On April 7, 2020 Ascentage Pharma (6855.HK), a globally focused, clinical-stage biotechnology company engaged in developing novel therapies for cancers, chronic hepatitis B (CHB), and age-related diseases, reported that it has received approval from the Center for Drug Evaluation of the China National Medical Products Administration for the Phase Ib/II study of APG-2575, the company’s novel Bcl-2‒selective inhibitor, for the treatment of patients with relapsed/refractory chronic lymphocytic leukemia or small lymphocytic lymphoma (r/r CLL/SLL) (Press release, Ascentage Pharma, APR 7, 2020, View Source;small-lymphocytic-lymphoma-in-china-301036549.html [SID1234556188]).

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This open-label, Phase Ib/II dose-escalation and dose-expansion study is designed to evaluate the safety, tolerability, and preliminary anticancer activity of APG-2575 as a single agent or in combination therapies for the treatment of patients with r/r CLL/SLL in China.

APG-2575 is a novel, orally administered Bcl-2‒selective inhibitor being developed by Ascentage Pharma. APG-2575 is designed to treat several hematologic malignancies by selectively blocking Bcl-2 to restore the normal apoptosis process in cancer cells.

Recent studies have demonstrated that Bcl-2 inhibitors are effective in CLL/SLL, and Bcl-2 inhibitors in combination with Bruton tyrosine kinase inhibitors or CD20 monoclonal antibodies can deepen patient responses and prolong patient survival. These findings provide the rationale for investigating APG-2525 as a single agent or in combination therapies. The Phase I study of APG-2575 monotherapy, previously commenced in the US, Australia, and China, has shown a favorable safety profile and promising anticancer activity, and the preliminary data support the continued clinical investigation of APG-2575.

CLL/SLL is a hematologic malignancy caused by mature B-cell neoplasms, and it mainly affects middle-aged and elderly populations. The condition is characterized by accumulation of cancerous lymphocytes in peripheral blood, bone marrow, spleen, and lymph nodes. CLL/SLL is the most common type of adult leukemia in North America and Europe but is less common in Asian countries such as China and Japan. However, with an aging population, as well as dietary and lifestyle changes in China, CLL/SLL incidences have been increasing, and more patients develop the condition at younger ages and display greater disease progression. Despite significant initial responses to current first-line treatments, many patients with CLL/SLL need continued treatment to maintain these responses, and relapse often portends a poor prognosis. At present, there are even fewer treatment options for patients with CLL/SLL in China than in Europe or North America, hence representing an urgent clinical need for more effective and safe therapies.

"APG-2575 is a key drug candidate in our development pipeline targeting apoptosis, and the first China-made Bcl-2‒selective inhibitor to enter clinical studies, with great potential as a monotherapy and in combination therapies in several hematologic malignancies. Upon receiving recent approvals and clearances for several Phase Ib/II studies of APG-2575 in China, Australia, and the US, we are now advancing its clinical development globally," said Dr. Yifan Zhai, Chief Medical Officer of Ascentage Pharma. "There is an enormous unmet clinical need in the treatment of CLL/SLL globally. We will soon initiate this Phase Ib/II study of APG-2575 in China, which hopefully will provide a new treatment option to patients with CLL/SLL."

On April 7, 2020 Kazia Therapeutics Limited (ASX: KZA;NASDAQ: KZIA), an Australian oncology-focused biotechnology company, reported to share positive interim data from its ongoing phase II study of paxalisib (formerly GDC-0084) in glioblastoma, the most common and most aggressive form of primary brain cancer (Press release, Kazia Therapeutics, APR 7, 2020, View Source [SID1234556187]).

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Key Points

Interim analysis of Part A (escalation cohort) (n=9) showed median overall survival (OS) of 17.7 months, representing a clinically meaningful extension of life when compared to the 12.7 months associated with the existing standard of care, temozolomide
Interim analysis of all evaluable patients (Part A = 9; Part B = 21) shows median progression-free survival (PFS) of 8.5 months, broadly in line with previous analysis, and comparing favourably to the 5.3 months associated with temozolomide
The longest-treated patient remains progression-free 19 months after diagnosis
Approximately half the enrolled patients remain on drug and both OS and PFS figures may further improve as the trial progresses toward conclusion
Kazia expects to present further data in 2H CY2020 and final data in 1H CY2021
Summary of Paxalisib Data in Comparison to Temozolomide (existing standard of care)

Temozolomide
(FDA-approved treatment)

Paxalisib

(interim phase II data)

Progression-Free Survival (PFS)

Measures ability of a drug to slow
growth of a tumour

5.3 months

8.5 months

Overall Survival (OS)

Measures ability of a drug to
prolong life

12.7 months

17.7 months

Kazia CEO, Dr James Garner, commented, "This is an excellent result, and we are delighted with the emerging data. The ‘gold standard’ for any new cancer treatment is the ability to extend life – an especially challenging goal in a disease such as glioblastoma – and this data provides our first evidence that paxalisib may achieve this objective in a very challenging patient population."

He continued, "There have not been any new drug treatments for newly-diagnosed glioblastoma patients for over twenty years, and we aspire to change that situation. We believe that paxalisib is rapidly becoming one of the most promising drug candidates in the global pipeline for this very challenging disease and we will be working strenuously to make it available to patients as quickly and efficiently as possible. The study continues to follow a number of patients who remain on treatment, and we currently expect to report a further data read-out in the second half of the year."

Background

The reported overall survival (OS) figure of 17.7 months represents a strong signal of clinical efficacy. The existing, FDA-approved standard of care, temozolomide, is associated with an OS of 12.7 months in this patient population[1]. Comparison between different studies is always imprecise, but the magnitude of the numerical difference provides powerful evidence that treatment with paxalisib may extend life in this patient group.

The reported progression-free survival (PFS) figure of 8.5 months is slightly better than the figure of 8.4 months previously reported in November 2019. Temozolomide is associated with a PFS of 5.3 months in this patient population.

Before losing patent protection, temozolomide achieved peak sales in excess of US$ 1 billion per annum, which provides an indication of the commercial opportunity associated with a new treatment for glioblastoma.

Thirty patients were enrolled to this study, comprising 9 in Part A, and 21 in Part B. Data reported here are provisional figures from Part A (for OS) and from the entire study population (for PFS), but may change as ongoing patients proceed through the study. The study has been conducted at leading centers of excellence in the United States.

The safety of paxalisib remained broadly consistent with prior experience, with hyperglycaemia (raised blood sugar), oral mucositis (mouth ulcers), and low-grade rash among the most common drug-related toxicities.

In addition to this phase II study in glioblastoma, four other studies are underway with paxalisib in different forms of brain cancer, and it is anticipated that several of these will provide initial efficacy data during CY 2020.

This data had been accepted for presentation at the annual meeting of the American Association of Cancer Research (AACR) (Free AACR Whitepaper) in San Diego, CA from 24 -29 April 2020. However, given the cancellation of the AACR (Free AACR Whitepaper) meeting due to the COVID-19 outbreak, Kazia has determined to provide an update to investors via this ASX announcement.

Next Steps

The phase II study remains ongoing, with approximately half of the total enrolled patient population still receiving drug at the time of analysis and a number of additional patients still in follow-up. At this stage, Kazia expects to present further data in 2H CY2020, and final data in 1H CY2021.

On April 7, 2020 Quest Diagnostics Incorporated (NYSE: DGX), the world’s leading provider of diagnostic information services, reported that it will report first quarter 2020 results on Wednesday, April 22, 2020, before the market opens (Press release, Quest Diagnostics, APR 7, 2020, View Source [SID1234556186]). It will hold its quarterly conference call to discuss the results beginning at 8:30 a.m. Eastern Time on that day.

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The company also will provide an update on the impact of the COVID-19 pandemic. On March 31, 2020, Quest Diagnostics filed a Current Report on Form 8-K with the Securities and Exchange Commission to outline the impact of the pandemic on its operating results, cash flows and financial condition and withdraw its previously announced guidance for full year 2020.

The conference call can be accessed by dialing 888-455-0391 within the U.S. and Canada, or 773-756-0467 internationally, using the passcode: "Investor." The earnings release and live webcast will be posted on www.QuestDiagnostics.com/investor. The company suggests participants dial in approximately 10 minutes before the call.

A replay of the call may be accessed online at www.QuestDiagnostics.com/investor or by phone at 800-839-1170 for domestic callers or 402-998-0559 for international callers; no passcode is required. Telephone replays will be available from approximately 10:30 a.m. Eastern Time on April 22, 2020 until midnight Eastern Time on May 6, 2020.

Anyone listening to the call is encouraged to read the company’s periodic reports on file with the Securities and Exchange Commission, including the discussion of risk factors and historical results of operations and financial condition in those reports.

On April 7, 2020 Alpha Tau Medical – the developer of breakthrough alpha-radiation cancer therapy Alpha DaRT, and Medison Canada – a fully owned subsidiary of Medison Pharma, reported that they have entered into an exclusive distribution arrangement (Press release, Alpha Tau Medical, APR 7, 2020, View Source [SID1234556185]). Medison is a specialized pharmaceutical and biotech company purely focused on licensing highly innovative, cutting edge therapeutics for patients in need of life-saving therapies in rest-of-the-world markets. Medison has exclusive commercial distribution rights in Israel, and according to the new arrangement, Medison shall also have the exclusive rights to commercialize and distribute the Alpha DaRT in Canada. The arrangement for Canada is for a 15-year period from Marketing Approval of the product in Canada. The parties have agreed on a split of revenues between both sides, and Alpha Tau will be entitled to receive upfront, regulatory and milestone sales-based fees of up to US$9 million.

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Alpha Tau CEO, Uzi Sofer, commented, "This arrangement meaningfully furthers our important mission to bring our live-saving cancer therapy to thousands of cancer patients in Canada who may not have other treatment options remaining. We are confident that with Medison’s capabilities, their outstanding team on the ground, and their track record of commercial success around the world, we will build together a successful commercial launch for Alpha DaRT in Canada. Even in these challenging times, we continue to push forward our mission to help cancer patients around the world, with active ongoing patient recruitment and treatment, and we appreciate Medison’s vote of confidence in uncertain times."

Raphi Levy, CFO of Alpha Tau, added, "We are very excited to have allied ourselves with such a leading partner in the Canadian therapeutics market. Canada is assuming increased importance for Alpha Tau as a nexus for our pre-clinical and clinical research. We are excited to work with Medison on bringing our therapy to Canadian clinicians, who are already learning about the Alpha DaRT and are eager to deploy it."

Meir Jakobsohn, Founder and CEO of Medison Pharma, commented, "Alpha DaRT is a great addition to our Canadian portfolio, and we believe that Alpha Tau’s breakthrough technology together with Medison’s on-the-ground extensive commercial capabilities can make a real change in patients’ lives in Canada."

Joe O’Neill, General Manager Medison Canada, added: "We are very excited to partner with Alpha Tau to bring this important treatment option to Canadian patients and their healthcare providers. Canada continues to be a leader in providing clinical trial support to innovative R&D companies in the oncology field and Medison Canada is proud that Alpha Tau is working with one of our leading hospitals, CHUM in Montreal, to develop this important treatment."

As part of its extensive global clinical trial plan, Alpha Tau has recently launched a clinical trial for patients with advanced pancreatic cancer in Montreal, conducted at CHUM (Montreal University Hospital Center). The company is also engaged in a research projects on alpha radiation dosing with leading universities and institutions in Montreal and Israel.

About Alpha DaRT
Alpha DaRT(Diffusing Alpha-emitters Radiation Therapy) enables highly potent and conformal alpha-irradiation of solid tumors. The treatment is delivered by intratumoral insertion of radium-224 impregnated seeds. When the radium decays, its short-lived daughters are released from the seed, and disperse while emitting high-energy alpha particles that destroy the tumor. Since the alpha-emitting atoms diffuse only a short distance, Alpha DaRT mainly affects the tumor, sparing the healthy tissue around it.

On April 7, 2020 SIRION Biotech GmbH reported that Beam Therapeutics licensed rights to use SIRION Biotech’s LentiBOOST for use in their CAR-T cell products (Press release, SIRION Biotech, APR 7, 2020, View Source [SID1234556183]).

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CAR-T cell therapy represents a promising and future-defining shift in cancer treatment. Beam Therapeutics is developing a new generation of CAR-T product candidates using its proprietary base editing technology.

Under the terms of this agreement, SIRION agreed to provide Beam with non-exclusive access to its proprietary lentiviral transduction enhancer LentiBOOST for clinical development and commercialization of Beam’s portfolio of CAR-T programs. SIRION will be entitled to undisclosed upfront and milestone payments and is eligible to receive royalties on future product net sales plus license fees tied to commercial success.

Dr. Christian Thirion, CEO and founder of SIRION Biotech GmbH explains: "LentiBOOST was engineered to improve lentiviral transduction of difficult cell types like T-cells and hematopoietic stem cells. This technology enables robust upscaling of the T-cell production process, and helps to reduce manufacturing costs by lowering the amount of lentiviral vectors needed for production of the cell product while at the same time improving clinical efficacy by increasing vector copy numbers (VCN) per cell. We are delighted that the LentiBOOST technology may help Beam further enhance the clinical success of its CAR-T pipeline."

"LentiBOOST is used in an increasing number of clinical trials in the US and in Europe and the technology is more and more considered as a gold standard in manufacturing of cell products. Our non-exclusive licensing strategy makes our technology available to a wide range of companies and research hospitals to boost the efficiency of their various clinical programs," says SVP of Business Development & Licensing, Dr. Sabine Ott.