License, Acquisition, Research and Collaboration and Sponsored Research Agreements |
3 Months Ended |
|---|---|
Mar. 31, 2026 | |
| Business Combination [Abstract] | |
| License, Acquisition, Research and Collaboration and Sponsored Research Agreements | 12. License, Acquisition, Research and Collaboration and Sponsored Research Agreements Adverum Biotechnologies—On January 25, 2021, the Company entered into an exclusive license agreement with Adverum Biotechnologies Inc. (“Adverum”) to in-license materials and technology related to the treatment of cardiomyopathy due to Friedreich's ataxia ("FA") (as amended in February 2022, the “Adverum Agreement”). In connection with the Adverum Agreement, the Company gained access to a portfolio of inventions, patent rights, technology, and licensed methods that the Company continues to develop, and the Company will assume all development and commercialization activities worldwide. Pursuant to the Adverum Agreement, the Company paid a one-time up-front non-refundable fee of $7.5 million, and is obligated to pay aggregate development and regulatory milestones of up to $17.5 million including a $3.5 million development milestone that was achieved and paid in the first quarter of 2023, and aggregate sales event and commercialization milestones of up to $49.0 million. The Company is obligated to pay Adverum tiered royalties ranging from high single-digits to sub teens based on annual aggregate worldwide net sales of Products (as defined in the Adverum Agreement). As of March 31, 2026, there were no research and development expenses recorded by the Company or payments made to Adverum under the terms of the Adverum Agreement other than the one-time up-front non-refundable fee of $7.5 million and the $3.5 million development milestone that was achieved and paid in the first quarter of 2023. The Adverum Agreement remains in effect until termination at the date of the last royalty term to expire. The Company can terminate the Adverum Agreement with 120 days’ written notice. The Adverum Agreement can also be terminated as a result of a patent challenge, material breach of contractual terms, or insolvency by either party. Cornell University—On May 28, 2020, the Company entered into two exclusive license agreements with Cornell University (“Cornell”) (each, as amended, the “First Cornell License Agreement” and the “Second Cornell License Agreement,” respectively; collectively the “May 2020 Cornell License Agreements”). The First Cornell License Agreement is for the in-license of technology related to portfolios for APOE-associated Alzheimer’s disease and Anti-Tau, although the Company’s license is not restricted by such indications and it includes assignment to the Company of Cornell’s IND for the use of AAVrh10.hAPOE2 vector to treat APOE4 homozygous patients who are at risk for or have Alzheimer’s disease to support development of the Company’s LX1001 program. The Second Cornell License Agreement is for the in-license of technology related to a portfolio for FA although the Company’s license is not restricted by such indications, and it includes assignment to the Company of Cornell’s IND for the use of AAVrh.10cUhCLN2 to treat children with CLN2 Batten disease to support development of the Company’s LX1004 program. Through the May 2020 Cornell License Agreements, the Company gains access to a portfolio of inventions, patent rights, technology, and licensed methods that the Company continues to develop. Under the terms of the May 2020 Cornell License Agreements, the Company has assumed all development and commercialization activities worldwide with respect to the licensed technology. As initial consideration for the May 2020 Cornell License Agreements, the Company paid Cornell an upfront payment in cash of $0.3 million and issued $1.3 million of notes (the “Cornell Notes”). In November 2020, the Cornell Notes with outstanding principal of $1.3 million were cancelled in exchange for 1,337,610 shares of series A convertible preferred stock, which converted into 126,258 shares of common stock upon the closing of the Company's IPO on November 7, 2023. As additional consideration, the Company is required to pay Cornell up to $8.4 million upon the achievement of specific clinical and regulatory milestones under the First Cornell License Agreement and up to $4.3 million in two portfolios and up to $0.6 million for a third portfolio upon the achievement of specific clinical and regulatory milestones under the Second Cornell License Agreement. In the second quarter of 2022, a clinical and regulatory milestone of $0.1 million was recognized and paid to Cornell in connection with the Second Cornell License Agreement. The Company is also required to pay Cornell a flat royalty in the mid-single-digits based on net sales of the products covered by the licenses, subject to certain adjustments. Upon expiration of the royalty term of a given licensed product in a country, the respective license becomes non-exclusive and royalty-free. In addition, each of the May 2020 Cornell License Agreements may be terminated by the Company for any reason upon ninety (90)-days’ advance notice to Cornell and by Cornell upon the Company’s material uncured breach, and all licenses and rights granted by either party under such agreement will concurrently terminate. On April 21, 2024, the Company entered into the Third License Agreement (as amended, the “Third Cornell License Agreement,” together with the May 2020 Cornell License Agreements, the “Cornell License Agreements”) with Cornell. Pursuant to the Third Cornell License Agreement, Cornell has granted the Company an exclusive license to practice under certain patent rights generated in animal studies conducted by Cornell on behalf of the Company and a non-exclusive license to know-how concerning a gene therapy for FA cardiomyopathy and current and future data generated in an ongoing investigator-initiated Phase 1A trial of AAVrh.10hFXN to treat FA cardiomyopathy. Both licenses are worldwide and cover products with human and non-human prophylactic and therapeutic uses. Cornell has also granted the Company a right of reference to Cornell’s Investigational New Drug application for a gene therapy for FA cardiomyopathy. Under the Third Cornell License Agreement, the Company paid a license issue fee and an initial data transfer fee to Cornell totaling $0.6 million. Additionally, the Company will be paying an annual data transfer fee of $50,000 until data is no longer being gathered. The Company has agreed to pay annual license maintenance fees ranging from $2,500 to $25,000 until such time the Company commercializes a licensed product. In addition, the Company will pay Cornell up to an aggregate of $2.1 million in regulatory milestones and up to an aggregate of $100 million in commercial milestones, plus low single digit royalties on net sales. The Third Cornell License Agreement contains other customary license terms including terms related to sublicensing, development, commercialization, milestones, royalties, intellectual property, and termination. Upon expiration of the applicable royalty term for a product in a given country, the Company shall retain a non-exclusive, royalty free license to the data and know-how, including to continue selling such product in that country. Cornell may terminate the Third Cornell License Agreement if the Company (i) breaches the Third Cornell License Agreement (subject to a cure period), (ii) participates in any proceeding challenging the validity of the licensed patents, (iii) publishes the licensed data without Cornell’s prior written consent, or (iv) does not reach certain milestones. Cornell may also terminate the Third Cornell License Agreement in part on a product-by-product basis if the Company does not diligently develop and sell a product. The Company may terminate the Third Cornell License Agreement, in whole or in part with respect to the right of reference, or the licensed data, know-how, or patent rights, with 90 days’ prior written notice to Cornell. On December 17, 2025, the Company executed a Second Amendment to the Third Cornell License Agreement (the “Second Amendment to the Third Cornell License Agreement”), which added a one-time fee of $0.1 million for additional data transfer and processing. During the three months ended March 31, 2026, the Company incurred $0.4 million of research and development costs and paid $0 in connection with the Cornell License Agreements. During the three months ended March 31, 2025, the Company incurred $30,000 of research and development costs and paid $0 in connection with the Cornell License Agreements. Stelios Therapeutics, Inc.—Stelios Therapeutics, Inc. (“Stelios”) was an early-stage company developing novel adeno-associated AAV-based gene therapies for rare cardiac conditions including arrhythmogenic cardiomyopathy and TNNI3-associated hypertrophic cardiomyopathy. On July 16, 2021, the Company acquired 100% of the outstanding stock of Stelios that was accounted for as an asset acquisition pursuant to FASB ASC 805, Business Combinations. The Company is required to pay up to a remaining aggregate of $12.5 million to the selling shareholders of Stelios upon the achievement of certain development milestones, excluding a $6.0 million development milestone that was achieved in the third quarter of 2024 and paid in the fourth quarter of 2024. Dr. Eric Adler, the Company's former Chief Medical Officer and Head of Research, was a co-founder of Stelios and a selling shareholder. Of the $6.0 million development milestone payment, Dr. Adler received approximately $1.3 million. Stelios was merged into the Company on December 15, 2022 and ceased to exist. The Regents of the University of California, San Diego—Stelios entered into an exclusive worldwide license agreement on April 23, 2020 (as amended, the “First UCSD Agreement”) with The Regents of UCSD to in-license materials and intellectual property related to a gene therapy for arrhythmogenic right ventricular cardiomyopathy. The First UCSD Agreement relates to the Company’s development efforts for its LX2021 program. In connection with the First UCSD Agreement, the Company gained access to inventions, patent rights, technology, and licensed methods that it continues to develop, and it has assumed all worldwide development and commercialization activities with respect to the licensed technology. The First UCSD Agreement required Stelios to pay one-time up-front non-refundable cash fees of $20,000 for each agreement and requires the Company to pay aggregate development and commercialization milestones of up to $4.8 million, and low- to mid-single digit royalties and low-single digit royalties based on aggregate net sales. The only research and development expense incurred by Stelios or the Company, and payment made to The Regents of UCSD through December 31, 2025 under the terms of the First UCSD Agreement, was the one-time up-front non-refundable cash fee of $20,000. The Company has the right to terminate the First UCSD Agreement at any time upon sixty (60)-days’ written notice to The Regents of UCSD. Stelios entered into an exclusive worldwide license agreement on August 6, 2020 (as amended, the “Second UCSD Agreement”) with The Regents of UCSD to in-license materials and intellectual property related to a gene therapy for hypertrophic cardiomyopathy. On November 14, 2025, the Company entered into an amended and restated license agreement with UCSD, which amended and restated in its entirety, the Second UCSD Agreement (the "Amended and Restated Second UCSD Agreement"). The Amended and Restated Second UCSD Agreement maintained the Company's prior exclusive worldwide license to materials and intellectual property related to a gene therapy for hypertrophic cardiomyopathy, replaced Stelios with the Company as licensee, and exclusively in-licensed additional materials and intellectual property related to the LX2022 program. Stelios previously paid a one-time up-front non-refundable cash fee of $20,000 under the Second UCSD Agreement. The Amended and Restated Second UCSD Agreement requires the Company to pay aggregate development and commercialization milestones of up to $2.4 million, and low-single digit royalties based on aggregate net sales. The only research and development expense incurred by Stelios or the Company, and payment made to The Regents of UCSD through March 31, 2026 under the terms of the Second UCSD Agreement and the Amended and Restated Second UCSD Agreement, was the one-time up-front non-refundable cash fee of $20,000. The Company has the right to terminate the Amended and Restated Second UCSD Agreement at any time upon sixty (60)-days’ written notice to The Regents of UCSD. On October 4, 2021, the Company entered into an exclusive worldwide license agreement (as amended, the “Third UCSD Agreement” and collectively with the First UCSD Agreement and the Amended and Restated Second UCSD Agreement, the “UCSD Agreements”) with The Regents of UCSD to in-license materials and intellectual property related to LX2020, a gene therapy for arrhythmogenic right ventricular cardiomyopathy. The Third UCSD Agreement relates to the Company’s development efforts for its LX2020 program. In connection with the Third UCSD Agreement, the Company gained access to inventions, patent rights, technology, and licensed methods that it continues to develop, and it has assumed all worldwide development and commercialization activities with respect to the licensed technology. The Third UCSD Agreement required the Company to pay a one-time up-front non-refundable cash fee of $20,000 and requires the Company to pay aggregate development and commercialization milestones of up to $4.0 million, and low- to mid-single digit royalties based on aggregate net sales. Research and development expenses incurred by the Company and payments made to The Regents of UCSD under the terms of the Third UCSD Agreement include the one-time up-front non-refundable cash fee of $20,000 as well as a $0.1 million development milestone that was achieved during the third quarter of 2024. The Company has the right to terminate the Third UCSD Agreement at any time upon sixty (60)-days’ written notice to The Regents of UCSD. On December 3, 2021, the Company entered into two sponsored research agreements with The Regents of UCSD (the "First UCSD SRA", the "Second UCSD SRA", and collectively, the “Initial UCSD SRAs”) for the Company’s LX2020, LX2021 and LX2022 programs in connection with the UCSD Agreements. Under the terms of the Initial UCSD SRAs, the Company has the first rights to obtain non-exclusive or exclusive, sublicensable, royalty-bearing, perpetual and transferable worldwide licenses in any resulting inventions owned by The Regents of UCSD or resulting inventions jointly owned between the Company and The Regents of UCSD, and the Company retains the rights to any resulting inventions owned by the Company. The Initial UCSD SRAs each have a two-year term and may be terminated early by the Company at any time upon the giving of thirty (30)-days’ written notice to The Regents of UCSD. The total costs to be invoiced to the Company over the terms of the Initial UCSD SRAs are $5.6 million. On April 13, 2024, the Company entered into a third sponsored research agreement with The Regents of UCSD (as amended, the “Third UCSD SRA”) for the Company’s LX2022 program in connection with the Second UCSD Agreement. Under the terms of the Third UCSD SRA, the Company has the first rights to obtain non-exclusive or exclusive, sublicensable, royalty-bearing, perpetual and transferable worldwide licenses in any resulting inventions owned by The Regents of UCSD or resulting inventions jointly owned between the Company and The Regents of UCSD, and the Company retains the rights to any resulting inventions owned by the Company. The Third UCSD SRA has a two-year term and may be terminated early by the Company at any time upon the giving of thirty (30)-days’ written notice to The Regents of UCSD. The costs to be invoiced to the Company over the term of the Third UCSD SRA are $0.7 million, and the Company may incur additional costs of $0.6 million under the Third UCSD SRA if certain study objectives are met. On April 13, 2024, the Company also entered into an amendment to the Second UCSD SRA (as amended, the "Amended Second UCSD SRA") for the Company’s LX2022 program that extended the term of the Second UCSD SRA to December 2024. On each of April 19, 2024 and September 27, 2024, the Company also entered into an amendment to the First UCSD SRA (as amended, the "Amended First UCSD SRA", and together with the Amended Second UCSD SRA and the Third UCSD SRA, as amended, the "UCSD SRAs") for the Company’s LX2021 program in connection with the First UCSD Agreement. The Amended First UCSD SRA extends the term of the First UCSD SRA to December 2026 and provides for additional research and development studies and expenses. The total costs to be invoiced to the Company under the Amended First UCSD SRA are $0.7 million. During the three months ended March 31, 2026, the Company did not incur any research and development expenses and paid $0.5 million to The Regents of UCSD in connection with the Amended UCSD SRAs. During the three months ended March 31, 2025, the Company incurred $34,000 of research and development expenses and paid $0.1 million to The Regents of UCSD in connection with the UCSD SRAs. The Company has incurred and paid cumulative totals of $5.7 million to The Regents of UCSD as of March 31, 2026, in connection with the UCSD SRAs. Myoventive, Inc.—In June 2025, the Company entered into a Collaboration and License Agreement with Tilia Therapeutics, Inc., now known as Myoventive, Inc. ("Myoventive" and the "Myoventive Agreement"), to develop therapies for genetic cardiac diseases utilizing a novel non-viral RNA platform. Under the terms of the Myoventive Agreement, the Company contributed expertise in developing cardiac genetic medicines, certain existing preclinical intellectual property, and technology, which will be combined with a novel non-viral delivery platform. As consideration for its contributions, the Company received a double-digit percentage equity position in Myoventive and will be entitled to future milestone payments, royalties, and opt-in rights to certain program(s). Abiomed, Inc. (Johnson & Johnson subsidiary)—In December 2025, the Company entered into a Collaboration Agreement with Abiomed, Inc. ("Abiomed" and the "Abiomed Agreement"), to investigate localized cardiac delivery of gene therapy. Under the Abiomed Agreement, the parties intend to advance the potential efficacy and safety profile of gene therapy for genetically mediated cardiovascular diseases by concentrating AAV-vector delivery to the heart by investigating cutting-edge routes of administration using the ImpellaTM heart pump technology. The Company did not incur or pay any research and development costs in connection with the Abiomed Agreement for the three months ended March 31, 2026. |