On February 23, 2022 Ayala Pharmaceuticals, Inc. (Nasdaq: AYLA), a clinical-stage oncology company focused on developing and commercializing small molecule therapeutics for patients suffering from rare tumors and aggressive cancers, reported that it has completed patient enrollment in Part A of the Phase 2/3 RINGSIDE clinical trial evaluating AL102 in desmoid tumors (Press release, Ayala Pharmaceuticals, FEB 23, 2022, View Source [SID1234608871]). AL102 is a potent, selective, oral gamma-secretase inhibitor (GSI). The Company expects to report interim results from Part A by mid-2022.

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"Completion of enrollment in Part A of the RINGSIDE trial is an important milestone in our AL102 clinical program for desmoid tumors," said Roni Mamluk, Ph.D., Chief Executive Officer of Ayala. "Part A will help us determine the optimal dose for the randomized portion of the study, we will be looking for safety and signs of anti-tumor activity based on MRI. We are extremely grateful to the patients who are participating and we look forward to sharing top line data around mid-year."

A total of 36 patients have been enrolled in Part A of the RINGSIDE study which is evaluating the safety, tolerability of AL102, as well as tumor volume by MRI at 16 weeks. Three dosing regimens of AL102 are being tested to determine the optimal dose regimen to advance forward.

In Part A, the effect of food on AL102 absorption was tested in a sub-study and PK results indicate that no food restrictions are required.

Part B of the study will be double-blind, placebo-controlled, and will start immediately after dose selection from Part A, enrolling up to 156 patients age 12 and up with progressive disease, randomized between AL102 or placebo. The study’s primary endpoint will be progression-free survival (PFS) with secondary endpoints including objective response rate (ORR), duration of response (DOR,) and patient-reported Quality of Life (QOL) measures. Patients who participated in Part A are eligible to enroll into an open-label extension study at the selected dose, and long-term efficacy and safety will be monitored.

For more information on the RINGSIDE Phase 2/3 study with AL102 for the treatment of desmoid tumors, please visit ClinicalTrials.gov and reference Identifier NCT04871282.

About AL102

AL102 is a potent, selective, oral gamma secretase inhibitor (GSI). AL102 is currently being developed for the treatment of desmoid tumors, as well as in combination with Novartis’ B-cell maturation antigen (BCMA)-targeting agents for the treatment of multiple myeloma (MM).

About Desmoid Tumors

Desmoid tumors, also called aggressive fibromatosis or desmoid-type fibromatosis, are rare connective tissue tumors that typically arise in the upper and lower extremities, abdominal wall, head and neck area, mesenteric root and chest wall with the potential to arise in additional parts of the body. Desmoid tumors do not metastasize, but often aggressively infiltrate neurovascular structures and vital organs. People living with desmoid tumors are often limited in their daily life due to chronic pain, functional deficits, general decrease in their quality of life and organ dysfunction. Desmoid tumors have an annual incidence of approximately 1,700 patients in the United States and typically occur in patients between the ages of 15 and 60 years. They are most commonly diagnosed in young adults between 30-40 years of age and are more prevalent in females. Today, surgery is no longer regarded as the cornerstone treatment of desmoid tumors due to high rate of recurrence post-surgery and there are currently no FDA-approved systemic therapies for the treatment of unresectable, recurrent or progressive desmoid tumors.

On February 23, 2022 Arcus Biosciences, Inc. (NYSE:RCUS), a clinical-stage, global biopharmaceutical company focused on developing differentiated molecules and combination therapies for people with cancer, reported financial results for the fourth quarter and year ended December 31, 2021 and provided a corporate update on its six clinical-stage molecules targeting TIGIT, the adenosine axis (CD73 and dual A2a/A2b receptor), HIF-2a and PD-1 across common cancers, including lung, colon, pancreatic and prostate (Press release, Arcus Biosciences, FEB 23, 2022, View Source [SID1234608870]).

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"Arcus is starting 2022 with a strong cash position and late-stage pipeline that includes two ongoing, and soon to be four, registrational Phase 3 studies for the anti-TIGIT antibody, domvanalimab," said Terry Rosen, Ph.D., Chief Executive Officer of Arcus. "Our strategy is to efficiently investigate and advance novel combinations of our six clinical-stage molecules in areas of high unmet need. We look forward to presenting, later this year, randomized datasets from our trials in lung and pancreatic cancer, both settings with large patient populations with great need for better treatment options."

Anti-TIGIT program (domvanalimab and AB308)

Recent Updates:

First patient was dosed in PACIFIC-8, a registrational Phase 3 study being conducted in collaboration with AstraZeneca. PACIFIC-8 is the second Phase 3 study for domvanalimab and is evaluating domvanalimab plus durvalumab, an anti-PD-L1 antibody, in unresectable Stage 3 NSCLC with curative intent, where durvalumab is standard of care.
2022 Milestones:

Data from ARC-7, an ongoing randomized 150-patient three-arm study in first-line PD-L1≥50% NSCLC, including progression-free survival data for all three arms, are expected to be presented in 2H22.
Arcus and Gilead plan to initiate two new Phase 3 studies for domvanalimab and zimberelimab in lung and gastrointestinal (GI) cancers, as well as additional clinical studies of domvanalimab-based combinations, in 2022.
Presentation of initial data from the Phase 1/1b ARC-12 study evaluating AB308, an Fc-enabled anti-TIGIT antibody, in combination with zimberelimab in advanced malignancies.
Etrumadenant (A2a/A2b adenosine receptor antagonist)

2022 Milestones:

Data from the etrumadenant-containing arm of ARC-7 are anticipated to be presented in 2H22.
Data from the randomized cohort of ARC-6 evaluating etrumadenant plus zimberelimab and docetaxel versus docetaxel in second-line metastatic castrate-resistant prostate cancer (CRPC) are anticipated in 2H22.
Additional clinical studies for etrumadenant, including combinations with domvanalimab, are being planned with Gilead.
Quemliclustat (small molecule CD73 inhibitor)

2022 Milestones:

Results from the randomized portion of ARC-8, including data on progression-free survival, are expected to be presented in 2H22.
Full enrollment of the ARC-8 cohort in 2L pancreatic cancer, an area of high unmet need, is on track to be completed in 1H22.
Additional clinical studies for quemliclustat are being planned with Gilead.
AB521 (HIF-2a inhibitor)

Recent Updates:

Initiated a Phase 1 study (ARC-14) in healthy volunteers to confirm Arcus’s PK/PD and tolerability expectations for AB521 and support rapid advancement into cancer patients.
2022 Milestones:

Preclinical data for AB521, alone and in combination with cabozantinib, as well as initial PK/PD data from the evaluation of AB521 in healthy volunteers, will be presented at the ESMO (Free ESMO Whitepaper) Targeted Anticancer Therapies Congress on March 7, 2022.
A Phase 1/1b study to explore AB521 in clear-cell renal cell carcinoma, alone and in combination with other molecules, including those targeting the CD73-adenosine axis, is anticipated to be initiated in mid-2022.
Discovery Programs:

Recent Updates:

In January, Arcus met the first milestone under an agreement with BVF, which is focused on the discovery and development of a potentially first-in-class small molecule designed to treat a wide range of inflammatory conditions.
Arcus recently selected AB598 (CD39 antibody) as a development candidate, which is advancing into IND-enabling studies; several other oncology discovery programs continue to progress.
Financial Results for the Fourth Quarter and Full Year Ended December 31, 2021

Cash, cash equivalents and investments were $681.3 million as of December 31, 2021, compared to $735.1 million as of December 31, 2020. The decrease was primarily due to cash used in operations and purchases of property and equipment totaling $26.1 million, partially offset by gross proceeds of $220.4 million received under the Amended and Restated Stock Purchase Agreement with Gilead in February 2021. Upon the receipt of $725 million in option exercise payments from Gilead in January 2022, Arcus’s cash and cash equivalents and investments totaled approximately $1.4 billion. Arcus expects cash, cash equivalents and marketable securities on-hand to be sufficient to fund operations into 2026.
Revenues: Collaboration and license revenues were $354.5 million for the three months ended December 31, 2021, compared to $9.5 million for the same period in 2020. The increase was primarily driven by license revenue recognized upon closing of Gilead’s exercise of its options. Collaboration and license revenues were $382.9 million for the full year ended December 31, 2021, compared to $77.5 million for the same period in 2020.
R&D Expenses: Research and development expenses were $49.9 million for the three months ended December 31, 2021, compared to $48.7 million for the same period in 2020. The increase was primarily driven by costs incurred to support Arcus’s expanded clinical and development activities including increased compensation costs related to a larger employee base, increased clinical trial costs, and increased early-stage research costs. Approximately $3.2 million of the increase in compensation costs is related to non-cash stock-based compensation. Research and development expenses were $256.3 million for the full year ended December 31, 2021, compared to $159.3 million for the same period in 2020.
G&A Expenses: General and administrative expenses were $23.3 million for the three months ended December 31, 2021, compared to $12.8 million for the same period in 2020. The increase was driven by the increased complexity of supporting Arcus’s expanding clinical pipeline and collaboration obligations, as well as compliance costs associated with Arcus’s growth. Arcus’s growing employee base and 2021 stock awards drove an increase in employee compensation costs, including approximately $2.7 million of increased non-cash stock-based compensation. General and administrative expenses were $72.3 million for the full year ended December 31, 2021, compared to $42.4 million for the same period in 2020.
Net Income: Net income was $279.4 million for the three months ended December 31, 2021, compared to net loss of $51.9 million for the same period in the prior year. Net income was primarily due to license revenue recognized upon closing of Gilead’s exercise in December 2021 of its options. Net income was $52.8 million for the full year ended December 31, 2021, compared to net loss of $122.9 million for the same period in 2020.
Arcus Clinical Study Overview

Carbo/pem: carboplatin/pemetrexed; dom: domvanalimab; durva: durvalumab; etruma: etrumadenant; gem/nab-pac: gemcitabine/nab-paclitaxel; quemli: quemliclustat; R/R: relapsed/refractory; SOC: standard of care; zim: zimberelimab CRC: colorectal cancer; CRPC: castrate-resistant prostate cancer; NSCLC: non-small cell lung cancer; PDAC: pancreatic ductal adenocarcinoma

On February 23, 2022 Aulos Bioscience, an immuno-oncology company working to revolutionize cancer care through the development of potentially best-in-class IL-2 therapeutics, reported that it has obtained approval from the Monash Health Human Research Ethics Committee (HREC) to initiate a Phase 1/2, first-in-human trial of AU-007, a monoclonal antibody computationally designed by Biolojic Design (Press release, Aulos Bioscience, FEB 23, 2022, View Source [SID1234608866]). AU-007 leverages a highly differentiated approach to harnessing the power of interleukin-2 (IL-2) to eradicate solid tumors.

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The Phase 1/2 study will be conducted in partnership with Monash Health, the largest public health service provider in Victoria, Australia. The study will evaluate the safety, tolerability and immunogenicity of AU-007 as a monotherapy and in combination with aldesleukin in patients with unresectable locally advanced or metastatic cancer. Aulos anticipates initiating enrollment and dosing in the first half of 2022, with preliminary data expected by year end.

"We are excited to move forward with the clinical development of AU-007 as a novel approach to treating cancers," said Aron Knickerbocker, Aulos Bioscience’s chief executive officer. "New preclinical data show strong evidence of anti-cancer activity, including complete tumor elimination when dosed in combination with checkpoint inhibitors. These data provide further validation that AU-007 can tip the delicate balance toward immune activation and away from immune suppression without eliciting IL-2’s immunosuppressive and toxic effects, and we look forward to sharing the results in the future."

Aulos will be attending the Gordon Research Conference "Antibody Biology and Engineering" meeting, being held March 13-18, 2022, in Ventura, California.

About AU-007

AU-007 is a computationally designed, human IgG1 monoclonal antibody that is highly selective to the CD25-binding portion of IL-2. With a mechanism of action unlike any other IL-2 therapeutic in development, AU-007 leverages the body’s own IL-2 to reinforce anti-tumor immune effects. This is achieved by preventing IL-2, either exogenous or secreted by T effector cells, from binding to trimeric receptors on T regulatory cells while still allowing IL-2 to bind and expand T effector and NK cells. This prevents the negative feedback loop caused by other IL-2-based treatments and biases the immune system toward activation over suppression. AU-007 also prevents IL-2 from binding to trimeric receptors on vasculature and pulmonary endothelium, which may significantly reduce the vascular leak syndrome and pulmonary edema associated with high-dose IL-2 therapy.

On February 23, 2022 PTC Therapeutics, Inc. (NASDAQ: PTCT) reported that management will present a company update at the following conferences (Press release, PTC Therapeutics, FEB 23, 2022, View Source [SID1234608865]):

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Cowen Virtual 42nd Annual Health Care Conference
Monday, March 7 at 1:30 p.m. ET

Barclays Global Healthcare Conference
Thursday, March 17 at 8 a.m. ET

The presentations will be webcast live on the Events and Presentations page under the Investor section of PTC Therapeutics’ website at View Source and will be archived for 30 days following the presentation. It is recommended that users connect to PTC’s website several minutes prior to the start of the webcast to ensure a timely connection.

On February 23, 2022 Oasmia Pharmaceutical AB (Oasmia), an oncology-focused specialty pharmaceutical company, reported that the first patient has now fully completed the study in the Investigator-Initiated Phase 1b trial of Docetaxel micellar for advanced prostate cancer (trial SAKK 67/20 of the Swiss Group for Clinical Cancer Research SAKK) (Press release, Oasmia, FEB 23, 2022, View Source [SID1234608863]). Furthermore, the first of three dosing groups in the trial has been successfully recruited and the first patient has started in the second dose group.

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Francois Martelet, MD, Chief Oasmia Officer, Oasmia, commented: "We are grateful for the significant effort of the Swiss Group for Clinical Cancer Research (SAKK), our investigators and clinics have made in progressing the study so far. SAKK has informed us that it is well-positioned to complete recruitment this year and secure data crucial for the continued development of Docetaxel micellar."

Oasmia’s Docetaxel micellar is a solvent-free formulation of docetaxel developed to avoid the need for the solubility enhancers in solvent-based docetaxel, and the mandatory high-dose steroid premedication, while providing an effective treatment option. Prostate cancer is a significant and increasingly prevalent health problem worldwide and is the leading cause of male cancer deaths.

The SAKK 67/20 trial (NCT04629781) is an open-label, multicenter, single-stage Phase 1b trial at major hospitals in Switzerland, recruiting 18 chemotherapy-naïve patients with metastatic castration resistant prostate cancer (mCRPC) with adequate bone marrow, liver, and renal function. The primary objective of this trial is to determine the maximum tolerated dose of Docetaxel micellar in patients with mCRPC and the secondary objectives are to evaluate safety, assess the preliminary anti-tumor activity, and to characterize the pharmacokinetics in this population.