Atossa Genetics Inc Stock

Atossa Therapeutics, Inc. operates as a clinical-stage biopharmaceutical company. The company is developing proprietary innovative medicines in areas of significant unmet medical need in oncology, with a focus on breast cancer and other breast conditions.

The company’s lead drug candidate under development is oral (Z)-endoxifen which it is developing in two settings: one to treat breast cancer by reducing tumor cell activity prior to surgery and another to reduce breast tissue density in women. More than ten million women in the U. S and millions more worldwide have elevated breast density.

The company has been granted two U.S. patents covering its proprietary (Z)-endoxifen and it has numerous applications pending in the U.S. and in other countries.

The company’s business strategy is to advance its programs through clinical studies, including with partners, and opportunistically add programs in areas of high unmet medical need through acquisition, minority investment, collaboration or internal development.

Leading Programs

(Z)-endoxifen. (Z)-endoxifen is an active metabolite of tamoxifen, which is an FDA-approved drug to treat and prevent breast cancer in high-risk women. The company is developing a proprietary form of (Z)-endoxifen which is administered orally for the potential treatment of breast cancer and reduction of breast density. The company has completed four Phase 1 clinical studies (including a study in men) and two Phase 2 clinical studies with its proprietary (Z)-endoxifen (including oral and topical formulations). The company has also completed significant pre-clinical development and have established clinical manufacturing capabilities through qualified third-parties.

(Z)-endoxifen for Women with Measurable Breast Density: Mammographic breast density (MBD) is an emerging public health issue affecting over ten million women in the U.S. alone.

In December 2021, the company commenced a Phase 2 study of its proprietary oral (Z)-endoxifen. The study, known as the Karisma-(Z)-endoxifen study, is a Phase 2, randomized, double-blind, placebo-controlled, dose-response study of the company’s proprietary oral (Z)-endoxifen in healthy premenopausal women with measurable breast density. The company expects to complete enrollment in this study by the end of 2023.

(Z)-endoxifen for Neoadjuvant Treatment of Breast Cancer: The company is also developing (Z)-endoxifen to treat estrogen receptor positive (ER+) / human epidermal growth factor receptor 2 negative (HER2-) breast cancer in the neoadjuvant setting, which is the administration of a therapy before the main treatment, which is usually surgery.

In October 2022, the company received authorization from the U.S. FDA for its Investigational New Drug (IND) application for oral (Z)-endoxifen. In February 2023, the company enrolled the first patient in this study.

In March 2023, the company announced that (Z)-endoxifen will be evaluated in a new study arm of the ongoing I-SPY endocrine program. This portion of the study targets patients with newly ER+ invasive cancer. The I-SPY trial is a collaborative effort among academic investigators from 20 major cancer research centers across the U.S., Quantum Leap Healthcare Collaborative, the FDA, and the Foundation for the National Institutes of Health (FNIH) Cancer Biomarkers Consortium. The company will supply (Z)-endoxifen and provide financial support to Quantum Leap for this study.

Programs Under Development

(Z)-endoxifen Programs

In 2020, the company completed a Phase 1, randomized double-blinded, placebo-controlled study in 12 healthy females in Australia to evaluate safety, tolerability and pharmacokinetics of 4 mg delayed release tablets.

In 2019, the company completed a double-blinded, parallel design Phase 1 study in 24 healthy females randomized to either active or placebo in Australia to evaluate pharmacokinetics of its 4 mg capsule (Z)-endoxifen and a new 4 mg modified-release tablet form of the company’s (Z)-endoxifen, as well as to assess safety and tolerability. Study results showed that the capsule and tablet were safe and well-tolerated.

In 2018, the company completed a placebo controlled, three arm, Phase 1, dose escalation study of its topical (Z)-endoxifen (2, 6 and 10 mg) for 28 days in 24 healthy males in Australia.

In 2017, the company completed a Phase 1 study in 49 healthy women in Australia using both the topical and oral capsule forms of its proprietary (Z)-endoxifen to assess the pharmacokinetics of its proprietary (Z)-endoxifen dosage forms as single (oral) and repeat (oral capsule 1, 2 and 4 mg and topical 2, 6 and10 mg) doses, as well as to assess safety and tolerability.

In October 2022, the company received authorization from the FDA to initiate its Phase 2 study of neoadjuvant (Z)-endoxifen in premenopausal women with ER+/HER2- breast cancer. This study is open for enrollment. In February 2023, the company enrolled its first patient in the study.

In December 2021, the company began to enroll participants in a Phase 2 clinical study of oral (Z)-endoxifen in women with elevated MBD. The study, known as the Karisma-Endoxifen study, is a Phase 2, randomized, double-blind, placebo-controlled, dose-response study of the company’s proprietary (Z)-endoxifen in healthy pre-menopausal women with elevated breast density.

In February 2021, the company concluded a Phase 2 study of its oral (Z)-endoxifen in Australia.

In April 2019, the company completed a randomized, double-blinded placebo-controlled Phase 2 study of its topical endoxifen in women with measurable MBD in Stockholm, Sweden.

In June 2019, the company reported preliminary analysis from its Phase 2 study of proprietary daily topical endoxifen to reduce MBD, showing significant (p=0.02) and rapid reduction in MBD at the 20 mg daily dose level. MBD was reduced by an average of 14.3% in the group applying 20 mg daily topical endoxifen, which was statistically significant (p = 0.02).

AT-H201 consists of a proprietary combination of two drugs previously approved by the FDA to treat other diseases. AT-H201 is intended to be inhaled via nebulizer with the goal of preventing or reducing lung injury from COVID-19. In July 2022, the company completed dosing in a placebo-controlled Phase 1/2a study of AT-H201 in healthy participants in Australia.

Other Programs; Immunotherapy/CAR-T Programs

Investment in CAR-T Company: On December 23, 2022, the company closed its previously announced investment in DCT, a privately-held, venture-capital backed, developer of CAR-T therapies. DCT is in the pre-clinical phase of developing controllable CAR-T cells to address difficult-to-treat cancers.

The company’s immunotherapy/CAR-T programs are in early stage of development. On November 26, 2020, the company entered into a sponsored research agreement with Dana-Farber Cancer Institute, Inc. The agreement provides that the company will support research of cytokine-coated nanoparticles for the potential treatment of breast cancer, which was conducted by Carl Novina, MD, Ph.D.

The company has filed patent applications on a novel method to deliver CAR-T cells or other types of immunotherapy into the milk ducts of the breast, the location where most breast cancers originate for the potential targeted treatment of breast cancer.

Research and Development

The company’s research and development expenses for the year ended December 31, 2022, were approximately $15.1 million.

Intellectual Property

The company owns patents directed to (Z)-endoxifen and patent applications directed to (Z)-endoxifen, therapies for respiratory conditions, other therapies, and immunotherapies, such as CAR-T therapies. The company commonly seeks patent claims directed to compositions of matter of therapeutics, including (Z)-endoxifen, as well as methods of using such compositions. For each of its products, the company has filed multiple patent applications and expect to file multiple additional patent applications. As of January 31, 2023, based on a review of its patent estate, the company owned two issued patents (one U.S. patent and one international patent) directed to its (Z)-endoxifen therapies, therapies for respiratory conditions, other therapies, immunotherapies, such as CAR-T therapies, and viral detection programs and are pursuing 75 pending patent applications (19 U.S. applications, including one allowed U.S. application, and 56 international applications, including two allowed international applications). Subsequent to January 31, 2023, the company was issued an additional U.S. patent directed to its (Z)-endoxifen therapies.

Government Regulation

The company is subject to extensive regulation by the FDA and other federal, state, and local regulatory agencies. The Federal Food, Drug, and Cosmetic Act, or the FDCA, and its implementing regulations set forth, among other things, regulations for the execution of clinical studies, and the requirements for the testing, development, manufacture, quality control, safety, effectiveness, approval, labeling, storage, record keeping, reporting, distribution, import, export, advertising and promotion of the company’s products. The company’s business activities outside of the U.S. are subject to anti-bribery or anti-corruption laws, regulations, industry self-regulation codes of conduct, and physicians’ codes of professional conduct or rules of other countries in which it operates, including the U.K. Bribery Act of 2010.

The company is regulated by the FDA under the Federal Food Drug and Cosmetics Act, as well as by other U.S. and foreign federal, state and local agencies. Specific health-care laws and regulations that the company is subject to include:

the federal Physician Self-Referral Law, which prohibits a physician from making referrals for certain designated health services payable by Medicare to an entity with which he or she (or an immediate family member) has a financial relationship, and prohibits the entity from presenting or causing to be presented claims to Medicare for those referred services;

the federal civil and criminal false claims laws, including the False Claims Act (FCA), which prohibits, among other things, individuals or entities from knowingly presenting, or causing to be presented, claims for payment from Medicare, Medicaid or other federal healthcare programs that are false or fraudulent. Moreover, the government may assert that a claim including items or services resulting from a violation of the federal Anti-Kickback Statute constitutes a false or fraudulent claim for purposes of the FCA;

the federal Civil Monetary Penalties Law, which prohibits, among other things, offering or transferring remuneration to a federal healthcare beneficiary that a person knows or should know is likely to influence the beneficiary’s decision to order or receive items or services reimbursable by the government from a particular provider or supplier; and

the federal Physician Payments Sunshine Act which requires certain applicable manufacturers of drugs, devices, biologics and medical supplies for which payment is available under certain federal healthcare programs, to monitor and report to CMS, certain payments and other transfers of value to physicians (defined to include doctors, dentists, optometrists, podiatrists and chiropractors); certain other healthcare providers, including physician assistants, nurse practitioners, clinical nurse specialists, certified nurse anesthetists, certified nurse-midwives, and teaching hospitals; as well as ownership and investment interests held by physicians and their immediate family members.

History

The company was founded in 2008. It was incorporated in Delaware in 2009. The company was formerly known as Atossa Genetics Inc. and changed its name to Atossa Therapeutics, Inc. in 2020.