On March 28, 2022 Ayala Pharmaceuticals, Inc. (Ayala or the Company) (Nasdaq: AYLA), a clinical-stage oncology company focused on developing and commercializing small molecule therapeutics for patients suffering from rare and aggressive cancers reported full-year 2021 financial results and provided a corporate update (Press release, Ayala Pharmaceuticals, MAR 28, 2022, View Source [SID1234611050]).

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"2022 is off to a very promising start for Ayala, following a number of important accomplishments across our pipeline of innovative gamma-secretase inhibitors in 2021," said Roni Mamluk, Ph.D., Chief Executive Officer of Ayala. "We are pleased with the progress of our AL102 program in desmoid tumors, having recently completed enrollment in Part A of the open label RINGSIDE study. We are encouraged by initial positive feedback received from investigators so far. We plan to announce initial interim data from Part A around mid-year 2022 and intend to move into Part B, the randomized portion of the study, immediately thereafter. We look forward to announcing additional data readouts and updates on our other clinical programs throughout 2022 including further data on AL101 in adenoid cystic carcinoma."

2021 and Recent Business and Clinical Highlights

Completed enrollment of Part A of the Phase 2/3 RINGSIDE study of AL102 in desmoid tumors: 36 patients have been enrolled in Part A of the RINGSIDE study, which is evaluating the safety and tolerability of AL102, as well as tumor volume by MRI at 16 weeks. Ayala expects to report an initial interim data read-out around mid-2022, with Part B of the study commencing immediately thereafter.
Initiated "Window of Opportunity" study of AL101 in adenoid cystic carcinoma (ACC): The study is focused on determining the effects of AL101 for the treatment of ACC and other cancers. The goals of the study are to better understand the mechanism of AL101, determine the best treatment regime and generate data for the future development strategy. The study is being conducted in collaboration with M.D. Anderson Cancer Center and the Adenoid Cystic Carcinoma Research Foundation.
Presented positive preliminary clinical data from the ongoing ACCURACY trial in ACC: Updated interim data from the 6mg cohort of the Phase 2 ACCURACY study of AL101 in recurrent/metastatic adenoid cystic carcinoma (R/M ACC) were presented at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress 2021 demonstrating partial responses in three subjects (9%) and stable disease in 20 subjects (61%). At ESMO (Free ESMO Whitepaper), the Company also presented preclinical proof of concept data on AL101 in combination with approved cancer therapies in patient-derived ACC tumor models
Initiated Phase 1 trial of AL102 in combination with Novartis’ B-cell maturation antigen (BCMA) targeting agent WVT078 in relapsed/refractory multiple myeloma (MM): inhibition of gamma-secretase with AL102 prevents the cleavage and shedding of BCMA, which is ubiquitously expressed on MM cells. Preclinical data have demonstrated that treatment with AL102 increases the expression of membrane-bound BCMA on the surface of MM cells and could enhance the activity of WVT078. Ongoing patient enrollment continues as planned.
AL101 in Notch-activated triple negative breast cancer: The Company has elected to discontinue the Phase 2 TENACITY study as part of its efforts to focus its resources on the more advanced programs and studies including the RINGSIDE study in desmoid tumors and the ACCURACY study for ACC.
Upcoming Milestones

Initial interim data from the pivotal Phase 2/3 RINGSIDE trial in desmoid tumors (Mid-2022): Ayala expects to report an initial interim data read-out from Part A of the Phase 2/3 RINGSIDE trial of AL102 in desmoid tumors around mid-2022. Part B of the study will be a double-blind placebo-controlled study and will start immediately after dose selection from Part A.
Additional data from the Phase 2 ACCURACY trial of AL101 in ACC: The ongoing ACCURACY trial is an open-label, single-arm Phase 2 clinical trial evaluating AL101 as monotherapy for the treatment of R/M ACC patients with Notch-activated mutations. The first cohort of the trial included 45 subjects dosed at 4 mg of AL101 IV once weekly. Final data from the 4 mg cohort and additional data from the 6 mg cohort,which includes 42 subjects are expected to be reported in the second half of 2022.
Initiate Phase 2 Clinical Trial Evaluating AL102 in T-cell Acute Lymphoblastic Leukemia (T-ALL): Ayala plans to begin a Phase 2 clinical trial evaluating AL101 in R/R T-ALL in the second half of 2022.
Full Year 2021 Financial Results

Cash Position: Cash and cash equivalents were $37.3 million as of December 31, 2021, as compared to $42.4 million as of December 31, 2020.

Collaboration Revenue: Collaboration revenue was $3.5 million for the full year of 2021, as compared to $3.7 million for the full year of 2020.

R&D Expenses: Research and development expenses were $29.9 million for the full year 2021, compared to $22.4 million in 2020. The increase was primarily driven by additional costs in connection with the initiation and advancement of the Phase 2/3 RINGSIDE pivotal study for desmoid tumors.

G&A Expenses: General and administrative expenses were $9.3 million as of December 31, 2021, compared to $7.4 million as of December 31, 2020. The increase was primarily due to higher expenses in connection with our operations as a public company, including director and officer insurance, increased headcount, and stock-based compensation.

Net Loss: Net loss was $40.3 million for the full year 2021, resulting in basic and diluted net loss per share of ($2.80). This compares with a net loss was $30.1 million for the full year of 2020, equivalent to basic and diluted net loss per share of ($3.06).

For further details on the Company’s financial results, refer to our Annual Report on Form 10-K, for the year ended December 31, 2021 filed with the U.S. Securities and Exchange Commission (SEC) on March 28, 2022.

On March 28, 2022 Kinnate Biopharma Inc. (Nasdaq: KNTE) ("Kinnate"), a biopharmaceutical company focused on the discovery and development of small molecule kinase inhibitors for difficult-to-treat, genomically defined cancers, reported financial results for the full year ended December 31, 2021 (Press release, Kinnate Biopharma, MAR 28, 2022, View Source [SID1234611049]).

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"The continuing advancement across our pipeline last year, including the first patient being dosed in our Phase 1 trial evaluating KIN-2787 in adult patients with BRAF-mutant advanced and metastatic tumors, positions the company to further deliver on several key upcoming milestones in 2022," said Nima Farzan, Chief Executive Officer of Kinnate Biopharma. "For KIN-2787, we are on target to report initial monotherapy data from the ongoing KN-8701 Phase 1 trial expected in the third quarter of 2022 and initial data from the combination of KIN-2787 with binimetinib in NRAS-mutant melanoma by year end 2022. Beyond KIN-2787, we were pleased to receive IND clearance for KIN-3248, a next-generation pan-FGFR inhibitor, from the FDA earlier this year and initiated the Phase 1 trial in the first quarter. Our goal of generating one IND filing per year from our Kinnate Discovery Engine remains an important focus for the company as we look to improve upon current targeted oncology outcomes."

Recent Business Highlights and Corporate Update:

KIN-2787, pan-RAF inhibitor

Announced that three abstracts highlighting data from KIN-2787 have been accepted for presentation at the upcoming American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2022, to be held April 8-13, in New Orleans, Louisiana. KIN-2787 is an orally available small molecule pan-RAF inhibitor being developed for the treatment of patients with lung cancer, melanoma, and other solid tumors. The three abstracts include:
Occurrence of BRAF class II and III alterations is common across solid tumors and is associated with inferior clinical outcomes in NSCLC and melanoma (PAN# 4122);
Design and rationale of a first in human (FIH) Phase 1/1b study evaluating KIN-2787, a potent and highly selective pan-RAF inhibitor, in adult patients with BRAF- and NRAS-mutation positive solid tumors (PAN# CT248); and
Antitumor activity of KIN-2787, a next-generation pan-RAF inhibitor, in preclinical models of human RAF/RAS mutant melanoma (PAN# 2674).
Presented findings from a collaborative study with Tempus investigating the prevalence of Class II and Class III alterations among patients with BRAF-mutated solid tumors. These findings were presented as an e-Poster at the virtual European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Targeted Anticancer Therapies Congress (TAT). The meeting took place March 7-9, 2022.
Delivered an oral presentation of KIN-2787 preclinical data at the virtual IASLC 2022 Targeted Therapies of Lung Cancer meeting which took place February 22-26, 2022. In this study, KIN-2787 activity was assessed by suppression of downstream MAPK pathway signaling and subsequent cell growth inhibition across BRAF-altered and/or RAS-altered versus wild type panels of human non-small cell lung cancer (NSCLC) cell lines.
KIN-3248, FGFR Inhibitor

Announced the presentation of updates from preclinical studies evaluating its Fibroblast Growth Factor Receptor (FGFR) inhibitor candidate, KIN-3248, a next-generation pan-FGFR inhibitor being developed for intrahepatic cholangiocarcinoma (ICC) and urothelial carcinoma (UC) and other solid tumors. These findings were presented during a poster session at the ASCO (Free ASCO Whitepaper) Gastrointestinal Cancers Symposium which took place January 20-22, 2022.
On January 18, 2022 announced that the U.S Food and Drug Administration (FDA) has cleared the company’s Investigational New Drug (IND) application for KIN-3248. The Phase 1 trial initiated in the first quarter of 2022 and will evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics and anti-cancer activity of KIN-3248 in FGFR inhibitor naïve and pretreated cancer patients with FGFR2 and/or FGFR3 gene alterations.
Full-Year 2021 Financial Results

Full year net loss for 2021 was $89.8 million, compared to $35.8 million in 2020.
Full year research and development expenses for 2021 were $67.2 million, compared to $29.2 million in 2020.
Full year general and administrative expenses for 2021 increased to $22.9 million, compared to $6.8 million in 2020.
As of December 31, 2021, the total of cash and cash equivalents and short-term investments was $324.9 million, exclusive of the joint venture’s cash.
Key Upcoming 2022 Milestone Targets
KIN-2787

Initial monotherapy data from the ongoing KN-8701 Phase 1 trial expected in the third quarter of 2022.
Initiation of the combination portion of KN-8701 to study KIN-2787 with binimetinib in NRAS-mutant melanoma expected in the first half of 2022 with initial data expected by year end 2022.
Initiation of a Phase 1 trial in Greater China by Kinnjiu expected in mid-2022.
KIN-3248

Initial data from ongoing KN-4802 Phase 1 trial expected in the second half of 2023.
Early Discovery Pipeline

Goal to generate one IND filing a year from the company’s Kinnate Discovery Engine.
Announcement of the company’s next pipeline target expected in the second half of 2022.

On March 28, 2022 Checkpoint Therapeutics, Inc. ("Checkpoint") (NASDAQ: CKPT), a clinical-stage immunotherapy and targeted oncology company, reported financial results for the full-year ended December 31, 2021 and recent corporate highlights (Press release, Checkpoint Therapeutics, MAR 28, 2022, View Source [SID1234611047]).

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James F. Oliviero, President and Chief Executive Officer of Checkpoint, said, "The past year represented a truly transformational period for Checkpoint Therapeutics, with the foundation laid for multiple significant potentially value enhancing catalysts in 2022. Following the positive topline results from our ongoing registrational trial of cosibelimab in metastatic cutaneous squamous cell carcinoma announced earlier this year, we look forward to a planned Biologics License Application submission for cosibelimab later in 2022." Mr. Oliviero continued, "We remain focused on expeditiously advancing our pipeline of product candidates with the goal of expanding patient access globally to potentially life-saving novel oncology therapies through a disruptive pricing strategy."

2021 and Recent Corporate Highlights:

In January 2022, Checkpoint announced positive topline results from the ongoing registration-enabling clinical trial evaluating the safety and efficacy of its anti-PD-L1 antibody, cosibelimab, administered as a fixed dose of 800 mg every two weeks in patients with metastatic cutaneous squamous cell carcinoma ("cSCC"). The study met its primary endpoint, with cosibelimab demonstrating a confirmed objective response rate of 47.4% (95% CI: 36.0, 59.1) based on independent central review of 78 patients enrolled in the metastatic cSCC cohort using Response Evaluation Criteria in Solid Tumors version 1.1 criteria. Checkpoint intends to submit a Biologics License Application for cosibelimab in late 2022, followed by a Marketing Authorization Application submission in Europe and other territories worldwide. With a potentially favorable safety profile versus anti-PD-1 therapy and a plan to commercialize at a substantially lower price, Checkpoint believes cosibelimab has the potential to be a market disruptive product in the $30 billion and growing PD-(L)1 class.
In December 2021, Checkpoint announced the initiation of the CONTERNO study, a global, randomized Phase 3 trial of cosibelimab in combination with pemetrexed and platinum chemotherapy for the first-line treatment of patients with non-squamous non-small cell lung cancer.
During the second quarter of 2021, Checkpoint had productive interactions with the FDA regarding its ongoing development program for olafertinib (formerly CK-101), a third-generation epidermal growth factor receptor inhibitor being evaluated by its partner in an ongoing double-blind, randomized Phase 3 study in China.
In March 2021, Checkpoint announced the formation of a Scientific Advisory Board comprised of clinical and scientific thought leaders in oncology. Members include Wayne A. Marasco, M.D., Ph.D., F. Stephen Hodi, Jr., M.D., Bruce E. Johnson, M.D., Roy S. Herbst, M.D., Ph.D., David Miller, M.D., Ph.D., and Emily Ruiz, M.D., M.P.H.
Financial Results:

Cash Position: As of December 31, 2021, Checkpoint’s cash and cash equivalents totaled $54.7 million, compared to $40.8 million at December 31, 2020, an increase of $13.9 million.
R&D Expenses: Research and development expenses for the year ended December 31, 2021, were $48.5 million, compared to $16.4 million for the year ended December 31, 2020, an increase of $32.1 million. Research and development expenses for the year ended December 31, 2021 included $7.3 million of non-cash stock expenses, compared to $5.2 million in non-cash stock expenses for the year ended December 31, 2020.
G&A Expenses: General and administrative expenses for the year ended December 31, 2021 were $8.5 million, compared to $7.9 million for the year ended December 31, 2020, an increase of $0.6 million. General and administrative expenses for the year ended December 31, 2021 included $3.5 million of non-cash stock expenses, compared to $3.1 million in non-cash stock expenses for the year ended December 31, 2020.
Net Loss: Net loss attributable to common stockholders for the year ended December 31, 2021 was $56.7 million, or $0.75 per share, compared to a net loss of $23.1 million, or $0.41 per share, for the year ended December 31, 2020.

On March 28, 2022 Chugai Pharmaceutical Co., Ltd. (TOKYO: 4519) reported that it obtained regulatory approval from the Ministry of Health, Labour and Welfare (MHLW) for the anti-HER2 humanized monoclonal antibodies, Perjeta for intravenous infusion 420mg/14 mL [generic name: pertuzumab] (hereafter, Perjeta) and Herceptin for intravenous infusion 60 and 150 [generic name: trastuzumab] (hereafter, Herceptin), for the additional indication of advanced or recurrent HER2-positive colon cancer or rectal cancer not amenable to curative resection that has progressed after cancer chemotherapy (Press release, Chugai, MAR 28, 2022, View Source [SID1234611046]).

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"There are many effective treatments available for colorectal cancer, however, recent genetic studies have revealed that some patients do not respond adequately to standard treatment. We are very pleased that now we have the opportunity to offer the combination therapy of Perjeta and Herceptin to these patients, as a new treatment option based on personalized medicine," said Chugai’s President and CEO, Dr. Osamu Okuda. "HER2-positive colorectal cancer is a rare subtype that accounts for 1~5% of all colorectal cancers. This approval opens the way for personalized medicine for this subtype for the first time in Japan and overseas. I would like to thank everyone who supported us for this approval, especially those who participated in the investigator-initiated study which formed the basis of this trial, including patients, their families and medical professionals. We are committed to providing information on the proper use of this combination therapy so that it may contribute to the treatment of colorectal cancer."

The approval is based on the results of an investigator-initiated phase II clinical trial (TRIUMPH study) conducted in Japan that evaluated the efficacy and safety of the combination of Perjeta and Herceptin in 30 patients with HER2-positive, curatively unresectable advanced or recurrent colorectal cancer who had previously undergone chemotherapy. The primary endpoint was the objective response rate as determined by the investigator’s judgment. Objective response was observed in 29.6% of patients confirmed HER2 expression in tumor tissue and 28.0% of patients diagnosed with HER2-positive and RAS wild-type by liquid biopsy. Major adverse events included infusion-related reactions, diarrhea, decreased appetite, nausea, stomatitis and nasopharyngitis.

HER2 protein overexpression and gene amplification should be determined with the pathological testing kit VENTANA ultraView Pathway HER2 (4B5) provided by Roche Diagnostics K.K. and Pathvision HER-2 DNA Probe Kit provided by Abbott Japan Joint company. Both tests obtained regulatory approval on March 17, 2022 and March 10, 2022, respectively, as companion diagnostics for Perjeta and Herceptin to identify patients with HER2 positive colorectal cancer who may benefit from the combination therapy.

[Approval Information (Perjeta)] * This additional section only

Indications

Advanced or recurrent HER2-positive colon cancer or rectal cancer not amenable to curative resection that has progressed after cancer chemotherapy
Dosage and administration

For cases of advanced or recurrent HER2-positive colon cancer or rectal cancer not amenable to curative resection that has progressed after cancer chemotherapy, the usual adult dosage is a loading dose of 840 mg pertuzumab (genetical recombination) and subsequent doses of 420 mg every 3 weeks, each administered by intravenous infusion over 60 minutes, in combination with trastuzumab (genetical recombination). If the first infusion is well tolerated, subsequent infusions may be administered over a shorter time of at least 30 minutes.
[Approval Information (Herceptin)] * This additional section only

Indications

Advanced or recurrent HER2-positive colon cancer or rectal cancer not amenable to curative resection that has progressed after cancer chemotherapy
Dosage and administration

Use Regimen B for advanced or recurrent HER2-positive colon cancer or rectal cancer not amenable to curative resection that has progressed after cancer chemotherapy, in combination with pertuzumab (genetical recombination).
Regimen B: The usual adult dosage is a loading dose of 8 mg/kg (body weight) trastuzumab (genetical recombination) and subsequent doses of 6 mg/kg every 3 weeks, each administered by intravenous infusion over at least 90 minutes.

If the first infusion is well tolerated, subsequent infusions may be administered over a shorter time of at least 30 minutes.

About Perjeta

Perjeta is a humanized monoclonal antibody that targets human epidermal growth factor receptor type 2 (HER2), which is involved in the growth of tumor cells. Perjeta in combination with Herceptin blocks the HER-signaling system more extensively. The drug was launched in 2013 for "inoperable or recurrent HER2-positive breast cancer." The indication was amended as "HER-2 positive breast cancer," after obtaining regulatory approval for the additional indication of "neoadjuvant and adjuvant therapy in HER2-positive breast cancer" in 2018.

About Herceptin

Herceptin, like Perjeta, is a humanized monoclonal antibody that targets human epidermal growth factor receptor type 2 (HER2), which is involved in the growth of tumor cells. Herceptin was launched in 2001 for "metastatic breast cancer overexpressing HER2." In 2011, it was approved for the treatment of patients with "advanced or recurrent gastric cancer overexpressing HER2 not amenable to curative resection," and in 2021 for "advanced or recurrent HER2-positive salivary gland cancer not amenable to curative resection."

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On March 28, 2022 Abeona Therapeutics Inc. (Nasdaq: ABEO), a fully-integrated leader in gene and cell therapy, reported that Vish Seshadri, Ph.D., Chief Executive Officer of Abeona Therapeutics, will participate in a panel discussion titled, "Cell Therapy: How the Definition has Expanded with Time, and the Potential Applications for Rare Diseases," at the Cantor Fitzgerald Virtual Rare Disease Summit on Wednesday, March 30, 2022 at 4:00 p.m. ET (Press release, Abeona Therapeutics, MAR 28, 2022, View Source [SID1234611045]).

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Those interested in participating in the Cantor Fitzgerald Virtual Rare Orphan Disease Summit are encouraged to contact their Cantor Fitzgerald representative.