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CHMP issues positive opinion on Type II variation to extend the therapeutic indication of Pepaxti based on OCEAN trial results

On September 14, 2023 Oncopeptides, a biotech company focused on difficult-to-treat cancers, reported that the Committee for Medicinal Products for Human Use (CHMP), part of the European Medicines Agency (EMA), has, following their scientific assessment, adopted a positive opinion on Oncopeptides’ application for earlier lines of treatment for patients with relapsed, refractory multiple myeloma (RRMM) (Press release, Oncopeptides, SEP 14, 2023, View Source [SID1234646783]). The opinion from the CHMP will now be sent to the European Commission for a final decision.

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Based on findings from its OCEAN trial, Oncopeptides submitted, on 28 Nov 2022, an application to extend the therapeutic indication of Pepaxti to the treatment of adult patients with multiple myeloma who have received at least two prior lines of therapies, whose disease is refractory to lenalidomide and the last line of therapy.

With their opinion, CHMP recommends that the use of Pepaxti could be expanded to earlier lines of treatment and also peripheral administration, meaning delivery of treatment through peripheral rather than central veins, a less invasive way of administration.

Oncopeptides will as a next step assess the current market dynamics of the rapidly evolving multiple myeloma landscape, including the competition in different lines of treatment and the effect that extending Pepaxti into earlier lines of treatment would have on Oncopeptides´ ability to receive a reimbursed price that reflects its innovation.

"The positive opinion from the CHMP further validates the scientific data on the efficacy, safety and increased quality of life that Pepaxti is able to bring to patients," says Sofia Heigis, CEO of Oncopeptides. "While we are convinced that our drug best serves patients in later lines of treatment where the unmet need and our chances to receive a price that reflects our innovation are high, we will closely evaluate the CHMP opinion and our potential next steps, always keeping value for patients and our shareholders as paramount priorities."

For more information, including a Q&A for investors, please visit Oncopeptides’ web site.

AI-Biopharma Auransa Announces Successful First Patient Dosed in Phase 1 Clinical Trial for Liver Cancer

On September 14, 2023 Auransa, a clinical-stage, AI-native biopharma that mines massive data for therapeutic gold, reported dosing of the first patient in its Phase 1 clinical trial to evaluate AU-409 as a treatment for patients with advanced liver cancers or solid tumors affecting the liver (Press release, Auransa, SEP 14, 2023, View Source [SID1234635626]). The patient was treated at the USC Norris Comprehensive Cancer Center where Anthony El-Khoueiry, MD is the principal investigator on the trial.

Specifically, the Phase 1 trial will evaluate the safety and pharmacokinetics of single agent AU-409 in cancer patients and help define potential doses for follow-on Phase 2 studies. For more information you can view the trial on ClinicalTrials.gov here.

"Our focus is on advancing novel therapies that raise the standard of care for cancer patients, leveraging AI and massive, available data," said Pek Lum, PhD, Founder-CEO, Auransa. "Too many patients are still treated with drugs that were not designed for them, will not completely reverse their cancer, and can lead to dangerous side effects. With the first patient dosed in our Phase 1 clinical trial for liver cancer, we take one step closer to helping these patients and proving the AI platform that discovered AU-409 will generate more novel treatments for cancer and cancer-related diseases."

Patients with advanced liver cancer, such as hepatocellular carcinoma and biliary tract cancer, continue to have limited survival rates and despite advances in treatment. For hepatocellular carcinoma, immunotherapy combinations, such as atezolizumab and bevacizumab, have become standard, first-line treatment options. Historically tyrosine kinase inhibitors (TKIs) were the standard of treatment.

"Despite the higher response rates seen with immunotherapy combinations, when compared to TKIs, most patients will develop resistance to treatment," said Dr. El-Khoueiry. "In second line treatment and beyond, TKIs like sorafenib offer limited efficacy and the potential for significant toxicity. This leaves a substantial gap for new, improved therapies for liver cancer. We look forward to seeing how the AU-409 program progresses to address these unmet patient needs."

Beyond AU-409 for liver cancer, Auransa has a growing pipeline of therapies for cancer and cancer-related diseases, with the goal of advancing towards the investigational new drug application stage, a critical entry point for FDA trials. This includes programs for heart-safe chemotherapy, late-stage prostate cancer, triple negative breast cancer, and head and neck cancer.

To build its pipeline, Auransa takes a fundamentally different approach from other TechBio companies using AI to decode biology. Instead of generating its own data on which to train algorithms, Auransa harnesses publicly available human disease data, applies algorithms that separate "signal from noise" by looking for recurrent disease signatures, and discovers novel compounds, targets, and disease models for the patient populations that will respond best. The goal is smarter, more efficient drug discovery that maximizes the chances of success in clinical trials and generates precision medicines for patients in need of a better standard of care.

For more information on Auransa, its pipeline and partnerships, please visit www.auransa.com.

About AU-409 for liver cancer

AU-409 is a novel small molecule with oral activity in models of liver cancer. In preclinical studies, AU-409 has been shown to modulate transcription of certain genes thereby altering the gene expression profile of liver cancer cells. The mechanism of action of AU-409 is distinct from that of current drugs approved for liver cancer, including tyrosine kinase inhibitors (TKIs) such as sorafenib or regorafenib. Non-clinical safety, toxicology and genetic toxicology studies support the first in-human clinical studies of AU-409.

About the AU-409 clinical program

AU-409-Clinical-2022-01 is the first-in-human study titled: First in Human Dose Escalation Study of AU-409 in Patients with Advanced Primary Liver Cancers or Advanced Solid Tumor with Liver Predominant Metastatic Disease, now being conducted at the USC Norris Comprehensive Cancer Center with Principal Investigator Anthony El-Khoueiry, MD. Primary objectives of the study include: (1) determine the maximum tolerated dose of AU-409 and the recommended Phase 2 dose and (2) characterize the safety and tolerability of AU-409 by assessing toxicities. Secondary objectives include: (1) determine the pharmacokinetics of AU-409 in patients with advanced-stage solid tumors treated with AU-409 and (2) obtain a preliminary assessment of anti-tumor activity of AU-409. If the trial is successful, USC may receive royalty payments under the collaboration agreement with Auransa.

For more information you can view the Phase 1 trial on ClinicalTrials.Gov here.

Generate:Biomedicines Announces Close of $273M Series C Financing to Advance Its Generative AI Pipeline of Preclinical and Clinical Protein Therapeutics

On September 14, 2023 Generate:Biomedicines, a clinical-stage biotherapeutics company pioneering a machine-learning-powered generative biology platform, reported that it has raised $273 million in Series C financing (Press release, Generate Biomedicines, SEP 14, 2023, View Source [SID1234635256]). This financing round attracted many new investors including Amgen; NVentures, NVIDIA’s venture capital arm; MAPS Capital (Mirae Asset Group); and Pictet Alternative Advisors. Additionally, company founder, Flagship Pioneering, and every Series B investor, including a wholly-owned subsidiary of the Abu Dhabi Investment Authority (ADIA); Fidelity Management & Research Company; funds and accounts advised by T. Rowe Price Associates, Inc.; ARCH Venture Partners; and March Capital, participated in the round.

"Our Series C round further propels The Generate Platform to increase significantly the precision, speed, and probability of success of novel therapeutics – positioning us to tackle even more complex targets and unmet patient needs," said Mike Nally, Chief Executive Officer of Generate:Biomedicines. "With our first program now in the clinic and multiple programs expected to enter clinical studies over the next couple of years, we are beginning to realize the enormous potential of generative biology to save and improve the lives of patients in areas where the burden of disease is greatest."

"We are thrilled to have the support of so many investors who believe in our ability to pioneer a new era of programmable protein-based therapeutics using generative AI," said Jason Silvers, M.D., Chief Financial Officer of Generate:Biomedicines. "The closing of our Series C financing provides us with a multi-year cash runway and will further enhance our ability to invest aggressively in The Generate Platform, pioneer growth through new program starts, advance multiple programs through clinical trials, and opportunistically consider additional value-creating collaborations."

Since the company’s Series B financing round in 2021, Generate:Biomedicines has made notable progress as a leader in the field of generative biology. Specific achievements include:

Initiated its first-in-human trial for GB-0669, a monoclonal antibody targeting a highly conserved region of the spike protein, in SARS-CoV‑2. Insights gleaned from these efforts enable the company to respond to future pandemics and develop more effective treatments targeting COVID variants.
Positioned to file a Clinical Trial Application by early Q4 2023 for its anti-TSLP monoclonal antibody, in asthma, which is expected to enter clinical trials shortly thereafter.
Entered into collaboration agreements with Amgen and The University of Texas MD Anderson Cancer Center.
Expanded The Generate Platform into new modalities, including into bi-specifics, enzymes, T‑cell engagers, and cell therapy, as well as achieved the structural confirmation of its first de novo generated binders.
Built an exceptional and diverse team of more than 280 highly talented and motivated employees.
"Since its founding, Generate:Biomedicines has worked diligently to turn biology into a truly engineerable discipline, transforming therapeutic development from a probabilistic endeavor to a deterministic one," said Noubar Afeyan, Ph.D., Co-founder and Chairman of the Board for Generate:Biomedicines and Founder and Chief Executive Officer of Flagship Pioneering. "The company has made significant advancements over the last five years, and I am confident this funding will enable us to realize a future where drug development is no longer a game of chance."

Generate:Biomedicines has a robust pipeline of 17 programs in preclinical and clinical development across oncology, immunology, and infectious disease. The company will leverage Series C funding to further its pipeline, including filing multiple INDs in 2024 and initiating multiple clinical trials annually thereafter. Additionally, the company will fully validate the breadth of its platform capabilities and establish its leadership in de novo therapeutic development.

About Generative Biology

Generative biology represents a fundamental shift in therapeutic development driven by artificial intelligence (AI) and machine learning. This approach creates never-before-seen therapeutic molecules targeted to specific biological processes involved in disease that can be modulated with a wide range of protein modalities—from short peptides to complex antibodies, enzymes, and cytokines. But the promise of generative biology goes beyond existing proteins found in nature and can create novel proteins that are purpose-built to address an existing or emerging therapeutic need. As a result, generative biology promises to leave trial-and-error drug discovery methods behind to usher in a new era of programmable drug generation that’s faster, cheaper, and better tailored to specific conditions.

Transgene Announces Upcoming Investor Meetings

On September 14, 2023 TRANSGENE (Paris: TNG), a biotech company that designs and develops virus-based immunotherapies for the treatment of cancer, today announces that Management will participate in several investor events, as set out below.

Cantor Global Healthcare Conference, New York, USA: September 26, 2023
Investor Access Forum, Paris, France: October 9, 2023
HealthTech Innovation Days 2023, Paris, France: October 24 & 25, 2023

Natera Announces Expanded I-SPY 2 Collaboration to Evaluate Signatera™ for Breast Cancer Patients in the Neoadjuvant Setting

On September 14, 2023 Natera, Inc. (NASDAQ: NTRA), a global leader in cell-free DNA testing, reported a new study as part of the I-SPY 2 trial, sponsored and operated by Quantum Leap Healthcare Collaborative, that will use Signatera, Natera’s personalized and tumor-informed molecular residual disease (MRD) test, to monitor response to neoadjuvant therapy in breast cancer patients across all subtypes (Press release, Natera, SEP 14, 2023, View Source [SID1234635175]).

This prospective study, in which 600 patients will be monitored in real time, will support efforts to establish circulating tumor DNA (ctDNA) as a composite endpoint and generate additional data on post-surgical ctDNA status in patients treated with neoadjuvant therapy. It expands on the existing collaboration with the ISPY-2 consortium that has led to the publication of multiple studies validating the use of Signatera for neoadjuvant response monitoring and predicting recurrence risk, including studies published in Cancer Cell1 and Annals of Oncology.2

"We are excited to expand our collaboration and help generate a large, prospective dataset to support the incorporation of ctDNA testing into future interventional trials in neoadjuvant breast cancer treatment," said Laura Esserman, MD, MBA, and Laura van ‘t Veer, PhD, professors at the University of California, San Francisco, and principal investigators of the I-SPY study. "We believe this study, in combination with our prior work in I-SPY 2, will help us understand the role that ctDNA can play in decision making for predicting response and improving our ability to determine who needs less therapy and who needs more. This is of utmost importance to patients."

"There is a significant unmet need for more accurate tools to enable precision care for patients diagnosed with breast cancer," said Minetta Liu, MD, chief medical officer of oncology at Natera. "I-SPY 2 serves as an excellent platform to investigate Signatera’s utility in the neoadjuvant and adjuvant settings. This extended collaboration will enhance our understanding of Signatera’s ability to assess therapy response, predict clinical outcomes, and advance a more personalized treatment strategy for patients with breast cancer."

About Signatera

Signatera is a custom-built circulating tumor DNA (ctDNA) test for treatment monitoring and molecular residual disease (MRD) assessment in patients previously diagnosed with cancer. The test is available for both clinical and research use, and has been granted four Breakthrough Device Designations by the FDA for multiple cancer types and indications. The Signatera test is personalized and tumor-informed, providing each individual with a customized blood test tailored to fit the unique signature of clonal mutations found in that individual’s tumor. Signatera is intended to detect and quantify cancer left in the body, at levels down to a single tumor molecule in a tube of blood, to identify recurrence earlier and to help optimize treatment decisions. The test has not been cleared or approved by the US Food and Drug Administration (FDA).