On September 13, 2022 Syros Pharmaceuticals, Inc. (NASDAQ:SYRS), a leader in the development of medicines that control the expression of genes, reported that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to SY-5609 for the treatment of pancreatic cancer (Press release, Syros Pharmaceuticals, SEP 13, 2022, View Source [SID1234619515]). SY-5609, a highly selective and potent oral cyclin-dependent kinase 7 (CDK7) inhibitor, is currently being evaluated in combination with chemotherapy for the treatment of patients with relapsed metastatic pancreatic cancer.

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"This orphan drug designation underscores the urgency of our efforts to develop SY-5609 for patients with pancreatic cancer, one of the most devastating and difficult to treat malignancies," said David A. Roth, M.D., Chief Medical Officer of Syros. "Based on the early data we reported last year, which demonstrated single-agent activity in heavily pre-treated patients, as well as compelling preclinical data and a strong mechanistic rationale, we believe SY-5609 could deliver meaningful benefit to people with pancreatic cancer, whose tumors have otherwise eluded therapeutic intervention. We look forward to sharing initial data from the safety lead-in portion of our ongoing Phase 1 study later this year."

The FDA’s Office of Orphan Drug Products grants orphan status to support development of medicines for the treatment of rare diseases that affect fewer than 200,000 people in the United States. Orphan drug designation may provide certain benefits, including a seven-year period of market exclusivity if the drug is approved, tax credits for qualified clinical trials and an exemption from FDA application fees.

Syros’ ongoing Phase 1 trial is evaluating SY-5609 in combination with chemotherapy in pancreatic cancer patients who have progressed following treatment with FOLFIRINOX. Patients were randomized to receive either SY-5609 in combination with gemcitabine, or SY-5609 in combination with gemcitabine and nab-paclitaxel, at the approved doses of the combination agents. The study is assessing safety and tolerability, as well as efficacy measures such as disease control rate and progression free survival. Safety and clinical activity data from the safety lead-in portion of the trial are expected in the second half of 2022.

Under an existing clinical supply agreement with Roche, Syros is also supplying SY-5609 for a combination dosing cohort in Roche’s ongoing Phase 1/1b INTRINSIC trial. This cohort is evaluating the combination of SY-5609 and atezolizumab in patients with BRAF-mutant colorectal cancer. Roche is the sponsor of the INTRINSIC trial.

On September 13, 2022 SonALAsense reported Diffuse intrinsic pontine glioma (DIPG) is a rare and deadly pediatric brain cancer with a 2% five-year survival rate (Press release, SonALAsense, SEP 13, 2022, View Source [SID1234619514]). To provide therapeutic options for patients and families, SonALAsense is partnering with clinicians at Children’s National Hospital, Ivy Brain Tumor Center at Barrow Neurological Institute, Phoenix Children’s Hospital and University of California San Francisco (UCSF) Medical Center to conduct a phase 1/2 clinical trial for sonodynamic therapy (SDT) . This is the first SDT trial for children.

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Roger Packer, M.D., co-leads the Children’s National clinical trial site with Lindsay Kilburn, M.D., and Hasan Syed, M.D., who treated the study’s first patient, a 5-year-old girl, two weeks ago. The trial will assess safety, overall patient health and tumor size.

"DIPG is a relentless and sadly fatal disease afflicting children, and there’s currently very little we can do to stop it," said Dr. Packer, who directs the Brain Tumor Institute at Children’s National. "Most children survive less than a year. Sonodynamic therapy offers us an innovative and promising, noninvasive way to directly target the tumors in these children."

"With sonodynamic therapy, we are using focused ultrasound to activate SonALAsense’s drug candidate, which is administered to the patient, and selectively killing tumor cells while leaving normal cells intact," said treating neurosurgeon, Dr. Syed. "With this, we are hoping to see improved outcomes for both tumor regression and patient health during treatment."

As many as 300 children are diagnosed with DIPG in the U.S. each year and there are no effective treatments. DIPG infiltrates the pons, which is located in the brainstem. The condition is difficult to diagnose and treat because tumor cells often diffuse throughout the pons. Radiation therapy is the current standard of care, but it can only delay the cancer, not stop it.

"SDT is a targeted and nonsurgical investigational approach for kids who have almost no therapeutic options," said SonALAsense founder, Chief Medical Officer and Chief Science Officer Stuart Marcus, M.D., Ph.D. "Surgery is contraindicated, chemotherapy and directed therapies are ineffective and have significant side effects."

SDT combines SonALAsense’s drug candidate, SONALA-001, a proprietary intravenous formulation of aminolevulinic acid (ALA), with Insightec’s magnetic resonance-guided focused ultrasound to destroy cancer cells. As the SDT pioneer, SonALAsense is the first and only company to advance SDT into clinical trials in DIPG.

"SDT is a completely novel approach to an incredibly challenging pediatric cancer," said Dr. Sabine Mueller, Pacific Pediatric Neuro-oncology Consortium (PNOC) Lead, and co-Leader of the Pediatric Malignancies Program, UCSF Helen Diller Family Comprehensive Cancer Center.

"DIPG is a devastating disease and, right now, we have no viable treatments for it," said patient advocate Lisa Ward, whose son Jace died from DIPG complications in 2021 at age 22. "We need to explore encouraging new treatments, like SDT, to help patients and families who are suffering with this condition."

The trial is supported, in part, by a $2 million National Cancer Institute Small Business Innovation Research grant to SonALAsense with a subcontract to Children’s National. The trial site in Phoenix is led by Nader Sanai, M.D., at the Ivy Brain Tumor Center, who also led the first-in-human Phase 0/1 clinical trial of SDT in recurrent high-grade glioma patients, along with Lindsey Hoffman, D.O., at Phoenix Children’s. The UCSF trial site is being led by Sabine Mueller, M.D., Ph.D.

On September 13, 2022 RayzeBio, Inc., a targeted radiopharmaceutical company developing an innovative pipeline against validated solid tumor targets, reported significant progress in clinical development of its lead product candidate, RYZ101 (Press release, RayzeBio, SEP 13, 2022, View Source [SID1234619513]). RYZ101 is an investigational, targeted radiopharmaceutical drug designed to deliver Actinium-225 (Ac225), a highly potent alpha-emitting radioisotope, to solid tumors expressing the somatostatin receptor (SSTR).

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The Company is enrolling patients in the Phase 1b portion of the ACTION-1 Phase 1b/3 trial of RYZ101 (NCT05477576). Safety, tolerability, and pharmacokinetics of RYZ101 will be evaluated in patients SSTR+ gastroenteropancreatic neuroendocrine tumors (GEP-NETs) who have progressed on Lu-177 labelled somatostatin analogue therapy. Enrollment is progressing as planned and RayzeBio expects to announce data from the trial in 2023.

Additionally, RayzeBio received Investigational New Drug Application (IND) clearance from the U.S. Food and Drug Administration (FDA) to investigate RYZ101 for first-line treatment of patients with SSTR+ extensive-stage small cell lung cancer (ES-SCLC) in combination with standard of care.

RYZ101 is positioned to be the first approved Ac225 radiopharmaceutical therapy for the industry.
"Clinicians and patients are very excited about the mechanism of action of RYZ101 and the advantages it offers. Significant interest is growing to explore Ac225, a highly potent radioisotope, for treatment of solid tumors. We believe RYZ101 has strong scientific rationale for treatment of both GEP-NETs and small cell lung cancer. We are very encouraged with the level of interest we have seen in the program and look forward to reporting results from RYZ101 in due course," said Susan Moran, M.D., M.S.C.E., Chief Medical Officer of RayzeBio.

"Ac225 has the potential to provided increased efficacy compared to existing beta particle therapy, and we are excited to see the first randomized phase 3 trial evaluating an actinium labeled compound (RYZ101). ACTION-1 will study this treatment in patients with advanced neuroendocrine tumors of the GI tract and pancreas, who have limited effective treatment options. This trial will be the next step forward in improving treatment of these patients," said Dr. Thomas Hope, M.D., Vice Chair of Clinical Operations and Strategy in the Department of Radiology. Dr. Hope is the chair of the steering committee for the phase 3 portion of ACTION-1 study.

As referenced in another press release issued today, RayzeBio has raised $418 million in equity capital since inception in 2020, including $160 million raised in a Series D financing, to advance targeted radiopharmaceutical therapies for cancer.

About gastroenteropancreatic neuroendocrine tumors

Gastroenteropancreatic neuroendocrine tumors, or GEP-NETs, are rare tumors with an incidence of approximately 18,000 patients annually in the United States. Despite this low incidence, many GEP-NETs follow an indolent disease course and thus the prevalence of patients with GEP-NETs in the United States is considerably higher, but still considered an orphan disease. The prognosis for patients with GEP-NET tumors depends on tumor grade and other histopathologic characteristics. Approximately 80% of GEP-NETs express the somatostatin receptor type 2 (SSTR2). Lutathera is a targeted radiopharmaceutical therapy comprised of a somatostatin analog peptide labeled with the beta-emitting radioisotope Lutetium-177 (Lu177), which received regulatory approval for treatment of adult patients with SSTR+ GEP-NETs in Europe and the United States in 2017 and 2018, respectively. However, most patients who receive Lu177-based somatostatin therapies eventually experience tumor progression and have limited subsequent treatment options.

About RYZ101

RYZ101 is an investigational targeted radiopharmaceutical therapy, designed to deliver a highly potent radioisotope, Actinium-225 (Ac225), to tumors expressing SSTR2. RYZ101 is being evaluated in clinical studies for patients with SSTR+ GEP-NETs who have previously been treated with Lu177-based somatostatin therapies and also in patients with extensive stage small cell lung cancer. Details of the study can be found at View Source

On September 13, 2022 City of Hope, one of the largest cancer research and treatment organizations in the United States, reported that it has received a $25 million gift from Julia and George Argyros, visionary philanthropists and longtime City of Hope supporters (Press release, City of Hope, SEP 13, 2022, View Source [SID1234619512]). The transformational gift, announced by Argyros Family Foundation president Julia A. Argyros during the Sept. 10 Hope Gala, advances City of Hope’s mission to deliver world-renowned research, treatment and cancer cures to Orange County. The gala raised $2.3 million, in addition to the announcement of the Argyros family’s transformational $25 million gift to City of Hope Orange County.

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The Argyros family gift continues a decades-long legacy of extraordinary generosity and support from the family to City of Hope that has been pivotal to its evolution into a leading cancer research and treatment system.

Argyros is a treasured name at City of Hope, one that is deeply connected to the daily rhythms of the first City of Hope campus near Los Angeles. The stunning Argyros Family Garden of Hope is a testament to the importance of caring for the whole person. Julia Argyros recalls sitting in the serene space in Duarte, observing patients and families pause and reflect in a place that represents healing.

Now, in recognition of the transformational gift, the Argyros name will grace the healing garden at Orange County’s most advanced comprehensive cancer center, extending a shared commitment to healing mind, body and spirit.

The Argyros family has made deep investments in health care, education, childhood development and arts foundations across Southern California.

"We are honored to be in the position to support City of Hope, which is saving lives and expanding access to its advanced cancer care. We believe it is our privilege to give back to the community. We hope that this gift helps people get the care they need and that everyone who walks through the healing gardens at the new cancer center is filled with peace and hope," Julia Argyros said.

Robert Stone, CEO, City of Hope, and the Helen and Morgan Chu Chief Executive Officer Distinguished Chair, said Julia and George Argyros have been exceptional supporters of City of Hope for nearly 40 years, including during George’s 20-year tenure as Chair of the Arnold and Mabel Beckman Foundation.

"City of Hope’s ability to provide advanced breakthroughs in cancer care to the people of Orange County is due to visionary supporters like Julia and George Argyros who empower our scientists and physicians to rapidly discover and develop lifesaving therapies for patients," Stone said. "We remain profoundly grateful for such an important and longstanding partnership in our mission to eradicate cancer."

City of Hope, now one of the largest cancer research and treatment organizations in the United States, recently opened City of Hope Orange County Lennar Foundation Cancer Center in Irvine. It is home to acclaimed physician-scientists who solely focus on preventing, treating and curing cancer. The Argyros gift demonstrates City of Hope’s strong base of philanthropic support in Orange County.

"City of Hope came to Orange County to answer a pressing need in the community, and we are grateful for those foresighted supporters who help us ignite a movement that is changing the health care landscape," said Annette M. Walker, president, City of Hope Orange County. "Supporters like Julia and George Argyros, and everyone whose contributions are helping to make this vision possible, are evidence of the incredible support for City of Hope in Orange County. Together, we can achieve our goal of making our communities cancer-free."

A Gala Filled With Hope

The gala at the Segerstrom Center for the Arts in Costa Mesa, California, brought together leaders and luminaries to honor Julia A. Argyros and celebrate the partnership of City of Hope’s many philanthropic supporters in Orange County and nationally. The gala featured a performance by Grammy-nominated recording artist Nick Jonas.

"I am honored to lend my voice to the important cause of expanding access to world-renowned cancer care," said Jonas, whose father is a cancer survivor.

Julia and George Argyros and George Wall and family were the evening’s presenting sponsors. The Wall family supported the gala in tribute to Cathy "Cate" Roth Wall, who received exceptional care at City of Hope.

Kristin J. Bertell, chief philanthropy officer, City of Hope, expressed gratitude for the overwhelming number of philanthropic contributions to City of Hope Orange County.

"Orange County recognizes that philanthropy speeds cancer advances and makes health care transformation possible," Bertell said. "Our patients and their families cannot afford to wait when it comes to receiving lifesaving cancer treatments and care. We are deeply grateful to Julia and George Argyros, George Wall and family, Nick Jonas, and everyone who is showing remarkable generosity in helping us get tomorrow’s cures to patients who need them today."

City of Hope Is Orange County’s Most Advanced Cancer Care

City of Hope, one of only 52 National Cancer Institute-designated comprehensive cancer centers in the country, is delivering advanced treatment, research and cancer cures to Orange County’s 3.2 million people.

Patients at City of Hope Orange County Lennar Foundation Cancer Center receive fully integrated, multidisciplinary care — from prevention through survivorship — in one convenient location where every physician and staff member is solely focused on treating and curing cancer. They have access to City of Hope’s 575 physicians and more than 1,000 researchers and scientists who only focus on cancer and nearly 1,000 Phase 1-3 clinical trials conducted at City of Hope each year.

The outpatient cancer center features a suite of market-leading technology, 67 spacious exam and treatment rooms, an infusion center designed around patient preferences, and a full-service salon and specialty shopping experience. It will be seamlessly connected to Orange County’s only specialty cancer hospital, opening in 2025.

City of Hope’s Orange County cancer care network currently includes four regional clinics — two in Newport Beach, one in Huntington Beach and one in Irvine. The cancer center, the regional clinics and the planned hospital will create Orange County’s largest network dedicated exclusively to cancer treatment and cures.

To make an appointment, call 888-333-HOPE (4673). To give to City of Hope Orange County, go to CityofHope.org/giving.

On September 13, 2022 METiS Therapeutics, a Boston-area company integrating drug discovery and delivery with AI and machine learning, reported they have entered a license agreement with Voronoi, Inc., a Korean company focused on precision medicine (Press release, MêTiS Biotechnologies, SEP 13, 2022, View Source [SID1234619511]). Under the terms of this license agreement, METiS will receive exclusive license to develop and commercialize Voronoi’s pan-RAF inhibitor program worldwide. In exchange, Voronoi will receive up to $1.7 million in upfront cash and success-based near-term discovery milestones. Voronoi will also be eligible for success-based payment up to $480.5 million in development, regulatory and sales milestones and tiered royalties based on annual net sales.

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"We view this program as a potential best-in-class asset addressing high unmet clinical needs for Class II and Class III BRAF mutant cancer patients, particularly those with CNS metastasis," said Chris Lai, Co-Founder and CEO of METiS Therapeutics. "We see it as a compelling proof-of-concept opportunity for our AiTEM platform to improve compound pharmacokinetics and deliver a differentiated profile, including CNS penetration. In addition, we view this as a foundational asset for our oncology portfolio."

BRAF is a protein kinase involved in cancer cell growth and BRAF mutations are the most common mutation found in tumor cells1. While several BRAF v600E inhibitors have been approved, this mutation only represents about 45% of total BRAF mutations, compared to Class II and III mutations which represent 55 percent of total BRAF mutations. This underscores the need for new therapeutic options in this space.

"We developed our structurally distinguished type II pan-RAF inhibitor to address unmet medical needs, and this partnership brings us closer to our goal of delivering this candidate to patients," says Daekwon Kim, CEO of Voronoi. "We are excited to partner with METiS’ seasoned team to continue on development of our pan-RAF inhibitor," added Daekwon.

This news follows METiS’ announcement that the company had joined the Roche Accelerator earlier this year. In December, METiS closed an $86 million Series A financing round. The company recently leased 14,000 square feet of mixed office and laboratory space at 101 Cambridgepark Drive in Cambridge, MA.