On December 8, 2022 Autolus Therapeutics plc (Nasdaq: AUTL), a clinical-stage biopharmaceutical company developing next-generation programmed T cell therapies, reported that Blackstone Life Sciences (‘Blackstone’) has committed to make two pre-agreed milestone payments of $35m each to Autolus, totaling $70m (Press release, Autolus, DEC 8, 2022, View Source [SID1234625453]). The milestones are expected to be recognized in Autolus’ Q4 2022 cash balance.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The first Blackstone milestone of $35m is being paid earlier than anticipated as a result of the joint steering committee’s review of Autolus’ interim analysis of pivotal FELIX Phase 2 clinical trial of obecabtagene autoleucel (obe-cel) in relapsed/refractory (r/r) adult Acute Lymphoblastic Leukemia (ALL). The study has met its primary endpoint, the details of which have been announced in a separate press release today.

The second Blackstone milestone of $35m is a pre-agreed manufacturing milestone as a result of completion of planned activities demonstrating the performance and qualification of Autolus’ obe-cel’s manufacturing process.

"Receiving these two $35m milestone payments highlights the continuing strength and collaborative nature of our partnership with Blackstone," said Dr. Christian Itin, CEO of Autolus. "We are delighted to have demonstrated the potential merits of obe-cel’s clinical profile and our evolving robust manufacturing process to the Blackstone team, and look forward to continued progress together as we focus on our goal of submitting a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) by the end of 2023."

"We are pleased with Autolus’ progress —we continue to believe obe-cel has the potential to become a transformative therapy for relapsed/refractory adult ALL patients," said Nicholas Simon, Senior Managing Director with Blackstone Life Sciences. "Our investments in these next generation cell therapies with Autolus exemplifies our conviction in the quality and promise of the life sciences sector in the UK."

As previously announced, Autolus and Blackstone entered into a strategic collaboration and financing agreement in November 2021, whereby funds managed by Blackstone agreed to provide up to $250 million in equity and product financing to support Autolus’ advancement of obe-cel, its CD19 CAR T cell investigational therapy product candidate, as well as next generation product candidates of obe-cel in B-cell malignancies.

On December 8, 2022 Eigen Therapeutics, the biotech startup on a mission to make cancer easier to find and eliminate, reported their launch today (Press release, Eigen Therapeutics, DEC 8, 2022, View Source [SID1234624978]). They also announced a $7 million seed round led by Josh Kopelman at First Round Capital, with participation from Builders VC, Kevin Mahaffey, Hawktail, Matthew De Silva (founder at Notable Labs), Varsha Rao, Bioverge, Alumni Ventures, Mount Pleasant Ventures, and others.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Eigen was founded by an engineer and a scientist, Transon Nguyen and Kamran Ali, who have assembled a diverse team of experts in technology and biological sciences. Both founders are veterans of Notable Labs, where they gained deep experience in the cancer drug discovery industry, and where they began to grapple with the gaps in targeted therapy success rates.

"Targeted therapies, including antibody-drug conjugates and CAR T-cell therapies, are incredibly effective in some instances and have significantly moved cancer treatment forward," explains Eigen CEO Transon Nguyen. "But they could be far more effective than they are today. There’s a lot of focus on developing better, smarter targeted therapies, but we noticed there hasn’t been much attention on the other side of the equation. So we asked: What if we also made cancer cells easier for targeted therapies to eliminate?"

Eigen focuses on priming therapies that make cancer cells more visible—so they can be identified faster. The company takes a two-step approach to treating cancer. The first step is a priming therapy, which essentially shines a spotlight on the cancer cells to make them more recognizable. They do this by increasing target expression across cancer cells.

"Once the cancer has been primed and highlighted, the targeted therapeutic can more easily find and attack the tumor cells and eliminate the cancer," says Eigen CTO Kamran Ali. "When priming therapies are paired with targeted therapies, cancer treatment can become far more effective."

Eigen will use the capital from their seed round to apply their novel high-throughput platform toward the development of priming therapies. Their initial programs are focused on hematological malignancies; in the future, their platform will allow them to quickly spin up programs for other cancer indications, such as solid tumors.

For Nguyen and Ali, the platform-based approach is a logical choice, as Notable was one of the first companies to prove out the thesis that a full-stack approach integrating robotics, software, and biology is a viable strategy for scaling up cancer drug discovery. Transon was Notable’s founding engineer, Head of Laboratory Automation, and VP of Engineering; Kamran was Notable’s Head of Platform and Discovery.

"Priming therapies have the potential to improve targeted therapies, and ultimately, the lives of cancer patients," says Josh Kopelman, founding partner at First Round Capital. "However, it’s a widely unsearched field. Eigen’s team and the platform they’ve built are the perfect match to systematically search this field and develop more priming therapies."

Eigen’s high-throughput platform takes a combinatorial functional screening approach to exploring the space of priming therapies. It uses proprietary machine learning algorithms to discover novel associations, new classes of drugs, and new potential therapies that change a cell’s state to make it more vulnerable to a targeted therapy. The Eigen platform represents the intersection of biology and engineering, and includes robotic systems, laboratory operations software, and machine learning analysis pipelines—all developed in-house—with the ultimate goal of saving more lives.

On December 8, 2022 Scorpion Therapeutics, Inc. ("Scorpion"), a pioneering oncology company redefining the frontier of precision medicine through its Precision Oncology 2.0 strategy, reported preclinical data demonstrating that STX-478, the Company’s highly differentiated, allosteric and central nervous-system ("CNS") penetrant inhibitor of mutant phosphoinositide-3-kinase alpha ("PI3Kα"), has a potentially best-in-class product profile, with the ability to improve outcomes in patients harboring tumors with prevalent PI3Kα kinase or helical domain mutations (Press release, Scorpion Therapeutics, DEC 8, 2022, View Source [SID1234624977]). The data is being presented for the first time today in a poster session at the San Antonio Breast Cancer Symposium ("SABCS") in San Antonio, Texas.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"We are delighted to share new preclinical data on STX-478 that further demonstrates its potential best-in-class product profile and supports its advancement into first-in-human clinical trials," said Axel Hoos, M.D., Ph.D., Chief Executive Officer of Scorpion. "In these studies, STX-478 showed robust anti-tumor activity as both a monotherapy and in combination with standard of care agents in models with kinase or helical domain mutations. Importantly, this activity was coupled with key features that differentiate STX-478 from other PI3Kα inhibitors. STX-478 exhibited high mutant selectivity, without the dose-limiting metabolic dysfunction associated with currently available treatments. It also displayed an ability to reach the CNS, which could address the brain metastases that often occur in patients with solid tumors, but are poorly treated by existing options. We look forward to filing our IND application in the first quarter of 2023 and initiating our Phase 1 study in patients soon thereafter."

PI3Kα is one of the most highly mutated targets in all of cancer. In the United States, over 166,000 patients per year present with cancers, including breast, gynecological and head and neck, which harbor PI3Kα mutations.1 Scorpion specifically designed STX-478 to deliver superior safety, tolerability and efficacy compared to currently marketed PI3Kα therapies and certain drug candidates known to be in development, while exhibiting excellent drug-life properties and a favorable therapeutic index.

In the preclinical data presented at SABCS, STX-478 demonstrated exceptional selectivity for PI3Kα, inhibiting the viability of both kinase and helical domain mutated cell lines in vitro. In addition, STX-478 demonstrated robust monotherapy activity in vivo, as well as combination activity with relevant co-treatments, across a variety of cancer types in xenograft studies. This includes a T47D ER+, HER2- breast cancer model, in which STX-478 treatment caused deep tumor regressions alone and in combination with fulvestrant; a panel of ER+ BrCa patient derived xenograft ("PDX") models, in which STX-478 was highly efficacious alone and in combination with fulvestrant or palbociclib; and an ER+ PDX model carrying a helical domain mutation, in which STX-478 inhibited tumor cell growth.

Importantly, STX-478 also demonstrated promising tolerability. Chronic dosing of STX-478 did not cause metabolic dysfunction in murine models at efficacious doses, nor did it affect blood glucose levels in higher species, even at doses that provide exposure well-above the efficacious exposure in murine models.

"We are particularly encouraged to present data for the first time showcasing STX-478’s activity against PI3Kα helical domain mutations, which could enable our investigational therapy to treat a large number of patients," said Darrin Stuart, Ph.D., Chief Scientific Officer of Scorpion. "This further showcases the demonstrated ability of our drug development platform to bring together scientific breakthroughs in biology, chemistry and data sciences to create superior product candidates that can extend the reach of precision medicine to many more people battling cancer. We look forward to initiating our first Phase 1 study of STX-478 next year."

Scorpion has completed IND-enabling studies for STX-478 and expects to file an IND application with the U.S. Food and Drug Administration in the first quarter of 2023. Scorpion anticipates evaluating STX-478 as a monotherapy and in relevant combinations with approved standard-of-care agents in patients harboring PI3Kα mutations in either the kinase or helical domain.

The poster presentation is now available here and under "Media" in the News section of Scorpion’s website: View Source Scorpion previously presented preclinical proof-of-concept data showcasing the exceptional selectivity of STX-478 for PI3Kα against the kinome, as well as pharmacokinetic analyses suggesting that STX-478 demonstrates from outstanding drug-like properties with adequate CNS exposure to treat brain tumors or metastases, at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting in April 2022.

On December 8, 2022 Agendia, Inc., a leader in gene expression profiling for early-stage breast cancer, reported that it will present late-breaking research at the 2022 San Antonio Breast Cancer Symposium (SABCS) that proves MammaPrint is the first FDA-cleared gene expression profiling test to predict an early-stage breast cancer patient’s benefit from extended endocrine therapy (EET) (Press release, Agendia, DEC 8, 2022, View Source [SID1234624976]). The study, which analyzed MammaPrint results of over 500 patients enrolled in the IDEAL trial, proved MammaPrint test results can effectively identify which post-menopausal, hormone receptor positive (HR+), human epidermal growth factor receptor 2 negative (HER2-) breast cancer patients will benefit from five years of EET. This research highlights the value of advanced gene expression profiling tests to gather personalized insights that inform a patient’s treatment plan beyond solely predicting their response to chemotherapy.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Prescribing EET with more precision can not only help avoid unnecessary patient suffering, but also give those who are most likely to benefit from EET the encouragement to remain adherent. Agendia’s research found MammaPrint can identify which HR+ post-menopausal patients are at risk of late distant metastasis and would benefit most from EET, and for whom treatment compliance is important. Additionally, MammaPrint helps identify those who will realize minimal to no benefit, offering patients the power of choice when discussing their treatment plan with their provider.

Laura van ‘t Veer, PhD, Co-Inventor of MammaPrint and a Professor of Laboratory Medicine and Director of Applied Genomics at the University of California San Francisco’s Helen Diller Family Comprehensive Cancer Center, will present during SABCS on December 9th at 12pm CT. She will highlight the following findings among HR+ post-menopausal women with early-stage breast cancer:

Tumors classified with a MammaPrint Low Risk result exhibited the largest benefit of five additional years of EET compared to 2.5 years, with 9.8% improvement for distant metastasis, 9.8% improvement for recurrence free interval, and 8.8% improvement for breast cancer free interval.
In contrast, tumors with a MammaPrint High Risk result do not benefit from five additional years of EET because they exhibit an early recurrence risk profile, and therefore have reduced sensitivity to the therapy after the initial 5 years of treatment.
Tumors with a MammaPrint UltraLow Risk result also do not benefit from EET due to their excellent prognosis and low risk of late recurrence.
MammaPrint test results have now been used in level 1B evidence for extended endocrine treatment decisions, providing high confidence of its net benefit that providers and patients can rely on when making EET treatment decisions.
"The results of this study reaffirm the critical role MammaPrint plays in guiding treatment decisions with the unprecedented precision that patients deserve. Physicians and patients need to be equipped with gene expression profiling solutions that can offer a comprehensive view of their tumor’s predicted response to various therapies, not just chemotherapy," said Dr. Laura van ‘t Veer. "No patient should have to be on a difficult therapy longer than necessary, and those who will benefit from a longer duration need encouragement to stay on track. Agendia is empowering women with need-to-know answers and physicians with tools to guide shared decision-making."

"Genomic information continues to transform the way we deliver cancer care, and this latest research is no exception. Being able to decipher which patients see a significant benefit and which can forgo EET is a monumental accomplishment in our aim toward precision medicine," said Dr. Gerrit-Jan Liefers, surgeon and head of the Geriatric Oncology Group at Leiden University Medical Center, and lead author of the study.

To learn more about Agendia’s solutions being showcased at booth #315 at SABCS, visit View Source Follow Agendia on Twitter, Facebook and LinkedIn for updates throughout the conference.

On December 8, 2022 Dana-Farber Cancer Institute reported that Multiple myeloma is a cancer of the plasma cells in bone marrow that affects more than 30,000 new patients in the United States each year (Press release, Dana-Farber Cancer Institute, DEC 8, 2022, View Source [SID1234624975]). Today, multiple myeloma is still considered incurable.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Nearly all people with multiple myeloma have early warning signs called "precursor conditions." Health leaders like the Dana-Farber Cancer Institute, one of the world’s leading centers of cancer research and treatment, can better treat this condition if it is detected early, and therefore reduce mortality. Now with their recent announcement about the ambitious PROMISE study, Dana-Farber aims to make multiple myeloma a cancer that is preventable.

By collecting and analyzing data from people who test positive for the early precursor conditions, Dana-Farber will be able to study people before they develop the condition, in order to discover how to better treat – and ultimately prevent – this deadly cancer.

A study of this magnitude requires extensive data collection, as well as an approach to enroll and engage participants from communities traditionally under-represented in biomedical and clinical research. To achieve these aims, Dana-Farber chose to collaborate with Vibrent Health, a digital technology company known for its innovation in precision medicine research solutions and providing researchers a better way to provide eConsent and collect data across electronic medical records, genomics, wearables, surveys, and more.

Dana-Farber will use Vibrent’s platform for informed eConsent to enroll a highly diverse pool of research participants nationwide. Dana-Farber study staff will also use Vibrent’s platform to perform novel data collection through virtual methods and computer-assisted telephone interviewing (CATI).

The Research Institute will implement patient engagement through Vibrent’s robust privacy-protecting participant portal and comprehensive researcher tools, as well as provide educational content that can be personalized to the patient’s specific and unique needs.

"Using this technology to interact directly with our study participants is so powerful," said the study’s principal investigator Irene Ghobrial, MD, of Dana-Farber. "Not only will this improve the quality of the research we are able to conduct, but it puts the tools in our participants hands to help us make multiple myeloma a preventable disease."

For Vibrent Health, this partnership builds on the company’s commitment to improving research data and health outcomes for traditionally underrepresented populations in clinical research.

"Clinical research has long faced limitations because of the available participants," said Vibrent Health CEO Praduman "PJ" Jain. "Our solutions remove the barriers – whether they are a person’s location, internet connectivity, or physical limitations – so they can contribute to the important work of researchers. By including their data, researchers can create health outcomes that better help these populations."

The PROMISE Study is currently enrolling participants with a target goal of 30,000 individual participants nationwide, in order to identify 3,000 with the precursor conditions. The resulting dataset will help Dana-Farber to identify the factors that are associated with disease progression and develop potential methods for prevention.

"Five years ago, we told people with precursor conditions of the disease to watch and wait until they experienced symptoms," said Ghobrial. "By empowering our community, we are understanding who is most likely to develop multiple myeloma in their lifetime and where early interception can be most effective".