Omeros Corporation Reports Second Quarter 2024 Financial Results

On August 7, 2024 Omeros Corporation (Nasdaq: OMER), a clinical-stage biopharmaceutical company committed to discovering, developing and commercializing small-molecule and protein therapeutics for large-market and orphan indications targeting immunologic disorders including complement-mediated diseases, as well as cancers and addictive and compulsive disorders, reported recent highlights and developments as well as financial results for the second quarter ended June 30, 2024, which include (Press release, Omeros, AUG 7, 2024, View Source [SID1234645499]):

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● Net loss for the second quarter of 2024 was $56.0 million, or $0.97 per share, compared to a net loss of $37.3 million, or $0.59 per share for the second quarter of 2023. For the six months ended June 30, 2024, our net loss was $93.2 million, or $1.60 per share, compared to a net loss of $71.0 million, or $1.13 per share in the prior year period. The second quarter of 2024 includes a $17.6 million charge for narsoplimab drug substance delivered during the quarter, the manufacturing of which commenced in October 2023, a $21.2 million payment for term loan-related debt repurchase, and $1.9 million of term loan-related transaction costs. These significant cash outlays, representing a total of $40.7 million dollars, are not expected to be repeated in the foreseeable future.

● On June 3, 2024, we entered into a Credit and Guaranty Agreement (the "Credit Agreement") with funds managed by Athyrium Capital Management (collectively, "Athyrium") and funds managed by Highbridge Capital Management (collectively, "Highbridge") as lenders (the "Lenders"). Under the Credit Agreement, we entered into an initial senior secured term loan of $67.1 million (the "Initial Term Loan") and paid $21.2 million to the Lenders in exchange for $118.1 million aggregate principal amount of Omeros’ existing 5.25 percent convertible senior notes due on February 15, 2026 (the "2026 Notes") held by the Lenders and representing 55 percent of our total 2026 Notes. The Credit Agreement also provides for a $25.0 million delayed draw term loan available to be drawn on request by Omeros on or prior to June 3, 2025 contingent on regulatory approval of narsoplimab in hematopoietic stem cell transplant-associated thrombotic microangiopathy ("TA-TMA"). All loans under the Credit Agreement have a stated maturity date of June 3, 2028.

ο The purchase price in the debt exchange and repurchase of our outstanding 2026 Notes represents a discount from notional value of approximately 25 percent.

ο Neither the Initial Term Loan nor the delayed draw term loan includes equity consideration for the Lenders, preventing any shareholder dilution as a consequence of these transactions.

ο After giving effect to the repurchase, we had approximately $98 million principal amount of our 2026 Notes outstanding.

● At June 30, 2024, we had $158.9 million of cash and short-term investments available for operations and debt servicing, a decrease of $12.9 million from December 31, 2023.

● As previously disclosed, we submitted an analysis plan to assess our existing clinical trial data along with other evidence proposed to be included in a resubmission of our biologics license application ("BLA") for narsoplimab in TA-TMA. We are engaged in ongoing discussions with the FDA regarding the analysis plan and the other evidence proposed to be included in the submission. An additional meeting with FDA has been scheduled and we expect to provide a further update on our plans for resubmission and anticipated timing when more definitive information becomes available.

● We continued advancing our lead MASP-3 inhibitor antibody zaltenibart (also known as OMS906) through a Phase 2 development program in paroxysmal nocturnal hemoglobinuria ("PNH") comprised of two fully enrolled clinical trials and a long-term extension study in which patients who have completed either of the first two studies are eligible to enroll. Patients continue to accrue to the extension study and we remain on track to initiate our Phase 3 program for zaltenibart in PNH later this year.

● Enrollment is ongoing in our Phase 2 clinical trial evaluating zaltenibart for the treatment of complement 3 glomerulopathy ("C3G"). A Phase 3 program in C3G is planned to begin in early 2025.

"Throughout the second quarter, we continued rapidly progressing our clinical programs while significantly strengthening our balance sheet," said Gregory A. Demopulos, M.D., Omeros’ chairman and chief executive officer. "Through the term loan and note repurchase transaction completed in June, we reduced by more than half the outstanding balance of our 2026 convertible notes at a substantial discount to par value without diluting shareholders. With the new secured term loan in place, a substantial portion of our outstanding debt is now maturing in 2028, the Company is well positioned to address the remaining balance of our 2026 convertible notes, and we have access to an additional term loan of up to $25 million to fund the commercial launch of narsoplimab in TA-TMA. Although the lengthy regulatory process is a continuing source of frustration for our team, our shareholders and, most especially, the TA-TMA patients in need of an effective treatment for this often-lethal condition, we believe that the evidence we have proposed to submit with our BLA is highly compelling and we remain dedicated to making narsoplimab the first approved product for the treatment of TA-TMA. We look forward to providing a further update on the outcome of our ongoing discussions with FDA. In parallel, our MASP-3 inhibitor zaltenibart continues to advance rapidly, generating consistently – and compared to other marketed and developing alternative pathway inhibitors – strong data, and it remains on track to initiate a Phase 3 program in PNH later this year and, in C3G, early in 2025."

Second Quarter and Recent Clinical Developments

● Recent developments regarding narsoplimab, our lead monoclonal antibody targeting mannan-binding lectin-associated serine protease-2 ("MASP-2"), include the following:

o We previously submitted to FDA an analysis plan to assess already existing clinical trial data, existing data from an historical control population available from an external source, data from the narsoplimab expanded access (i.e., compassionate use) program, and data directed to the mechanism of action of narsoplimab. We are having ongoing discussions with the agency regarding the proposed analysis plan and FDA’s requirements for our resubmission of our BLA. An additional meeting with FDA has been scheduled and we expect to provide a further update on our plans for resubmission and the anticipated timing when more definitive information becomes available.

o FDA recently announced the establishment of the Rare Disease Innovation Hub, which will serve as a single point of connection and engagement within the FDA to support the development of treatments and products for rare diseases. The hub will have a particular focus on products intended for smaller populations or for diseases where the natural history is variable and not fully understood. FDA’s focus on these issues and their responsiveness to the rare disease community’s advocacy is encouraging, and we view the establishment of the hub as a tangible demonstration of FDA’s commitment to recognizing and addressing the unique challenges faced in developing therapies for these conditions.

ο In addition to previous publications on narsoplimab in TA-TMA, international transplant experts are preparing two manuscripts – one directed to the results of a survival comparison between our pivotal trial of narsoplimab in TA-TMA and an external control population of TA-TMA patients and the second detailing the survival data obtained from narsoplimab treatment of TA-TMA patients in our expanded access program. Physicians continue to request access to narsoplimab under this program for their patients with TA-TMA. Given that there is no approved treatment for this life-threatening condition, we continue to do what we can to help these patients.

● Recent developments regarding OMS1029, our long-acting, next-generation MASP-2 inhibitor, include:

o Both the single- and multiple-ascending-dose Phase 1 studies of OMS1029 have now been completed. The results support once-quarterly dosing, administered either subcutaneously or intravenously. Data from the multiple-ascending-dose study will be utilized to inform dose selection for continued clinical development. Consistent with the results of the single-ascending-dose Phase 1 clinical trial of OMS1029 completed in early 2023, OMS1029 was generally well tolerated at all doses evaluated in the multiple-ascending-dose study, with no significant safety concern identified to date.

o We continue to evaluate large market indications for Phase 2 clinical development of OMS1029. These include neovascular age-related macular degeneration, sometimes referred to as "wet AMD." MASP-2 inhibition previously showed efficacy in a pre-clinical murine model of wet AMD and we are currently engaged in a primate study comparing OMS1029 to Eylea (afibercept), a product currently approved to treat wet AMD. If shown to be effective, OMS1029 administered systemically (e.g., either intravenously or subcutaneously) could represent a significantly more attractive treatment experience compared to Eylea and other currently approved treatments for wet AMD, which require frequent injections directly into the posterior chamber of the eye.

● Recent developments regarding OMS906, our lead monoclonal antibody targeting mannan-binding lectin-associated serine protease-3 ("MASP-3"), the key activator of the alternative pathway, include:

o The United States Adopted Names Council ("USAN"), in consultation with the World Health Organization’s International Nonproprietary Names Expert Committee ("INN"), recently selected the nonproprietary name "zaltenibart" for OMS906. The USAN Council, by working closely with the INN Programme of the World Health Organization and various national nomenclature groups, aims for global standardization and unification of drug nomenclature to ensure that drug information is communicated accurately and unambiguously. Going forward, we will use the name zaltenibart to refer to our lead MASP-3 antibody in publications, at conferences and across other forums.

o Our Phase 2 trial evaluating two doses of zaltenibart in PNH patients who have had an unsatisfactory response to the C5 inhibitor ravulizumab has continued to produce encouraging data. The study utilizes a "switch-over" design, enrolling PNH patients who are receiving ravulizumab and adding zaltenibart to provide combination therapy with ravulizumab for 24 weeks. Those patients who demonstrate a hemoglobin response with the combination therapy are then switched to zaltenibart monotherapy. In June, at the annual congress of the European Hematology Association (EHA) (Free EHA Whitepaper), interim analysis results from the combination therapy portion of the trial were presented by Dr. Morag Griffin, an internationally recognized PNH expert from St. James University Hospital in England. During the adjunctive therapy period, the statistically significant mean hemoglobin improvement from baseline was 3.27 g/dL and 10 of 12 patients advanced to monotherapy. Absolute reticulocyte count also demonstrated statistically significant improvement. Zaltenibart was safe and well tolerated. An abstract providing results of the zaltenibart monotherapy stage has been submitted to the American Society of Hematology (ASH) (Free ASH Whitepaper) for presentation at their annual meeting in December. The efficacy and safety profiles of zaltenibart as monotherapy remain strong, including demonstration of sustained and clinically meaningful improvements in hemoglobin levels and absolute reticulocyte counts, as well as prevention of both extravascular and intravascular hemolysis.

o Our Phase 2 study of zaltenibart in PNH patients who have not previously received treatment with a complement inhibitor (i.e., naïve patients) is also ongoing and continues to progress well. Results from an interim analysis of subcutaneous zaltenibart treatment were presented at the American Society of Hematology (ASH) (Free ASH Whitepaper) meeting held in December 2023. Following that presentation, we amended the study protocol to identify the plasma concentrations and the level of MASP-3 inhibition required to inhibit breakthrough hemolysis. These data, in combination with data derived from our "switch-over" PNH study and from our Phase 1 studies in healthy subjects, are expected to provide all the data needed to finalize selection of the zaltenibart dose for our upcoming Phase 3 clinical trials.

o We plan to conduct two trials in our Phase 3 program for zaltenibart in PNH. Similar to our Phase 2 program, one will enroll complement inhibitor-naïve patients and the other will employ a "switch over" design. Through our recent advisory boards with experts in PNH and focus-group PNH patients, we have received valuable input to inform the design of our Phase 3 studies and our positioning of zaltenibart in the marketplace, if approved. The zaltenibart drug substance necessary to supply our Phase 3 clinical trials has been manufactured, upcoming pre-Phase 3 meetings with both European and U.S. regulators have been scheduled or requested, and the other activities required to initiate our Phase 3 program have also been completed or are progressing as planned. We remain on track to initiate the program later this year.

● Recent developments regarding OMS527, our phosphodiesterase 7 ("PDE7") inhibitor program focused on addictions and compulsive disorders as well as movement disorders, include:

o In April 2023 we were awarded a three-year, $6.69 million grant by the National Institute on Drug Abuse ("NIDA") to pursue development of our lead orally administered PDE7 inhibitor compound for the treatment of cocaine use disorder ("CUD"). We expect to complete by the end of this year a grant-funded preclinical cocaine interaction study, which is a safety prerequisite to initiation of the randomized, placebo-controlled, inpatient clinical study evaluating the safety and effectiveness of OMS527 in patients with CUD, which is also contemplated to be funded with proceeds of the NIDA award.

Agenus Publishes Seminal Study on Botensilimab’s Activity in Treatment-Resistant Cancers

On August 7, 2024 Agenus Inc. (NASDAQ: AGEN), a leader in developing novel immunological agents to treat various cancers, reported the publication of a seminal study in the prestigious Cancer Discovery, a journal of the American Association for Cancer Research (AACR) (Free AACR Whitepaper), detailing the novel mechanism of action and effectiveness of botensilimab, an investigational, novel multifunctional anti-CTLA-4 antibody, in various treatment-resistant cancers (Press release, Agenus, AUG 7, 2024, View Source [SID1234645521]).

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The paper, entitled "Botensilimab, an Fc-Enhanced Anti-CTLA-4 Antibody, Is Effective Against Tumors Poorly Responsive to Conventional Immunotherapy," highlights several key findings:

Demonstrated Activity Across Multiple Cancers: Botensilimab has shown increased activity in multiple treatment-resistant cancers, including those that have progressed on prior checkpoint inhibitors.
Fc-Enhanced Design for Multifunctional Immune Activation: Unlike traditional anti-CTLA-4 antibodies, botensilimab’s Fc-enhanced design allows it to leverage multiple immune-activating mechanisms simultaneously. This includes enhanced T cell priming, reduction of intratumoral regulatory T cells, and activation of antigen-presenting cells, leading to a robust anti-tumor response.
Activity Independent of Conventional Limitations: Botensilimab shows clinical activity regardless of factors that typically limit conventional immunotherapy efficacy, such as tumor neoantigen burden and FcγRIIIA genotype. This broadens its potential applicability across diverse patient populations.
Remodeling the Tumor Microenvironment: The antibody uniquely remodels the tumor microenvironment, transforming "cold" tumors that are currently unresponsive to immune therapies into "hot" immunologically active tumors. This is achieved by reducing regulatory T cells and increasing T cell inflammation gene signatures within the tumor microenvironment.
Promise in Difficult-to-Treat Cancers: Botensilimab demonstrates significant promise in treating over nine difficult-to-treat cancers, including microsatellite stable colorectal cancer and may extend clinical benefits to patient populations historically unresponsive to conventional immune checkpoint inhibitors.
"We are thrilled to share these groundbreaking findings with the scientific community," said Dhan Chand, PhD, lead author and Vice President of Research at Agenus. "The compelling preclinical and clinical evidence generated with botensilimab reveal an actionable pathway to improve treatment outcomes and extend survival to patient populations historically unresponsive to conventional immune checkpoint inhibitors."

This paper underscores the potential of botensilimab to overcome some of the most challenging hurdles in cancer treatment, offering new hope to patients with limited options. Agenus is committed to advancing this promising therapy through further clinical development and regulatory pathways to ensure rapid and broad patient access.

About Cancer Discovery

Cancer Discovery publishes high-impact, peer-reviewed articles describing major advances in research and clinical trials. As the premier cancer information resource, the Journal also presents Review Articles, Perspectives and Commentaries, News stories, and Research Watch summaries of important journal articles to its readers to keep them informed about the latest findings in the field. Topics span the spectrum of cancer research and medicine from the laboratory to the clinic and epidemiologic studies.

About Botensilimab

Botensilimab is an investigational human Fc-enhanced CTLA-4 blocking antibody designed to boost both innate and adaptive anti-tumor immune responses. Its novel design leverages mechanisms of action to extend immunotherapy benefits to "cold" tumors which generally respond poorly to standard of care or are refractory to conventional PD-1/CTLA-4 therapies and investigational therapies. Botensilimab augments immune responses across a wide range of tumor types by priming and activating T cells, downregulating intratumoral regulatory T cells, activating myeloid cells and inducing long-term memory responses.

Approximately 1,100 patients have been treated with botensilimab in phase 1 and phase 2 clinical trials. Botensilimab alone, or in combination with Agenus’ investigational PD-1 antibody, balstilimab, has shown clinical responses across nine metastatic, late-line cancers. For more information about botensilimab trials, visit www.clinicaltrials.gov with the identifiers NCT03860272, NCT05608044, NCT05630183, and NCT05529316.

Assertio Reports Second Quarter 2024 Financial Results

On August 7, 2024 Assertio Holdings, Inc. ("Assertio" or the "Company") (Nasdaq: ASRT), a pharmaceutical company with comprehensive commercial capabilities oering dierentiated products to patients, today reported nancial results for the second quarter ended June 30, 2024 (Press release, Assertio Holdings, AUG 7, 2024, View Source [SID1234647065]).

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"In my rst two months at Assertio, I have been excited to work closely with the team, meet our growing list of oncology clinic customers and focus on how best to maximize our results going forward. The second quarter’s performance illustrates how we continue to diligently execute the three pillars of Assertio’s business plan: driving the nancial performance of Assertio’s key assets, including our lead asset Rolvedon, generating cash ow and identifying new assets to create further value for our stockholders," said Brendan O’Grady, who was appointed Chief Executive Ocer on May 29, 2024.

"Rolvedon has been well received by physicians and continues to increase its share of the oncology G-CSF market,
generating its sixth consecutive quarter of demand growth since launch. As a potential opportunity for further
dierentiation, we have also completed enrollment of Rolvedon’s same-day dosing trial and expect to present the
initial data at a medical conference later this year. Combined with the rest of our platform and pipeline of
prospective business development opportunities, I am excited about the potential for Assertio."

Personalis Reports Second Quarter 2024 Financial Results

On August 7, 2024 Personalis, Inc. (Nasdaq: PSNL), a leader in advanced genomics for precision oncology, reported financial results for the second quarter ended June 30, 2024, and provided recent business accomplishments (Press release, Personalis, AUG 7, 2024, View Source [SID1234645500]).

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Recent Business Accomplishments


Highlighted clinical performance of NeXT Personal at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) meeting in May
o
Compelling breast cancer detection results were presented by Dr. Isaac Garcia-Murillas (Institute of Cancer Research, London) and Prof. Nicolas Turner (Royal Marsden NHS Foundation Trust UK). In this study, they found:

NeXT Personal detected cancer recurrence approximately 15 months before imaging

100% of patients that recurred were detected with NeXT Personal and 100% of patients that were ctDNA negative were cancer-free at follow up time points
o
A presentation by Dr. Rodrigo Toledo of the Vall d’Hebron Institute of Oncology highlighted the importance of NeXT Personal’s use for immunotherapy monitoring. This data showed:

Baseline levels of ctDNA and the changes detected by NeXT Personal predict therapy response and clinical outcomes for late-stage cancer patients receiving immunotherapy

NeXT Personal detected cancer progression 81 days before imaging on average

Delivered 561 total molecular tests in the second quarter of 2024, a 66% increase compared with the prior quarter, and commenced NeXT Personal Dx commercialization efforts with Tempus AI, Inc. (Tempus)

Announced a cross-license agreement with Myriad Genetics, Inc. covering patent estates for tumor-informed approaches to detect minimal residual disease (MRD)

Successfully settled the IP lawsuit with Foresight Diagnostics, Inc. (Foresight) with Foresight agreeing to license Personalis’ MRD patents

Second Quarter 2024 Results Compared to Second Quarter 2023


Reported total company revenue of $22.6 million in the second quarter of 2024, an increase of 35% compared with $16.7 million
o
Revenue from pharma testing and services of $13.2 million in the second quarter of 2024, an increase of 117% compared with $6.1 million

o
Revenue from Enterprise customers of $8.0 million in the second quarter of 2024, an increase of 8% compared with $7.4 million
o
Revenue from population sequencing for the U.S. Department of Veterans Affairs Million Veterans Program of $1.3 million in the second quarter of 2024, a decrease of 57% compared with $3.0 million

Other Income of $3.0 million in the second quarter of 2024, which consists of a non-cash gain of $3.0 million related to fair-value accounting for the outstanding warrants issued to Tempus

Net loss of $12.8 million in the second quarter of 2024, a decrease of 47% compared with $24.0 million

Cash, cash equivalents, and short-term investments of $87.0 million as of June 30, 2024

"The revenue growth depicts growing customer confidence in both our technology and platform. Importantly, the growth in our clinical testing volume means that we can see the impact on patient lives. Now that our collaborators have presented compelling NeXT Personal clinical evidence, we expect to leverage that towards obtaining Medicare coverage and materially increasing the number of patients we can help," said Chris Hall, Chief Executive Officer. "As we continue to execute on our Win-in-MRD strategy, we are more confident than ever in our ability to drive broad adoption for patient testing."

Third Quarter and Revised Full Year 2024 Outlook

Personalis expects the following for the third quarter of 2024:


Total company revenue in the range of $21.0 to $22.0 million

Revenue from pharma tests, enterprise sales, and other customers in the range of $17.0 to $18.0 million

Revenue from population sequencing of approximately $4.0 million

Personalis expects the following for the full year of 2024:


Total company revenue in the range of $79.0 to $81.0 million, an increase from $76.0 to $78.0 million

Revenue from pharma tests, enterprise sales, and all other customers in the range of $71.0 to $73.0 million, an increase from prior guidance of $68.0 to $70.0 million

Revenue from population sequencing of approximately $8.0 million

Non-GAAP net loss of approximately $75.0 million, a decrease from our prior guidance of $77.0 million and excludes any non-cash gain or loss from the outstanding warrants issued to Tempus

Cash usage of approximately $60.0 million, a decrease from our prior guidance of $62.0 million

Webcast and Conference Call Information

Personalis will host a conference call to discuss the second quarter of 2024 financial results, as well as plans for 2024, after market close on Wednesday, August 7, 2024, at 2:00 p.m. Pacific Time / 5:00 p.m. Eastern Time. The conference call can be accessed live by dialing 800-717-1738 for domestic callers or 646-307-1865 for international callers. The live webinar can be accessed at View Source A replay of the webinar will be available shortly after the conclusion of the call and will be archived on the company’s website.

IDRx Announces $120 Million Series B Financing to Advance Potential Best-in-class New Treatment for Gastrointestinal Stromal Tumor (GIST)

On August 7, 2024 IDRx, Inc., a clinical-stage biopharmaceutical company dedicated to transforming cancer treatment with purpose-built precision therapies, reported the completion of an oversubscribed $120 million Series B Preferred Stock financing (Press release, IDRx, AUG 7, 2024, View Source [SID1234645522]). The financing was led by RA Capital Management, Commodore Capital, and Blackstone Multi-Asset Investing with additional new investors, including Rock Springs Capital and a U.S.-based healthcare-focused fund. Existing investors, including Andreessen Horowitz (a16z) Bio + Health, Casdin Capital, Nextech Invest Ltd. (on behalf of one or more funds managed by it), Forge Life Science Partners, co-founder Nick Lydon, Ph.D., and strategic partner Merck KGaA, Darmstadt, Germany also participated in the financing. Derek DiRocco, Ph.D., Partner at RA Capital Management, has joined the IDRx Board of Directors.

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IDRx plans to use the proceeds from the financing to support the ongoing Phase 1/1b StrateGIST 1 study of its lead product candidate IDRX-42, a potent, oral, highly selective KIT inhibitor targeting all major categories of activating and resistance mutations in patients with KIT-mutant GIST. Additionally, proceeds will be used to fund the expected initiation of the first pivotal study for IDRX-42 in patients with second-line GIST.

At the 2024 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) annual meeting, IDRx presented preliminary Phase 1 data from the ongoing Phase 1/1b StrateGIST 1 trial. The data support the best-in-class potential of IDRX-42 in patients with GIST. This included a 23% objective response rate (ORR) across all patients (median four prior lines of therapy) and a 43% ORR in second-line patients, including those with either or both ATP binding site and activation loop mutations, with a favorable tolerability profile consistent with use in front-line and second-line settings. IDRx is now enrolling patients in the Phase 1b portion of the trial. In addition, the U.S. Food and Drug Administration (FDA) has recently granted Fast Track designation to IDRX-42 for the treatment of GIST after disease progression on or intolerance to imatinib.

"We are thrilled to announce this financing, which includes support from a top-tier syndicate of investors and positions us to accelerate the development of IDRX-42 for a broad population of patients with GIST, including in the second-line and front-line settings, where patients haven’t seen a new treatment option in over 15 years," said Tim Clackson, Ph.D., Chief Executive Officer of IDRx. "IDRX-42 was designed to overcome the twin challenges of on-target treatment resistance and off-target driven adverse events, which limit the clinical benefit of currently available TKIs. We are extremely encouraged by the clinical data generated with IDRX-42 to date and are focused on rapidly advancing this potential new treatment option that could elevate the standard of care for GIST patients."

"RA Capital Management is excited to co-lead this round to support the advancement of IDRX-42 as a differentiated new treatment for GIST," said Derek DiRocco, Ph.D., Partner of RA Capital Management and board member of IDRx. "We have been extremely impressed by what the IDRx team has accomplished in a short time, and I look forward to contributing to IDRx’s future growth and success as the company prepares to execute on its late-stage clinical development activities for IDRX-42."

About GIST

GIST is the most common subtype of soft tissue sarcoma. Approximately 80% of cases arise from gain of function mutations in the KIT receptor tyrosine kinase, driving the malignancy through constitutive activation of aberrant signaling. Resistance mutations in KIT emerge in ~90% of patients treated with imatinib, the current standard of care for GIST. In unresectable or metastatic GIST, clinical benefits from existing treatments can vary by mutation type.

About the StrateGIST 1 Study

StrateGIST 1 is an ongoing, open-label, first-in-human Phase 1/1b study designed to evaluate the safety, tolerability, pharmacokinetics, and preliminary antitumor activity of IDRX-42 in patients with metastatic and/or surgically unresectable GIST after failure of imatinib and other approved drugs. The study is currently enrolling patients with documented pathogenic mutation in KIT or any platelet-derived growth factor receptor alpha (PDGFRA) mutation (other than PDGFRA exon 18) at sites in the U.S., United Kingdom, Belgium, The Netherlands, France, Germany, Italy, Spain, and South Korea. The Phase 1b portion has been initiated and includes four expanded exploratory cohorts based on defined lines of prior TKI therapy, including front line, second line, third line + (with standard approved TKIs only), and third line + (including investigational TKIs).

About IDRX-42

IDRX-42 is a potent, oral, highly selective KIT inhibitor targeting all major categories of activating and resistance mutations in patients with KIT-mutant GIST (including variants in exons 9, 11, 13 and 17). In preclinical studies, IDRX-42 demonstrated superior antitumor activity compared to imatinib, the current first-line of therapy, in GIST human xenograft models expressing mutations in KIT exons 9 and 11. In xenograft models expressing secondary resistance mutations in KIT exon 13 or 17, IDRX-42 treatment resulted in potent and dose-dependent antitumor activity superior to the second-line standard of care agent, sunitinib. IDRX-42 is currently being evaluated in StrateGIST 1, a first-in-human Phase 1/1b study. IDRX-42 was granted Orphan Drug designation by the FDA for the treatment of GIST.