On November 14, 2024 Merck (NYSE: MRK), known as MSD outside of the United States and Canada, reported that Joseph Romanelli, president, Human Health International, and Dr. Marjorie Green, senior vice president and head of oncology, global clinical development, Merck Research Laboratories, are scheduled to participate in a fireside chat at the Jefferies London Healthcare Conference on Thursday, Nov. 21, 2024, at 6:30 a.m. EST / 11:30 a.m. GMT (Press release, Merck & Co, NOV 14, 2024, View Source [SID1234648406]).

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Investors, analysts, members of the media and the general public are invited to listen to a live audio webcast of the presentation at this weblink.

On November 14, 2024 CureLab Oncology, a clinical-stage biotech company dedicated to pioneering innovative therapies, reported a patent application for an innovative chronic pain treatment centered on its p62/SQSTM1 DNA plasmid (Press release, CureLab Oncology, NOV 14, 2024, View Source [SID1234648423]). This development, which leverages CureLab’s chronic inflammation therapeutic, Elenagen, signifies a potential paradigm shift in addressing chronic pain, a pervasive health challenge that affects millions globally.

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With this patent, CureLab Oncology aims to address the limitations of existing chronic pain treatments, such as NSAIDs (nonsteroidal anti-inflammatory drugs), acetaminophen, COX-2 inhibitors, antidepressants, anti-seizure medications, and opioids. While these medications offer varying degrees of pain relief, they often come with a range of side effects, including gastrointestinal problems, kidney problems, stomach bleeding, ulcers, dizziness, drowsiness, nausea, and potential addiction. The patent emphasizes the urgent need for more effective chronic pain medications with fewer side effects.

The patent application encompasses the utilization of p62/SQSTM1 DNA to combat a wide spectrum of chronic pain conditions, including arthritis, back pain, headaches, cancer pain, neuropathic pain, and fibromyalgia. This therapeutic strategy operates by modulating the body’s inflammatory response, a critical factor in the development and persistence of chronic pain.

"Chronic pain often stems from persistent inflammation, a complex biological response that can disrupt the body’s natural healing processes," explained Dr. Alexander Shneider, CEO of CureLab Oncology. "Elenagen, our innovative p62-based therapy, targets this underlying inflammation, promoting resolution and potentially alleviating pain at its source."

This groundbreaking therapy holds immense promise for both humans and animals suffering from chronic pain. Preclinical investigations employing animal models have illuminated the efficacy of this therapy in mitigating pain severity and augmenting the quality of life. Notably, the therapy exhibits a favorable safety profile, with no substantial adverse effects detected in the studies.

The global chronic pain treatment market is expected to reach $131.4 billion by 2030, growing at a compound annual growth rate (CAGR) of 4.3% from 2023 to 2030. This growth is driven by the increasing prevalence of chronic pain, the aging population, and the rising demand for effective and non-addictive pain management options. With its potential for fewer side effects and broader applications compared to current treatments, CureLab’s p62-based therapy is positioned to meet the growing demand of the growing chronic pain market.

"Our patenting strategy for this chronic pain treatment aligns with our successful approach for other therapies," said Ilya Lapshin, general counsel for CureLab Oncology. "By initially filing a provisional patent application in the USA and subsequently seeking worldwide protection, we aim to secure comprehensive intellectual property rights for this groundbreaking innovation."

To see CureLab’s portfolio of patents, visit curelaboncology.com/ip and curelabveterinary.com/ip.

About Elenagen
CureLab’s lead investigational compound is code-named Elenagen, an experimental DNA therapy that consists of a circular piece of DNA called a plasmid that includes a gene for a human protein called p62/SQSTM1. In clinical studies conducted ex-US, Elenagen demonstrated desirable safety profile and statistically significant clinical benefit for cancer patients enhancing the anti-cancer effects of chemotherapy. Experimental results also demonstrate mitigation of chronic inflammation, anti-aging effects, and stimulation of an immune response to the tumor.

On November 14, 2024 Immutep Limited (ASX: IMM; NASDAQ: IMMP) ("Immutep" or "the Company"), a clinical-stage biotechnology company developing novel LAG-3 immunotherapiesfor cancer and autoimmune disease, reported the presentation of new data from EFTISARC-NEO, a Phase II investigator-initiated trial of eftilagimod alpha (efti) in combination with radiotherapy plus KEYTRUDA (pembrolizumab) for patients with soft tissue sarcoma (STS), at the Connective Tissue Oncology Society (CTOS) 2024 Annual Meeting (Press release, Immutep, NOV 14, 2024, View Source [SID1234648305]). Based on preliminary analysis among 21 patients available for primary endpoint assessment, the triple combination therapy demonstrates significant efficacy in the neoadjuvant setting for resectable STS.

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Katarzyna Kozak, M.D., Ph.D., and Paweł Sobczuk, M.D., Ph.D., medical oncologists at the Department of Soft Tissue/Bone Sarcoma and Melanoma at MSCNRIO (Warsaw) and the trial’s principal investigators, stated: "Our belief in efti’s unique mechanism of action to complement radiotherapy and pembrolizumab in order drive better outcomes for patients with this rare aggressive disease was the foundation of the EFTISARC-NEO trial. These very encouraging results we are presenting today build our confidence in the synergistic effects of this new therapeutic approach and its potential to treat these patients in dire need of more effective therapies. In particular, the high level of hyalinization/fibrosis achieved with this novel combination therapy, three-times above historical results from standard radiotherapy, demonstrates remarkable efficacy in patients with resectable soft tissue sarcomas."

In the neoadjuvant setting for patients with resectable STS, the combination achieved a greater than threefold increase in tumour hyalinization/fibrosis (median 50%) at the time of surgical resection as compared to a historical median 15% from standard radiotherapy alone. In addition to being the primary endpoint of the EFTISARC-NEO study, the tumour hyalinization/fibrosisrate has also been identified as an important predictor of overall survival for STS patients.

The EFTISARC-NEO trial, with a data cut-off of 20 October 2024, also showed 71.4% of patients achieved a pathologic response defined as ≥35% of hyalinization/fibrosis and 9.5% of patients achieved a complete pathologic response. The triple combination therapy is safe with no grade ≥3 toxicities related to efti and pembrolizumab.

Dr. Frédéric Triebel, CSO of Immutep, said: "We are pleased with the strength of these preliminary results in this difficult-to-treat cancer. To see 71.4% of soft tissue sarcoma patients achieving a pathologic response defined as ≥35% of hyalinization/fibrosis combined with low viable tumour cells at 8% is very promising, especially as strong efficacy has been observed in different STS subtypes. We look forward to further evaluation of efti’s potential as neoadjuvant immunotherapy to help drive improved clinical outcomes.

The ongoing open-label EFTISARC-NEO Phase II study, conducted by the Maria Skłodowska-Curie National Research Institute of Oncology (MSCNRIO) in Warsaw, is expected to reach the planned enrolment of 40 patients in Q1 CY2025. The trial is primarily funded with an approved grant from the Polish government awarded by the Polish Medical Research Agency program. For more information, visit clinicaltrials.gov (NCT06128863)

On November 14, 2024 bluebird bio, Inc. (NASDAQ: BLUE) ("bluebird bio" or the "Company") reported third quarter results and business highlights for the quarter ended September 30, 2024, including recent commercial and operational progress (Press release, bluebird bio, NOV 14, 2024, View Source [SID1234648391]).

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"Patient starts more than doubled from our second to third quarter update, providing clear evidence that our commercial launches continued to accelerate," said Andrew Obenshain, chief executive officer. "This momentum, coupled with steps we took in the third quarter to increase manufacturing capacity for ZYNTEGLO and optimize our cost structure, is propelling bluebird forward on our path to becoming a sustainable commercial gene therapy company. We remain focused on securing additional cash resources to extend our runway, which we believe would enable us to achieve this vision and reach cash flow break-even in the second half of 2025."

COMMERCIAL LAUNCH UPDATES

Continued commercial momentum across the portfolio

57 patient starts completed to date in 2024 (35 ZYNTEGLO, 17 LYFGENIA, 5 SKYSONA).
17 additional starts scheduled through the remainder of 2024.
Evidence of strong commercial demand, with 30 patient starts already scheduled in 2025, supporting the potential for cash flow breakeven in the second half of 2025.
More than 70 activated QTCs, with 40% having initiated or completed treatment for at least one patient.
Validated access and reimbursement strategy is driving favorable coverage landscape

To date, more than half of all states have affirmed coverage for LYFGENIA through a preferred drug list or published coverage criteria.
Nearly 50% of Medicaid-insured individuals with sickle cell disease in the U.S. live in a state that has already completed prior authorization approval for the use of LYFGENIA for at least one patient.
Multiple outcomes-based agreements are published and in place for LYFGENIA with national commercial payer organizations, representing more than 200 million U.S. lives.
DATA PRESENTATIONS AT ASH (Free ASH Whitepaper) 2024

Updated data from the Company’s lentiviral vector (LVV) gene addition programs in patients with sickle cell disease who have a history of vaso-occlusive events and patients with beta-thalassemia who require regular blood transfusions will be presented at the 66th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition. The meeting will take place December 7-10, 2024 at the San Diego Convention Center and online.

SICKLE CELL DISEASE DATA

Oral Presentation [#511]: An Update on Lovotibeglogene Autotemcel (lovo-cel) Clinical Trials for Sickle Cell Disease (SCD) and Analysis of Early Predictors of Response to Lovo-cel
Presenting Author: Dr. Stacey Rifkin-Zenenberg (Hackensack)
Date/Time: Sunday, December 8, 2024, 9:30 a.m. – 11:00 a.m. PT
Poster Presentation [#3576]: Participants with a History of Stroke in Lovotibeglogene Autotemcel (lovo-cel) Clinical Trials
Presenting Author: Dr. Jen Jaroscak (The Medical University of South Carolina)
Date/Time: Sunday, December 8, 2024, 6:00 p.m. – 8:00 p.m. PT
BETA-THALASSEMIA DATA

Poster Presentation [#2194]: Betibeglogene Autotemcel (beti-cel) Gene Addition Therapy results in durable Hemoglobin A (HbA) Production with up to 10 Years of Follow-Up in Participants with Transfusion-Dependent β-Thalassemia
Presenting Author: Dr. Alexis A Thompson (Children’s Hospital of Philadelphia)
Date/Time: Saturday, December 7, 2024, 5:30 p.m. – 7:30 p.m. PT
Abstracts outlining bluebird bio’s accepted data at ASH (Free ASH Whitepaper) 2024 are available on the ASH (Free ASH Whitepaper) conference website.

THIRD QUARTER FINANCIAL HIGHLIGHTS

Cash Position: The Company’s cash, cash equivalents and restricted cash balance was approximately $118.7 million, including restricted cash of approximately $48.0 million, as of September 30, 2024.

bluebird and Hercules are engaging collaboratively as bluebird works to secure adequate cash runway to obtain additional financing and reach cash flow break-even. Based on current forecasts, which assume continued cost-saving initiatives, successfully renegotiating key contracts, and continued collaborative engagement from Hercules, we expect our existing cash and cash equivalents will enable us to fund our operations into the first quarter of 2025.

The Company anticipates quarterly cash flow break-even in the second half of 2025, assuming it scales to approximately 40 drug product deliveries per quarter and obtains additional cash resources to extend its runway.
Revenue, net: Total revenue, net was $10.6 million for the three months ended September 30, 2024, compared to $12.3 million for the three months ended September 30, 2023, driven by quarter-to-quarter variability in drug product infusions.

Revenue for the third quarter includes revenue from LYFGENIA, following the completion of the first infusion for sickle cell disease.

bluebird previously guided to an anticipated reduction of net revenue in the third quarter; the Company now anticipates net revenue of at least $25 million in the fourth quarter 2024, as previously reported patient starts are infused.
Cost of Product Revenue: Cost of product revenue was $11.8 million for the three months ended September 30, 2024, compared to $9.1 million for the three months ended September 30, 2023.
SG&A Expenses: Selling, general and administrative expenses were $39.8 million for the three months ended September 30, 2024, compared to $40.8 million for the three months ended September 30, 2023. The decrease of $1.0 million was primarily driven by decrease in employee compensation, benefit, and other headcount related expenses, commercial expenses, and facility fees, partially offset by increased professional services fees.
R&D Expenses: Research and development expenses were $23.2 million for the three months ended September 30, 2024, compared to $58.5 million for the three months ended September 30, 2023. The decrease of $35.3 million was primarily driven by material production shift to inventory and cost of product revenue as well as decreased employee compensation, benefit, and other headcount related expenses, consulting fees, and facility and information technology fees.
Net income (loss): Net loss was $60.8 million for the three months ended September 30, 2024, compared to a net loss of $87.2 million for the three months ended September 30, 2023.
CONFERENCE CALL DETAILS

bluebird will hold a conference call to discuss its third quarter 2024 results and business updates today, Wednesday, November 14, 2024, at 8:00 am ET.

To access the live conference call via telephone, please register at this link to receive a dial in number and unique PIN.

To access the live webcast, please visit the "Events & Presentations" page within the Investors & Media section of the bluebird bio website at View Source A replay of the webcast will be available on the bluebird bio website for 90 days following the event.

On November 14, 2024 Moleculin Biotech, Inc., (Nasdaq: MBRX) ("Moleculin" or the "Company"), a late-stage pharmaceutical company with a broad portfolio of drug candidates targeting hard-to-treat tumors and viruses, reported it has amended the clinical trial protocol with the U.S. Food and Drug Administration ("FDA") for its Phase 3 pivotal trial protocol evaluating Annamycin in combination with Cytarabine (also known as "Ara-C" and for which the combination of Annamycin and Ara-C is referred to as "AnnAraC") for the treatment of AML patients who are refractory to or relapsed after induction therapy (R/R AML) (MB-108) (Press release, Moleculin, NOV 14, 2024, View Source [SID1234648407]). This Phase 3 "MIRACLE" trial (derived from Moleculin R/R AML AnnAraC Clinical Evaluation) will be a global trial, including sites in the US. Additionally, the Company released a Virtual Investor "What This Means" segment to discuss the amended protocol. Access the segment here.

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"Our team has been thoughtful and strategic with the design of the MIRACLE trial, which may allow for possible accelerated approval of Annamycin in combination with cytarabine for the treatment of relapsed or refractory AML. This amended protocol enables us to share definitive data earlier, which helps to partially de-risk financing the trial and potentially accelerates the timeline for strategic partnering. We believe that the unblinding of data at 45 subjects will enable us to begin assessing all three arms of the study and provide us with a clear path forward in understanding the potential of Annamycin for AML patients. This change now puts us potentially less than 12 months away from definitive unblinded data that could be a strong indicator of our likelihood of approval, and the kind of data that is likely to drive advanced partnering discussions," commented Walter Klemp, Chairman and Chief Executive Officer of Moleculin.

The MIRACLE study, subject to appropriate future filings with and potential additional feedback from the FDA and their foreign equivalents, is expected to initially utilize an adaptive design whereby the first 75 to 90 subjects will be randomized in Part A of the trial to receive high dose cytarabine (HiDAC) combined with either placebo, 190 mg/m2 of Annamycin, or 230 mg/m2 of Annamycin, such doses were specifically recommended by the FDA in the Company’s end of Phase 1B/2 meeting. The amended protocol will allow for the unblinding of preliminary primary efficacy data (CR) and safety/tolerability of the three arms at 45 subjects. This early unblinding will yield 30 subjects with Annamycin (190mg/m2 and 230/m2) and HiDAC and 15 subjects with just HiDAC. The Company expects to reach 45 subjects in the second half of 2025, in addition to the planned unblinding expected in 2026 of the next 30-45 subjects.

For Part B of the trial, approximately 244 additional subjects will be randomized to receive either HiDAC plus placebo or HiDAC plus the optimum dose of Annamycin. The selection of the optimum dose will be based on the overall balance of safety, pharmacokinetics and efficacy, consistent with the FDA’s new Project Optimus initiative. This increase from 240 to 244 subjects represents the statistical "cost" of the additional unblinding.

The amended protocol is currently being reviewed by the Institutional Review Board (IRB). Once approved, the amended protocol will be filed with the amendment for the Company’s Initial New Drug (IND) application in the US with the FDA.

Annamycin currently has Fast Track Status and Orphan Drug Designation from the FDA for the treatment of relapsed or refractory acute myeloid leukemia, in addition to Orphan Drug Designation for the treatment of soft tissue sarcoma. Furthermore, Annamycin has Orphan Drug Designation for the treatment of relapsed or refractory acute myeloid leukemia from the European Medicines Agency (EMA).