On January 17, 2023 BeiGene (NASDAQ: BGNE; HKEX: 06160; SSE: 688235), a global biotechnology company,reported that it will share clinical data and patient-reported outcomes for its PD-1 inhibitor, tislelizumab, at the 2023 ASCO (Free ASCO Whitepaper) Gastrointestinal Cancers Symposium, notably, an oral presentation for interim results of the global pivotal phase 3 trial RATIONALE 305 of tislelizumab in combination with chemotherapy in first-line gastric or gastroesophageal junction (G/GEJ) cancer (Press release, BeiGene, JAN 17, 2023, View Source [SID1234626282]). At the interim analysis, RATIONALE 305 trial met one of the primary endpoints of overall survival (OS) in patients with G/GEJ whose tumors express PD-L1.

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"With more than one million gastric cancer cases and approximately 770,000 deaths in 2020, we have a meaningful opportunity to advance care for these patients. We are pleased to report a significant survival benefit for tislelizumab and chemotherapy at this interim analysis of the RATIONALE 305 trial," Mark Lanasa, M.D., Chief Medical Officer, Solid Tumors at BeiGene. "We look forward to sharing additional results as the study continues, including results for the intent-to-treat population."

Analyses from additional studies of tislelizumab as monotherapy and in combination will be presented at the conference.

Tislelizumab presentations 2023 ASCO (Free ASCO Whitepaper) Gastrointestinal Cancers Symposium

Abstract 286 Phase 3 Study of Tislelizumab Plus Chemotherapy vs Placebo Plus Chemotherapy as First-line Treatment of Advanced Gastric or Gastroesophageal Junction Adenocarcinoma

Jan 19, 2023 Oral Presentation (4:15pm, PST)
Abstract 340 Randomized, global, phase 3 study of tislelizumab plus chemotherapy versus placebo plus chemotherapy as first-line treatment for advanced or metastatic esophageal squamous cell carcinoma (RATIONALE-306): non-Asia subgroup

Jan 19, 2023 Poster Session A (12:00pm and 4:45pm, PST)
Abstract 495 Tislelizumab versus sorafenib in first-line treatment of unresectable hepatocellular carcinoma: Impact on health-related quality of life in RATIONALE-301 population

Jan 20, 2023 Rapid Abstract Session (7:00am – 7:45am, PST)
About Tislelizumab

Tislelizumab is a humanized IgG4 anti-PD-1 monoclonal antibody specifically designed to minimize binding to Fcγ receptors on macrophages, helping to aid the body’s immune cells to detect and fight tumors.

Tislelizumab is the first investigational medicine from BeiGene’s immuno-oncology biologics program and is being evaluated in solid tumor and hematologic malignancies, as monotherapy and in combination.

The global tislelizumab clinical development program includes more than 11,500 subjects enrolled to-date in 21 registration-enabling trials, from more than 30 countries and regions.

Biologics License Applications (BLA) for tislelizumab are under review with U.S. and European Union (E.U.) regulators; for unresectable recurrent locally advanced or metastatic esophageal squamous cell carcinoma (ESCC) in the U.S.; for non-small cell lung cancer and unresectable recurrent locally advanced or metastatic ESCC in the E.U.

Tislelizumab is approved by the China NMPA as a treatment for nine indications and additional three supplemental BLAs under review at CDE. Tislelizumab is not approved for use outside of China.

About the Clinical Trials

RATIONALE 301 (NCT03412773) is a global, Phase 3, randomized, open-label study of tislelizumab compared with sorafenib as a first-line treatment in adult patients with unresectable hepatocellular cancer (HCC), G/GEJ adenocarcinoma. The primary endpoint of the study is non-inferiority of Overall Survival between the two treatment groups. The key secondary endpoint is Overall Response Rate, as assessed by Blinded Independent Review Committee (BIRC) per RECIST v1.1. Other secondary endpoints include other efficacy assessments such as Progression Free Survival, Duration of Response, and Time to Progression per BIRC, as well as measures of health-related quality of life, and safety and tolerability.

RATIONALE 305 (NCT03777657) is a randomized, double-blind, placebo-controlled global Phase 3 trial comparing the efficacy and safety of tislelizumab combined with chemotherapy and placebo combined with chemotherapy as a first-line treatment for patients with locally advanced, unresectable or metastatic G/GEJ adenocarcinoma. The primary endpoint of the trial is OS. Secondary endpoints include progression-free survival (PFS), overall response rate (ORR), duration of response (DoR), and safety. A total of 997 patients from 13 countries and regions globally, including close to 50 percent from outside of China, were enrolled and randomized 1:1 to receive either tislelizumab and chemotherapy or placebo and chemotherapy.

RATIONALE 306 (NCT03783442) is a randomized, placebo-controlled, double-blind, global Phase 3 study to evaluate the efficacy and safety of tislelizumab in combination with chemotherapy as a first-line treatment in patients with advanced or metastatic ESCC. The primary endpoint of the trial is overall survival (OS). Secondary endpoints include progression free survival, overall response rate, and duration of response per RECIST v1.1, as well as health-related quality of life measures and safety.

The trial enrolled 649 patients at research centers across Asia-Pacific, Europe, and North America. Patients were randomized 1:1 to receive either tislelizumab plus chemotherapy or placebo plus chemotherapy.

On January 17, 2023 Alkermes plc (Nasdaq: ALKS) reported that nemvaleukin alfa (nemvaleukin), the company’s investigational, novel engineered interleukin-2 (IL-2) variant immunotherapy, has been granted an Innovation Passport for the treatment of mucosal melanoma under the Innovative Licensing and Access Pathway (ILAP) by the Medicines and Healthcare products Regulatory Agency (MHRA), the regulatory body of the United Kingdom (UK) (Press release, Alkermes, JAN 17, 2023, View Source [SID1234626281]). The Innovation Passport designation is the entry point to the ILAP, which aims to accelerate time to market and facilitate patient access to medicines in the UK for life-threatening or seriously debilitating conditions, or conditions for which there is a significant patient or public health need. Benefits of ILAP include access to a range of development tools, such as the potential for a 150-day accelerated Marketing Authorization Application (MAA) assessment, rolling review and a continuous benefit-risk assessment.

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"We believe in the potential of nemvaleukin to help address persistent unmet needs faced by patients with mucosal melanoma," said Craig Hopkinson, M.D., Chief Medical Officer and Executive Vice President of Research & Development at Alkermes. "The granting of the Innovation Passport represents an exciting step in the clinical development program of our IL-2 candidate, and we look forward to working closely with the MHRA and the ILAP partner agencies in an effort to bring nemvaleukin to people living with mucosal melanoma in the UK."

The U.S. Food and Drug Administration previously granted Orphan Drug designation and Fast Track designation to nemvaleukin for the treatment of mucosal melanoma.

About Innovation Passport and ILAP

ILAP was launched by the MHRA in January 2021 with an aim to accelerate the development of and facilitate patient access to medicines. The Innovation Passport is granted by the UK’s ILAP Steering Group, which consists of representatives from MHRA, the National Institute for Health and Care Excellence (NICE), the Scottish Medicines Consortium (SMC), the All Wales Therapeutics and Toxicology Centre (AWTTC) and the National Health Service (NHS) England. It is the first step in the ILAP process and awarded to companies developing therapies with the potential to offer significant benefit to patients who have conditions that are life-threatening or seriously debilitating. A single Innovation Passport can cover multiple future indications for the same molecule.

About Nemvaleukin Alfa (nemvaleukin)

Nemvaleukin is an investigational, novel, engineered fusion protein comprised of modified interleukin-2 (IL-2) and the high affinity IL-2 alpha receptor chain, designed to preferentially expand tumor-killing immune cells while avoiding the activation of immunosuppressive cells by selectively binding to the intermediate-affinity IL-2 receptor complex. The selectivity of nemvaleukin is designed to leverage the proven anti-tumor effects of existing IL-2 therapy while mitigating certain limitations. Nemvaleukin is currently the most advanced IL-2-based immuno-therapy in clinical development, with two actively recruiting, potentially registrational studies, ARTISTRY-6 and ARTISTRY-7 in mucosal melanoma and platinum-resistant ovarian cancer, respectively.

About the ARTISTRY Clinical Development Program

ARTISTRY is an Alkermes-sponsored clinical development program evaluating nemvaleukin as a potential immunotherapy for cancer. The ARTISTRY program is comprised of multiple clinical trials evaluating intravenous and subcutaneous dosing of nemvaleukin, both as a monotherapy and in combination with the anti-PD-1 therapy KEYTRUDA (pembrolizumab) in patients with advanced solid tumors. Trials in the ARTISTRY program include: ARTISTRY-1, ARTISTRY-2, ARTISTRY-3, ARTISTRY-6 and ARTISTRY-7.

On January Orphagen Pharmaceuticals, Inc., a biotech company pioneering the screening, discovery, and development of small molecule ligands that modulate orphan or unexplored members of the nuclear receptor family, reported that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation (RPDD) for OR-449 for the treatment of pediatric adrenocortical carcinoma (ACC) (Press release, Orphagen, JAN 17, 2023, View Source [SID1234626280]).

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OR-449 is a selective, first-in-class, potent and orally bioavailable small molecule antagonist to steroidogenic factor-1 (SF-1 or NR5A1), an orphan nuclear receptor and transcription factor that is essential for the growth and development of the adrenal gland. Orphagen is developing OR-449 for both the adult and pediatric forms of ACC as well as other cancers known to express a high level of SF-1.

"We are gratified that OR-449 has received an RPDD from the FDA. We currently plan to file an Investigational New Drug (IND) application with the FDA later this year to support initiation of a Phase 1 clinical trial," said Orphagen CEO, Scott Thacher, Ph.D. "We speak to clinicians regularly who remind us of the urgent need for an improved therapy for patients with ACC, and this drives our commitment to the clinical development of OR-449, our first internal program to reach this stage."

About ACC and SF-1
ACC is a rare and aggressive cancer of the adrenal gland. Surgical removal of an affected adrenal gland is effective treatment if the tumor has not metastasized. Once an ACC tumor becomes metastatic, as it does for most patients, it is difficult to control and five-year survival in both adult and pediatric patients with metastatic disease is low, about 10-20%. The estimated annual incidence of ACC in the U.S. is 600 patients/year. In clinical practice, SF-1 is widely used as a marker for ACC, and it is recognized as a potential therapeutic target for both adult and pediatric ACC. SF-1 is commonly amplified at the gene level in pediatric ACC, and SF-1 is recognized as a cell lineage marker in the FDA’s Pediatric Cancer Target List.

Rare Pediatric Disease Designation
A rare pediatric disease is defined by the Federal Food, Drug, and Cosmetic Act to include a serious or life-threatening disease, which primarily affects individuals aged from birth to 18 years and affects fewer than 200,000 people in the U.S. To address the challenges that drug companies face when developing treatments for these unique patient populations, Congress reauthorized the Creating Hope Act. Since first enacted by Congress in 2012, the Creating Hope Act has been extended multiple times, most recently on December 27, 2020. Under this Act, companies are eligible to receive a Priority Review Voucher (PRV) following approval of a product with an RPDD if the marketing application receives FDA approval by September 30, 2026, under current legislation, or at a later date if the Act is extended by Congress. If issued, a sponsor may redeem a PRV for priority review of a subsequent marketing application for a different product candidate, or the PRV can be sold or transferred to another sponsor.

On January 17,2023 AIM ImmunoTech Inc. (NYSE American: AIM) ("AIM" or the "Company"), an immuno-pharma company focused on the research and development of therapeutics to treat multiple types of cancers, immune disorders, and viral diseases – including COVID-19, the disease caused by the SARS-CoV-2 virus – reported it has entered into an external sponsored collaborative clinical research agreement with Erasmus MC and AstraZeneca (Press release, AIM ImmunoTech, JAN 17, 2023, View Source [SID1234626277]).

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Under the agreement, Erasmus MC is planning to perform an investigator-initiated clinical study, entitled "Combining anti-PD-L1 immune checkpoint inhibitor durvalumab with TLR-3 agonist rintatolimod in patients with metastatic pancreatic ductal adenocarcinoma for therapy effect. DURIPANC Study," in which it will use both Study Drugs provided by AstraZeneca and AIM ImmunoTech.

"We have been diligently working to advance the development of Ampligen as a synergistic combination therapy with checkpoint inhibitors based on the promising data demonstrated to date. In the previously conducted human clinical studies, Ampligen has shown synergistic potential with PD-1 checkpoint blockade therapeutics. We are thrilled to take this important step forward in clinical development in collaboration with AstraZeneca and its PDL-1 therapy, durvalumab, and further unlock the potential of Ampligen for the treatment of pancreatic cancer where there remains significant unmet need," commented Thomas K. Equels, MS JD, Chief Executive Officer of AIM.

Prof. Casper H.J. van Eijck, MD, PhD, Pancreato-biliary Surgeon at Erasmus MC, added, "PD-L1 expression levels in the malignant tumors of pancreatic cancer patients correlates to poor prognosis. While immune checkpoint inhibitors targeting PD1/PDL1 have shown promise in other solid tumors, they have shown limited efficacy thus far in ductal cancer of the pancreas. This may be in part due to limited CD8+ T-cell maturation necessary for the immune system to combat the cancer. We are excited about the promise of combining Ampligen with durvalumab as previous data has shown that Ampligen can prime dendritic cells and enhance the maturation of T-cells essentially activating the immune system through its activation of TLR3 while durvalumab will bind PD-L1 and ‘uncloak’ the tumor cells in the tumor microenvironment allowing them to be recognized and attacked by the immune system. We believe this one-two punch of TLR-3 stimulation and PD-L1 blockade could lead to synergistic anti-tumor immunity in patients and extend overall and progression free survival in patients with metastatic pancreatic cancer."

Ampligen is AIM’s dsRNA drug currently being developed for globally important cancers. Ampligen has shown therapeutic synergy with checkpoint inhibitors, including increasing survival rates and efficacy, in the treatment of animal tumors when used in combination with checkpoint blockade therapies. The first detection of Ampligen’s synergistic potential with checkpoint blockade therapeutics was witnessed in pre-clinical mouse models of melanoma and pancreatic cancers. Additionally, there is published data from two recent clinical studies – in triple-negative breast cancer and advanced recurrent ovarian cancer – that further support Ampligen’s potential to enhance checkpoint blockade therapy efficacy.

About AIM ImmunoTech Inc.

On January 1, 2023 Kazia Therapeutics Limited (ASX: KZA; NASDAQ: KZIA), an Australian oncology-focused biotechnology company, reported a placement of KZA shares, to institutional and sophisticated investors in Australia, at a price of A$0.11 per share (Press release, Kazia Therapeutics, JAN 17, 2023, View Source [SID1234626276]). The placement will raise A$4.5 million (exclusive of costs).

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Placement

The Placement to professional and sophisticated investors consists of an:

A$2,792,572 unconditional institutional placement of 25,387,018 New Shares, representing approximately 16% of the total shares of Kazia on issue prior to the issue of the New Shares; and

A$1,707,428 institutional placement of 15,522,075 New Shares, conditional on Kazia shareholder approval for the purposes of ASX Listing Rule 7.1.

The price of both the Unconditional Placement and the Conditional Placement will be A$0.11 which represents a 13% premium to the 15-day volume-weighted average price (VWAP) on the Australian Securities Exchange (ASX) up to and including 11 January 2023 of A$0.097.

New Shares to be issued under the Unconditional Placement will be issued without shareholder approval under the Company’s existing placement capacity under ASX Listing Rules 7.1. Settlement and issuance of shares is expected to occur on or around January 16, 2023, with quotation expected on January 17, 2023 in Australia. The New Shares will rank equally with the Company’s existing shares on issue.

An Extraordinary General Meeting (EGM) is expected to be held on 24 February 2023 to seek approval for the issuance of New Shares under the Conditional Placement. Kazia will provide eligible shareholders with a notice of meeting and explanatory materials over the coming days.

The Placement was not underwritten.

Funding will be used to drive Kazia’s clinical program toward several critical inflection points, including the final data read out on the paxalisib GBM AGILE study.