On January 19, 2023 Bellicum Pharmaceuticals, Inc. (Nasdaq: BLCM), a leader in developing novel, controllable cellular immunotherapies for cancers, reported that an abstract on early results from a Phase 1 clinical trial for BPX-601, its lead GoCAR-T product candidate, has been accepted for poster presentation at the 2023 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Genitourinary Cancers Symposium (ASCO GU) (Press release, Bellicum Pharmaceuticals, JAN 19, 2023, View Source [SID1234626351]). The meeting is being held February 16-18, 2023 in San Francisco and virtually. The ongoing trial is being conducted in patients with metastatic castration-resistant prostate cancer.

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Details of the poster presentation are as follows:

Title: Early Results from a Phase 1, Multicenter Trial of PSCA-Specific GoCAR-T Cells (BPX-601) in Patients with Metastatic Castration-Resistant Prostate Cancer (mCRPC)
Poster Board: E1, Abstract 140
Presenter: Mark N. Stein, M.D.
Time/Location: Thursday, February 16, 2023, 2:30 p.m. ET / 11:30 a.m. PT, Level 1, West Hall, Moscone Center

On january 19, 2023 BeiGene (NASDAQ: BGNE; HKEX: 06160; SSE: 688235), a global biotechnology company, reported that the Medicines and Healthcare products Regulatory Agency (MHRA) has granted marketing authorizations for BRUKINSA (zanubrutinib) in Great Britain for both the treatment of adult patients with chronic lymphocytic leukemia (CLL) and the treatment of adult patients with marginal zone lymphoma (MZL) who have received at least one prior anti-CD20-based therapy (Press release, BeiGene, JAN 19, 2023, View Source [SID1234626350]).

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"BRUKINSA is a highly selective BTK inhibitor that has demonstrated clinically meaningful improvements over the first generation of BTK inhibitor in relapsed CLL," said Dr Renata Walewska, Department of Hematology, University Hospitals Dorset, Bournemouth, UK. "The authorization of BRUKINSA for MZL and CLL in Great Britain is a significant step forward for eligible patients and their physicians as there is no targeted treatment currently authorized for MZL patients other than chemoimmunotherapy, and it represents an alternative to current BTKi treatments for patients with CLL."

The MHRA authorization for CLL is based on two global Phase 3 clinical trials: SEQUOIA (NCT03336333)1, comparing BRUKINSA against bendamustine plus rituximab (BR) in patients with previously untreated CLL, and ALPINE (NCT03734016)2, comparing BRUKINSA against IMBRUVICA (ibrutinib) in patients with relapsed/refractory (R/R) CLL.

The MHRA authorization for MZL is based on results from the multicenter, global, single-arm, open-label, Phase 2 MAGNOLIA3 trial in patients with R/R MZL who received at least one anti-CD-20 based regimen.

"As a BTK inhibitor designed to maximize BTK occupancy and minimize off-target binding, we believe BRUKINSA presents a very promising treatment option for eligible patients with MZL and CLL," said Mehrdad Mobasher, M.D., M.P.H., Chief Medical Officer, Hematology at BeiGene.

"As we strive for delivering cancer medicines more quickly to more patients around the world, we are pleased with the progress we’ve made in bringing BRUKINSA to more eligible patients with hematological malignancies in Great Britain following these authorizations," added Dr. Robert Mulrooney, General Manager, UK & Ireland at BeiGene.

Earlier this year, the National Institute for Health and Care Excellence (NICE), recommended BRUKINSA for the treatment of Waldenström’s macroglobulinemia (WM) in adults who have had at least one treatment, only if bendamustine plus rituximab is also suitable. BRUKINSA has also been recommended by the Scottish Medicines Consortium for the treatment of adult patients with WM who have received at least one prior therapy, or in first-line treatment for patients unsuitable for chemoimmunotherapy.

BRUKINSA is currently authorized in the European Union, and Northern Ireland, as per rulings set out in the Northern Ireland Protocol,for the treatment of adult patients with WM who have received at least one prior therapy or as the first-line treatment for patients unsuitable for chemoimmunotherapy; for the treatment of adult patients with CLL and for adult patients with MZL who have received at least one prior anti-CD20-based therapy.

About Chronic Lymphocytic Leukemia

A life-threatening cancer of adults, CLL is a type of mature B-cell malignancy in which abnormal leukemic B lymphocytes (a type of white blood cells) arise from the bone marrow and flood peripheral blood, bone marrow, and lymphoid tissues4,5. CLL is the most common type of leukemia in adults, accounting for about one-quarter of new cases of leukemia6. In Europe, the estimated incidence is 4.92/100,000 persons per year7,8.

About Marginal Zone Lymphoma

MZL is a group of ultra-rare, slow growing B-cell malignancies that begin in the marginal zones of lymph tissue9. Epidemiological data from Europe is limited, but the incidence rate of MZL is estimated to range between 20 and 30 persons per million per year10,11,12. There are three different subtypes of MZL: extranodal marginal zone B-cell lymphoma, or mucosa-associated lymphoid tissue (MALT), which is most common; nodal marginal zone B-cell lymphoma which develops in the lymph nodes and is rare; and splenic marginal zone B-cell lymphoma which develops in the spleen, bone marrow, or both, and is the rarest form of the disease13.

About BRUKINSA

BRUKINSA is a small-molecule inhibitor of Bruton’s tyrosine kinase (BTK) discovered by BeiGene scientists that is currently being evaluated globally in a broad clinical program as a monotherapy and in combination with other therapies to treat various B-cell malignancies. BRUKINSA was specifically designed to deliver targeted and sustained inhibition of the BTK protein by optimizing bioavailability, half-life, and selectivity. With differentiated pharmacokinetics compared to other approved BTK inhibitors, BRUKINSA has been demonstrated to inhibit the proliferation of malignant B cells within a number of disease-relevant tissues.

BRUKINSA is supported by a broad clinical program which includes more than 4,700 subjects in 35 trials in more than 30 geographies. To date, BRUKINSA is approved in over 60 markets, including the United States, China, the European Union, Great Britain, Canada, Australia, South Korea, Iceland, Norway and Switzerland.

On January 19, 2023 bluebird bio, Inc. (Nasdaq: BLUE) ("bluebird") reported the pricing of its underwritten public offering of 20,000,000 shares of its common stock at a public offering price of $6.00 per share, before deducting underwriting discounts and commissions (Press release, bluebird bio, JAN 19, 2023, View Source [SID1234626344]). bluebird also granted the underwriters a 30-day option to purchase up to an additional 3,000,000 shares of its common stock at the public offering price per share, less underwriting discounts and commissions. The gross proceeds from the public offering are expected be $120 million, before deducting underwriting discounts and commissions and offering expenses payable by bluebird and assuming no exercise of the underwriters’ option to purchase additional shares of common stock. All shares in the offering are to be sold by bluebird.

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Goldman Sachs & Co. LLC and J.P. Morgan Securities LLC are acting as joint book running managers for the offering.

bluebird intends to use the net proceeds of the offering (i) to support commercialization and manufacturing for its two approved gene therapies, ZYNTEGLO and SKYSONA; (ii) to accelerate future commercialization activities for its gene therapy candidate, lovotibeglogene autotemcel (lovo-cel) for sickle cell disease, if approved; and (iii) to fund working capital and other general corporate purposes.

The offering is expected to close on or about January 23, 2023, subject to customary closing conditions.

The offering is being made pursuant to an effective shelf registration statement on Form S-3, including a prospectus, that was filed with the U.S. Securities and Exchange Commission (the "SEC") on February 18, 2020 and was automatically effective upon filing. A preliminary prospectus supplement describing the terms of the offering has been filed with the SEC. A final prospectus supplement will be filed with the SEC and will form a part of the effective registration statement. Copies of the final prospectus supplement and accompanying prospectus relating to the offering may be obtained, when available, by contacting Goldman Sachs & Co. LLC, Attention: Prospectus Department, 200 West Street, New York, NY 10282, by phone at (866) 471-2526, or by email at [email protected]; or J.P. Morgan Securities LLC, Attention: c/o Broadridge Financial Solutions, 155 Long Island Avenue, Edgewood, NY 11717, by phone at (866) 803-9204, or by email at [email protected].

This press release shall not constitute an offer to sell or the solicitation of an offer to buy these securities, nor shall there be any sale of these securities in any state or other jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such state or jurisdiction.

Forward-Looking Statements

This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Examples of forward-looking statements in this press release include, without limitation, statements regarding the consummation of the offering, the terms of the offering and the anticipated use of the net proceeds from the offering. All statements that are not statements of historical facts are, or may be deemed to be, forward-looking statements. Such forward-looking statements are based on historical performance and current expectations and projections about our future financial results, goals, plans and objectives and involve inherent risks, assumptions and uncertainties, including internal or external factors that could delay, divert or change any of them in the next several years, that are difficult to predict, may be beyond our control and could cause our future financial results, goals, plans and objectives to differ materially from those expressed in, or implied by, the statements. No forward-looking statement can be guaranteed. Forward-looking statements in this press release should be evaluated together with the many risks and uncertainties that affect bluebird bio’s business, particularly those identified in the risk factors discussion in bluebird bio’s Annual Report on Form 10-K, as updated by our subsequent Quarterly Reports on Form 10-Q, Current Reports on Form 8-K and other filings with the Securities and Exchange Commission. These risks include, but are not limited to: the risk that we may not realize expected cost savings from the restructuring, including the anticipated decrease in operational expenses, at the levels we expect; we may encounter additional delays in the development of our programs, including the imposition of new clinical holds or delays in resolving existing clinical holds, that may impact our ability to meet our expected timelines and increase our costs; the internal and external costs required for our ongoing and planned activities, and the resulting impact on expense and use of cash, may be higher than expected which may cause us to use cash more quickly than we expect or change or curtail some of our plans or both; our expectations as to expenses, cash usage and cash needs may prove not to be correct for other reasons such as changes in plans or actual events being different than our assumptions; the risk that the efficacy and safety results from our prior and ongoing clinical trials will not continue or be seen in additional patients treated with our product candidates; the risk that additional insertional oncogenic or other reportable events associated with lentiviral vector, drug product, or myeloablation will be discovered or reported over time; the risk that our eli-cel, beti-cel and lovo-cel programs may be subject to further delays in their development, including but not limited to the imposition of new clinical holds; the risk that lovo-cel may not be approved within the priority review timeframe or at all; and the risk that any one or more of our products and product candidates, including eli-cel, beti-cel or lovo-cel, will not be successfully developed, approved or commercialized. The forward-looking statements included in this press release are made only as of the date of this press release and except as otherwise required by applicable law, bluebird bio undertakes no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events, changed circumstances or otherwise.

On January 19, 2023 Encysce Biosciences presented its investor presentation (Presentation, Ensysce Biosciences, JAN 19, 2023, View Source [SID1234626338]).

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On January 18, 2023, ImmuneOnco Biopharmaceuticals (Shanghai) Inc. (hereinafter referred to as "ImmuneOnco") reported that the Phase Ib/IIa clinical study of the first domestic SIRPαFc fusion protein drug targeting human CD47 (project number: IMM01) combined with boron tezomib and dexamethasone in the treatment of multiple myeloma has been approved by the National Medical Products Administration (NMPA), further establishing ImmuneOnco’s position as a leader in the research and development of CD47-targeted drugs (Press release, ImmuneOnco Biopharma, JAN 18, 2023, View Source [SID1234655675]).

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IMM01 has entered phase II clinical development for multiple indications, covering hematological tumors and solid tumors, involving different drug combinations (including AZA, PD1 antibody and other drugs), and the clinical development of multiple indications fully reflects the differentiation layout.

Dr. Tian Wenzhi, Founder and Chairman of ImmuneOnco, said, "I am very pleased to know that the phase Ib/IIa clinical study of IMM01 combined with bortezomib and dexamethasone in the treatment of multiple myeloma has been approved by NMPA. Due to the differential design of our drug molecules, IMM01 has shown no interaction with red blood cells in vitro Combined, it will not cause severe anemia events. At the same time, due to the glycosylation modification, the immunogenicity of the drug is greatly reduced, the pharmacokinetic parameters are improved, and the bioavailability of the drug is significantly improved, so that the dose can be climbed in the single-drug clinical phase I. The slope stage showed good safety and efficacy. During the single-drug dose escalation process, 2 cases of complete remission (CR) and 1 case of partial remission (PR) appeared in patients with relapsed and refractory lymphoma who had undergone repeated treatment. One of the patients has been in long-term remission for more than 2 years. In addition, it is particularly important that complete remission has been observed in the previously enrolled CMML, MDS and AML patients, which is expected to bring long-term survival opportunities to patients We will work closely with clinical experts and subjects to accelerate the clinical trial research of IMM01 and benefit cancer patients as soon as possible."