On April 21, 2021 Sermonix Pharmaceuticals Inc., a privately held biopharmaceutical company developing innovative therapeutics to treat ESR1-mutated metastatic breast and gynecological cancers, reported a preclinical collaboration with researcher Jay Gertz, Ph.D., to examine the potential effects of lasofoxifene, Sermonix’s lead investigational drug, on endometrial cancer (Press release, Sermonix Pharmaceuticals, APR 21, 2021, View Source [SID1234578341]).

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The project will investigate the effects of lasofoxifene on unique models of endometrial cancer that carry ESR1 mutations. Lasofoxifene has shown novel activity in ESR1 mutations, and Sermonix is currently enrolling patients in two Phase 2 Evaluation of Lasofoxifene in ESR1 Mutations (ELAINE) studies within the breast cancer arena.

"Sermonix is committed to exploring ESR1 mutations across gynecological tumor types and examining lasofoxifene’s potential to serve the unmet medical needs of more women fighting cancer," said Barry Komm, Ph.D., chief scientific officer of Sermonix. "The unique ESR1 model system is state-of-the-art for this exploration and Dr. Gertz is an esteemed partner with whom it is an honor for Sermonix to collaborate."

Dr. Gertz is a cancer researcher at Huntsman Cancer Institute and associate professor of oncological sciences at the University of Utah. His lab studies how transcription regulation is altered in cancer with a particular focus on the roles of steroid hormone signaling in endometrial and breast cancers.

"The Gertz Lab has a major emphasis in advancing the study of new drugs for endometrial cancer, an aggressive disease with limited treatment options," said Dr. Gertz. "We look forward to learning more about the efficacy of lasofoxifene in the context of ESR1-mutated endometrial cancer."

About Lasofoxifene
Lasofoxifene is an investigational, nonsteroidal selective estrogen receptor modulator (SERM), which Sermonix licensed globally from Ligand Pharmaceuticals Inc. (NASDAQ: LGND) and has been studied in previous comprehensive Phase 1-3 non-oncology clinical trials in more than 15,000 postmenopausal women worldwide. Lasofoxifene’s bioavailability and activity in mutations of the estrogen receptor could potentially hold promise for patients who have acquired endocrine resistance due to ESR1 mutations, a common finding in the metastatic setting and an area of high unmet medical need. Lasofoxifene’s novel activity in ESR1 mutations was discovered at Duke University and Sermonix has exclusive rights to develop and commercialize the product in this area. Lasofoxifene, a potent, oral SERM could, if approved, play a critical role in the targeted precision medicine treatment of advanced ER+ breast cancer.

On April 20, 2021 Kaleido Biosciences, Inc. (Nasdaq: KLDO), a clinical-stage healthcare company with a small molecule driven approach to targeting the microbiome to treat disease and improve human health, reported a research collaboration with researcher Robert Jenq, M.D. Professor of Genomic Medicine, at The University of Texas MD Anderson Cancer Center, to explore the potential of Kaleido’s novel Microbiome Metabolic Therapies (MMT) in preventing febrile neutropenia—a serious complication associated with hematopoietic stem cell transplantations (HSCT) (Press release, Kaleido Biosciences, APR 20, 2021, View Source [SID1234578253]).

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Approximately 60,000 patients undergo HSCT annually in the U.S. and Europe, which includes patients with myeloid disorders, lymphoid disorders and plasma cell disorders. Recent scientific findings have demonstrated that treatment regimens given to HSCT patients have a profound detrimental effect on their microbiome, putting them at increased risk of bacterial infections, febrile neutropenia and ultimately, graft-versus-host disease (GVHD).

"Today, standard-of-care for patients with hematological cancers includes radiation and chemotherapy to eliminate malignant cells. This may lead to significant side effects including severe damage to the gut epithelium, and major disruption to a patient’s microbiome," said Johan van Hylckama Vlieg, Chief Scientific Officer at Kaleido Biosciences. "Additionally, treatment combinations with broad-spectrum antibiotics used in the conditioning process, leave patients increasingly more susceptible to neutropenic fever. We are eager to work with Dr. Jenq to explore the potential of Kaleido’s MMTs to selectively support the restoration of the microbiome and gut barrier integrity, with the aim of ultimately improving treatment outcomes for patients undergoing HSCT."

Within the collaboration, selected MMTs will be evaluated in disease animal models developed by Dr. Jenq, to further explore the molecular mechanisms by which MMTs act on the microbiome and gut barrier function and support the identification of a lead compound from Kaleido’s compound library.

About Microbiome Metabolic Therapies (MMT)
Kaleido’s Microbiome Metabolic Therapies, or MMTs, are designed to drive the function and distribution of the microbiome organ’s existing microbes in order to decrease or increase the production of metabolites, or to advantage or disadvantage certain bacteria in the microbiome community. The Company’s initial MMT candidates are targeted glycans that are orally administered, have limited systemic exposure, and are selectively metabolized by enzymes in the microbiome. Kaleido utilizes its human-centric discovery and development platform to study MMTs in microbiome samples in an ex vivo setting, followed by advancing MMT candidates rapidly into clinical studies in healthy subjects and patients. These human clinical studies are conducted under regulations supporting research with food, evaluating safety, tolerability and potential markers of effect. For MMT candidates that are further developed as therapeutics, the Company conducts clinical trials under an Investigational New Drug (IND) or regulatory equivalent outside the U.S., and in Phase 2 or later development.

On April 20, 2021 Vanda Pharmaceuticals Inc. (Vanda) (Nasdaq: VNDA) reported it will release results for the first quarter 2021 on Wednesday, May 5, 2021, after the market closes (Press release, Vanda Pharmaceuticals, APR 20, 2021, View Source [SID1234578269]).

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Vanda will host a conference call at 4:30 PM ET on Wednesday, May 5, 2021, during which management will discuss the first quarter 2021 financial results and other corporate activities. To participate in the conference call, please dial 1-866-688-9426 (domestic) or 1-409-216-0816 (international) and use passcode 5709209.

The conference call will be broadcast simultaneously and archived on Vanda’s website, www.vandapharma.com. Investors should go to the website at least 15 minutes early to register, download, and install any necessary audio software.

A replay of the call will be available on Wednesday, May 5, 2021, beginning at 7:30 PM ET and will be accessible until Wednesday, May 12, 2021, at 7:30 PM ET. The replay call-in number is 1-855-859-2056 for domestic callers and 1-404-537-3406 for international callers. The passcode number is 5709209.

On April 20, 2021 Lineage Cell Therapeutics, Inc. (NYSE American and TASE: LCTX), a clinical-stage biotechnology company developing novel cell transplants for serious medical conditions, reported a worldwide license and development collaboration agreement with Immunomic Therapeutics, Inc., ("ITI"), a privately-held clinical-stage biotechnology company pioneering the study of nucleic acid immunotherapy platforms (Press release, Lineage Cell Therapeutics, APR 20, 2021, View Source [SID1234578238]). The collaboration will generate a novel product candidate derived from Lineage’s VAC allogeneic cancer immunotherapy platform and targeting a proprietary Tumor Associated Antigen (TAA) construct provided by ITI, for the treatment of glioblastoma multiforme (GBM). Lineage and ITI will collaborate in the manufacturing and clinical development of a novel VAC product candidate. Following the full development and delivery of Current Good Manufacturing Practice (cGMP) VAC product material, ITI will assume full and independent clinical and commercial responsibility and further advancement of the program. Under the terms of the agreement, Lineage will be entitled to upfront payments totaling $2 million anticipated in the first year and up to $67 million in development and commercial milestones across multiple indications and territories. Lineage also will be eligible to receive royalties up to 10% on net sales of future products.

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"The VAC platform provides us with the opportunity to generate a broad pipeline of product candidates, each targeting a different type of cancer," stated Brian Culley, Lineage CEO. "This collaboration represents the first of many partnerships we hope to enter into with our platform and we believe it helps further validate VAC as a promising new therapeutic vaccine platform. Our objective is to leverage our technology to generate additional VAC-derived cell therapies for our pipeline, as well as in collaboration with partners, capitalizing on the strength of Lineage’s recent manufacturing and cell transplant success. These alliances also will diversify our oncology pipeline across more programs, providing new opportunities for success without the financial burden of independent development. We appreciate ITI selecting our antigen delivery platform for this collaboration and look forward to a productive partnership on this new VAC-derived product candidate. We also are eager to collaborate with additional partners on future versions of VAC."

"We’re very pleased to collaborate with Lineage, a well-recognized cell therapy company, to expand our pipeline with the development of a novel product candidate to treat GBM," commented Dr. William Hearl, CEO of ITI. "Over the last several years, ITI has invested significant capital and development resources to identifying multiple novel paths forward in GBM. By teaming up with Lineage, we are hoping to expand our efforts in this difficult to treat indication and look forward to the benefit that the VAC immunotherapy platform can bring to our antigen constructs."

About Glioblastoma multiforme (GBM)

Glioblastoma multiforme (GBM) (also called glioblastoma) is a fast-growing glioma that develops from star-shaped glial cells (astrocytes and oligodendrocytes) that support the health of the nerve cells within the brain. GBM is often referred to as a grade IV astrocytoma. These are the most invasive type of glial tumors, rapidly growing and commonly spreading into nearby brain tissue. GBMs can arise in the brain "de novo" or evolve from lower-grade astrocytomas or oligodendrogliomas. In adults, GBM occurs most often in the cerebral hemispheres, especially in the frontal and temporal lobes of the brain. GBM is a devastating brain cancer that typically results in death in the first 15 months after diagnosis, with only 25% of glioblastoma patients surviving more than one year, and only 5% of patients surviving more than five years.

About VAC2

VAC2 is an allogeneic, or non-patient specific "off-the-shelf," cancer vaccine product candidate designed to stimulate patient immune responses to an antigen commonly expressed in cancerous cells but not in normal adult cells. VAC2, which is produced from a pluripotent cell technology using a directed differentiation method, is comprised of a population of nonproliferating mature dendritic cells. As the most potent type of antigen presenting cell in the body, dendritic cells instruct the body’s immune system to attack and eliminate harmful pathogens and unwanted cells. Because the tumor antigen is loaded exogenously into the dendritic cells prior to administration, VAC2 is a platform technology that can be modified to carry selected antigens, including patient-specific tumor neo-antigens or viral antigens. VAC2 is currently being tested in a Phase 1 study in adult patients with non-small cell lung cancer (NSCLC) in the advanced and adjuvant settings (NCT03371485), conducted by Cancer Research UK.

On April 20, 2021 Case Western Reserve University and Atelerix Life Sciences Inc., a Charlottesville, Virginia-based biotechnology company, reported that they have signed a two-year option to license a novel family of small molecules called Active Thiol-Based Compounds (ATBC’s) to prevent or reverse life-threatening opioid-related side effects (Press release, Case Western Reserve University, APR 20, 2021, View Source [SID1234578254]).

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The lead compound, ATLX–0199 (also called sudaxine), will be first targeted at opioid-induced respiratory depression (OIRD) in the hospital setting.

A recent study by the medical technology company Medtronic found that 46% of surgical patients receiving opioids for post-operative pain-relief suffer one or more episodes of OIRD, presenting perhaps the most significant obstacle to discharge from intensive care units and the hospital generally.

"No compound now on the market is able to reverse OIRD without also eliminating pain relief—an unacceptable trade-off in the hospital setting," said David Kalergis, Atelerix CEO, co-founder and biotechnology entrepreneur. "This unmet need is well-recognized by medical practitioners, speeding the prospect of rapid adoption and reimbursement in this potential $1 billion market."

The ATBC family and ATLLX-0199 are the result of several decades of collaborative scientific discovery and research among the company’s three co-founders: Stephen Lewis, a professor in the Department of Pediatrics at the Case Western Reserve School of Medicine, Benjamin Gaston, formerly of Case Western Reserve and now Billie Lou Wood Professor of Pediatrics at Indiana University’s School of Medicine, and James Bates, associate professor of Anesthesia at the University of Iowa’s Carver College of Medicine. Bates will become chief medical officer of Atelerix.

Development of the technology has been supported by funding and technical assistance from the National Institutes of Health, Harrington Discovery Institute at University Hospitals and the National Heart, Lung, and Blood Institute’s Catalyze Program.

The ATBC technology harnesses new, cutting-edge understanding of the molecular pathways involved in the effects of opioids, both as a pain reliever and a source of life-threatening side-effects like suppressed breathing. Rather than affecting the opioid receptors directly, they target the inhibition of pathways responsible for the side effects, allowing a more selective therapeutic intervention.

Studies on animal models have shown that ATLX–0199 can be injected intravenously to immediately reverse the negative effects of opioids on breathing without causing withdrawal or reversing the effects of pain control. Long-term, the company expects to expand the technology for use in trauma and overdoses, as well as administering the drug nasally, through patches or other routes.

"As a practicing anesthesiologist, I deal with the problem of opioid-induced depression of breathing in my patients every day," Bates said. "In my new role as chief medical officer of Atelerix Life Sciences, I will be directly involved in developing a solution to this serious unmet medical need by helping speed sudaxine through the U.S. Food and Drug Administration-approval process and into widespread use in the hospital setting."

The two-year option to license agreement, managed through Case Western Reserve’s Technology Transfer Office (TTO), will allow pre-clinical testing of the technology, including safety profiling and feasibility of scaled-up manufacturing.

"After several years of supporting this project within the university, we believe our timely partnership with Atelerix will provide a product that will address the real needs of patients receiving opioids," said Stephanie Weidenbecher, TTO’s senior licensing manager. "Follow-on programs in trauma, military and overdose settings for sudaxine and other molecules in the ATBC family will present comparable opportunities."