Atossa Therapeutics and Quantum Leap Healthcare Announce New Study Arm to Evaluate (Z)-Endoxifen in the Ongoing I-SPY 2 Clinical Trial

Atossa Therapeutics, Inc. (Nasdaq: ATOS), a clinical stage biopharmaceutical company developing innovative medicines in areas of significant unmet medical need in oncology, and Quantum Leap Healthcare Collaborative™ today announce that Atossa’s proprietary Selective Estrogen Receptor Modulator (SERM),(Z)-endoxifen, will be evaluated in a new study arm of the ongoing I-SPY 2clinical trial.

SEATTLE and SAN FRANCISCO, March 21, 2023 — Atossa Therapeutics, Inc. (Nasdaq: ATOS), a clinical stage biopharmaceutical company developing innovative medicines in areas of significant unmet medical need in oncology, and Quantum Leap Healthcare Collaborative™ today announce that Atossa’s proprietary Selective Estrogen Receptor Modulator (SERM),(Z)-endoxifen, will be evaluated in a new study arm of the ongoing I-SPY 2clinical trial. The I-SPY 2 TRIAL evaluates neoadjuvant treatments for locally advanced breast cancer and is a collaborative effort among academic investigators from major cancer research centers across the United States, Quantum Leap Healthcare Collaborative, the U.S. Food and Drug Administration, and the Foundation for the National Institutes of Health (FNIH) Cancer Biomarkers Consortium. Approximately 20 patients will be treated with (Z)-endoxifen for up to 24 weeks prior to surgery.

The new study arm evaluating(Z)-endoxifen is part of the ongoing I-SPY 2 Endocrine Optimization Pilot Protocol (EOP), which targets patients with newly diagnosed estrogen receptor-positive (ER+) invasive breast cancer whose tumors are predicted to be sensitive to endocrine therapy but for whom chemotherapy is expected to provide little or no benefit. These patients have substantial risk for recurrence, often after five years, and need novel agents that are more tolerable and effective than the current standard of care and address the risk of recurrence. 

“Atossa is proud to partner with Quantum Leap Healthcare Collaborative and excited that(Z)-endoxifen has been added to their unprecedented I-SPY clinical trial, which is designed to quickly and efficiently bring new drug therapies to breast cancer patients who need them urgently,” said Dr. Steven Quay, Atossa’s President and Chief Executive Officer. “Data from this trial will supplement data generated through our ongoing Phase 2 EVANGELINE trial, which is investigating (Z)-endoxifen as a neoadjuvant treatment for premenopausal women with ER+ / HER2- breast cancer. There remains a critical need for more effective and tolerable treatment options for this patient population.”

“(Z)-endoxifen is one of the most active metabolites of tamoxifen. Tamoxifen is known to be effective in treating and preventing ER+ breast cancer, but may be most effective in those patients who can adequately metabolize it, ”said Dr. Laura Esserman of the University of California San Francisco, founder and leader of the I-SPY TRIAL. “(Z)-endoxifen may overcome some of the shortcomings of tamoxifen, as it is already the active metabolite. Perhaps most exciting is that it may be effective in premenopausal women without the need for ovarian suppression. Ovarian suppression in premenopausal women and aromatase inhibitors in postmenopausal women are associated with both short-term and potential long-term side effects that diminish adherence.(Z)-endoxifen holds promise of being a more effective and tolerable alternative to targeting the estrogen receptor in women with early-stage breast cancer.”

Atossa is currently evaluating (Z)-endoxifen in two ongoing Phase 2 studies: 

The EVANGELINE (Endoxifen Versus exemestANe GosEreLIn) trial is a randomized non-inferiority study of (Z)-endoxifen compared to exemestane plus goserelin as a neoadjuvant treatment for premenopausal women with Grade 1 or 2 ER+ / HER2- breast cancer. Participants receive neoadjuvant treatment for up to six months, followed by surgery. The primary objective of the EVANGELINE study is to determine whether the endocrine sensitive disease (ESD) rate, measured by Ki-67 (a proliferation marker prognostic for disease free survival), after four weeks of treatment with (Z)-endoxifen is non-inferior to the ESD rate following treatment with current standard of care, exemestane plus goserelin. Exemestane is an aromatase inhibitor designed to block the synthesis of estrogen and slow the growth of ER+ cancers. Goserelin is a medication given to block the ovaries from making estrogen, which in premenopausal women is associated with significant morbidity and inadequate compliance, which compromises efficacy and increases the risk of mortality.

The Karisma-Endoxifen trial is a randomized, double-blind, placebo-controlled efficacy study of oral(Z)‑endoxifen in premenopausal women with measurable breast density. Participants receive daily doses of (Z)-endoxifen for six months, over the course of which mammograms are conducted to measure reduction in mammographic breast density(MBD). Participants also have a mammogram at 24 months to assess the durability of the MBD changes. MBD affects more than 10 million women in the United States and many millions more worldwide. Increased MBD reduces the ability of mammograms to detect cancer. Studies have also shown that women with MBD have an increased risk of developing breast cancer and that the higher the MBD, the higher the incidence of breast cancer.

Atossa will supply (Z)-endoxifen and provide financial support to Quantum Leap for this study. Quantum Leap, as sponsor, will provide the clinical sites and clinical expertise. Currently, there are 41 open sites, all of which have the EOP program open.

About Premenopausal Patients with ER+ / HER2- Breast Cancer

Breast cancer is the most frequently diagnosed cancer in premenopausal women worldwide and accounts for almost half of the cancers that occur in women aged 15-49. An overwhelming majority (75%) of premenopausal breast cancer falls under luminal A (ER+/HER2-) or B (ER+/HER2+) subtypes. Ovarian function suppression, when combined with either tamoxifen or an aromatase inhibitor, is the standard of care for the endocrine management of stage 2 and 3 premenopausal ER+/HER2-breast cancer. The I SPY Endocrine Optimization Pilot (EOP) specifically targets women of all ages with molecularly low risk stage 2 and 3 breast cancer.

About(Z)-Endoxifen

(Z)-endoxifen is the most active metabolite of the FDA approved Selective Estrogen Receptor Modulator (SERM), tamoxifen. Studies by others have demonstrated that the anti-estrogenic effects of tamoxifen are driven in a concentration-dependent manner by(Z)-endoxifen. In addition to its potent anti-estrogen effects, (Z)-endoxifen at higher concentrations has been shown to target PKCβ1, a known oncogenic protein.

Atossa has developed a proprietary oral formulation of (Z)-endoxifen that does not require liver metabolism to achieve therapeutic concentrations and is encapsulated to bypass the stomach as acidic conditions converts a greater proportion of (Z)-endoxifen to the inactive (E)-endoxifen. Atossa’s(Z)-endoxifen has been shown to be well tolerated in Phase 1 studies and in a small Phase 2 study of women with breast cancer. We are currently studying our(Z)-endoxifen in healthy women with measurable breast density and premenopausal women with ER+/HER2- breast cancer. Atossa’s (Z)-endoxifen is protected by two issued U.S. patents and numerous pending patent applications.

About Atossa Therapeutics

Atossa Therapeutics, Inc. is a clinical-stage biopharmaceutical company developing innovative medicines in areas of significant unmet medical need in oncology with a current focus on breast cancer and lung injury caused by cancer treatments. For more information, please visit www.atossatherapeutics.com

About Quantum Leap Healthcare Collaborative

Quantum Leap Healthcare Collaborative is a 501c(3) charitable organization established in 2005 as a collaboration between medical researchers at University of California, San Francisco and Silicon Valley entrepreneurs. Our mission is to integrate care and research, and to foster high-impact trials with embedded clinical processes and systems technology and improved data management, greater access to clinical trial matching, and greater benefit to patients, providers, and researchers. Our goal is to improve and save lives. Quantum Leap provides operational, financial, and regulatory oversight to I-SPY. For more information, visit https://www.quantumleaphealth.org/

About the I-SPY TRIALs

The I-SPY TRIAL (Investigation of Serial studies to Predict Your Therapeutic Response with Imaging And moLecular analysis 2) (I-SPY 2 TRIAL) was designed to rapidly screen promising experimental treatments and identify those most effective in specific patient subgroups based on molecular characteristics (biomarker signatures).  The Endocrine Optimization Pilot (EOP) is developing better endpoints and new endocrine targeted agents for stage 2/3molecularly low risk breast cancer. The trial is a unique collaborative effort by a consortium that includes the Food and Drug Administration (FDA), industry, patient advocates, philanthropic sponsors, and clinicians from 30 major U.S. cancer research centers. Under the terms of the collaboration agreement, Quantum Leap Healthcare Collaborative is the trial sponsor and manages all study operations. For more information, visit www.ispytrials.org.

Atossa Therapeutics Contacts:
Kyle Guse
General Counsel and Chief Financial Officer
kyle.guse@atossainc.com

Eric Van Zanten
VP, Investor and Public Relations
610-529-6219
eric.vanzanten@atossainc.com

Quantum Leap Healthcare Collaborative Media Contact:
Jacqueline Murray
Director, Marketing and Communications
(415)839-8082
j.murray@quantumleaphealth.org

Forward Looking Statements
Forward-looking statements in this press release, which Atossa undertakes no obligation to update, are subject to risks and uncertainties that may cause actual results to differ materially from the anticipated or estimated future results, including the risks and uncertainties associated with any variation between interim and final clinical results, actions and inactions by the FDA, the outcome or timing of regulatory approvals needed by Atossa including those needed to commence studies of (Z)-endoxifen, lower than anticipated rate of patient enrollment, estimated market size of drugs under development, the safety and efficacy of Atossa’s products, performance of clinical research organizations and investigators, obstacles resulting from proprietary rights held by others such as patent rights, whether reduction in breast density or in Ki-67 or any other result from a neoadjuvant study is an approvable endpoint for (Z)-endoxifen, whether Atossa can complete acquisitions, and other risks detailed from time to time in Atossa’s filings with the Securities and Exchange Commission, including without limitation its periodic reports on Form 10-K and 10-Q, each as amended and supplemented from time to time.

For more information, email karyn.digiorgio@quantumleaphealth.org

Contact Us
QLHC in the News
Working at QLHC
Contact Us

Tracey Heather

Advocate Liaison

As Patient Engagement Lead at Quantum Leap, I manage follow-up data projects for the I-SPY 2 Trial, develop strategies to strengthen patient engagement, and oversee the I-SPY Advocate group. After six successful years raising funds to support Quantum’s mission, I embraced this new role last summer, drawn by the opportunity to make a direct impact on the patients we serve. Each day brings new challenges and insights, and I especially enjoy supporting and collaborating with our inspiring I-SPY Advocates.

Outside of work, I am pursuing a Master of Public Administration with a focus on nonprofit management at SF State. I’m passionate about trail running, skiing, live music, camping, traveling, and collegiate gymnastics—my daughter competes at Oregon State. For the past 5 years, I have volunteered with SF CASA as a mentor to an incredible 18-year-old foster youth whose resilience continually reminds me of life’s true priorities.

Carolyn Clark Beedle

Carolyn Clark Beedle, a 2023 breast cancer survivor, joined the advocate program after successful completion of treatment with the UCSF Breast Oncology Program. Her experience working with a patient advocate during her TNBC treatment led to an understanding that empowering women to advocate for their own health and healthcare will contribute to improved health outcomes and broader access to care. Carolyn began advocating for cancer patients and their families during her treatment, now is a member of the Breast Science Advocacy Core (BSAC) with the UCSF Breast Oncology Program, and currently shares information and research with CFNP associates at La Clinica in the Bay Area to inform and empower their patient population.

Carolyn is continuing her on the ground training as an advocate reviewer with both Quantum Health and BSAC and is enrolled in the Patient Advocacy Training in Health Science course with Stanford Medicine. Her 30+ career in corporate marketing/communications, program development and non-profit leadership augmented by her breast cancer treatment experience serves Carolyn well in representing and communicating the patient voice and perspective.

She received her BA (History/English Lit) and MA (Public History/Research and Record Management) from the University of San Diego, is a proud fifth generation San Franciscan, and active board member with numerous non-profits that support social work and the arts.

Silver Alkhafaji

Silver Alkhafaji is a PhD candidate in the Pharmaceutical Sciences and Pharmacogenomics (PSPG) program at UCSF. She received her Bachelor of Science in Chemical Biology from UC Berkeley. Prior to UCSF, she worked in the Clinical Pharmacology Department at Genentech. Silver’s current research focuses on non-invasive liquid biopsies to predict response and side effects of immunotherapies and endocrine therapies in early-stage breast cancer participants in I-SPY 2.

Silver is interested in clinical outcomes research to advance precision medicine and improve cancer patients’ quality of life. She is passionate about health equity, inclusive research, patient advocacy, and women’s health.
Silver volunteers at the Patient and Family Cancer Support Center at UCSF where she assists in patient navigation and connecting patients and their families with resources that improve their healthcare experience while receiving cancer treatments and/or during survivorship. 

Through her DEI work in her PhD program, Silver raises awareness around issues related to social justice and community building through organizing community-centered events. Additionally, she is a member of the Life Sciences Career Advisory Council at Thrive Scholars, where she enjoys supporting college students of color from economically disadvantaged communities in providing the opportunities they need to thrive at top colleges and in high-trajectory careers. 

Silver is a member of the American Association of University Women (AAUW) Alameda Branch where she focuses her efforts on increasing membership of community college women coming from exceptional backgrounds: student parents, low-income, and first-generation college students.

In her free time, she writes poetry and prose on emotional healing, radical acceptance, and patience. Writing has helped her process difficult situations and connect with people on a deeper level.

Jane Mortimer

Jane is a breast cancer survivor and advocate dedicated to positively impacting the lives of women affected by the disease. Diagnosed with triple negative breast cancer in 2012, she participated in the I-SPY 2 trial at UCSF and has been cancer free for more than ten years.

Her advocacy journey began in 2003 at UCSF as a volunteer with the Patient and Family Cancer Support Center and Decision Support Services and she previously managed the Peer Support program at UCSF. Drawing on her experience in marketing and media strategy, she uses her skills to make a meaningful impact by supporting advocacy and research that improves outcomes for women living with breast cancer.

Jan Tomlinson

In March of 2023 , Jan was diagnosed with a large aggressive triple negative breast cancer and informed that her cancer was the” bad girl” of cancer and offered standard chemotherapy for 24 weeks. Devasted by the diagnosis Jan felt like she had a dire prognosis. After seeking several opinions, she opted to join a Clinical Trial program for her treatment. The trial consisted of significantly less chemotherapy, and monitored closely over a 12-week period, The data predicted a complete pathological response , and she then went immediately to surgery. Pathology reports supported that she had a successful outcome reaching PCR meaning the tumor was gone, and no residual cancer was found in the surrounding tissue or lymph nodes. Jan was thrilled when her surgeon advised her of the results. The experience made Jan want to give back and share information that she received when she was at a critical juncture in her diagnosis. She is so passionate about making sure that everyone knows that the standard of care is one treatment option.

As she says, “ clinical trials have to be on the table” Because she achieved PCR, she expects a great outcome. She wants to share her story and encourage other women to strongly consider and participate in clinical trials. Jan is a UCSF Patient Advocate, involved in several programs they lead. Jan also is a BLACC Cab Member. Jan recently was in Washington DC to participate on a panel on Clinical Trials for ISPY at the National Press Club. UCSF will be hosting the RISE Up For Breast Cancer event where Jan will share her experience with clinical trials.

Deborah Collyar

Deb is a connector who founded Patient Advocates in Research (PAIR) “where research meets reality,” bringing ideas and people together for medical advances that offer real results for diverse patients and families.

Her vast experience between the worlds of tech, communication, strategy, management, policy, and equity bridges gaps between patients, scientists, medical providers, payers, governments, and non-profits.

Deb infuses patient engagement into projects, gathers relevant patient input, and encompasses many diseases, programs and policies at grassroots, national and international levels through companies, academia, and governments.

Key patient insights are delivered throughout discovery, development, clinical trials, results reporting, data-sharing, standards, genomics, and into practice.

Her experience spans translational and clinical research, epidemiology, health outcomes, and health delivery research with academia, federal agencies, companies, and patient communities.