Interim data from Part A of the RINGSIDE study demonstrated substantial initial anti-tumor activity for AL102 as a single agent and supports continued development

More advanced and comprehensive data set from RINGSIDE to be presented at ESMO

REHOVOT, Israel and WILMINGTON, Del., Aug. 15, 2022 (GLOBE NEWSWIRE) -- Ayala Pharmaceuticals, Inc. (Nasdaq: AYLA), a clinical-stage oncology company focused on developing and commercializing small molecule therapeutics for patients suffering from rare and aggressive cancers, primarily in genetically defined patient populations, today announced second-quarter 2022 financial results and provided a corporate update.

“We continue to make considerable progress across our pipeline, and we were particularly excited to announce interim results from the ongoing Phase 2/3 RINGSIDE trial evaluating AL102 in desmoid tumors. Although early, the results showed substantial initial anti-tumor activity and a favorable side effect profile,” said Roni Mamluk, Ph.D., Chief Executive Officer of Ayala. “Based on these positive results, our efforts are focused on finalizing the Part A of the study shortly followed by immediate initiation of Part B. For AL101, we were pleased with the latest interim results from the ACCURACY trial presented at ASCO, which demonstrated anti-tumor monotherapy activity and evidence of improved progression-free survival (PFS) in patients with recurrent/metastatic ACC.”

Second-quarter 2022 and Recent Business Highlights

• In July, announced positive interim data from Part A of the Phase 2/3 RINGSIDE study of AL102 in desmoid tumors: Data showed tumor shrinkage in substantially all patients who were evaluable at 16 weeks. AL102 was well tolerated at all three dosing regimens with no dose-limiting toxicities and no Grade 4/5 adverse events. The results from Part A will be used to determine the dose of AL102 to be evaluated in Part B of RINGSIDE, the randomized portion of the study, which Ayala is on track to initiate in 3Q 2022. More advanced and comprehensive data from RINGSIDE is expected to be presented at ESMO.
• Data on AL101 in adenoid cystic carcinoma (ACC) presented at ASCO 2022 Annual Meeting: In a poster at ASCO, the company provided an update from the 4mg and 6 mg AL101 cohorts in the ACCURACY study of AL101, a selective gamma-secretase inhibitor, in subjects with recurrent/metastatic (R/M) adenoid cystic carcinoma (ACC) harboring Notch activating mutations. An overall disease control rate of 66.7% was observed. The median PFS in each of the 4mg and 6mg dose cohorts was 3.7 months and 6.7 months among the patients who had a partial response.

Upcoming Milestones

• More advanced data from the RINGSIDE trial in desmoid tumors: Ayala expects to present a more comprehensive data set from Part A of the RINGSIDE trial of AL102 at ESMO.
• Initiation of Part B of the RINGSIDE trial: Part B will be a double-blind placebo-controlled study enrolling up to 156 patients with progressive disease, randomized between AL102 or placebo. The primary endpoint will be PFS with secondary endpoints including objective response rates, duration of response, and patient-reported quality of life measures.
• Initiate Phase 2 clinical trial evaluating AL102 in T-cell acute lymphoblastic leukemia (T-ALL): Ayala plans to begin an investigator-initiated Phase 2 clinical trial evaluating AL102 in R/R T-ALL around year-end.
  

Second-Quarter 2022 Financial Results

Cash Position: Cash and cash equivalents were $20.1 million as of June 30, 2022.

Collaboration Revenue: Collaboration revenue was $38 thousand for the second quarter of 2022, as compared to $761 thousand for the corresponding quarter in 2021.

R&D Expenses: Research and development expenses were $5.6 million for the second quarter of 2022, compared to $8.1 million for the corresponding quarter in 2021. The decrease is mainly due to the winding down of the ACCURACY study.

G&A Expenses: General and administrative expenses were $2.3 million for the second quarter of 2022, compared to $2.5 million for the second quarter of 2021.

Net Loss: Net loss was $8.2 million for the second quarter of 2022, resulting in basic and diluted net loss per share of $0.54. This compares with a net loss of $10.8 million for the second quarter of 2021 or basic and diluted net loss per share of $0.75 for that quarter.

For further details on the company’s financial results, refer to form Quarterly Report on Form 10-Q for the three months ended June 30, 2022, filed with the SEC on August 15, 2022.

About Ayala Pharmaceuticals

Ayala Pharmaceuticals, Inc. is a clinical-stage oncology company focused on developing and commercializing small molecule therapeutics for patients suffering from rare tumors and aggressive cancers. Ayala’s approach is focused on predicating, identifying and addressing tumorigenic drivers of cancer through a combination of its bioinformatics platform and next-generation sequencing to deliver targeted therapies to underserved patient populations. The company has two product candidates under development, AL101 and AL102, targeting the aberrant activation of the Notch pathway with gamma secretase inhibitors to treat a variety of tumors including desmoid tumors, adenoid cystic carcinoma and T-cell acute lymphoblastic leukemia (T-ALL). AL101, has received Fast Track Designation and Orphan Drug Designation from the U.S. FDA and is currently in a Phase 2 clinical trial for patients with ACC (ACCURACY) bearing Notch activating mutations. AL102 is currently in a Pivotal Phase 2/3 clinical trials for patients with desmoid tumors (RINGSIDE). For more information, visit www.ayalapharma.com.

Contacts:

Investors:
Joyce Allaire
LifeSci Advisors LLC
+1-617-435-6602
jallaire@lifesciadvisors.com 

Ayala Pharmaceuticals:
+1-857-444-0553
info@ayalapharma.com 

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including statements relating to our development of AL101 and AL102, the promise and potential impact of our preclinical or clinical trial data, the timing of and plans to initiate additional clinical trials of AL101 and AL102, the timing and results of any clinical trials or readouts, our participation at scientific or medical conferences, the sufficiency of cash to fund operations, and the anticipated impact of COVID-19, on our business. These forward-looking statements are based on management's current expectations. The words ”may,” “will,” “should,” “expect,” “plan,” “anticipate,” “could,” “intend,” “target,” “project,” “estimate,” “believe,” “predict,” “potential” or “continue” or the negative of these terms or other similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words.

These statements are neither promises nor guarantees, but involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements, including, but not limited to, the following: we have incurred significant losses since inception and anticipate that we will continue to incur losses for the foreseeable future. We are not currently profitable, and we may never achieve or sustain profitability; we will require additional capital to fund our operations, and if we fail to obtain necessary financing, we may not be able to complete the development and commercialization of AL101 and AL102; we have identified conditions and events that raise substantial doubt about our ability to continue as a going concern; we have a limited operating history and no history of commercializing pharmaceutical products, which may make it difficult to evaluate the prospects for our future viability; we are heavily dependent on the success of AL101 and AL102, our most advanced product candidates, which are still under clinical development, and if either AL101 or AL102 does not receive regulatory approval or is not successfully commercialized, our business may be harmed; due to our limited resources and access to capital, we must prioritize development of certain programs and product candidates; these decisions may prove to be wrong and may adversely affect our business; the outbreak of COVID-19, may adversely affect our business, including our clinical trials; our ability to use our net operating loss carry forwards to offset future taxable income may be subject to certain limitations; our product candidates are designed for patients with genetically defined cancers, which is a rapidly evolving area of science, and the approach we are taking to discover and develop product candidates is novel and may never lead to marketable products; we were not involved in the early development of our lead product candidates; therefore, we are dependent on third parties having accurately generated, collected and interpreted data from certain preclinical studies and clinical trials for our product candidates; enrollment and retention of patients in clinical trials is an expensive and time-consuming process and could be made more difficult or rendered impossible by multiple factors outside our control; if we do not achieve our projected development and commercialization goals in the timeframes we announce and expect, the commercialization of our product candidates may be delayed and our business will be harmed; our product candidates may cause serious adverse events or undesirable side effects, which may delay or prevent marketing approval, or, if approved, require them to be taken off the market, require them to include safety warnings or otherwise limit their sales; the market opportunities for AL101 and AL102, if approved, may be smaller than we anticipate; we may not be successful in developing, or collaborating with others to develop, diagnostic tests to identify patients with Notch-activating mutations; we have never obtained marketing approval for a product candidate and we may be unable to obtain, or may be delayed in obtaining, marketing approval for any of our product candidates; even if we obtain FDA approval for our product candidates in the United States, we may never obtain approval for or commercialize them in any other jurisdiction, which would limit our ability to realize their full market potential; we have been granted Orphan Drug Designation for AL101 for the treatment of ACC and may seek Orphan Drug Designation for other indications or product candidates, and we may be unable to maintain the benefits associated with Orphan Drug Designation, including the potential for market exclusivity, and may not receive Orphan Drug Designation for other indications or for our other product candidates; although we have received Fast Track designation for AL101, and may seek Fast Track designation for our other product candidates, such designations may not actually lead to a faster development timeline, regulatory review or approval process; we face significant competition from other biotechnology and pharmaceutical companies and our operating results will suffer if we fail to compete effectively; we are dependent on a small number of suppliers for some of the materials used to manufacture our product candidates, and on one company for the manufacture of the active pharmaceutical ingredient for each of our product candidates; any future collaborations will be, important to our business. If we are unable to maintain our existing collaboration or enter into new collaborations, or if these collaborations are not successful, our business could be adversely affected; enacted and future healthcare legislation may increase the difficulty and cost for us to obtain marketing approval of and commercialize our product candidates, if approved, and may affect the prices we may set; if we are unable to obtain, maintain, protect and enforce patent and other intellectual property protection for our technology and products or if the scope of the patent or other intellectual property protection obtained is not sufficiently broad, our competitors could develop and commercialize products and technology similar or identical to ours, and we may not be able to compete effectively in our markets; we may engage in acquisitions or in-licensing transactions that could disrupt our business, cause dilution to our stockholders or reduce our financial resources; and risks related to our operations in Israel could materially adversely impact our business, financial condition and results of operations.

These and other important factors discussed under the caption “Risk Factors” in our Quarterly Report on Form 10-Q for the three months ended June 30, 2022 filed with the U.S. Securities and Exchange Commission (SEC) on August 15, 2022 and our other filings with the SEC, could cause actual results to differ materially from those indicated by the forward-looking statements made in this press release. Any such forward-looking statements represent management's estimates as of the date of this press release. New risk factors and uncertainties may emerge from time to time, and it is not possible to predict all risk factors and uncertainties. While we may elect to update such forward-looking statements at some point in the future, except as required by law, we disclaim any obligation to do so, even if subsequent events cause our views to change. Although we believe the expectations reflected in such forward-looking statements are reasonable, we can give no assurance that such expectations will prove to be correct. These forward-looking statements should not be relied upon as representing our views as of any date subsequent to the date of this press release.