On November 22, 2024 Nanjing Leads Biolabs Co., Ltd. (hereinafter referred to as "Leads Biolabs") reported that LBL-024, an anti-PD-L1/4-1BB bispecific antibody independently developed by the company with global intellectual property rights for the treatment of neuroendocrine cancer, has obtained Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA) (Press release, Nanjing Leads Biolabs, NOV 22, 2024, View Source [SID1234648556]). This marks another significant milestone following the Breakthrough Therapy Designation granted to LBL-024 by the Center for Drug Evaluation (CDE) of National Medical Products Administration (NMPA) in China.

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Dr. Charles Cai, Chief Medical Officer of Leads Biolabs, said "LBL-024 obtained Breakthrough Therapy Designation from CDE in October this year for the treatment of advanced extrapulmonary neuroendocrine cancer. Our clinical data to date has been very encouraging, suggesting that LBL-024 could meaningfully improve outcomes of patients living with this devastating disease. The grant of ODD from FDA further underscores the exceptional potential of LBL-024 to address a critical gap in this therapeutic area. These policy supports for obtaining Orphan Drug qualification will greatly expedite the commercialization of LBL-024, potentially changing the treatment landscape for patients with advanced neuroendocrine cancer who currently have few therapeutic options."

Dr. Xiaoqiang Kang, founder, chairman and CEO of Leads Biolabs, said "The receipt of ODD for LBL-024 from FDA represents a pivotal milestone in our global strategy. This designation not only enables LBL-024 to receive additional policy support and resource allocation during its development, accelerating its path to market and positioning it as a potential first-in-class therapeutic antibody targeting 4-1BB worldwide, but also provides us with greater market opportunities and avenues for growth on a global scale."

About LBL-024

LBL-024 is a tetravalent bispecific antibody that simultaneously targets PD-L1 and 4-1BB, serving dual functions: blocking the immunosuppressive PD-1/PD-L1 pathway, and selectively co-stimulating 4-1BB in the tumor microenvironment to enhance immune responses. The dual functions of LBL-024—lifting PD-1/PD-L1 immune inhibition and intensifying 4-1BB modulated T cell activation—synergistically enhance the anti-tumor immune response.

LBL-024 received IND approvals from both FDA and NMPA on July 30, 2021 and September 9, 2021 respectively to conduct phase Ⅰ/Ⅱ clinical research, and has achieved outstanding results. Sponsored by Leads Biolabs, led by Professor Shen Lin from Beijing Cancer Hospital with participation of multiple clinical trial centers, the current clinical data demonstrate that LBL-024 monotherapy has more than doubled both the Objective Response Rate (ORR) and Overall Survival (OS) compared to existing treatments for this disease. Based on the current treatment status and the available safety and efficacy data, LBL-024 has entered into a single-arm pivotal trial for extrapulmonary neuroendocrine carcinomas in July 2024 and stands as the globally first 4-1BB-targetd drug candidates to have reached pivotal stage, according to Frost & Sullivan.

About Neuroendocrine Cancer

Neuroendocrine carcinoma (NEC) is a class of poorly differentiated, high-grade neuroendocrine neoplasms (NEN), which originate in the diffuse neuroendocrine cell system and may occur in many different sites. Its molecular characteristics are significantly different from those of neuroendocrine tumors (NET). NEC can be divided into pulmonary NEC and extrapulmonary NEC, among which pulmonary NEC includes small cell lung cancer (SCLC) and pulmonary large cell neuroendocrine carcinoma (p-LCNEC). According to previous data collected, the number of new cases of SCLC is about 98,193, p-LCNEC is about 19,639, and extrapulmonary NECs is about 9,820 in the United States every year. As a result, there are an estimated 127,652 new cases of NECs in the United States each year, meeting the FDA’s definition of a rare disease.

Symptoms of NEC can vary depending on the type of tumor, its location in the body and the hormone released. The survival rate of NEC varies according to the type of cancer and whether it spreads. Specifically, the 5-year survival rate of pulmonary NECs is 5.6%, digestive tract NECs is 13.1%, and other primary NECs are 26.0%. Due to the limited effective treatment options available for patients, the overall survival rate of NEC is low, and more effective new treatment options are urgently needed.

About Orphan Drug Designation

Orphan drugs are medications used for the prevention, treatment, and diagnosis of rare diseases. According to the Orphan Drug Act of the United States, Orphan Drug Designation is established to encourage the development of drugs for treating rare diseases. It provides a series of incentives for new drug development, including but not limited to: (1) tax credits for clinical trial expenses; (2) specific guidance from FDA on all stages of clinical research; (3) exemption of the application fee for new drug registration; and (4) 7 years of market exclusivity after listing.

On November 21, 2024 Allink Biotherapeutics, a clinical-stage biotechnology company pioneering next-generation bispecific antibody and ADC therapeutics, reported two significant milestones in the development of its lead program ALK201 (Press release, Shanghai Allink Biotherapeutics, NOV 21, 2024, View Source [SID1234648782]). The U.S. Food and Drug Administration (FDA) just cleared the company’s Investigational New Drug (IND) application, and first patient has been successfully dosed in Australia.

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Aberrations in FGF/FGFR2 signaling pathways are extensively documented in multiple malignancies, including gastroesophageal adenocarcinoma and breast cancer, correlating with poor clinical outcomes. ALK201 is an innovative ADC candidate targeting FGFR2b a critical oncogenic driver that has demonstrated clinical validation in various solid tumors . Leveraging the company’s proprietary hydrophilic linker and rationally selected payload, extensive preclinical studies of ALK201 have demonstrated robust anti-tumor activity and a promising therapeutic window, supporting its development as a potentially transformative ADC for FGFR2b-associated malignancies.

"The FDA’s IND clearance and first patient dosing in Australia represent significant milestones of ALK201, our first ADC program, advances into clinical development," said Hui Feng, Ph.D., Chief Executive Officer of AllinkBio. "The rapid advancement from first preclinical candidate to clinical stage exemplifies our R&D team’s operational efficiency. Encouraged by robust preclinical data demonstrating the anti-tumor efficacy in FGFR2b-expressing solid tumors. We are strategically advancing our clinical development to swiftly bring this potential therapeutic option to address unmet medical needs worldwide."

The Phase 1 multicenter clinical trial was designed to investigate ALK201, evaluating its safety, tolerability, pharmacokinetic profile, and preliminary antitumor activity in patients with advanced solid tumors. The study consists of two sequential segments: a dose-escalation phase to determine the maximum tolerated dose (MTD), followed by a clinical expansion phase in which biomarker-guided patient populations will be further investigated.

AllinkBio team’s strategic vision and outstanding R&D execution capability have enabled the company growing from a preclinical to clinical stage biotech in about one year. The company’s deep expertise in molecule discovery and drug design, combined with a sophisticated biomarker-driven approach, has empowered swift development of a number of promising therapeutic candidates. AllinkBio is highly committed to bringing innovative precision therapeutics to patients in need.

About ALK201
ALK201 is a novel FGFR2b targeted ADC, in development as a potential treatment for multiple solid tumors. FGFR2b is a promising target with its mutations and overexpression comprehensively implicated in oncogenesis across diverse solid tumor landscapes. ALK201 is currently being evaluated in a Phase 1 study.

On November 21, 2024 Intensity Therapeutics, Inc. (Nasdaq: INTS), ("Intensity" or the "Company") a late-stage clinical biotechnology company focused on the discovery and development of proprietary, novel immune-based intratumoral cancer therapies designed to kill tumors and increase immune system recognition of cancers, reported that it has entered into a securities purchase agreement with a single healthcare focused institutional investor for the issuance and sale of 1,237,113 shares of its common stock in a registered direct offering at a purchase price of $2.425 per share (Press release, Intensity Therapeutics, NOV 21, 2024, View Source [SID1234648578]).

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In a concurrent private placement (the "Private Placement" and together with the Registered Offering, the "Offerings"), the Company also agreed to issue to the same investor warrants to purchase up to 1,237,113 shares of its common stock (the "Common Warrants"). The Common Warrants have an exercise price of $2.95 per share, will be exercisable commencing six months from the date of issuance, and will expire five and one-half years following the date of issuance.

The gross proceeds from the Offerings, before deducting the placement agent’s fees and other offering expenses payable by the Company, are expected to be approximately $3 million. The Company expects to use the net proceeds from the Offerings for general working capital.

A.G.P./Alliance Global Partners is acting as lead placement agent for the Offerings and Brookline Capital Markets, a division of Arcadia Securities, LLC, is acting as a co-placement agent for the Offerings.
The Offerings are expected to close on or about November 22, 2024, subject to the satisfaction of customary closing conditions.

The shares (or common stock equivalents in lieu thereof) offered to the institutional investor described above are being offered pursuant to a registration statement on Form S-3 (File No. 333-280681), which was declared effective by the Securities and Exchange Commission (the "SEC") on July 11, 2024. The Offering is being made only by means of a prospectus which is a part of the effective registration statement. The Common Warrants will be issued in a concurrent private placement. A final prospectus supplement and the accompanying prospectus relating to the registered direct offering will be filed with the SEC and will be available on the SEC’s website at www.sec.gov. Additionally, when available, electronic copies of the final prospectus supplement and the accompanying prospectus may be obtained from A.G.P./Alliance Global Partners, 590 Madison Avenue, 28th Floor, New York, NY 10022, or by telephone at (212) 624-2060, or by email at [email protected], or Brookline Capital Markets, a division of Arcadia Securities, LLC, 600 Lexington Avenue, 20th Floor, New York, NY 10022, or by telephone at (646) 256-5258, or by email at [email protected].
The private placement of the Common Warrants and the shares underlying the Common Warrants offered to the institutional investor will be made in reliance on an exemption from registration under Section 4(a)(2) of the Securities Act of 1933, as amended (the "Securities Act") and Regulation D promulgated thereunder. Accordingly, the securities issued in the concurrent private placements may not be offered or sold in the United States except pursuant to an effective registration statement or an applicable exemption from the registration requirements of the Securities Act and such applicable state securities laws.

On November 21, 2024 BPGbio, Inc., a leading biology-first, AI-powered clinical-stage biopharma company focused on mitochondrial biology and protein homeostasis, reported that it will co-present two pivotal studies with Stanford Medicine’s Department of Neurology at the Society for Neuro-Oncology (SNO) 2024 Annual Meeting, taking place November 21-24, 2024, in Houston, Texas (Press release, BPGbio, NOV 21, 2024, View Source [SID1234648568]). The presentations will highlight BPM31510 and BRG399—potential glioblastoma multiforme (GBM) therapies that were identified by the company’s proprietary NAi Interrogative Biology Platform, a causal AI-powered system designed to reveal hidden cause-and-effect relationships within patient biology.

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The first study, titled "Optimizing brain cancer therapy: balancing tumor eradication and normal tissue preservation with BPM31510," explores BPM31510’s superior ability to preserve healthy cells compared to standard cancer treatments in mouse, rat, and human glioblastoma cell lines. The study demonstrates BPM31510’s ability to effectively target and diminish cancer cells while selectively allowing the growth of healthy brain cells, an effect that standard treatments fail to achieve. Researchers also showed that the BPM31510 significantly improved survival in glioma implanted mice and rats compared to the control group, mirroring the results from the ongoing phase 2b trial on BPM31510 for GBM and highlighting BPM31510’s potential in brain cancer therapy.

The second study, titled "Improved anti-glioblastoma efficacy by BRG399, a novel oral microtubule binding agent," presents findings on BRG399’s anti-cancer activity. BRG399, a novel pan-cancer therapy, works by disrupting the cell division process in both solid and liquid tumors. BRG399 was also found to induce an immune memory response, protecting against tumor recurrence. The study confirmed that BRG399 can cross into the brain, target tumor cells, and improve survival in rat glioma models.

"Standard cancer treatments that focus solely on destroying tumors often harm healthy tissue in the process," said Seema Nagpal, M.D., principal investigator of the BPM31510 GBM phase 2b trial and Clinical Professor of Neurology and Neurological Sciences at Stanford Medicine. "These new treatments are taking novel targeted approaches on glioma cancer cells and we are excited to share the encouraging results from our studies with our peers and industry researchers."

"Our ongoing clinical and preclinical trials for BPM31510 and BRG399 are continuously deepening our understanding of these promising therapies," said Niven R. Narain, Ph.D., President and CEO of BPGbio. "By putting biology first, our NAi Platform has enabled us to optimize every aspect of these therapies, from dosing and timing to patient selection and potential indication expansion opportunities."

Poster Presentation Details:

Optimizing brain cancer therapy: balancing tumor eradication and normal tissue preservation with BPM31510
Date and Time: November 22, 2024, 7:30 p.m. – 9:30 p.m. CST
Location: Hall B3, George R. Brown Convention Center, Houston, Texas
Presenter: Abbas Khojasteh, Ph.D.
Abstract Number: DDDR-13

Improved anti-glioblastoma efficacy by BRG399, a novel oral microtubule binding agent
Date and Time: November 22, 2024, 7:30 p.m. – 9:30 p.m. CST
Location: Hall B3, George R. Brown Convention Center, Houston, Texas
Presenter: Stephane Gesta, Ph.D.
Abstract Number: DDDR-14
About BPM31510

BPM31510IV is BPGbio’s lead candidate in late-stage development for aggressive solid tumors such as glioblastoma multiforme (GBM) and pancreatic cancer. Other topical and oral formulations of the investigational agent are also being developed as a potential treatment for several rare diseases. The compound has demonstrated a tolerable safety profile and shown potential clinical benefits across multiple disease indications. Validated by BPGbio’s NAi Interrogative Biology platform, BPM31510 induces a hallmark shift in the tumor microenvironment (TME) by modulating mitochondrial oxidative phosphorylation in aggressive tumors, leading to cancer cell death. In many mitochondrial diseases, restoring CoQ10 levels can overcome the effect of mutations in genes that lead to mitochondrial dysfunction. BPM31510 has been granted Orphan Drug Designation by the FDA for GBM, pancreatic cancer, and epidermolysis bullosa (EB), as well as Rare Pediatric Disease Designation for primary CoQ10 deficiency and EB.

About BRG399

BRG399 is a BPGbio-developed candidate being studied for its therapeutic potential as a treatment for solid and liquid tumor cancers as well as diseases associated with inflammation. This experimental drug, a first-in-class, anti-mitotic agent with broad-spectrum anti-cancer activity and favorable pharmacological properties for clinical testing, is being designed for oral delivery. BRG399 is leading the new oncology drug pipeline for BPGbio which includes drug candidates uniquely targeting E2 enzymes.

On November 21, 2024 Novocure (NASDAQ: NVCR) reported that the U.S. Food and Drug Administration (FDA) approved its new Head Flexible Electrode (HFE) transducer arrays for use with Optune Gio for the treatment of adult patients with glioblastoma multiforme (GBM) (Press release, NovoCure, NOV 21, 2024, View Source [SID1234648567]).

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"At Novocure we recognize product innovation must deliver meaningful results for our patients"

Optune Gio is a wearable, portable device that produces alternating electric fields known as Tumor Treating Fields (TTFields), which are delivered through non-invasive, wearable arrays. TTFields exert physical forces on the electrically charged components of dividing cancer cells, which disrupt the rapid cell division exhibited by cancer cells.

The new HFE arrays utilize a flexible polymer material in place of the ceramic discs used in the current Optune Gio arrays, making them one-third lighter and 50% thinner.

"At Novocure we recognize product innovation must deliver meaningful results for our patients," said Mukund Paravasthu, Chief Operating Officer, Novocure. "The newly FDA approved HFE arrays are lighter, thinner and designed to be more comfortable, clear benefits for the patient. We look forward to introducing the new arrays in the U.S. and will continue our work to deliver product innovations that prioritize the needs of people using our treatments."

Novocure plans to convert Optune Gio users in the U.S. to the new HFE arrays through the first half of 2025 through a controlled transition plan.

Important Safety Information

What is Optune Gio approved to treat?

Optune Gio is a wearable, portable, FDA-approved device indicated to treat a type of brain cancer called glioblastoma multiforme (GBM) in adult patients 22 years of age or older.

Newly diagnosed GBM

If you have newly diagnosed GBM, Optune Gio is used together with a chemotherapy called temozolomide (TMZ) if:

Your cancer is confirmed by your healthcare professional AND
You have had surgery to remove as much of the tumor as possible
Recurrent GBM

If your tumor has come back, Optune Gio can be used alone as an alternative to standard medical therapy if:

You have tried surgery and radiation and they did not work or are no longer working AND
You have tried chemotherapy and your GBM has been confirmed by your healthcare professional
Who should not use Optune Gio?

Optune Gio is not for everyone. Talk to your doctor if you have:

An implanted medical device (programmable shunt), skull defect (missing bone with no replacement), or bullet fragment. Optune Gio has not been tested in people with implanted electronic devices, which may cause the devices not to work properly, and Optune Gio has not been tested in people with skull defects or bullet fragments, which may cause Optune Gio not to work properly
A known sensitivity to conductive hydrogels (the gel on the arrays placed on the scalp like the ones used on EKGs). When Optune Gio comes into contact with the skin, it may cause more redness and itching or may rarely cause a life-threatening allergic reaction
Do not use Optune Gio if you are pregnant or are planning to become pregnant. It is not known if Optune Gio is safe or effective during pregnancy.

What should I know before using Optune Gio?

Optune Gio should only be used after receiving training from qualified personnel, such as your doctor, a nurse, or other medical staff who have completed a training course given by Novocure, the maker of Optune Gio.

Do not use any parts that did not come with the Optune Gio Treatment Kit sent to you by Novocure or given to you by your doctor
Do not get the device or transducer arrays wet
If you have an underlying serious skin condition on the scalp, discuss with your doctor whether this may prevent or temporarily interfere with Optune Gio treatment
What are the possible side effects of Optune Gio?

Most common side effects of Optune Gio when used together with chemotherapy (temozolomide, or TMZ) were low blood platelet count, nausea, constipation, vomiting, tiredness, scalp irritation from the device, headache, seizure, and depression. The most common side effects when using Optune Gio alone were scalp irritation (redness and itchiness) and headache. Other side effects were malaise, muscle twitching, fall and skin ulcers. Talk to your doctor if you have any of these side effects or questions.

Please click here for the Optune Gio Instructions For Use (IFU) for complete information regarding the device’s indications, contraindications, warnings, and precautions.

About Tumor Treating Fields

Tumor Treating Fields (TTFields) are electric fields that exert physical forces to kill cancer cells via a variety of mechanisms. TTFields do not significantly affect healthy cells because they have different properties (including division rate, morphology, and electrical properties) than cancer cells. These multiple, distinct mechanisms work together to target and kill cancer cells. Due to these multimechanistic actions, TTFields therapy can be added to cancer treatment modalities in approved indications and demonstrates enhanced effects across solid tumor types when used with chemotherapy, radiotherapy, immune checkpoint inhibition, or targeted therapies in preclinical models. TTFields therapy provides clinical versatility that has the potential to help address treatment challenges across a range of solid tumors.