On January 9, 2023 SpringWorks Therapeutics, Inc. (Nasdaq: SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, will present today at the 41st Annual J.P. Morgan Healthcare Conference at 7:30 a.m. PT (10:30 a.m. ET), and a live webcast will be available at ir.springworkstx.com. Ahead of the presentation, the Company highlighted its 2022 accomplishments and reported its anticipated key milestones for 2023 (Press release, SpringWorks Therapeutics, JAN 9, 2023, View Source [SID1234626110]).

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2022 Accomplishments

Presented positive Phase 3 DeFi data of nirogacestat, an investigational oral gamma secretase inhibitor, in adult patients with desmoid tumors at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress.
Submitted a New Drug Application (NDA) to the United States Food and Drug Administration (FDA) for nirogacestat for the treatment of adults with desmoid tumors, which is being reviewed under the FDA’s Real-Time Oncology Review (RTOR) program.

Expanded and strengthened its intellectual property portfolio, including a new patent issued by the United States Patent and Trademark Office in the fourth quarter of 2022 directed to pharmaceutical compositions of nirogacestat (U.S. Patent No. 11,504,354), which expires in 2042 and represents what is expected to be the seventh Orange Book-listable patent for nirogacestat.

Participated in a Type C meeting with the FDA to align on the statistical analysis plan for the Phase 2b ReNeu trial evaluating mirdametinib, an investigational MEK inhibitor, in adult and pediatric patients with NF1-associated plexiform neurofibromas (NF1-PN) and expectations for an anticipated NDA submission in this indication.

Initiated a Phase 2 trial evaluating nirogacestat in patients with ovarian granulosa cell tumors (OvGCT).
Expanded global, non-exclusive license and collaboration agreement with GSK plc (GSK) to evaluate nirogacestat in combination with belantamab mafodotin in patients with multiple myeloma, including in earlier lines of treatment.

Demonstrated clinical proof of concept for BGB-3245, a selective RAF dimer inhibitor being developed by MapKure, LLC, a joint venture between SpringWorks and BeiGene, as well as for the combination of mirdametinib and BeiGene’s RAF dimer inhibitor, lifirafenib.

Advanced BGB-3245 monotherapy into dose expansion cohorts and announced plans for a new Phase 1/2a study evaluating BGB-3245 in combination with mirdametinib.
Nominated TEAD inhibitor development candidate (SW-682).

Strengthened financial position to over $650 million in cash, cash equivalents and marketable securities, as of September 30, 2022, which is expected to provide runway into 2026.

Established commercial infrastructure to support anticipated first U.S. product launch for nirogacestat in patients with desmoid tumors.

Anticipated 2023 Key Milestones

Obtain regulatory approval from the FDA for nirogacestat in adults with desmoid tumors and launch nirogacestat as the first approved therapy for these patients in the second half of 2023.

Highlight publication of Phase 3 DeFi trial data in a peer-reviewed journal and present additional analyses from the study at upcoming medical meetings.

Present topline data from the pediatric and adult cohorts of the Phase 2b ReNeu trial in NF1-PN in the second half of 2023. Pending these data, SpringWorks anticipates submitting an NDA for mirdametinib as a treatment for NF1-PN in the first half of 2024.

Continue enrollment in the Phase 2 trial of nirogacestat in patients with OvGCT.

Expand data set with additional clinical data of nirogacestat in combination with BCMA-directed therapies and initiate additional planned Phase 1 collaborator studies.

Present additional clinical data from the Phase 1 trial evaluating BGB-3245 in adult patients with RAF mutant solid tumors at a medical conference in the first half of 2023.

Present additional data from BeiGene-sponsored Phase 1b/2 trial evaluating mirdametinib in combination with lifirafenib in adult patients with RAS/RAF mutant and other MAPK pathway aberrant solid tumors at a medical conference in the first half of 2023.

Dose first patient in BGB-3245 + mirdametinib combination study in MAPK-mutant solid tumors in the first half of 2023.
File Investigational New Drug Application for TEAD inhibitor SW-682.
"We expect 2023 to not only mark SpringWorks’ transition into a commercial-stage company, but also to be a year of continued development execution and value-creating data readouts across our portfolio," said Saqib Islam, Chief Executive Officer of SpringWorks. "We are very pleased with our progress towards our goal of having two marketed products by 2025, given the overwhelmingly positive Phase 3 DeFi data for nirogacestat in desmoid tumors that were disclosed last year and our expectations for a successful topline readout for the Phase 2b ReNeu trial in children and adults with NF1-PN. Our strong execution in 2022, coupled with a financial position that funds us well into 2026 and durable IP protections for our lead assets, positions us well for continued performance on behalf of patients with devastating diseases."

Presentation at the 41st Annual J.P. Morgan Healthcare Conference
SpringWorks will webcast its presentation from the 41st Annual J.P. Morgan Healthcare Conference today, Monday, January 9, 2023 at 7:30 a.m. PT (10:30 a.m. ET). To access the live webcast, please visit the Events & Presentations page within the Investors & Media section of the company’s website at View Source A replay of the webcast will be available on SpringWorks’ website for a limited time following the conference.

On January 9, 2023 Sutro biopharma presented its corporate presentation (Presentation, Sutro Biopharma, JAN 9, 2023, View Source [SID1234626111]).

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On January 9, 2023 Sonnet BioTherapeutics Holdings, Inc. (NASDAQ:SONN), a clinical-stage company developing targeted immunotherapeutic drugs, reported a clinical collaboration agreement with Roche (Press release, Sonnet BioTherapeutics, JAN 9, 2023, View Source [SID1234626109]). A clinical trial (SB221) will be conducted to assess the safety and preliminary efficacy of SON-1010 (IL12-FHAB) in combination with Roche’s atezolizumab in patients with platinum-resistant ovarian cancer (PROC). Interleukin-12 (IL-12) is a cytokine, or an immune cell-signaling protein, that enhances the activity of natural killer (NK) cells and T cells. SON-1010 is a proprietary version of native human IL-12, configured using Sonnet’s fully human albumin binding (FHAB) platform, which targets the tumor microenvironment (TME) and extends the pharmacokinetics (PK) and subsequent pharmacodynamics (PD) of the molecule. Atezolizumab is an immune checkpoint inhibitor approved for the treatment of some of the most aggressive and difficult-to-treat forms of cancer. The characteristics of ovarian cancer present a unique opportunity to assess the combination of these two agents in an indication that persists as a large unmet medical need.

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"As this is Sonnet’s first combination clinical study and an opportunity to use our lead FHAB-derived candidate, SON-1010, with atezolizumab, it is a very important milestone for the company. We believe that the combination of our best-in-class IL12-FHAB immune-enhancer candidate with atezolizumab could enable the next generation of cancer treatment." said Pankaj Mohan, Ph.D., Sonnet’s Founder and Chief Executive Officer. "We anticipate initiating the clinical study during the second calendar quarter of 2023 and will look to a successful safety evaluation for the opportunity to expand the collaboration".

Sonnet and Roche have entered into a Master Clinical Trial and Supply Agreement (MCSA), along with ancillary Quality and Safety Agreements, to study the safety and efficacy of the combination of SON-1010 and atezolizumab in a platinum-resistant ovarian cancer (PROC) patient setting. Further, the companies would provide SON-1010 and atezolizumab, respectively, for use in the Phase 1b/Phase 2a safety and efficacy study.

"The extended PK of SON-1010, along with its ability to target and be retained within the TME, makes it a potentially best-in-class version of IL-12", said Richard Kenney, M.D., Sonnet’s Chief Medical Officer. "Ovarian cancer has a high expression of proteins that bind albumin, which will concentrate SON-1010 in the TME. The induced immune responses can make this relatively ‘cold’ tumor immunologically ‘hot’. Ovarian cancer patients who are resistant to platinum compounds have very few options for successful treatment. This combination provides a novel alternative that may improve their rate of response."

SB221 is a global Phase 1b/2a multicenter, dose-escalation and randomized proof-of-concept study to assess the safety, tolerability, PK, PD, and efficacy of SON-1010 administered subcutaneously (SC), either alone or in combination with atezolizumab given intravenously (IV). The study is designed in Part 1 to rapidly establish the maximum tolerated dose (MTD) of the combination in patients with advanced solid tumors in small dose-escalation groups and to expand the dataset at the recommended Phase 2 dose (RP2D). This would be followed in Part 2 by an assessment in patients with PROC of the potential for improved efficacy of the combination over SON-1010 alone or the standard of care. Both companies look forward to this collaboration as an opportunity to improve outcomes for patients with ovarian cancer.

On January 9 2023 Selecta Biosciences, Inc. (NASDAQ: SELB), a biotechnology company leveraging its clinically validated ImmTOR platform to develop tolerogenic therapies for autoimmune diseases and gene therapies, reported a corporate update, including its roadmap for 2023 (Press release, Selecta Biosciences, JAN 9, 2023, View Source [SID1234626107]).

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Key 2023 Anticipated Milestones

Report top-line data from Phase 3 DISSOLVE I & II programs of SEL-212 in chronic refractory gout in Q1 2023
Preliminary Phase 1 SEL-302 data in gene therapy for MMA
Initiate IND enabling studies with the selected IL-2 candidate to further advance and expand the immune tolerance platform in autoimmune disease
Begin IND enabling studies with the selected IgA protease candidate from IGAN Biosciences
"In 2022, we delivered on key milestones that further validated the value and breadth of our innovative ImmTOR and ImmTOR-IL immune tolerance platforms, continued to advance our diversified clinical pipeline and established strategic collaborations that will propel our next-generation programs toward multiple IND filings," said Carsten Brunn, Ph.D., President and Chief Executive Officer of Selecta. "Building on the momentum of our recently announced deal with Astellas Gene Therapies’ for Xork in Pompe disease, the initiation of the Phase 1/2 trial in methylmalonic acidemia, the identification of an IL-2 candidate and selection of an IgA protease candidate, we also expect joint topline data from the Phase 3 DISSOLVE clinical program investigating SEL-212 in chronic refractory gout in Q1 2023. We are at a pivotal moment in the Company’s growth trajectory and as we look ahead, we believe we are well positioned to take a potentially generational leap forward for our precision immune tolerance platform, advance our pipeline in autoimmune disease and continue to explore additional collaborations to maximize the value of our ImmTOR platform and pipeline."

Clinical Development Overview

Tolerogenic Therapies for Autoimmune Disease:

ImmTOR-IL: In December 2022, the Company opted into an agreement for an identified IL-2 candidate and is currently negotiating the terms of the license. The IL-2 candidate will be studied in combination with ImmTOR to further advance and expand the pipeline in autoimmune disease. The combination of ImmTOR and IL-2 (ImmTOR-IL) represents an evolution of the precision immune tolerance platform to further enhance the magnitude and duration of antigen-specific immune tolerance for the treatment of patients with autoimmune diseases.

The Company plans to initiate IND enabling studies in 2023 while also exploring multiple autoimmune indications that would be suitable for study with ImmTOR-IL.

Gene Therapies:

SEL-302 for Methylmalonic Acidemia (MMA): In December 2022, Selecta initiated ReiMMAgine, the Phase 1/2 clinical trial of SEL-302, an adeno-associated virus (AAV) gene therapy combined with ImmTOR for the treatment of MMA.

The ReiMMAgine trial is now enrolling patients and aims to evaluate the safety, tolerability and efficacy of SEL-302, a combination of ImmTOR and AAV gene therapy.
SEL-018 IgG Protease (Xork) for Pompe Disease: In January 2023, the Company announced an exclusive licensing and development agreement for IdeXork (Xork), a next-generation immunoglobulin G (IgG) protease, to be developed for use with AT845, Astellas Gene Therapies’ investigational AAV-based treatment for Late-Onset Pompe disease (LOPD) in adults.

Xork has the potential to expand access of life-changing gene therapies to more patients by addressing pre-existing immunity to AAV. Xork is differentiated by its low cross reactivity to pre-existing antibodies in human serum.
Under the terms of the agreement, Selecta will receive a $10M upfront payment and is eligible to receive up to $340M for certain additional development and commercial milestones plus royalties on commercial sales. Selecta is responsible for the early development activities and manufacturing of Xork and will maintain the rights for the development of additional indications beyond Pompe disease.

Biologic Therapies:

SEL-212 for chronic refractory gout: DISSOLVE, the Phase 3 development program of SEL-212, which has been licensed to Sobi continues to advance. DISSOLVE I & II trials are on track for joint topline data expected in Q1 2023.

ImmTOR with IgA Protease for IgA Nephropathy: In December 2022, the Company selected the next generation Immunoglobulin A (IgA) protease from IGAN Biosciences for the treatment of IgAN.

Identified a new class of IgA protease from commensal bacteria with a lower level of baseline anti-drug antibodies (ADAs). Combining ImmTOR with this next generation IgA protease candidate has the potential to mitigate the formation of new ADAs and address the underlying pathophysiology of IgAN.

Further Corporate Updates:

In November 2022, Blaine Davis was appointed as Chief Financial Officer. Mr. Davis brings more than 25 years of experience in investor relations, business development, corporate affairs and sales and marketing at life sciences companies, with a particular focus on rare diseases.

On January 9, 2023 Seres therapeutics presented its corporate presentation (Presentation, Seres Therapeutics, JAN 9, 2023, View Source [SID1234626108]).

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