BPGbio and University of Oxford Enter into Strategic Collaboration Focused on Breakthrough E2-based Protein Degradation Research in Oncology and CNS Diseases

On October 7, 2024 BPGbio, Inc., a leading biology-first, AI-powered, clinical stage biopharma focused on mitochondrial biology and protein homeostasis, and the University of Oxford’s Centre for Medicines Discovery, reported a five-year research collaboration focused on advancing novel protein degradation technologies, particularly in oncology and central nervous system (CNS) diseases, with the goal of unlocking new therapeutic pathways for conditions with limited treatment options (Press release, BPGbio, OCT 7, 2024, View Source [SID1234647077]). The organizations will work in phases, starting with the validation of BPGbio’s novel E2 TPD technology, and expanding into future study of the degradable target space, to identify novel targets and generate high quality publications.

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This collaboration builds on BPGbio’s first-in-class E2-based protein degradation program, which features a proprietary library of more than 1,000 Ro3 fragments discovered by BPGbio that are potential ligands and seed compounds to a variety of E2 targets. The collaboration will also utilize BPGbio’s proprietary ternary structures, its computational toolkit for E2 ligand design, and assays for rapidly attaining selectivity and specificity.

The partnership will leverage the Centre for Medicines Discovery’s expertise in translational research, including their groundbreaking efforts in neurological diseases such as Parkinson’s Disease. The team will utilize BPGbio’s proprietary NAi Interrogative Biology Platform, which integrates patient biology with AI-driven analysis, to accelerate biomarker discovery and therapeutics development.

"This collaboration represents a powerful alliance of biology-first science and cutting-edge translational research," said Niven R. Narain, Ph.D., President and CEO of BPGbio. "By harnessing our NAi Interrogative Biology Platform alongside the University of Oxford’s expertise, we aim to push the boundaries of protein degradation science and deliver transformative therapies for diseases like cancer and CNS disorders, where unmet medical needs remain significant."

"By partnering with BPGbio, we’re combining our world-class translational research expertise with their pioneering approach to protein degradation," said Prof. Paul Brennan, Ph.D., FRSC, Director of the Centre for Medicines Discovery at University of Oxford. "This collaboration has the potential to unlock new therapeutic strategies for diseases that have long resisted treatment, including challenging cancers and CNS disorders. We are excited to explore novel targets and bring forward breakthrough therapies that could make a profound difference for patients."

Equillium Announces Abstract Accepted for Poster Presentation at the Society for Immunotherapy of Cancer

On October 7, 2024 Equillium Inc. (Nasdaq: EQ), a clinical-stage biotechnology company leveraging a deep understanding of immunobiology to develop novel therapeutics to treat severe autoimmune and inflammatory disorders, reported that an abstract was accepted for poster presentation at the 39th Annual Meeting of the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) (Press release, Equillium, OCT 7, 2024, View Source [SID1234647076]). The conference will take place at the George R. Brown Convention Center in Houston, Texas from November 6 to 10, 2024.

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Title: Interleukin (IL)-15 and IL-21 synergistically enhance NK and CD8+ T cell responses
Presenting Author: Phoi Tiet, Senior Research Associate, Equillium, Inc.
Abstract Number: 908
Location: Exhibit Halls A & B
Date: Saturday, November 9, 2024

The abstract highlights that cytokines are a focus of anti-cancer therapeutic development due to their central role in regulating anti-tumoral immune responses, and that data demonstrates that IL-15 and IL-21 synergistically augment NK and CD8 T cells activities, which further enhanced their proliferation and cytolytic function. With the ability to rescue T cell dysfunction, this cytokine combination could be a promising treatment approach to stimulating anti-tumor immune responses.

About Multi-Cytokine Platform and Multi-Cytokine Inhibitors EQ101 & EQ302

Our proprietary multi-cytokine platform generates rationally designed composite peptides that selectively block key cytokines at the shared receptor level targeting pathogenic cytokine redundancies and synergies while preserving non-pathogenic signaling. This approach is expected to avoid the broad immuno-suppression and off-target safety liabilities that may be associated with other therapeutic classes, such as Janus kinase inhibitors. Many immune-mediated diseases are driven by the same combination of dysregulated cytokines, and we believe identifying the key cytokines for these diseases will allow us to target and develop customized treatment strategies for multiple autoimmune and inflammatory diseases.

Current platform assets include EQ101, a clinical stage, first-in-class, selective, tri-specific inhibitor of IL-2, IL-9, and IL-15 for intravenous and subcutaneous delivery and EQ302, a preclinical stage, first-in-class, selective, bi-specific inhibitor of IL-15 and IL-21 for oral delivery.

Scholar Rock Announces Proposed Public Offering of Common Stock and Pre-Funded Warrants

On October 7, 2024 Scholar Rock Holding Corporation (Nasdaq: SRRK), a late-stage biopharmaceutical company focused on advancing innovative treatments for spinal muscular atrophy (SMA), cardiometabolic disorders, and other serious diseases where protein growth factors play a fundamental role, reported that it has commenced an underwritten public offering for $275 million of shares of its common stock and, in lieu of common stock to investors who so choose, pre-funded warrants to purchase shares of its common stock (Press release, Scholar Rock, OCT 7, 2024, View Source [SID1234647075]). All of the shares and pre-funded warrants are being offered by Scholar Rock. In addition, Scholar Rock intends to grant the underwriters a 30-day option to purchase additional shares of its common stock in an amount up to 15% of the securities offered in the public offering. The offering is subject to market and other conditions, and there can be no assurance as to whether or when the offering may be completed, or as to the actual size or terms of the offering.

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Scholar Rock intends to use the net proceeds from the offering to support commercialization of apitegromab, to advance its ongoing and future clinical programs, to further develop its technology platform to continue to advance its clinical and preclinical pipeline, and for working capital and other general corporate purposes.

J.P. Morgan Securities LLC, Jefferies LLC and Piper Sandler & Co. are acting as joint book-running managers for the offering. BMO Capital Markets Corp., Wedbush Securities Inc. and Raymond James & Associates, Inc. are acting as co-managers for the offering.

An automatically effective shelf registration statement on Form S-3 relating to the offering of the securities described above was filed with the Securities and Exchange Commission (SEC) on October 7, 2024. A preliminary prospectus supplement and accompanying prospectus relating to the offering will be filed with the SEC and will be available on the SEC’s website located at www.sec.gov. Copies of the preliminary prospectus supplement and the accompanying prospectus relating to this offering may be obtained, when available, by contacting: J.P. Morgan Securities LLC, c/o: Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717, or by email at [email protected] and [email protected]; Jefferies LLC, Attention: Equity Syndicate Prospectus Department, 520 Madison Avenue, New York, NY 10022, by telephone at 877-821-7388, or by email at [email protected]; or Piper Sandler & Co., 800 Nicollet Mall, J12S03, Minneapolis, MN 55402, Attention: Prospectus Department, by telephone at 800-747-3924, or by email at [email protected].

BerGenBio Announces Safety Data from Dose Escalation Phase 1b in First Line NSCLC Patients

On October 7, 2024 BerGenBio ASA (OSE: BGBIO), a clinical-stage biopharmaceutical company developing novel, selective AXL kinase inhibitors for severe unmet medical needs, reported preliminary safety data from the Phase 1b portion of the BGBC016 study in first-line (1L) Non-Small Cell Lung Cancer (NSCLC) patients (Press release, BerGenBio, OCT 7, 2024, View Source [SID1234647074]).

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The Phase 1b part of the study evaluated three escalating doses of BerGenBio’s selective AXL inhibitor bemcentinib in combination with standard chemo-immunotherapy (CIT), doublet chemotherapy and pembrolizumab, Keytruda, for the 1L treatment of advanced/metastatic NSCLC patients. The primary endpoint was the assessment of the safety profile of the combination in NSCLC patients regardless of their STK11 mutational status.

Key conclusions include:

All three selected doses demonstrated that the triplet combination is well tolerated with no new safety signals identified, supporting the further clinical development of bemcentinib and CIT in 1L NSCLC patients.
No dose-related impact on electrocardiographic changes (QTc), a known class effect of tyrosine kinase inhibitors, was reported for bemcentinib during the observation period.
Pharmacokinetic analyses confirmed adequate plasma exposure of bemcentinib, achieving levels consistent with that previously observed in responders in BerGenBio’s BGBC008 study of bemcentinib and pembrolizumab in second-line NSCLC patients.

Martin Olin, Chief Executive Officer of BerGenBio, commented: "The data show that bemcentinib has a manageable safety profile and gives us increased confidence in continuing the BGBC016 clinical trial. The global trial is currently enrolling patients in Phase 2a targeting patients with a mutation in the STK11 gene, and we look forward to sharing the first preliminary data as they mature."

MediLink Announces Global Clinical Trial Collaboration and Supply Agreement on YL201 Combination Therapy

On October 7, 2024 MediLink Therapeutics (Suzhou) Co., Ltd. ("MediLink"), a clinical-stage biotech company, reported a global clinical trial collaboration and supply agreement with Amgen Inc. Amgen will lead a global clinical study to evaluate the therapeutic potential of the combination of MediLink’s B7-H3-targeting antibody-drug conjugate (ADC) YL201 and Amgen’s DLL3- and CD3-targeting bispecific T-cell engager (BiTE) IMDELLTRA in extensive-stage small cell lung cancer (ES-SCLC) under the clinical trial collaboration and supply agreement (Press release, Amgen, OCT 7, 2024, View Source [SID1234647073]). MediLink will provide the investigational drug YL201 for the combination study.

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This open-label, global, multi-center Phase Ib clinical study is designed to evaluate the safety, tolerability, pharmacokinetics, and efficacy of this combination regimen in ES-SCLC patients.

Both YL201 and IMDELLTRA have shown potential in ES-SCLC. In May of this year, IMDELLTRA received accelerated approval from the FDA and is currently marketed in the US for the treatment of adult patients with ES-SCLC with disease progression on or after platinum-based chemotherapy. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s). The efficacy of YL201 monotherapy is encouraging in ES-SCLC. MediLink has announced the data of a Phase I/II clinical trial of YL201 in patients with advanced solid tumors including SCLC as a selected oral presentation at the ESMO (Free ESMO Whitepaper) Congress 2024. This clinical trial collaboration aims to explore the potential of the two innovative drugs in the treatment of ES-SCLC and offer a novel and synergetic mechanism of action for clinical benefit.

About ES-SCLC

SCLC is an aggressive high-grade neuroendocrine carcinoma with extremely poor outcomes [1]. SCLC comprises roughly 15% (~0.36 million new cases) of the 2.4 million new lung cancer cases worldwide each year [2].

Approximately two-thirds of patients with SCLC present with extensive-stage (ES) disease at diagnosis, featuring tumors with distant metastasis or exceeding an area that can be treated within a single radiation field [3-5]. Patients with ES-SCLC, compared with patients with limited-stage (LS-SCLC), manifested with worse prognosis as the median expected overall survival is ~12 months following initial therapy and a 5-year overall survival rate of 3% [6-8].

About YL201

YL201 is an innovative ADC that specifically targets B7-H3. B7-H3 is overexpressed on different malignant cells and cancer-initiating cells of various tumor types, but has a restricted expression in normal tissue [9], indicating its potential as an ADC drug target. YL201 has been developed by utilizing MediLink’s Tumor Microenvironment Activable LINker-payload (TMALIN) conjugated with a highly specific B7-H3 antibody. Currently, YL201 is being investigated in four Phase I or II studies, including one global Phase I clinical study.

About IMDELLTRA

IMDELLTRA is a first-in-class, targeted immunotherapy engineered by Amgen researchers that binds to both DLL3 on tumor cells and CD3 on T cells, activating T cells to kill DLL3-expressing SCLC cells. This results in the formation of a cytolytic synapse with lysis of the cancer cell [10-11]. DLL3 is a protein that is expressed on the surface of SCLC cells in ~85-96% of patients with SCLC, but is minimally expressed on healthy cells, making it an exciting target [12-13].

IMPORTANT SAFETY INFORMATION

WARNING: CYTOKINE RELEASE SYNDROME AND NEUROLOGIC TOXICITY including IMMUNE EFFECTOR CELL-ASSOCIATED NEUROTOXICITY SYNDROME

Cytokine release syndrome (CRS), including serious or life-threatening reactions, can occur in patients receiving IMDELLTRATM. Initiate treatment with IMDELLTRATM using the step-up dosing schedule to reduce the incidence and severity of CRS. Withhold IMDELLTRATM until CRS resolves or permanently discontinue based on severity.
Neurologic toxicity, including immune effector cell-associated neurotoxicity syndrome (ICANS), including serious or life-threatening reactions, can occur in patients receiving IMDELLTRATM. Monitor patients for signs and symptoms of neurologic toxicity, including ICANS, during treatment and treat promptly. Withhold IMDELLTRATM until ICANS resolves or permanently discontinue based on severity.
WARNINGS AND PRECAUTIONS

Cytokine Release Syndrome (CRS): IMDELLTRATM can cause CRS including serious or life-threatening reactions. In the pooled safety population, CRS occurred in 55% of patients who received IMDELLTRATM, including 34% Grade 1, 19% Grade 2, 1.1% Grade 3 and 0.5% Grade 4. Recurrent CRS occurred in 24% of patients, including 18% Grade 1 and 6% Grade 2.
Most events (43%) of CRS occurred after the first dose, with 29% of patients experiencing any grade CRS after the second dose and 9% of patients experiencing CRS following the third dose or later. Following the Day 1, Day 8, and Day 15 infusions, 16%, 4.3% and 2.1% of patients experienced ≥ Grade 2 CRS, respectively. The median time to onset of all grade CRS from most recent dose of IMDELLTRATM was 13.5 hours (range 1 to 268 hours). The median time to onset of ≥ Grade 2 CRS from most recent dose of IMDELLTRATM was 14.6 hours (range: 2 to 566 hours).

Clinical signs and symptoms of CRS included pyrexia, hypotension, fatigue, tachycardia, headache, hypoxia, nausea, and vomiting. Potentially life-threatening complications of CRS may include cardiac dysfunction, acute respiratory distress syndrome, neurologic toxicity, renal and/or hepatic failure, and disseminated intravascular coagulation (DIC).

Administer IMDELLTRATM following the recommended step-up dosing and administer concomitant medications before and after Cycle 1 IMDELLTRATM infusions as described in Table 3 of the Prescribing Information (PI) to reduce the risk of CRS. Administer IMDELLTRATM in an appropriate health care facility equipped to monitor and manage CRS. Ensure patients are well hydrated prior to administration of IMDELLTRATM.

Closely monitor patients for signs and symptoms of CRS during treatment with IMDELLTRATM. At the first sign of CRS, immediately discontinue IMDELLTRATM infusion, evaluate the patient for hospitalization and institute supportive care based on severity. Withhold or permanently discontinue IMDELLTRATM based on severity. Counsel patients to seek medical attention should signs or symptoms of CRS occur.

Neurologic Toxicity, Including ICANS: IMDELLTRATM can cause serious or life-threatening neurologic toxicity, including ICANS. In the pooled safety population, neurologic toxicity, including ICANS, occurred in 47% of patients who received IMDELLTRATM, including 10% Grade 3. The most frequent neurologic toxicities were headache (14%), peripheral neuropathy (7%), dizziness (7%), insomnia (6%), muscular weakness (3.7%), delirium (2.1%), syncope (1.6%), and neurotoxicity (1.1%).
ICANS occurred in 9% of IMDELLTRATM-treated patients. Recurrent ICANS occurred in 1.6% of patients. Most patients experienced ICANS following Cycle 2 Day 1 (24%). Following Day 1, Day 8, and Day 15 infusions, 0.5%, 0.5% and 3.7% of patients experienced ≥ Grade 2 ICANS, respectively. The median time to onset of ICANS from the first dose of IMDELLTRATM was 29.5 days (range: 1 to 154 days). ICANS can occur several weeks following administration of IMDELLTRATM. The median time to resolution of ICANS was 33 days (range 1 to 93 days).

The onset of ICANS can be concurrent with CRS, following resolution of CRS, or in the absence of CRS. Clinical signs and symptoms of ICANS may include but are not limited to confusional state, depressed level of consciousness, disorientation, somnolence, lethargy, and bradyphrenia.

Patients receiving IMDELLTRATM are at risk of neurologic adverse reactions and ICANS resulting in depressed level of consciousness. Advise patients to refrain from driving and engaging in hazardous occupations or activities, such as operating heavy or potentially dangerous machinery, in the event of any neurologic symptoms until they resolve.

Closely monitor patients for signs and symptoms of neurologic toxicity and ICANS during treatment. At the first sign of ICANS, immediately evaluate the patient and provide supportive therapy based on severity. Withhold IMDELLTRATM or permanently discontinue based on severity.

Cytopenias: IMDELLTRATM can cause cytopenias including neutropenia, thrombocytopenia, and anemia. In the pooled safety population, decreased neutrophils occurred in 12% including 6% Grade 3 or 4 of IMDELLTRATM-treated patients. The median time to onset for Grade 3 or 4 neutropenia was 29.5 days (range: 2 to 213). Decreased platelets occurred in 33% including 3.2% Grade 3 or 4. The median time to onset for Grade 3 or 4 decreased platelets was 50 days (range: 3 to 420). Decreased hemoglobin occurred in 58% including 5% Grade 3 or 4. Febrile neutropenia occurred in 0.5% of patients treated with IMDELLTRATM.
Monitor patients for signs and symptoms of cytopenias. Perform complete blood counts prior to treatment with IMDELLTRATM, before each dose, and as clinically indicated. Based on the severity of cytopenias, temporarily withhold, or permanently discontinue IMDELLTRATM.

Infections: IMDELLTRATM can cause serious infections, including life-threatening and fatal infections. In the pooled safety population, infections, including opportunistic infections, occurred in 41% of patients who received IMDELLTRATM. Grade 3 or 4 infections occurred in 13% of patients. The most frequent infections were COVID-19 (9%, majority during the COVID-19 pandemic), urinary tract infection (10%), pneumonia (9%), respiratory tract infection (3.2%), and candida infection (3.2%).
Monitor patients for signs and symptoms of infection prior to and during treatment with IMDELLTRATM and treat as clinically indicated. Withhold or permanently discontinue IMDELLTRATM based on severity.

Hepatotoxicity: IMDELLTRATM can cause hepatotoxicity. In the pooled safety population, elevated ALT occurred in 42%, with Grade 3 or 4 ALT elevation occurring in 2.1%. Elevated AST occurred in 44% of patients, with Grade 3 or 4 AST elevation occurring in 3.2%. Elevated bilirubin occurred in 15% of patients; Grade 3 or 4 total bilirubin elevations occurred in 1.6% of patients. Liver enzyme elevation can occur with or without concurrent CRS. Monitor liver enzymes and bilirubin prior to treatment with IMDELLTRATM, before each dose, and as clinically indicated. Withhold IMDELLTRATM or permanently discontinue based on severity.
Hypersensitivity: IMDELLTRATM can cause severe hypersensitivity reactions. Clinical signs and symptoms of hypersensitivity may include, but are not limited to, rash and bronchospasm. Monitor patients for signs and symptoms of hypersensitivity during treatment with IMDELLTRATM and manage as clinically indicated. Withhold or consider permanent discontinuation of IMDELLTRATM based on severity.
Embryo-Fetal Toxicity: Based on its mechanism of action, IMDELLTRATM may cause fetal harm when administered to a pregnant woman. Advise patients of the potential risk to a fetus. Advise females of reproductive potential to use effective contraception during treatment with IMDELLTRATM and for 2 months after the last dose.
ADVERSE REACTIONS

The most common (> 20%) adverse reactions were CRS (55%), fatigue (51%), pyrexia (36%), dysgeusia (36%), decreased appetite (34%), musculoskeletal pain (30%), constipation (30%), anemia (27%) and nausea (22%). The most common (≥ 2%) Grade 3 or 4 laboratory abnormalities were decreased lymphocytes (57%), decreased sodium (16%), increased uric acid (10%), decreased total neutrophils (6%), decreased hemoglobin (5%), increased activated partial thromboplastin time (5%), decreased potassium (5%), increased aspartate aminotransferase (3.2%), decreased white blood cells (3.8%), decreased platelets (3.2%), and increased alanine aminotransferase (2.1%).
Serious adverse reactions occurred in 58% of patients. Serious adverse reactions in > 3% of patients included CRS (24%), pneumonia (6%), pyrexia (3.7%), and hyponatremia (3.6%). Fatal adverse reactions occurred in 2.7% of patients including pneumonia (0.5%), aspiration (0.5%), pulmonary embolism (0.5%), respiratory acidosis (0.5%), and respiratory failure (0.5%).
DOSAGE AND ADMINISTRATION: Important Dosing Information

Administer IMDELLTRATM as an intravenous infusion over one hour.

Administer IMDELLTRATM according to the step-up dosing schedule in the IMDELLTRATM PI (Table 1) to reduce the incidence and severity of CRS.
For Cycle 1, administer recommended concomitant medications before and after Cycle 1 IMDELLTRATM infusions to reduce the risk of CRS reactions as described in the PI (Table 3).
IMDELLTRATM should only be administered by a qualified healthcare professional with appropriate medical support to manage severe reactions such as CRS and neurologic toxicity including ICANS.
Due to the risk of CRS and neurologic toxicity, including ICANS, monitor patients from the start of the IMDELLTRATM infusion for 22 to 24 hours on Cycle 1 Day 1 and Cycle 1 Day 8 in an appropriate healthcare setting.
Recommend that patients remain within 1 hour of an appropriate healthcare setting for a total of 48 hours from start of the infusion with IMDELLTRATM following Cycle 1 Day 1 and Cycle 1 Day 8 doses, accompanied by a caregiver.
Prior to administration of IMDELLTRATM evaluate complete blood count, liver enzymes, and bilirubin before each dose, and as clinically indicated.
Ensure patients are well hydrated prior to administration of IMDELLTRATM.
Please see IMDELLTRA full Prescribing Information, including BOXED WARNINGS