On May 12, 2022 Sysmex Corporation (HQ: Kobe, Japan; Chairman and CEO: Hisashi Ietsugu) reported certain differences between its financial forecast on November 10, 2021, for the fiscal year ended March 31, 2022 (April 1, 2021, to March 31, 2022) (Press release, Sysmex, MAY 12, 2022, View Source [SID1234614269]). Furthermore, at a meeting of the Managing Board on May 12, 2022, Sysmex resolved to award dividends from surplus as described below, with a record date of March 31, 2022. We intend to propose this payment of dividends from surplus at the General Meeting of Shareholders scheduled for June 24, 2022.

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1. Change from Financial Forecasts
(1) Consolidated Financial Results for Fiscal Year from April 1, 2021, to March 31, 2022
(2) Reason Consolidated net sales were higher than previously expected, owing to favorable sales in the EMEA region and lower yen exchange rates than we had assumed earlier. On the profit front, operating profit, profit before tax and profit attributable to owners of the parent outpaced our previous expectations due to higher-than-expected consolidated net sales and a decline in selling, general and administrative expenses, owing to sales activities being constrained due to COVID-19. A change in the accounting treatment of cloud computing contracts during the fiscal year under review has been applied retroactively to the previous fiscal year’s figures, which are provided for reference. For details, please see the "Summary of Consolidated Financial Results for the Fiscal Year Ended March 31, 2022," disclosed today (May 12, 2022).

2. Dividend from Surplus
(1) Dividend
(2) Reason In terms of returns to shareholders, we intend to provide a stable dividend on a continuous basis and aim for a consolidated payout ratio of 30% under our basic policy of sharing the successes of our operations in line with business performance.

In accordance with this policy, we have set the ordinary year-end dividend for the fiscal year ended March 31, 2022, at ¥39 per share. Accordingly, annual total dividends will be ¥76 and the consolidated payout ratio will be 36.0%.

On May 12, 2022 Cogent Biosciences, Inc. (Nasdaq: COGT), a biotechnology company focused on developing precision therapies for genetically defined diseases, reported a poster presentation at the European Hematology Association (EHA) (Free EHA Whitepaper) Congress being held June 9-12, 2022 (Press release, Cogent Biosciences, MAY 12, 2022, View Source [SID1234614320]). The poster will review initial clinical data from the Company’s ongoing Phase 2 APEX trial with bezuclastinib. The abstract is available on the EHA (Free EHA Whitepaper) website at www.ehaweb.org and the poster will be published on the EHA (Free EHA Whitepaper) Congress platform on June 10, 2022.

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Presentation Title: A Phase 2 Study of Bezuclastinib (CGT9486), a Novel, Highly Selective, Potent KIT D816V inhibitor, in Adults with Advanced Systemic Mastocytosis (Apex): Methods, Baseline Data, and Early Insights
Session Type: Poster Session
Session Date and Time: Friday, June 10, 2022 – 16:30 – 17:45 CEST (10:30am-11:45am ET)
Abstract Number: P1049

On May 12, 2022 Regulus Therapeutics Inc. (Nasdaq: RGLS), a biopharmaceutical company focused on the discovery and development of innovative medicines targeting microRNAs (the "Company" or "Regulus"), reported financial results for the first quarter ended March 31, 2022 and provided a corporate update (Press release, Regulus, MAY 12, 2022, View Source [SID1234614336]).

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"The year is off to a great start as we continue to advance our pipeline, including the recent acceptance of our IND application for RGLS8429. We are thrilled to begin clinical development where we will investigate the safety, tolerability, pharmacokinetics, and preliminary efficacy of RGLS8429 for the treatment of ADPKD," stated Jay Hagan, CEO of Regulus. "We also entered into an exciting research collaboration with Brigham and Women’s Hospital, which will further our understanding into broader applications of miR-155 inhibitors and potentially bring us closer to improving outcomes for Amyotrophic Lateral Sclerosis (ALS) patients. We look forward to providing updates in the coming quarters regarding progress on these fronts."

Program Updates

RGLS8429 for ADPKD: As announced yesterday, the U.S. Food and Drug Administration (FDA) recently accepted the Company’s IND for RGLS8429 for the treatment of ADPKD. The Company plans to initiate a Phase 1 single-ascending dose (SAD) study in healthy volunteers to assess safety, tolerability and pharmacokinetics of RGLS8429. Following the SAD study, the Company plans to initiate a Phase 1b multiple ascending dose (MAD) study in adult patients with ADPKD to assess safety, tolerability and pharmacokinetics of RGLS8429, and to evaluate the dose response of RGLS8429 treatment on ADPKD biomarkers including polycystins, cystic kidney volume (htTKV), and overall kidney function. Top-line data from the healthy volunteer study are expected in the second half of 2022, and top-line biomarker data for the first cohort of RGLS8429-treated patients with ADPKD are expected in the first half of 2023.

Lademirsen (RG-012) for Alport syndrome: In February 2022, the Company announced completion of enrollment by Sanofi in the Phase 2 HERA clinical study evaluating lademirsen for the treatment of adult patients with Alport Syndrome under the Company’s Collaboration and License Agreement with Sanofi. Final data are expected in the first half of 2023 and, if successful, could provide further validation of the Company’s platform technology designed to address genetic kidney diseases and earn the Company a $25 million milestone.

Corporate Highlights

Collaboration Agreement with Brigham and Women’s Hospital: In March 2022, the Company announced a collaboration agreement with the laboratories of Oleg Butovsky, Ph.D., and Howard L. Weiner, M.D., at Brigham and Women’s Hospital to investigate the biologic effects of miR-155 inhibitors in both in vitro and in vivo models of ALS.

Financial Results

Cash Position: As of March 31, 2022, Regulus had $53.9 million in cash and cash equivalents.

Research and Development (R&D) Expenses: Research and development expenses were $3.7 million for the three months ended March 31, 2022, compared to $3.3 million for the same period in 2021. These amounts reflect internal and external costs associated with advancing our clinical and preclinical pipeline.

General and Administrative (G&A) Expenses: General and administrative expenses were $2.9 million for the three months ended March 31, 2022, compared to $2.5 million for the same period in 2021. These amounts reflect personnel-related and ongoing general business operating costs.

Net Loss: Net loss was $6.7 million, or $0.05 per share (basic and diluted), for the three months ended March 31, 2022, compared to $6.0 million, or $0.08 per share (basic and diluted), for the same period in 2021.

Conference Call and Webcast Information:
The Company will host a conference call and live audio webcast today at 5:00 p.m. Eastern Daylight Time to discuss its first quarter 2022 financial results and corporate update. To access the call, please dial (866) 652-5200 (domestic) or (412) 317-6060 (international). To access the telephone replay of the call, dial (877) 344-7529 (domestic) or (412) 317-0088 (international), passcode ID 6812601. The webcast and telephone replay will be archived on the Company’s website at www.regulusrx.com following the call.

About ADPKD

Autosomal Dominant Polycystic Kidney Disease (ADPKD), caused by the mutations in the PKD1 or PKD2 genes, is among the most common human monogenic disorders and a leading cause of end-stage renal disease. The disease is characterized by the development of multiple fluid filled cysts primarily in the kidneys, and to a lesser extent in the liver and other organs. Excessive kidney cyst cell proliferation, a central pathological feature, ultimately leads to end-stage renal disease in approximately 50% of ADPKD patients by age 60. Approximately 140,000 individuals are diagnosed with the disease in the United States alone, with an estimated global prevalence of 4 to 7 million.

About RGLS8429

RGLS8429 is a novel, next generation oligonucleotide designed to inhibit miR-17 and to preferentially target the kidney. Administration of RGLS8429 has shown robust data in preclinical models, where clear improvements in kidney function, size, and other measures of disease severity and has demonstrated a superior pharmacologic profile compared to Regulus’ first generation compound in preclinical studies. The U.S. Food and Drug Administration (FDA) accepted the Company’s IND for RGLS8429 for the treatment of ADPKD.

On May 12, 2022 Alpine Immune Sciences, Inc. (NASDAQ: ALPN), a leading clinical-stage immunotherapy company focused on developing innovative treatments for cancer and autoimmune and inflammatory diseases, reported financial results for the first quarter ended March 31, 2022 (Press release, Alpine Immune Sciences, MAY 12, 2022, View Source [SID1234614353]).

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"Our clinical pipeline of novel autoimmune and immuno-oncology candidates continues to make significant progress, as highlighted by the promising monotherapy dose escalation data presented at AACR (Free AACR Whitepaper) for davoceticept, a first-in-class CD28 costimulator and dual checkpoint inhibitor, in advanced malignancies and monotherapy expansion cohorts are currently open for enrollment," said Mitchell H. Gold, MD, Executive Chairman and Chief Executive Officer of Alpine. "For ALPN-303, our next-generation dual BAFF/APRIL inhibitor, we look forward to sharing preliminary data from our phase 1 healthy volunteer study as part of a poster at the 2022 EULAR congress and to initiating patient-based studies by the end of 2022."
First Quarter 2022 and Recent Pipeline Updates
•Davoceticept monotherapy dose escalation data (NEON-1) presented in an oral mini-symposium at the 2022 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting, demonstrating tumor volume reduction in 23% of evaluable participants despite a highly heterogeneous, heavily pretreated, advanced solid tumor population. Monotherapy expansion cohorts are currently open for enrollment.
•Key preclinical rationale for davoceticept published in Nature Communications, showing its novel, first-in-class approach to engaging CD28 to enhance activation of T cells and drive anti-tumor activity.
•NEON-2 trial of davoceticept in combination with pembrolizumab placed on a partial clinical hold. Participants previously enrolled in the NEON-2 trial were able to continue to receive study drug; the NEON-1 monotherapy trial was not affected.
•Preliminary data for the first-in-human, phase 1 study of ALPN-303 in healthy volunteers to be presented as part of a poster at the 2022 European Alliance of Associations for Rheumatology (EULAR) Congress. Initiation of a phase 2 study in systemic lupus erythematosus (SLE) and at least one basket study in renal, hematologic and/or dermatologic indications planned by the end of 2022.
First Quarter 2022 Financial Results
As of March 31, 2022, we had cash, cash equivalents, and investments totaling $219.1 million. Net cash provided by operating activities for the quarter ended March 31, 2022 was $5.4 million compared to net cash used in operating activities of $16.0 million for the quarter ended March 31, 2021. Cash provided by operating activities was driven by the receipt of a $25.0 million upfront payment from Horizon, which was recorded as current and noncurrent deferred revenue on our Condensed Consolidated Balance Sheets. The Company recorded net losses of $7.5 million and $10.6 million for the quarters ended March 31, 2022 and 2021, respectively.
Collaboration revenue for the first quarter ended March 31, 2022 was $13.6 million compared to $3.2 million for the first quarter ended March 31, 2021. The 2022 amounts were attributable to revenue recognized under our AbbVie and Horizon Agreements, while 2021 revenue recognized solely relates to AbbVie.
Research and development expenses for the first quarter ended March 31, 2022 were $16.3 million compared to $10.4 million for the first quarter ended March 31, 2021. The increase was primarily attributable to our Synergy, NEON, and ALPN-303 studies, and increased personnel costs and direct research activities. These increases were partially offset by a decrease in contract manufacturing and process development for ALPN-303.
General and administrative expenses for the first quarter ended March 31, 2022 were $4.8 million compared to $3.3 million for the first quarter ended March 31, 2021. The increase was primarily attributable to increases in personnel costs.
The Company expects that its current cash resources will be sufficient to fund its planned operations into 2024.

On May 12, 2022 NeuBase Therapeutics, Inc. (Nasdaq: NBSE) ("NeuBase" or the "Company"), a biotechnology platform company Drugging the Genome to address disease at the base level using a new class of precision genetic medicines, reported its financial results for the three-month period ended March 31, 2022, and other recent developments (Press release, NeuBase Therapeutics, MAY 12, 2022, View Source [SID1234614371]).

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"We are pleased with the progress being made across our development pipeline of therapeutic programs to treat DM1, HD, and KRAS-driven cancers. The new data we’ve announced to date this year have further validated the use of our PATrOL platform to design novel genetic medicines that target and rescue gene dysfunctions, with the potential for clinically impactful outcomes in both rare and common diseases," said Dietrich A. Stephan, Ph.D., Founder, Chief Executive Officer, and Chairman of NeuBase. "We continue to execute the development strategy for our DM1 program, which includes a series of IND-enabling studies scheduled to report data throughout CY2022. Last quarter, we presented pharmacodynamic data that illustrated a single intravenous (IV) dose or multiple subcutaneous (SC) doses of our DM1 development candidate resolves the genetic defect and myotonia in skeletal muscle of the gold-standard mouse model of the disease. Building off these results, we plan on announcing at ASGCT (Free ASGCT Whitepaper) additional pharmacokinetic (PK) data, which will illustrate the exposure levels of our development candidate when administered via systemic administration in skeletal muscles, heart, and brain, tissues that are affected in DM1. We expect these data to support further advancement of our lead candidate for DM1 and validate a differentiated whole-body solution for this disease. Considering this progress, we believe the submission of an IND application to the FDA is on track for the fourth quarter of CY2022."

Second Quarter of Fiscal Year 2022 and Recent Operating Highlights

Myotonic Dystrophy Type 1 (DM1) Program: NeuBase is making steady progress advancing IND-enabling studies for its development candidate in the DM1 program, which includes PK, absorption, distribution, metabolism, and excretion (ADME), and bioavailability via IV and SC routes of administration, exploratory and IND-enabling Good Laboratory Practice (GLP) toxicology, and mechanism of action studies. In addition, Good Manufacturing Practice (GMP) of NeuBase’s development candidate to support Phase 1/2 clinical trials has been successfully implemented via contract manufacturing organizations.
In March 2022, the Company presented a robust data package through posters and presentations at the 2022 MDA Clinical & Scientific Conference demonstrating that systemic administration of the Company’s lead DM1 candidate, NT-0231.F, in the HSALR model achieves clinically relevant molecular and functional rescue, including genetic target engagement, displacement of sequestered MBNL1, resolution of nuclear aggregates, rescue of the spliceopathy, and reversal of myotonia (delayed muscle relaxation after contraction). In PK studies of NT-0231.F in wild-type BALB/C mice, a single IV or SC dose showed high volume of distribution, suggesting wide tissue distribution.
The Company announced that two abstracts have been accepted for presentation at the ASGCT (Free ASGCT Whitepaper) 25th Annual Meeting. The presentations will include new preclinical data on the biodistribution in key tissues of NT-0231.F.
With continued positive results from in vitro and in vivo preclinical studies, the Company expects to file an IND application for NT-0231.F in the fourth quarter of CY2022.
Huntington’s Disease (HD) Program: The HD program is currently in preclinical development. In CY2022, NeuBase expects to present new preclinical data describing the pharmacology of a candidate compound in the brain after systemic administration, nominate a development candidate and initiate scale-up and toxicology activities.
KRAS Oncology Program: The Company is conducting in vitro mechanistic studies and in vivo pharmacology studies for the KRAS program (KRAS G12V and G12D mutations). Existing in vivo data show activity illustrating allele-selective engagement of mutant KRAS at the DNA and RNA levels, with abrogation of downstream hyperactive signaling through multiple RAS pathway members, resulting in anti-tumor activity.
Financial Results for the Second Fiscal Quarter Ended March 31, 2022

As of March 31, 2022, the Company had cash and cash equivalents of approximately $39.0 million, compared with approximately $52.9 million as of September 30, 2021.
For the fiscal quarter ended March 31, 2022, the Company reported a net loss of approximately $9.9 million, or a net loss of $0.30 per share, compared with a net loss of approximately $5.5 million, or a net loss of $0.24 per share, for the same period last year.
For the fiscal quarter ended March 31, 2022, total operating expenses were approximately $9.9 million, consisting of approximately $3.1 million in general and administrative expenses and $6.8 million of research and development expenses. This compares with total operating expenses of approximately $5.9 million for the same period last year, consisting of approximately $2.7 million in general and administrative expenses and $3.2 million in research and development expenses.
Financial Results for the Six-Month Period Ended March 31, 2022

For the six-month period ended March 31, 2022, the Company reported a net loss of approximately $17.7 million, or a net loss of $0.54 per share, compared with a net loss of approximately $9.6 million, or a net loss of $0.41 per share, for the same period last year.
For the six-month period ended March 31, 2022, total operating expenses were approximately $17.2 million, consisting of approximately $6.0 million in general and administrative expenses and $11.2 million of research and development expenses. This compares with total operating expenses of approximately $10.6 million for the same period last year, consisting of approximately $5.4 million in general and administrative expenses and $5.2 million in research and development expenses.