1stOncology™: Cancer Intelligence Service – Page 6554 – 1stOncology is recognized as a Top 10 Global Pharma Analytic Provider

Astex announces that the phase 3 ASCERTAIN – acute myeloid leukemia (AML) clinical study of oral hypomethylating agent decitabine and cedazuridine fixed-dose combination (ASTX727 or DEC-C) met the trial’s primary endpoint

On May 12, 2022 Astex Pharmaceuticals, Inc. reported top-line results from the ASCERTAIN phase 3 study evaluating oral decitabine and cedazuridine fixed-dose combination (ASTX727 or DEC-C) tablets versus IV decitabine in adults with AML who are not candidates to receive standard induction chemotherapy (Press release, Astex Pharmaceuticals, MAY 12, 2022, View Source [SID1234614375]).

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

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

Schedule Your 30 min Free Demo!

The study met its primary endpoint of decitabine exposure equivalence of 5-day dosing between orally administered ASTX727 and intravenous (IV) decitabine as per the protocol analysis plan with a high degree of confidence. Safety observations were similar to those observed with IV decitabine.

The full data will be presented on June 10 as part of EHA (Free EHA Whitepaper)2022. Astex plans to file a Marketing Authorization Application (MAA) with the European Medicines Agency (EMA) and similar applications in other countries where IV decitabine is indicated for the treatment of AML.

"We are delighted with the outcome of the ASCERTAIN – AML trial, and the demonstration that the fixed-dose oral combination of decitabine and cedazuridine provides exposure equivalence to IV decitabine in the AML population," said Harold Keer, MD, PhD, chief medical officer of Astex Pharmaceuticals, Inc. "Subject to regulatory review and approvals, ASTX727 could bring a new treatment option to patients with AML who are not candidates to receive standard induction chemotherapy. The ASCERTAIN – AML study was conducted during the height of the COVID-19 pandemic, and this experience helped to highlight the potential advantage of an orally administered therapy. We are extremely grateful to all the patients, caregivers, partner research and manufacturing organizations, as well as the healthcare professionals who contributed to this effort."

"Parenterally administered hypomethylating agents have been a cornerstone of the treatment of AML patients who are not candidates to receive standard induction chemotherapy for over 10 years," said Professor Klaus Geissler MD, principal investigator of the ASCERTAIN – AML phase 3 study and head of the Fifth Medical Department, Clinic Hietzing Hospital, Vienna, Austria. "Oral ASTX727 may deliver a treatment alternative for patients with AML which potentially reduces the number of office visits and offers an option for patients to take their medication from the convenience and comfort of their homes."

Based on the data from the ASCERTAIN clinical program, the oral decitabine and cedazuridine fixed-dose combination is also being investigated in combination with other agents in hematological malignancies. The first of these studies is investigating the all-oral combination of decitabine and cedazuridine with venetoclax for the treatment of AML.

Oral decitabine and cedazuridine fixed-dose combination is approved as INQOVI in the U.S. and Canada for the treatment of intermediate and high-risk myelodysplastic syndromes (MDS), including chronic myelomonocytic leukemia (CMML) and is the only approved oral hypomethylating agent that has demonstrated equivalent exposure to its IV form.

Commercialization of INQOVI in the U.S. and Canada for the above indication is conducted by Taiho Oncology, Inc., and by Taiho Pharma Canada, Inc., respectively. Astex, Otsuka and Taiho are all members of the Otsuka group of companies.

ABOUT THE ASCERTAIN – AML CLINICAL STUDY1

The ASCERTAIN – AML clinical trial was designed as a randomized crossover study comparing oral decitabine (35mg) and cedazuridine (100mg) fixed-dose combination tablet given once daily for 5 days on a 28-day cycle to IV decitabine (20mg/m2) administered as a daily 1-hour infusion for 5 days on a 28-day cycle, in the first 2 cycles in AML patients who were unfit to receive intensive chemotherapy. Patients continued to receive oral decitabine and cedazuridine from cycle 3 onwards. The primary endpoint for the study was total 5-day decitabine area-under-the-curve (AUC) equivalence of oral decitabine and cedazuridine and IV decitabine. See: View Source

ABOUT DECITABINE AND CEDAZURIDINE FIXED-DOSE COMBINATION (ASTX727)

ASTX727 is an orally administered, fixed dose combination of the approved anti-cancer DNA hypomethylating agent, decitabine, together with cedazuridine,2 an inhibitor of cytidine deaminase.3 By inhibiting cytidine deaminase in the gut and the liver, ASTX727 is designed to allow for oral delivery of decitabine over 5 days in a given cycle to achieve comparable systemic exposure to IV decitabine administered over 5 days.

ASTX727 has been evaluated in a phase 1/2 pharmacokinetics-guided dose escalation and dose confirmation study, and a phase 3 exposure equivalence study in patients with MDS and CMML. The phase 1 and phase 2 clinical study results have been published in Lancet Haematology4 and Blood,5 respectively, and the phase 3 results have been presented at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in December 20196 and the International Congress on Myelodysplastic Syndromes in September 2021.7

Astex is also expanding the evaluation of decitabine – cedazuridine combinations through a program of investigator-sponsored trials.

ASTX727 is an investigational compound and is not currently approved in any country other than the U.S., Canada, and Australia.

The EHA (Free EHA Whitepaper) abstract can be downloaded from the EHA (Free EHA Whitepaper) website at: View Source

Learn more about INQOVI at View Source

INDICATIONS

In the U.S., INQOVI (decitabine and cedazuridine) is indicated for treatment of adult patients with myelodysplastic syndromes (MDS), including previously treated and untreated, de novo and secondary MDS with the following French-American-British subtypes (refractory anemia, refractory anemia with ringed sideroblasts, refractory anemia with excess blasts, and chronic myelomonocytic leukemia [CMML]) and intermediate-1, intermediate-2, and high-risk International Prognostic Scoring System groups.8

IMPORTANT SAFETY INFORMATION

WARNINGS AND PRECAUTIONS

Myelosuppression: Fatal and serious myelosuppression can occur with INQOVI. Based on laboratory values, new or worsening thrombocytopenia occurred in 82% of patients, with Grade 3 or 4 occurring in 76%. Neutropenia occurred in 73% of patients, with Grade 3 or 4 occurring in 71%. Anemia occurred in 71% of patients, with Grade 3 or 4 occurring in 55%. Febrile neutropenia occurred in 33% of patients, with Grade 3 or 4 occurring in 32%. Myelosuppression (thrombocytopenia, neutropenia, anemia, and febrile neutropenia) is the most frequent cause of INQOVI dose reduction or interruption, occurring in 36% of patients. Permanent discontinuation due to myelosuppression (febrile neutropenia) occurred in 1% of patients. Myelosuppression and worsening neutropenia may occur more frequently in the first or second treatment cycles and may not necessarily indicate progression of underlying MDS.

Fatal and serious infectious complications can occur with INQOVI. Pneumonia occurred in 21% of patients, with Grade 3 or 4 occurring in 15%. Sepsis occurred in 14% of patients, with Grade 3 or 4 occurring in 11%. Fatal pneumonia occurred in 1% of patients, fatal sepsis in 1%, and fatal septic shock in 1%.

Obtain complete blood cell counts prior to initiation of INQOVI, prior to each cycle, and as clinically indicated to monitor response and toxicity. Administer growth factors, and anti‑infective therapies for treatment or prophylaxis as appropriate. Delay the next cycle and resume at the same or reduced dose as recommended.

Embryo-Fetal Toxicity: INQOVI can cause fetal harm. Advise pregnant women of the potential risk to a fetus. Advise patients to use effective contraception during treatment with INQOVI and for 6 months (females) or 3 months (males) after last dose.

ADVERSE REACTIONS

Serious adverse reactions in > 5% of patients included febrile neutropenia (30%), pneumonia (14%), and sepsis (13%). Fatal adverse reactions included sepsis (1%), septic shock (1%), pneumonia (1%), respiratory failure (1%), and one case each of cerebral hemorrhage and sudden death.

The most common adverse reactions (≥ 20%) were fatigue (55%), constipation (44%), hemorrhage (43%), myalgia (42%), mucositis (41%), arthralgia (40%), nausea (40%), dyspnea (38%), diarrhea (37%), rash (33%), dizziness (33%), febrile neutropenia (33%), edema (30%), headache (30%), cough (28%), decreased appetite (24%), upper respiratory tract infection (23%), pneumonia (21%), and transaminase increased (21%). The most common Grade 3 or 4 laboratory abnormalities (>50%) were leukocytes decreased (81%), platelet count decreased (76%), neutrophil count decreased (71%), and hemoglobin decreased (55%).

USE IN SPECIFIC POPULATIONS

Lactation: Because of the potential for serious adverse reactions in the breastfed child, advise women not to breastfeed during treatment with INQOVI and for 2 weeks after the last dose.

Renal Impairment: No dosage modification of INQOVI is recommended for patients with mild or moderate renal impairment (creatinine clearance [CLcr] of 30 to 89 mL/min based on Cockcroft-Gault). Due to the potential for increased adverse reactions, monitor patients with moderate renal impairment (CLcr 30 to 59 mL/min) frequently for adverse reactions. INQOVI has not been studied in patients with severe renal impairment (CLcr 15 to 29 mL/min) or end-stage renal disease (ESRD: CLcr <15 mL/min).

Please see the accompanying Full Prescribing Information.

ABOUT ACUTE MYELOID LEUKEMIA (AML)

AML is the most common form of acute leukemia in adults.9 An estimated 20,050 new cases of AML are projected to be diagnosed in the U.S. in 202210 and an estimated 11,540 patients are projected to die from AML in the US in 2022.10. Although 60 to 80 percent of AML patients less than 60 years of age may achieve a complete response (CR) with standard intensive induction chemotherapy,11 the outlook for patients 60 years of age or more is significantly worse, with response rates less than 50 percent, cure rates following transplant remaining at less than 10 percent and a median survival of less than one year.11-13. These figures have not significantly improved during the last three decades. These patients have few therapeutic options available.14-15. Effective, less toxic therapies are needed for the treatment of AML, particularly for elderly patients where comorbidities and other consequences of aging may often render them ineligible to receive intensive remission induction chemotherapy, thus denying them a potentially curative transplant.13

Chemomab Therapeutics Announces First Quarter 2022 Financial Results and Provides Corporate Update

On May 12, 2022 Chemomab Therapeutics, Ltd. (Nasdaq: CMMB), a clinical-stage biotechnology company focused on the discovery and development of innovative therapeutics for fibrotic and inflammatory diseases with high unmet need, reported financial and operating results for the first quarter ended March 31, 2022 and provided a corporate update (Press release, Chemomab, MAY 12, 2022, View Source [SID1234614374]).

"During the quarter we continued to make good progress in refining and implementing revisions to our CM-101 clinical program aimed at decreasing overall development risk, maximizing the clinical data obtained to facilitate clinical decision-making, and generating critical data to support advancement to registration trials," said Dale Pfost, PhD, Chief Executive Officer of Chemomab. "We are expanding our efforts in primary sclerosing cholangitis (PSC) with an enlarged clinical trial that is recruiting patients from new sites in the U.S. and Europe, while adding an important dose finding component and an open-label extension. We will be performing an interim analysis of the currently enrolling dose cohort in the PSC study to assess safety and to confirm the planned sample sizes for the CM-101 dose cohorts. This planned interim read-out is expected late this year."

Dr. Pfost added, "In systemic sclerosis (SSc), we expect the revised Phase 2 trial design to enable an expedited path to proof-of-concept data, and, importantly, to provide additional information on CM-101’s activity in modifying the skin, lung and vascular pathophysiology seen in SSc patients. We are designing this trial with the assistance of key SSc opinion leaders and are on track to launch the trial by the end of the year. I am also pleased to report that we have now completed enrollment in our Phase 2 safety, pharmacokinetic and biomarker liver fibrosis study, with final readouts expected near year’s end. We continue to use our capital efficiently and look forward to providing further details on our progress with the PSC and SSc Phase 2 trials this summer."

Recent Highlights:

Named Jack Lawler Vice President of Global Clinical Development Operations. Mr. Lawler, who is based in the U.S., brings the company more than 20 years of diverse global clinical drug development experience.
Chief Scientific Officer Dr. Adi Mor presented "Blocking CCL24, a novel target regulating inflammation fibrosis and endothelial damage, shows promising potential as treatment for Systemic Sclerosis" at the biennial International Rheumatology Conference in Israel. Study data from experimental models and patient samples demonstrated that CCL24, the target for CM-101, is overexpressed in skin and serum samples of diffuse SSc patients compared to healthy individuals. CCL24 levels also correlated with fibrotic biomarkers and disease progression. In an experimental mouse model of SSc, CM-101 profoundly reduced skin and lung fibrosis.
Professor Francesco Del Galdo of the University of Leeds presented "CCL24 as a Marker of Worse Prognosis in diffuse cutaneous SSc: a Promising Novel Biological Target" at the 7th Systemic Sclerosis World Congress. Professor Del Galdo’s findings support the role of CCL24 as a potential therapeutic target, demonstrating elevated serum levels of CCL24 in diffuse cutaneous SSc patients. High CCL24 serum levels were correlated with disease activity and worse prognosis as reflected by high fibrotic activity and deterioration of lung function over time in a longitudinal patient cohort.
Participated in the 32nd Annual Oppenheimer Healthcare Conference
At the Cantor Fitzgerald Rare Orphan Disease Summit, Dr. Adi Mor described how Chemomab’s CM-101 "pipeline in a product" strategy offers important synergies and efficiencies in the drug development process.
First Quarter 2022 Financial Highlights

Cash Position: Cash and cash equivalents were $57.5 million as of March 31, 2022, compared to $61.2 million as of December 31, 2021.
Research and Development (R&D) Expenses: R&D expenses were $2.7 million for the first quarter ended March 31, 2022, compared to $1.2 million for the same quarter in 2021.
General and Administrative (G&A) Expenses: G&A expenses were $2.6 million for the first quarter ended March 31, 2022, compared to $0.5 million for the same quarter in 2021. The current quarter figure includes a $0.9 million non-cash stock-based compensation payment.
Net Loss: Net loss was $5.1 million, or a net loss of $0.02 per basic and diluted share, for the first quarter ended March 31, 2022, compared to $1.7 million, or a net loss of $0.01 per basic and diluted share, for the quarter ended March 31, 2021. The weighted average number of Ordinary Shares outstanding, basic and diluted were 228,090,300 (equal to 11,404,515 American Depository Shares) and 156,751,771 (equal to 7,837,589 American Depository Shares) for the quarters ended March 31, 2022, and March 31, 2021, respectively.
For further details on the company’s financial results for the quarter ended March 31, 2022, refer to the Form 10-Q, which will be filed with the SEC today, May 12, 2022.

Conference Call
Chemomab management will host a conference call for investors today, Thursday, May 12, 2022, beginning at 8:00 a.m. Eastern Time to discuss these results and answer questions. Shareholders and other interested parties may participate in the conference call by clicking this Webcast link to access the live webcast or replay, or by dialing 877-407-9208 (in the U.S.) or 201-493-6784 (outside the U.S. and in Israel) and entering passcode 13728593. Ask for the Chemomab conference call. The call also will be webcast live on the company’s website at View Source

A replay of the call will be available on the company website for 90 days at www.chemomab.com.

Entry into a Material Definitive Agreement

On May 12, 2022, Athersys, Inc. (the "Company," "we," "us" or "our") reported that entered into a common stock purchase agreement (the "Purchase Agreement") with Aspire Capital Fund, LLC ("Aspire Capital"), which provides that, upon the terms and subject to the conditions and limitations set forth therein, Aspire Capital is committed to purchase up to an aggregate of $100 million of shares of the Company’s common stock over the 24-month term of the Purchase Agreement (Filing, 8-K, Athersys, MAY 12, 2022, View Source [SID1234614373]).

Concurrently with entering into the Purchase Agreement, the Company also entered into a registration rights agreement with Aspire Capital (the "Registration Rights Agreement"), pursuant to which the Company agreed to file with the Securities and Exchange Commission a prospectus supplement to the Company’s effective shelf registration statement on Form S-3 (File No. 333-238810) registering all of the shares of common stock that may be offered to Aspire Capital from time to time under the Purchase Agreement.

Under the Purchase Agreement, we have the right, in our sole discretion, to present Aspire Capital with a purchase notice (each, a "Purchase Notice"), directing Aspire Capital (as principal) to purchase up to 200,000 shares of our common stock per trading day, provided that the aggregate price of such purchase shall not exceed $500,000 per trading day, up to $100 million of our common stock in the aggregate. We and Aspire Capital also may mutually agree to increase the number of shares that may be sold to as much as an additional 2,000,000 shares per business day. The purchase price per share pursuant to such Purchase Notice (the "Purchase Price") is the lower of (i) the lowest sale price for the Company’s common stock on the date of sale and (ii) the arithmetic average of the three lowest closing sale prices for the Company’s common stock during the ten consecutive business days ending on the business day immediately preceding the purchase date of those securities. The applicable Purchase Price will be determined prior to delivery of any Purchase Notice.

In addition, on any date on which we submit a Purchase Notice to Aspire Capital in an amount of at least 100,000 shares, we also have the right, in our sole discretion, to present Aspire Capital with a volume-weighted average price purchase notice (each, a "VWAP Purchase Notice") directing Aspire Capital to purchase an amount of the Company’s common stock equal to a percentage (not to exceed 30%) of the aggregate shares of common stock traded on The Nasdaq Capital Market on the next business day (the "VWAP Purchase Date"), subject to a maximum number of shares determined by the Company (the "VWAP Purchase Share Volume Maximum"). The purchase price per share pursuant to such VWAP Purchase Notice (the "VWAP Purchase Price") shall be the lower of (i) the closing sale price on the date of sale and (ii) 95% of the volume weighted average price for the Company’s common stock traded on The Nasdaq Capital Market on (a) the VWAP Purchase Date if the aggregate shares to be purchased on that date does not exceeded the VWAP Purchase Share Volume Maximum and the sale price of our common stock has not fallen below the price set by us in the VWAP Purchase Notice (the "VWAP Minimum Price Threshold") (to be appropriately adjusted for any reorganization, recapitalization, non-cash dividend stock split, reverse stock split or other similar transaction) or (b) the portion of such business day until such time as the aggregate shares to be purchased will equal the VWAP Purchase Share Volume Maximum. Further, if the sale price of our common stock falls on the VWAP Purchase Date below the greater of (i) 90% of the closing price of our common stock on the business day immediately preceding the VWAP Purchase Date and (ii) the VWAP Minimum Price Threshold, the VWAP Purchase Price will be determined using the percentage in the VWAP Purchase Notice of the total shares traded for such portion of the VWAP Purchase Date prior to the time that the sale price of our common stock fell below the VWAP Minimum Price Threshold and the volume weighted average price of our common stock sold during such portion of the VWAP Purchase Date prior to the time that the sale price of our common stock fell below the VWAP Minimum Price Threshold.

The respective prices and share numbers in the preceding paragraphs shall be appropriately adjusted for any reorganization, recapitalization, stock dividend, stock split, reverse stock split or other similar transaction.

The Purchase Agreement provides that the Company and Aspire Capital shall not effect any sales under the Purchase Agreement on any purchase date where the closing sale price of the Company’s common stock is less than $0.25 per share. There are no trading volume requirements or restrictions under the Purchase Agreement, and we will control the timing and amount of any sales of our common stock to Aspire Capital. Aspire Capital has no right to require any sales by us, but is obligated to make purchases from us as we direct in accordance with the Purchase Agreement. The Company may deliver multiple Purchase Notices and VWAP Purchase Notices to Aspire Capital from time to time during the term of the Purchase Agreement, so long as the most recent purchase has been completed. There are no limitations on use of proceeds, financial or business covenants, restrictions on future fundings, rights of first refusal, participation rights, penalties or liquidated damages in the Purchase Agreement. The Purchase Agreement may be terminated by us at any time, at our discretion, without any penalty or cost to us. Also, Aspire Capital has agreed that neither it nor any of its agents, representatives and affiliates shall engage in any direct or indirect short-selling or hedging, which establishes a net short position with respect to our common stock during any time prior to the termination of the Purchase Agreement.

The Purchase Agreement provides for events of default, upon the occurrence of which Aspire Capital may terminate the Purchase Agreement. Such events of default include, without limitation:

•the lapse, or unavailability to Aspire Capital for the sale of shares of the Company’s common stock, of any registration statement that is required to be maintained effective pursuant to the terms of the Registration Rights Agreement, subject to specified cure periods;
•the suspension from trading or failure of the Company’s common stock to be listed on a Principal Market (as defined in the Purchase Agreement) for a period of three consecutive business days;
•the delisting of the Company’s common stock from the Principal Market, provided the Company’s common stock is not immediately thereafter trading on the New York Stock Exchange, the NYSE American, The Nasdaq Global Select Market, The Nasdaq Global Market or The Nasdaq Capital Market;
•the failure for any reason by the Company’s transfer agent to issue shares to Aspire Capital within five business days after the applicable purchase date that Aspire Capital is entitled to receive such shares;
•if any of the current Executive Officers (as defined in Section 16 of the Securities Exchange Act of 1934) of the Company ceases to be an Executive Officer or full-time employee of the Company for any reason;
•if any proceeding against the Company is commenced pursuant to or within the meaning of any bankruptcy law; and
•any breach by the Company of the representations, warranties, covenants or other term or condition contained in the Purchase Agreement or any related agreements that would reasonably be expected to have a material adverse effect, except, in the case of a breach of a covenant which is reasonably curable, only if such breach continues for a period of at least five business days.

The foregoing is a summary description of certain terms of the Purchase Agreement and the Registration Rights Agreement. For a full description of all terms, please refer to copies of the Purchase Agreement and the Registration Rights Agreement that are filed herewith as Exhibits 10.1 and 10.2, respectively, to this Current Report on Form 8-K and are incorporated herein by reference. All readers are encouraged to read the entire text of the Purchase Agreement and the Registration Rights Agreement.

ImaginAb announces a new supply agreement with Invicro to offer clinical doses of ImaginAb’s CD8 investigational ImmunoPET imaging agent (zirconium Zr 89 crefmirlimab berdoxam) for Immuno-Oncology (I-O) preclinical research and clinical trials.

On May 12, 2022 ImaginAb Inc., a market leading global biotechnology company focused on developing next generation ImmunoPET imaging agents and therapeutic radiopharmaceuticals (RPT), reported an agreement with Invicro LLC, a global, industry-leading imaging CRO, and part of REALM IDx, Inc., to supply clinical doses of ImaginAb’s investigational CD8 ImmunoPET agent (zirconium Zr 89 crefmirlimab berdoxam) for use in clinical trials as part of Invicro’s global core lab imaging service (Press release, Immudex, MAY 12, 2022, View Source [SID1234614372]). The agreement also allows Invicro to produce zirconium Zr 89 crefmirlimab for its preclinical offerings.

This offering is a response to the growth seen in I-O therapies and demand from pharmaceutical and biotech companies for advanced, non-invasive, whole-body imaging biomarkers. ImaginAb’s investigational CD8 ImmunoPET agent provides a number of potential benefits that may help inform early decision making through Phase I-IV I-O clinical trials.

"We are delighted with our agreement with Invicro and the operational benefits we hope it will bring to its customers," stated Ian Wilson, CEO of ImaginAb.

"Driven through discussions with pharma and biotech companies that currently license our technology, we believe this partnership will bring the best of both businesses together, to offer the potential benefits that our investigational CD8 ImmunoPET imaging agent may bring in understanding therapeutic efficacy and treatment outcomes in clinical trials more precisely and earlier."

This offering is intended to provide pharma and biotech customers the opportunity to receive clinical doses of ImaginAb’s investigational CD8 ImmunoPET agent directly from Invicro.

"Invicro’s vision to transform health care through advanced imaging technologies aligns with ImaginAb’s investigational CD8 technology and mission. We believe this agreement will offer deeper insights into immune biology and pharmacodynamics that will help advance the development of therapies within the I-O space." commented Dr. Matthew Silva, CEO of Invicro.

NeuBase Therapeutics Reports Business Update and Financial Results for the Second Quarter of Fiscal Year 2022

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]).

"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.