaq: SNSE), a clinical stage immuno-oncology company focused on the discovery and development of next-generation therapeutics for cancer patients, reported financial results for the first quarter ended March 31, 2023, and provided recent business updates (Press release, Sensei Biotherapeutics, MAY 9, 2023, View Source [SID1234631293]).

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"We are pleased with the progress across our pipeline, underscored by regulatory clearance to proceed with a Phase 1/2 clinical study of our lead candidate, SNS-101, in patients with advanced solid tumors. We look forward to commencing this trial in mid-2023 and expect to move swiftly to a recommended Phase 2 dose," said John Celebi, President and Chief Executive Officer of Sensei Biotherapeutics. "We are also maintaining superb progress and promising results from our other pipeline programs, each with breakthrough potential within their target classes, as we advance toward key decision points later this year."

Highlights and Milestones

SNS-101

Sensei continues to advance SNS-101, a conditionally active antibody targeting the immune checkpoint VISTA (V-domain Ig suppressor of T cell activation), which is implicated in resistance to cancer immunotherapy and whose expression correlates with poor survival across numerous cancers. Recent updates for SNS-101 include:

In April 2023, the U.S. Food and Drug Administration (FDA) cleared Sensei’s Investigational New Drug (IND) application for the planned Phase 1/2 clinical trial of SNS-101 in patients with advanced solid tumors. The Phase 1/2 clinical trial is designed to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of SNS-101 as both a monotherapy and in combination with Regeneron’s PD-1 inhibitor Libtayo (cemiplimab) in advanced solid-tumor cancer patients. Sensei expects to begin clinical trial enrollment and commence patient dosing at a starting dose of 0.3 mg/kg in mid-2023.
In February 2023, Sensei CSO Edward van der Horst presented key preclinical data supporting the mechanism of SNS-101 at the Keystone Symposia on Next Generation Antibody Therapeutics.
Under the recently signed CRADA with the National Cancer Institute (NCI), part of the National Institutes of Health, in February 2023, preclinical studies are in progress with the goal of further elucidating VISTA’s role in immune checkpoint resistance and expanding the potential of SNS-101 as a combination therapy beyond anti-PD-1. In addition, preparations are underway for the NCI to participate as a trial site in the clinical investigation of SNS-101.
In a multi-dose GLP toxicology study, SNS-101 was well tolerated and displayed a favorable multi-dose pharmacokinetic profile, with linear elimination kinetics and an absence of target-mediated drug disposition.
Additional TMAb Platform Updates

Through its Tumor Microenvironment Activated biologics (TMAb) platform, Sensei is also advancing several conditionally active antibody programs, including SNS-102 targeting VSIG4 (V-Set and Immunoglobulin Domain Containing 4), SNS-103 targeting ENTPDase1 (ecto-nucleoside triphosphate diphosphohydrolase-1, also known as CD39) and a recently initiated fourth program.

SNS-102: Eight parental pH-sensitive VSIG4 antibodies have been selected, which have undergone further lead optimization and are currently being characterized.
SNS-103: In April 2023, Sensei presented new preclinical data on SNS-103 at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting. Eight parental pH-sensitive CD39 antibodies have been selected, which have undergone further lead optimization and are currently being characterized.
Sensei remains on track to select product candidates for both SNS-102 and SNS-103 in 2023.
Sensei has initiated early discovery efforts for a fourth TMAB program focused on developing a conditionally active bispecific antibody.
Upon successful candidate selection, Sensei expects to advance one product candidate to IND-enabling studies.
First Quarter 2023 Financial Results

Cash Position: Cash, cash equivalents and marketable securities were $95.5 million as of March 31, 2023, as compared to $107.1 million as of December 31, 2022. Sensei expects its current cash balance to fund operations into the second half of 2025.

Research and Development (R&D) Expenses: R&D expenses were $5.2 million for the quarter ended March 31, 2023, compared to $7.5 million for the quarter ended March 31, 2022. The decrease in R&D expenses was primarily attributable to lower personnel costs due to the restructuring, lower expense relating to lab supply purchases and lower manufacturing related expense partially offset by higher expense associated with clinical trials.

General and Administrative (G&A) Expenses: G&A expenses were $5.8 million for the quarter ended March 31, 2023, compared to $5.0 million for the quarter ended March 31, 2022. The increase in G&A expense was primarily attributable to external professional services, including $1.5 million of non-recurring expenses associated with stockholder activism related to our upcoming 2023 annual meeting of stockholders.

Net Loss: Net loss was $10.2 million for the quarter ended March 31, 2023, compared to $12.4 million for the quarter ended March 31, 2022.

On May 9, 2023 Scholar Rock (NASDAQ: SRRK), a Phase 3 clinical-stage biopharmaceutical company focused on the treatment of serious diseases in which protein growth factors play a fundamental role, reported financial results and corporate updates for the first quarter ended March 31, 2023 (Press release, Scholar Rock, MAY 9, 2023, View Source [SID1234631292]).

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"We are executing across our business and leveraging Scholar Rock’s differentiated platform, which selectively targets latent forms of growth factors, such as myostatin and TGFβ1, to develop highly selective and potentially transformative therapies for patients who have high unmet medical need," said Jay Backstrom, M.D., M.P.H., President & Chief Executive Officer of Scholar Rock. "We continue to advance our two clinical programs in spinal muscular atrophy and oncology, and we look forward to sharing updates on our progress as we approach several key milestones, including presenting 36-month data on apitegromab from the Phase 2 TOPAZ trial, completing enrollment of our apitegromab Phase 3 SAPPHIRE trial, and providing biomarker and clinical updates from our SRK-181 Phase 1 DRAGON trial."

Recent Company Highlights and Upcoming Milestones

Apitegromab is an investigational antibody inhibiting myostatin activation by selectively binding the pro- and latent forms of myostatin and is being developed as the potential first muscle-targeted therapy for the treatment of spinal muscular atrophy (SMA).

Continued progress towards completion of enrollment for Phase 3 SAPPHIRE clinical trial. The randomized double-blind, placebo-controlled clinical trial evaluating apitegromab for patients with nonambulatory Types 2 and 3 SMA on either nusinersen or risdiplam, is actively enrolling SMA patients across sites in the U.S. and Europe. Enrollment completion is expected in 2023, with the top-line data readout expected in 2024. If successful and if approved, the company expects to initiate a commercial product launch in 2025.

36-month data from Phase 2 TOPAZ trial to be presented at Cure SMA conference in June. The company announced two upcoming oral presentations at the Cure SMA Research & Clinical Conference, taking place June 28-30 in Orlando, Florida.

24-month data from the TOPAZ trial presented at the 2023 Muscular Dystrophy Association (MDA) Clinical and Scientific Conference in March. The company shared data evaluating outcomes after 24 months of treatment with apitegromab, demonstrating sustained improvements in motor function in patients with nonambulatory Types 2 and 3 SMA.

SRK-181 is an investigational selective inhibitor of latent TGFβ1 activation and is being developed with the aim of overcoming resistance to checkpoint therapy in patients with advanced cancer.

Advancing Phase 1 DRAGON proof-of-concept trial. In the second half of 2023, the company expects to provide biomarker and clinical updates from Part B of the DRAGON trial. In March 2023, Scholar Rock presented encore data at the European Society for Medical Oncology Targeted Anticancer Therapies (ESMO TAT) Congress, which showed that SRK-181 continued to be generally well tolerated with early indications of efficacy (as of the data cut-off date of December 2, 2022).
Corporate

Announced the addition of Richard Brudnick to the Board of Directors. In April 2023, Mr. Brudnick joined Scholar Rock’s Board of Directors. As an accomplished biotechnology executive, he brings an extensive background in corporate development and strategy, with over 30 years of industry experience across multiple specialties, stages and therapeutic areas.

First Quarter 2023 Financial Results

For the quarter ended March 31, 2023, net loss was $39.4 million or $0.49 per share compared to a net loss of $8.0 million or $0.21 per share for the quarter ended March 31, 2022.

Revenue was $0 for the quarter ended March 31, 2023, compared to $33.2 million for the quarter ended March 31, 2022. The prior year revenue was related to the Gilead fibrosis-focused research collaboration, which was executed in December 2018 and concluded in December 2021.

Research and development expense was $29.7 million for the quarter ended March 31, 2023, compared to $29.4 million for the quarter ended March 31, 2022. The increase was primarily attributable to costs associated with clinical trials, including the Phase 3 SAPPHIRE pivotal trial for apitegromab in SMA, and the DRAGON trial for SRK-181. These increases were offset by decreases in employee compensation and benefits costs, resulting from the restructuring in May 2022.

General and administrative expense was $10.8 million for the quarter ended March 31, 2023, compared to $10.8 million for the quarter ended March 31, 2022.
As of March 31, 2023, Scholar Rock had cash, cash equivalents, and marketable securities of approximately $275 million, which is expected to fund the company’s anticipated operating and capital expenditure requirements into 2025.

"Scholar Rock is in a strong financial position, and we are relentlessly focused on achieving our goal of improving the lives of patients who may potentially benefit from our medicines," said Ted Myles, Chief Operating Officer and Chief Financial Officer of Scholar Rock. "We continue to execute on our plan by advancing our clinical programs towards key inflection points, while diligently managing our resources."

On May 9, 2023 Repare Therapeutics Inc. ("Repare" or the "Company") (Nasdaq: RPTX), a leading clinical-stage precision oncology company, reported financial results for the first quarter ended March 31, 2023 (Press release, Repare Therapeutics, MAY 9, 2023, View Source [SID1234631291]).

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"We continue to execute clinically and across our pipeline programs, including presenting initial clinical data of camonsertib in combination with various PARP inhibitors from the ongoing TRESR and ATTACC trials at this year’s AACR (Free AACR Whitepaper) conference," said Lloyd M. Segal, President and Chief Executive Officer of Repare. "These findings are consistent with our preclinical data demonstrating that low, intermittent dosing of the camonsertib and PARP inhibitor combinations appears well tolerated and exhibited antitumor activity, most notably in advanced ovarian cancer. In addition, we are on track to present initial Phase 1 monotherapy data for RP-6306 in June."

First Quarter 2023 Review and Operational Updates:

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Announced initial clinical data from the Phase 1/2 TRESR and ATTACC trials evaluating camonsertib (RP-3500/RG6526, partnered with Roche), a potent and selective oral small molecule inhibitor of ATR (Ataxia-Telangiectasia and Rad3-related protein kinase), in combination with three poly (ADP-ribose) polymerase (PARP) inhibitors in a Clinical Trials Plenary Session at the 2023 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting.
o
Camonsertib combinations appear to be well tolerated. Dose limiting toxicity in 68 patients treated with the proposed combination doses were related to myelotoxicity (Grade 3+ anemia 3%, thrombocytopenia 6%, neutropenia 7%, and febrile neutropenia 3%). No prophylactic growth factors were required when administering the PARP inhibitors at evaluated doses.
o
Camonsertib combination resulted in durable clinical benefit across tumor types and genomic alterations, regardless of choice of PARP inhibitor and presence of platinum resistance. Overall clinical benefit rate (CBR) for all patients was 48%. Patients with platinum-resistant tumors had an overall response rate (ORR) of 12% and CBR of 49% and benefited similarly to non-platinum-resistant tumors (ORR 13%, CBR 46%).

o
Compelling results were observed particularly in patients with advanced ovarian cancer (n = 19). In these patients, overall response was 32%, CBR was 58% and median progression-free survival was approximately 7 months with treatment greater than 16 weeks and ongoing in 9 patients.
o
Early circulating tumor DNA molecular responses in 66% (31/47) of evaluable patients confirm antitumor activity of low dose, intermittent PARP inhibitor + ATR inhibitor therapy. The molecular response rate (MRR) was significantly higher in patients with clinical benefit (83%) compared to those without (48%; p=0.015) and significantly higher than camonsertib monotherapy that was also administered at higher doses (43% or 27/63; p=0.02). Molecular responses were also observed in patients with prior PARP inhibitor exposure (57%) and platinum resistance (64%).
o
Repare is conducting dose optimization and efficacy assessments in tumor specific expansions in the ATTACC study in collaboration with Roche to support future clinical development plans for camonsertib combinations with PARP inhibitors.
•
Evaluating RP-6306, a first-in-class, oral PKMYT1 inhibitor as a monotherapy and in combinations in multiple early clinical studies.
o
Repare presented two poster presentations for RP-6306 at the 2023 AACR (Free AACR Whitepaper) Annual Meeting regarding the co-mutation landscape in CCNE1 amplifications and the tumor heterogeneity of copy number in ovarian and uterine cancers. Additionally, several collaborators presented preclinical findings on the potential benefits of combining a Wee1 inhibitor with RP-6306 and the effect of RP-6306 in triple negative breast cancer.
o
Repare expects to report initial Phase 1 monotherapy clinical data for RP-6306 for the treatment of molecularly selected advanced solid tumors (MYTHIC) in June 2023. The Company expects to report initial Phase 1 combination therapy clinical data for RP-6306 for the treatment of molecularly selected advanced solid tumors in the fourth quarter of the year.
o
Repare is working with clinical investigators to initiate clinical testing, as part of an investigator-sponsored trial (IST), of a fourth RP-6306 combination with carboplatin, with first patient dosing expected this year.
o
Repare is collaborating with the Canadian Cancer Trials Group for a basket Phase 2 IST to evaluate RP-6306 in patients with selected, advanced cancers receiving standard agents that is expected to begin this year. A sub-study under the master clinical trial protocol will evaluate RP-6306 in combination with gemcitabine in patients with CD4/6i-resistant ER+/HER2- metastatic breast cancer.
•
Advancing preclinical programs into clinical development.
o
Repare initiated IND-enabling studies in the first half of this year for a small molecule, now designated RP-1664, against an undisclosed target with potential to enter the clinic in late 2023 or early 2024.
o
Repare is also pursuing development of an inhibitor of polymerase theta (Polθ) that is expected to enter the clinic in 2024.
•
In April 2023, Repare announced the appointment of Susan Molineaux, Ph.D., to its Board of Directors, effective as of the date of the Company’s upcoming annual meeting of shareholders in June 2023. Concurrent with Dr. Molineaux’s appointment as of the date of the annual meeting, Jerel

Davis, Ph.D., Managing Director at Versant Ventures and a founding member of Repare’s Board of Directors, will step down from the Board. Additionally, Repare has expanded the senior leadership team with the appointment of Daniel Belanger as EVP Human Resources in May 2023.
First Quarter 2023 Financial Results:

•
Cash and cash equivalents and marketable securities: Cash and cash equivalents and marketable securities as of March 31, 2023 were $314.1 million.
•
Revenue from collaboration agreements: Revenue from collaboration agreements were $5.7 million and $0.4 million for the three months ended March 31, 2023 and 2022, respectively. The year-over-year increase in revenue was due to revenue recognized from our collaboration and license agreement with Roche.
•
Research and development expenses, net of tax credits (Net R&D): Net R&D expenses were $31.8 million and $26.5 million for the three months ended March 31, 2023 and 2022, respectively. The year-over-year increase in Net R&D expenses was primarily due to higher personnel related costs from headcount in support of our development activities, and direct external costs related to the advancement of preclinical programs into IND-enabling studies.
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General and administrative (G&A) expenses: G&A expenses were $8.6 million and $8.8 million for three months ended March 31, 2023 and 2022, respectively. The year-over-year decrease in G&A was primarily due to lower professional fees associated with our collaboration and license agreement with Roche and lower D&O insurance premiums, offset by higher personnel related costs.
•
Net loss: Net loss was $34.9 million, or $0.83 per share, in the three months ended March 31, 2023, and $34.8 million, or $0.83 per share in the three months ended March 31, 2022.
About Repare Therapeutics’ SNIPRx Platform

Repare’s SNIPRx platform is a genome-wide CRISPR-based screening approach that utilizes proprietary isogenic cell lines to identify novel and known synthetic lethal gene pairs and the corresponding patients who are most likely to benefit from the Company’s therapies based on the genetic profile of their tumors. Repare’s platform enables the development of precision therapeutics in patients whose tumors contain one or more genomic alterations identified by SNIPRx screening, in order to selectively target those tumors in patients most likely to achieve clinical benefit from resulting product candidates.

On May 9, 2023 Quanterix Corporation (NASDAQ: QTRX), a company fueling scientific discovery through ultrasensitive biomarker detection, reported financial results for the three months ended March 31, 2023 (Press release, Quanterix, MAY 9, 2023, View Source [SID1234631290]).

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"Our corporate transformation initiated in August 2022 to maximize Quanterix’s full potential is on track. We remain confident that we have taken the right steps to build a strong foundation in preparation for future growth," said Masoud Toloue, President and Chief Executive Officer of Quanterix. "This progress takes on added importance with the FDA’s recent decision to approve tofersen for the treatment of superoxide dismutase 1 amyotrophic lateral sclerosis, in large part based on strong surrogate neurofilament light chain biomarker data. This decision adds significant momentum to the use of biomarkers for predicting disease severity and clinical benefit in neurodegenerative diseases, and we believe Quanterix will continue to be at the forefront of these developments."

First Quarter 2023 Financial Highlights

● Q1 total revenue was $28.5 million versus prior year Q1 of $29.6 million, a decrease of 4% driven by a decline in instrument revenue. Q1 total revenue increased 10% from Q4 2022 driven by increased consumable revenue.
● Gross margin, a key success indicator of the strategic realignment, saw strong quarter over quarter improvement. Q1 2023 GAAP gross margin was 59.5% versus Q1 2022 GAAP gross margin of 49.3% and Q4 2022 GAAP gross margin of 48.8%. Q1 2023 non-GAAP gross margin was 53.1% versus Q1 2022 non-GAAP gross margin of 43.2% and Q4 2022 non-GAAP gross margin of 41.3%.
● Q1 net loss was $6.1 million versus prior year Q1 of $18.2 million, a decrease of 66%.
● Cash burn for Q1 was $9.1 million, leaving us with $329.4 million of unrestricted cash as of March 31, 2023.
For additional information on the non-GAAP financial measures included in this press release, please see "Use of Non-GAAP Financial Measures" and "Reconciliation of GAAP to Non-GAAP Financial Measures" below.

2023 Business Highlights

·

In January 2023, the Company expanded its lab developed test (LDT) menu with the launch of an NfL LDT, which can be used as an aid in the evaluation of individuals for possible neurodegenerative conditions or other causes of neuronal or central nervous system damage. Quanterix’s Simoa NfL is the most widely published NfL test with hundreds of research papers, demonstrating its validity for assessing neuronal damage, and Simoa NfL has become widely adopted in therapeutic clinical trial designs. We believe the recent FDA approval of tofersen, which relied on neurofilament light chain biomarker data, also validates the importance of this test.

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The Company participated in the 2023 Alzheimer’s Disease/Parkinson’s Disease conference in Gothenburg Sweden. Simoa-based biomarkers were highlighted in more than 11 panel discussions and over 30 poster presentations.

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Published discoveries enabled through Quanterix’s Simoa technology continue to illustrate industry reliance on the Company’s ultra-sensitive technology for breakthrough discovery in research and clinical applications. The technology was highlighted in more than 140 new publications in the first quarter 2023, bringing total Simoa-specific inclusions to over 2,200 as of March 31, 2023.

Full Year Business Outlook

We have made a modest increase to our guidance for the full year 2023. We expect revenues to be in the range of $104 to $111 million. We expect GAAP gross margin percentage to be in the high 40s and non-GAAP gross margin percentage to be in the mid 40s. Expected cash burn for 2023 is unchanged, and we expect an approximately 10% improvement over 2022.

Conference Call

In conjunction with this announcement, Quanterix Corporation will host a conference call on May 9, 2023 at 4:30 p.m. Eastern Time. Individuals interested in listening to the conference call may do so by pre-registering here and obtaining a dial-in number and passcode.

A live webcast will also be available at: View Source You may also access the live webcast by visiting the News & Events page within the Investors section of the Quanterix website at www.quanterix.com. The webcast will be available on the Company’s website for one year following completion of the call.