Expanded Access Policy

We are a clinical-stage company dedicated to the development of novel therapeutics for diseases with high unmet medical needs, including amyotrophic lateral sclerosis (ALS) and treatment-resistant metastatic breast cancer that can reach and positively impact everyone, including patients, their families and caregivers.

Before our therapies currently in development are fully reviewed and approved by regulatory authorities (including the U.S. Food and Drug Administration (FDA)), they can be accessed only through participation in our clinical trials. Clinical trials are controlled research studies designed to evaluate whether a new therapy is safe and effective for patients, and they provide the most direct opportunity for patients to access a new product before regulatory approval. While we are mindful of the serious impact that these devastating diseases have on those affected, and are committed to completing the regulatory review and approval process diligently through our ongoing clinical trials, at this time, we do not offer any Sponsor-initiated expanded access programs for our investigational therapies outside of our clinical trials. You can find additional information about our ongoing clinical trials by accessing https://www.leonabio.com/patients/ and https://clinicaltrials.gov/study/NCT05696626. You can learn more about expanded access, including the general criteria and how expanded access works, by visiting the FDA website at: Expanded Access | Information for Patients

However, we recognize that some individuals may have a serious or immediately life-threatening disease or condition for which we are developing an investigational therapy and for which the individual may have no satisfactory therapeutic alternatives and who do not qualify for participation in one of our clinical trials. In these rare cases, we may consider providing access to an investigational therapy outside of a clinical trial to a limited number of physicians who are treating such individuals through physician-initiated expanded access.

We will evaluate a request for expanded access on a case-by-case basis and only when such request is submitted by a licensed physician. The decision to make an investigational therapy available to an individual outside of a clinical trial through the expanded access process is complex and involves a number of factors. For example, we must ensure that expanded access can be provided in a fair manner that complies with current regulations and guidelines, including upholding ethical principles and minimizing potential risks to patients. As a general policy, we will not grant a request for expanded access unless:

• The investigational therapy is currently in clinical development and is currently being studied in humans for a particular use.
• The patient has a serious disease or condition, or whose life is immediately threatened by their disease or condition.
• It is impossible or not feasible for the patient to participate in a LeonaBio-sponsored clinical trial because, for example, the patient does not meet the criteria for participation.
• The patient has exhausted all available therapies.
• There is sufficient evidence that the potential benefit to the patient justifies the potential risks of treatment with the investigational drug, and those potential risks are not unreasonable in the context of the disease or condition to be treated, based on all currently available information for the investigational therapy.
• The grant of physician-initiated expanded access for the requested use will not interfere with LeonaBio’s clinical trials that may support marketing approval of the expanded access use or otherwise compromise the clinical development of the investigational therapy.

Requesting Physician-Initiated Expanded Access:

We encourage patients to first discuss with their treating physician about whether participation in a clinical trial is appropriate for them. If a treating physician believes expanded access may be an available and appropriate option for a patient, the physician may submit an inquiry for more information or a formal request for expanded access on behalf of the patient by contacting LeonaBio at clinicaltrials@leonabio.com.

LeonaBio expects to acknowledge receipt of an inquiry or a formal request for physician-initiated expanded access within 5 business days of receipt. Submitting a formal request or receiving LeonaBio’s acknowledgement of receipt of a request does not guarantee that the request will be granted. A request for physician-initiated expanded access can only be considered if the patient’s treating physician provides the required patient medical information and is fully committed to and is supportive of the requested treatment, including complying with all applicable regulatory requirements for a physician-initiated expanded access and being fully responsible for patient care. Even if a request for expanded access is granted by LeonaBio, the expanded access request remains subject to review by FDA. Physicians located in the United States may find additional information regarding physician-initiated expanded access by visiting FDA’s website at: Expanded Access | Information for Physicians.

We may revise this Expanded Access Policy at any time. The posting of this policy does not guarantee access to any specific investigational therapy for any individual.