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The forward-looking statements made in this filing relate only to events or information as of the date on which the statements are made in this Annual Report. You should not rely upon forward-looking statements as predictions of future events. Although we believe that the expectations reflected in the forward-looking statements are reasonable, we cannot guarantee that the future results, levels of activity, performance or events and circumstances reflected in the forward-looking statements will be achieved or occur. We undertake no obligation to update publicly any forward-looking statements for any reason after the date of this Annual Report to conform these statements to actual results or to changes in our expectations, except as required by law. We intend the forward-looking statements contained in this Annual Report to be covered by the safe harbor provisions for forward-looking statements contained in Section 27A of the Securities Act of 1933, as amended, or the Securities Act, and Section 21E of the Securities Exchange Act of 1934, as amended, or the Exchange Act.
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PART I
Item 1. Business
Overview
We are a precision oncology company focused on the development of targeted therapeutics for the treatment of cancer in genomically defined patient populations. Our vision is to elevate precision medicine to the forefront of every cancer treatment journey, as we believe that each patient living with cancer deserves the opportunity to benefit from a genomically-driven treatment decision. We utilize our deep expertise in developing drugs for rare, genomically defined patient populations and strategic collaborations with our diagnostic collaborators to work towards a future where each tumor’s unique genomic test result can be matched with a purpose-built precision medicine.
We are focused on identifying oncogenic drivers that are known to be predominantly mutually exclusive with other driver alterations, and pursuing innovations and efficiencies in the conduct of clinical trials that we believe may enable development of targeted therapeutics against those oncogenic drivers.
Our lead drug development candidate, seribantumab, is an Anti-HER3 monoclonal antibody and potential novel targeted therapy for solid tumors driven by neuregulin-1, or NRG1 fusions, a type of genomic alteration and oncogenic driver, and other receptor tyrosine-protein kinase erbB-, or HER3, genomically defined cancers. We have designed and initiated our registration-directed Phase 2 CRESTONE clinical trial to investigate the safety and efficacy of seribantumab in advanced solid tumors harboring an NRG1 fusion. We expect to complete enrollment of the first 20 patients in Cohort 1 of the CRESTONE study in mid-2022, and to present initial clinical data from approximately 10 patients from Cohort 1 of the CRESTONE study treated with seribantumab at 3 grams weekly at a major medical meeting in mid-2022.
We also plan to expand our drug development pipeline beyond seribantumab into additional genomically defined cancers by leveraging our value-driving partnerships with Caris Life Sciences and others, as well as by exploring further opportunities to selectively partner on additional precision oncology assets.
We are managed by a team with significant experience and track records for clinical and commercial execution in the oncology space and backed by a group of renowned scientific advisors and partners that bring diagnostic, clinical, and operational expertise to help optimize execution.
Our strategy
Our goal is to deliver safe and effective therapies for genomically defined patient populations with significant unmet clinical needs. Key elements of our strategy include:
| ● | Implement an innovative operational model tailored to the efficient development and potential regulatory approval of precision therapeutics for rare, genomically defined cancers, beginning with the investigation of seribantumab for any solid tumor driven by an NRG1 fusion. We have designed our business model around genomically defined cancers with smaller trial size requirements and the potential for accelerated approval. Rather than follow the traditional drug discovery path, we have focused on external development to identify potential assets that we can acquire at attractive economic terms and that will enable us to move efficiently through clinical development. We acquired seribantumab in July 2019 as a Phase 2-ready asset, enabling us to move rapidly into a potentially registration-directed Phase 2 trial. We have also employed innovative models to enhance traditional patient enrollment into our clinical trials, with opportunity for enrollment not only at core sites, but also through referrals through our diagnostic partnerships, and a “just-in-time” site initiation model. This enables us to reduce overhead costs for a trial and increase the availability of eligible patients, addressing two key risks in the development of therapies for rare, genomically defined patient populations. |
| ● | Prioritize oncogenic drivers that are predominantly mutually exclusive oncogenic alterations to maximize the probability of clinical success. We focus on oncogenic driver events that are most often mutually exclusive within a tumor, meaning that co-expression with other known driver alterations is not observed. We believe that |
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