Renal Cell Carcinoma

SAN DIEGO, CA, USA I May 05, 2025 I Janux Therapeutics, Inc. (Nasdaq: JANX) (Janux), a clinical-stage biopharmaceutical company developing a broad pipeline of novel immunotherapies by applying its proprietary technology to its Tumor Activated T Cell Engager (TRACTr) and Tumor Activated Immunomodulator (TRACIr) platforms, today announced the initiation of Phase 1b expansion studies in the ongoing ENGAGER-PSMA-01 trial. ENGAGER-PSMA-01 is a first-in-human, open-label, multicenter Phase 1 clinical trial designed to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary efficacy of JANX007 administered as monotherapy or in combination in adult patients with advanced metastatic castration-resistant prostate cancer (mCRPC). In December 2024, Janux reported positive interim clinical data from the Phase 1a dose escalation portion of the trial in 16 mCRPC patients with a median of four prior lines of therapy. At that time, the median radiographic progression-free survival (rPFS) reported was 7.4 months for all 16 patients.* As of April 21, 2025, updated results** have been achieved in the same 16 patients supporting the initiation of the Phase 1b expansion studies: *8/16 patients were noted as in-progress in the December 2024 reported results. **rPFS results based upon Kaplan-Meier estimate. In addition, Janux has selected a CRS-mitigation strategy to support the initiation of the Phase 1b expansion studies that is designed to maintain the CRS profile reported in December. The first Phase 1b expansion study will enroll taxane-naïve mCRPC patients and is designed to generate additional safety and efficacy data in this first and second line (1L/2L) patient population. This study will assess JANX007 monotherapy at two dose regimens (0.3/2/6mg and 0.3/2/9mg) with dosing administered once weekly or once every two weeks in mCRPC patients who have progressed on or after novel hormonal therapy (NHT). “Initiation of the taxane-naïve study marks an important step as we begin to evaluate JANX007 in earlier-line mCRPC patient populations,” said Zachariah McIver, D.O., Ph.D., Chief Medical Officer of Janux. “While therapeutic options for mCRPC have expanded, there remains a significant need for novel, non-chemotherapeutic approaches.” Janux plans to initiate three additional Phase 1b expansion studies, evaluating: “Improved efficacy and durability of responses has been observed by other prostate cancer drugs and TCEs when moving into earlier lines of therapy. There are also indications that safety with TCEs improve in earlier lines of therapy where disease burden is lower. We believe that these observations, coupled with the data seen in our Phase 1a dose escalation in later line patients, strongly support our decision to develop JANX007 in earlier lines of therapy,” said David Campbell, Ph.D., President and CEO of Janux. Additional data from JANX007 and JANX008 will be presented at future Janux events in the second half of 2025. Separately, Janux will host an R&D Day in mid-2025 highlighting product candidates identified from its preclinical pipeline to move into clinical trials. Janux’s TRACTr and TRACIr Pipeline Janux’s first clinical candidate, JANX007, is a TRACTr that targets prostate-specific membrane antigen (PSMA) and is being investigated in a Phase 1 clinical trial in adult patients with metastatic castration-resistant prostate cancer. Janux’s second clinical candidate, JANX008, is a TRACTr that targets epidermal growth factor receptor (EGFR) and is being studied in a Phase 1 clinical trial for the treatment of multiple solid cancers including colorectal carcinoma, squamous cell carcinoma of the head and neck, non-small cell lung cancer, renal cell carcinoma, small cell lung cancer, pancreatic ductal adenocarcinoma and triple-negative breast cancer. We are also generating a number of additional TRACTr and TRACIr programs for potential future development, some of which are at development candidate stage or later. We are currently assessing priorities in our preclinical pipeline. About Janux Therapeutics Janux is a clinical-stage biopharmaceutical company developing tumor-activated immunotherapies for cancer. Janux’s proprietary technology enabled the development of two distinct bispecific platforms: TRACTr and TRACIr. The goal of both platforms is to provide cancer patients with safe and effective therapeutics that direct and guide their immune system to eradicate tumors while minimizing safety concerns. Janux is currently developing a broad pipeline of TRACTr and TRACIr therapeutics directed at several targets to treat solid tumors. Janux has two TRACTr therapeutic candidates in clinical trials, the first targeting PSMA is in development for prostate cancer, and the second targeting EGFR is being developed for colorectal carcinoma, squamous cell carcinoma of the head and neck, non-small cell lung cancer, renal cell carcinoma, small cell lung cancer, pancreatic ductal adenocarcinoma and triple-negative breast cancer. For more information, please visit www.januxrx.com and follow us on LinkedIn. SOURCE: Janux Therapeutics

Taylor Tieden for BioSpace On the FDA’s docket this month are two expansion bids, one for GSK’s asthma drug Nucala into COPD and another for Merck’s oral cancer drug for a pair of rare tumors. Amid the growing macro-level headwinds in the vaccine space, the FDA this month is expected to decide on Moderna’s mRNA COVID-19 shot, alongside a handful of other big verdicts. Read below for more. GSK Asks FDA to Approve Asthma Injection Nucala for COPD GSK is proposing its subcutaneous biologic Nucala to treat adult patients with chronic obstructive pulmonary disease (COPD) with an eosinophilic phenotype. The FDA’s decision is due on May 7. A pulmonary disease, COPD is characterized by inflammation in the lungs, manifesting as breathlessness, coughs and airflow obstruction. According to GSK, some 40% of patients suffer from disease exacerbations caused by type 2 inflammation, which in turn is linked to high eosinophil counts in the blood. Nucala , a monoclonal antibody, targets and blocks the IL-5 cytokine, which is crucial to maintain the activity of eosinophils. GSK is supporting Nucala’s COPD application with data from the Phase III MATINEE trial. The pharma released a topline readout from the study in September 2024, noting that Nucala elicited a “statistically significant and clinically meaningful reduction” in yearly rates of moderate or severe disease exacerbations as opposed to placebo. Nucala is currently approved for eosinophilic granulomatosis with polyangiitis and hypereosinophilic syndrome, and as an add-on maintenance treatment for asthma and chronic rhinosinusitis with nasal polyps. Arcutis Seeks Zoryve Expansion into Body and Scalp Psoriasis By May 22, the FDA will release its verdict on Arcutis Biotherapeutics’ proposed expansion for Zoryve into body and scalp psoriasis. Arcutis is backing its expansion bid with data from the Phase III ARRECTOR trial, which found that 66.4% of Zoryve-treated patients achieved clear or almost-clear skin, as opposed to 27.8% in placebo patients. Results also showed that 65.3% of patients in the Zoryve arm saw significant improvements in itch, versus 30.3% in the placebo group. Arcutis also filed findings from a Phase IIb study as well as long-term efficacy and safety data for Zoryve in plaque psoriasis. Zoryve is a topical phosphodiesterase 4 inhibitor that works by helping to suppress inflammation in the skin. The drug won FDA approval in 2011 for the treatment of plaque psoriasis in patients aged 12 years and up. If approved for body and scalp psoriasis, Zoryve would be a “truly meaningful innovation” for patients who suffer from inadequately controlled symptoms, Arcutis CEO Frank Watanabe said in a September 2024 release. Sanofi Eyes Extension of Meningococcal Disease Shot to Infants, Toddlers Sanofi’s meningococcal disease vaccine MenQuadfi is currently approved for the primary immunization of children aged two years and up. As a booster, it can be used in those 13 years and older who continue to be at risk of meningococcal disease. Sanofi is seeking to expand MenQuadfi’s label to also include infants and toddlers from six weeks to 23 months of age. The FDA is currently reviewing this proposal and is due to release its decision on May 23. If approved, MenQuadfi could protect this younger population from the A, C, W and Y serogroups of Neisseria meningitidis . In an April presentation before the CDC’s Advisory Committee for Immunization Practices, Sanofi presented data from several trials, including the Phase III MET42 study. Results showed that MenQuadfi was safe to use in infants and toddlers, with most side effects being mild or moderate in severity. Efficacy-wise, MenQuadfi was able to match a commercially approved vaccine in immune response and seroprotection. Liquidia Awaits Final Yutrepia Approval in Interstitial Lung Disease On May 23, Liquidia is expecting to win final approval of its inhalation powder Yutrepia for the treatment of patients with pulmonary arterial hypertension associated with interstitial lung disease (PH-ILD). The FDA gave the drug tentative approval in August 2024, indicating that it had cleared all efficacy and safety hurdles and satisfied regulatory standards of product quality. However, Liquidia still needed to wait out regulatory exclusivity for a competing product. Yutrepia is Liquidia’s inhalable version of treprostinil, a vasodilator that has known activity against pulmonary hypertension. Yutrepia is backed by data from the Phase III INSPIRE study, which found that the drug was both safe and well-tolerated in patients who were naïve to treprostinil or who were switching to Yutrepia from nebulized treprostinil. The INSPIRE trial found that patients treated with Yutrepia saw symptoms stabilize or improve over one year of dosing. Merck Hopes to Tack Two Rare Cancers Onto Welireg’s Label Merck is hoping to expand its oral cancer drug Welireg into advanced pheochromocytoma and paraganglioma (PPGL). The FDA’s decision deadline is May 26. PPGL refers to two types of rare tumors of the adrenal gland. Diagnosed in some 2,000 patients per year, they are typically caused by specific genetic mutations. Symptoms include high blood pressure, palpitations and headaches. According to Merck, up to 25% of PPGL cases are already at the metastatic stage at diagnosis. Merck is supporting Welireg’s PPGL bid with data from Cohort A1 of the Phase II LITESPARK-015 study. The pharma did not provide specific data in a January news release , revealing only that the application is based on objective response rate and duration of response in PPGL patients after Welireg treatment. The FDA has granted the drug priority review in this indication. Welireg is an orally available inhibitor of the HIF-2α transcription factor, which is involved in oxygen sensing and in the body’s response to hypoxia. The drug is indicated for patients with renal cell carcinoma (RCC) and those with von Hippel-Lindau disease—a rare genetic disease involving the formation of tumors across various organs—who also require treatment for associated RCC. If approved, Welireg will become the only drug for advanced PPGL in the U.S., Merck said. After Delay, Eton Nears Approval for Oral Hydrocortisone By May 28, the FDA will release its verdict on Eton Pharmaceuticals’ ET-400, an oral formulation of hydrocortisone, for the treatment of adrenal insufficiency in infants. This new target action date comes after the FDA in February announced that it needed more time to review Eton’s data package for ET-400. Eton submitted supplemental information in December last year, in response to a request from the regulator. ET-400 is a proprietary oral formulation of hydrocortisone that, according to the biotech, is stable at room temperature. If approved, ET-400 will follow in the footsteps of Eton’s Alkindi Sprinkle, which also has hydrocortisone as an active ingredient, but is formulated as oral granules. Alkindi Sprinkle, approved in 2020 , is designed to rapidly deliver glucocorticoids to children with adrenocortical insufficiency. In a July 2024 announcement , Eton CEO Sean Brynjelsen said that the approval of ET-400 would allow the biotech to “capture a greater percentage of the oral hydrocortisone market” and, alongside Alkindi Sprinkle, would open up a peak opportunity of “more than $50 million annually.” Moderna Seeks Approval for Next-Generation COVID-19 Shot The FDA’s last big milestone for the month belongs to Moderna and its investigational next-generation COVID-19 vaccine mRNA-1283. The regulator is expected to release its decision on May 31. In June 2024, the company released Phase III data for the experimental shot, touting better efficacy than Spikevax, its currently approved COVID-19 vaccine. Moderna did not provide specific data, only claiming that mRNA-1283 elicited a “higher immune response” against SARS-CoV-2 than Spikevax. Of note, Moderna emphasized at the time that mRNA-1283 could induce robust responses against Omicron and original strains of the virus—a benefit the company claimed was most evident in seniors over 65 years of age. The CDC’s vaccine advisors were supposed to convene in late February to discuss various vaccines, including mRNA-1283, before the meeting was postponed . The panel finally convened last month.

Phase 1b expansion study initiated in taxane-naïve mCRPC patients Updated data from Phase 1a dose escalation support initiation of Phase 1b dose expansion Updates on JANX007 and JANX008 expected in the second half of 2025 Janux to host R&D Day in mid-2025 highlighting preclinical pipeline progression to the clinic SAN DIEGO--(BUSINESS WIRE)-- Janux Therapeutics, Inc. (Nasdaq: JANX) (Janux), a clinical-stage biopharmaceutical company developing a broad pipeline of novel immunotherapies by applying its proprietary technology to its Tumor Activated T Cell Engager (TRACTr) and Tumor Activated Immunomodulator (TRACIr) platforms, today announced the initiation of Phase 1b expansion studies in the ongoing ENGAGER-PSMA-01 trial. ENGAGER-PSMA-01 is a first-in-human, open-label, multicenter Phase 1 clinical trial designed to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary efficacy of JANX007 administered as monotherapy or in combination in adult patients with advanced metastatic castration-resistant prostate cancer (mCRPC). In December 2024, Janux reported positive interim clinical data from the Phase 1a dose escalation portion of the trial in 16 mCRPC patients with a median of four prior lines of therapy. At that time, the median radiographic progression-free survival (rPFS) reported was 7.4 months for all 16 patients.* As of April 21, 2025, updated results** have been achieved in the same 16 patients supporting the initiation of the Phase 1b expansion studies: Median rPFS of 7.5 months (n=16) Median rPFS of 7.9 months for patients treated at 6mg and 9mg target doses (n=9) 6-month rPFS of 65% (n=16) 6-month rPFS of 78% for patients treated at 6mg and 9mg target doses (n=9) Safety data remained consistent with the December 2024 data disclosure (n=16) *8/16 patients were noted as in-progress in the December 2024 reported results. **rPFS results based upon Kaplan-Meier estimate. In addition, Janux has selected a CRS-mitigation strategy to support the initiation of the Phase 1b expansion studies that is designed to maintain the CRS profile reported in December. The first Phase 1b expansion study will enroll taxane-naïve mCRPC patients and is designed to generate additional safety and efficacy data in this first and second line (1L/2L) patient population. This study will assess JANX007 monotherapy at two dose regimens (0.3/2/6mg and 0.3/2/9mg) with dosing administered once weekly or once every two weeks in mCRPC patients who have progressed on or after novel hormonal therapy (NHT). “Initiation of the taxane-naïve study marks an important step as we begin to evaluate JANX007 in earlier-line mCRPC patient populations,” said Zachariah McIver, D.O., Ph.D., Chief Medical Officer of Janux. “While therapeutic options for mCRPC have expanded, there remains a significant need for novel, non-chemotherapeutic approaches.” Janux plans to initiate three additional Phase 1b expansion studies, evaluating: JANX007 in combination with an androgen receptor inhibitor in taxane-experienced mCRPC patients JANX007 monotherapy in PARP inhibitor-resistant mCRPC patients JANX007 monotherapy in NHT- and taxane-experienced mCRPC patients designed to support OPTIMUS dose selection for registrational studies “Improved efficacy and durability of responses has been observed by other prostate cancer drugs and TCEs when moving into earlier lines of therapy. There are also indications that safety with TCEs improve in earlier lines of therapy where disease burden is lower. We believe that these observations, coupled with the data seen in our Phase 1a dose escalation in later line patients, strongly support our decision to develop JANX007 in earlier lines of therapy,” said David Campbell, Ph.D., President and CEO of Janux. Additional data from JANX007 and JANX008 will be presented at future Janux events in the second half of 2025. Separately, Janux will host an R&D Day in mid-2025 highlighting product candidates identified from its preclinical pipeline to move into clinical trials. Janux’s TRACTr and TRACIr Pipeline Janux’s first clinical candidate, JANX007, is a TRACTr that targets prostate-specific membrane antigen (PSMA) and is being investigated in a Phase 1 clinical trial in adult patients with metastatic castration-resistant prostate cancer. Janux’s second clinical candidate, JANX008, is a TRACTr that targets epidermal growth factor receptor (EGFR) and is being studied in a Phase 1 clinical trial for the treatment of multiple solid cancers including colorectal carcinoma, squamous cell carcinoma of the head and neck, non-small cell lung cancer, renal cell carcinoma, small cell lung cancer, pancreatic ductal adenocarcinoma and triple-negative breast cancer. We are also generating a number of additional TRACTr and TRACIr programs for potential future development, some of which are at development candidate stage or later. We are currently assessing priorities in our preclinical pipeline. About Janux Therapeutics Janux is a clinical-stage biopharmaceutical company developing tumor-activated immunotherapies for cancer. Janux’s proprietary technology enabled the development of two distinct bispecific platforms: TRACTr and TRACIr. The goal of both platforms is to provide cancer patients with safe and effective therapeutics that direct and guide their immune system to eradicate tumors while minimizing safety concerns. Janux is currently developing a broad pipeline of TRACTr and TRACIr therapeutics directed at several targets to treat solid tumors. Janux has two TRACTr therapeutic candidates in clinical trials, the first targeting PSMA is in development for prostate cancer, and the second targeting EGFR is being developed for colorectal carcinoma, squamous cell carcinoma of the head and neck, non-small cell lung cancer, renal cell carcinoma, small cell lung cancer, pancreatic ductal adenocarcinoma and triple-negative breast cancer. For more information, please visit www.januxrx.com and follow us on LinkedIn. Forward-Looking Statements This news release contains certain forward-looking statements that involve risks and uncertainties that could cause actual results to be materially different from historical results or from any future results expressed or implied by such forward-looking statements. Such forward-looking statements include statements regarding, among other things, Janux’s ability to bring new treatments to cancer patients in need, expectations regarding the timing, scope and results of Janux’s development activities, including its ongoing and planned preclinical studies and clinical trials, the timing of and plans for regulatory filings, the potential benefits of Janux’s product candidates and platform technologies, the timing of future data releases, and expectations regarding the use of Janux’s platform technologies to generate novel product candidates. Factors that may cause actual results to differ materially include the risk that compounds that appear promising in early research do not demonstrate safety and/or efficacy in later preclinical studies or clinical trials, the risk that Janux may not obtain approval to market its product candidates, uncertainties associated with performing clinical trials, regulatory filings and applications, risks associated with reliance on third parties to successfully conduct clinical trials, the risks associated with reliance on outside financing to meet capital requirements, the risks related to geopolitical events, including international tariffs, global economic, financial, and healthcare system disruptions and the current and potential future negative impacts to Janux’s business operations and development activities, and other risks associated with the process of discovering, developing and commercializing drugs that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such drugs. You are urged to consider statements that include the words “may,” “will,” “would,” “could,” “should,” “believes,” “estimates,” “projects,” “promise,” “potential,” “expects,” “plans,” “anticipates,” “intends,” “continues,” “designed,” “goal,” or the negative of those words or other comparable words to be uncertain and forward-looking. For a further list and description of the risks and uncertainties Janux faces, please refer to Janux’s periodic and other filings with the Securities and Exchange Commission, which are available at www.sec.gov. Such forward-looking statements are current only as of the date they are made, and Janux assumes no obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise. Investors: Andy Meyer Janux Therapeutics ameyer@januxrx.com (202) 215-2579 Media: Jessica Yingling, Ph.D. Little Dog Communications Inc. jessica@litldog.com (858) 344-8091