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CARMEL, Ind., Sept. 30, 2024 (GLOBE NEWSWIRE) -- MBX Biosciences, Inc. (NASDAQ: MBX), a clinical-stage biopharmaceutical company focused on the discovery and development of novel precision peptide therapies for the treatment of endocrine and metabolic disorders, today announced that both the rationale and design of the Phase 2 Avail™ trial of MBX 2109, the Company’s potential long-acting parathyroid hormone (PTH) peptide prodrug, in adults with hypoparathyroidism (HP) were featured in a poster presentation at the American Society for Bone and Mineral Research (ASBMR) 2024 Annual Meeting held in Toronto, ON, Canada September 27-30, 2024. “We are pleased to share more information on our ongoing Phase 2 MBX 2109 trial in patients with HP at ASBMR,” said Kent Hawryluk, President and Chief Executive Officer of MBX Biosciences. “MBX 2109 was designed to treat the underlying pathophysiology of HP. We believe this study will provide insight into its potential to provide patients with consistent and convenient relief from the burdensome symptoms caused by PTH deficiency and identify appropriate doses to be studied in the Phase 3 program. Dosing in the trial commenced in August 2024, and topline results are anticipated in the third quarter of 2025.” MBX 2109 is an investigational, PTH peptide prodrug designed as a once-weekly PTH replacement therapy with the potential to normalize serum calcium levels, reduce the need for active vitamin D and calcium supplementation, and improve patient convenience and treatment adherence. The Phase 2 Avail™ trial (NCT06465108) is a randomized, multicenter, 12-week, double-blind, placebo-controlled study evaluating the safety, pharmacokinetics, and efficacy of MBX 2109 in approximately 48 adults with HP. The primary endpoint of the Phase 2 clinical trial is the proportion of patients who can discontinue active vitamin D and reduce calcium supplements to less than or equal to 600 mg per day after 12 weeks of treatment while maintaining normal serum albumin-adjusted calcium levels (8.2-10.6 mg/dL). Secondary endpoints include safety and tolerability of MBX 2109 and characterization of its pharmacokinetics and pharmacodynamic activity (including urine calcium, serum phosphorus, 1,25 dihydroxyvitamin D and bone biomarkers) and the impact on quality of life using patient-reported outcome tools. The trial begins with an optimization period of up to 4 weeks in which doses of calcium, vitamin D, and/or magnesium supplements are adjusted to achieve normal serum calcium levels. Participants completing the optimization period are then randomized 1:1:1:1 to receive weekly doses of placebo or MBX 2109 at 400, 600, or 800 μg for 12 weeks. The treatment period is comprised of a 4-week fixed-dose period and an 8-week titration period. During the 8-week dose-adjustment period, doses of MBX 2109 and active vitamin D and calcium supplements are adjusted according to a prespecified protocol algorithm to reduce risk of hypo- or hypercalcemia. Participants completing the 12-week treatment period may enroll in a long-term, open-label extension study (NCT06531941). Participants who received MBX 2109 will maintain their existing dosage from the 12-week trial (200 to 1600 μg of MBX 2109) while patients on placebo will transition to MBX 2109 at the 400 μg dose. The first patient in the study was dosed in August 2024. The study is actively enrolling, and topline study results are expected to be available in the third quarter of 2025. About HypoparathyroidismHP is a rare endocrine disease caused by a deficiency of PTH released by the parathyroid glands that results in decreased calcium levels in the blood leading to hypocalcemia. Hypocalcemia can result in a variety of acute symptoms, such as muscle cramping or spasm, tingling, and neurological symptoms such as depression, confusion and cognitive impairment. More serious complications can occur, including seizures and cardiac arrhythmias. As a result, HP can interfere with daily activities, negatively impacting the quality of life for patients and we estimate that HP affects approximately 120,000 people in the United States and more than 250,000 in the United States and Europe. The most common cause for HP, in approximately 75% of cases, is the inadvertent removal or damage to the parathyroid glands during neck surgery. It can also be caused by certain autoimmune processes and genetic conditions. The current standard of care for HP does not address the PTH deficiency, which is the underlying cause of the disease. To avoid hypocalcemia and its symptoms due to PTH deficiency, the current standard of care consists primarily of high doses of oral calcium supplements and active vitamin D. About MBX 2109MBX 2109 is a parathyroid hormone peptide prodrug that is designed as a potential long-acting hormone replacement therapy for the treatment of HP. Leveraging the Company’s proprietary Precision Endocrine Peptide™ platform technology, MBX 2109 was designed to provide convenient, once-weekly administration and a continuous, infusion-like PTH exposure with lower daily peak-to-trough ratios than observed with daily PTH dosing regimens. MBX 2109 received orphan drug designation from the U.S. Food and Drug Administration for the treatment of HP. About MBX BiosciencesMBX Biosciences is a biopharmaceutical company focused on the discovery and development of novel precision peptide therapies for the treatment of endocrine and metabolic disorders. The Company was founded by global leaders with a transformative approach to peptide drug design and development. Leveraging this expertise, the Company designed its proprietary Precision Endocrine Peptide™ (PEP™) platform to overcome the key limitations of unmodified and modified peptide therapies and to improve clinical outcomes and simplify disease management for patients. PEPs are selectively engineered to have optimized pharmaceutical properties, including extended time-action profiles and consistent drug concentrations with low peak-to-trough concentration ratios, consistent exposure to target tissues, and less frequent dosing. The Company is advancing a pipeline of novel candidates for endocrine and metabolic disorders with clinically validated targets, established endpoints for regulatory approval, significant unmet medical needs and large potential market opportunities. The Company’s pipeline includes its lead product candidate MBX 2109, in Phase 2 development for the treatment of chronic hypoparathyroidism (HP); MBX 1416, in Phase 1 development for the treatment of post-bariatric hypoglycemia (PBH); and an obesity portfolio that includes MBX 4291, as well as multiple discovery and pre-clinical candidates in development for the treatment of obesity. The Company is based in Carmel, Indiana. To learn more, please visit the Company website at www.mbxbio.com and follow us on LinkedIn. Forward-Looking StatementsThis press release contains “forward-looking statements” within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934, each as amended. The words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “would” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. These forward-looking statements include, but are not limited to, express or implied statements regarding: MBX Biosciences’ expectations regarding the Phase 2 Avail™ trial of MBX 2109, including the timing of topline results and the ability to provide insight into MBX 2109’s potential to provide patients with consistent and convenient relief from the burdensome symptoms caused by PTH deficiency and identify appropriate doses to be studied in the Phase 3 program; the potential for MBX 2109 to normalize serum calcium levels, reduce the need for active vitamin D and calcium supplementation, and improve patient convenience and treatment adherence; and the potential for MBX 2109 to be a once-weekly PTH replacement therapy. Forward looking statements are based on management’s current expectations and are subject to risks and uncertainties that could negatively affect MBX Biosciences’ business, operating results, financial condition and stock value. Factors that could cause actual results to differ materially from those currently anticipated include: risks relating to the Company’s research and development activities; MBX Biosciences’ ability to execute on its strategy including obtaining the requisite regulatory approvals on the expected timeline, if at all; uncertainties relating to preclinical and clinical development activities; the Company’s dependence on third parties to conduct clinical trials, manufacture its product candidates and develop and commercialize its product candidates, if approved; MBX Biosciences’ ability to attract, integrate and retain key personnel; risks related to the Company’s financial condition and need for substantial additional funds in order to complete development activities and commercialize a product candidate, if approved; risks related to regulatory developments and approval processes of the U.S. Food and Drug Administration and comparable foreign regulatory authorities; risks related to establishing and maintaining MBX Biosciences’ intellectual property protections; and risks related to the competitive landscape for MBX Biosciences’ product candidates; as well as other risks described in “Risk Factors,” in MBX Biosciences’ Registration Statement on Form S-1 filed with the Securities and Exchange Commission (SEC), as well as subsequent filings with the SEC. MBX Biosciences expressly disclaims any obligation or undertaking to release publicly any updates or revisions to any forward-looking statements contained herein to reflect any change in its expectations or any changes in events, conditions or circumstances on which any such statement is based, except as required by law, and claims the protection of the safe harbor for forward-looking statements contained in the Private Securities Litigation Reform Act of 1995. Media Contact:Kate Burdick Inizio Evoke CommsKate.burdick@inizioevoke.com 860-462-1569 Investor Contact:Irina KofflerLifeSci Advisorsikoffler@lifesciadvisors.com917-734-7387

Topline results expected in Q3 2025CARMEL, Ind., Aug. 12, 2024 (GLOBE NEWSWIRE) -- MBX Biosciences, Inc., a clinical stage biopharmaceutical company focused on the discovery and development of novel precision peptide therapies for the treatment of endocrine and metabolic disorders, today announced that it has dosed the first patient in its Phase 2 Avail™ trial of MBX 2109, the Company’s parathyroid hormone (PTH) peptide prodrug that is designed to be long-acting and is in development for the treatment of chronic hypoparathyroidism (HP). “We are pleased to advance our innovative Precision Endocrine PeptideTM (PEPTM) candidate, MBX 2109, by dosing our first patient with HP,” said Kent Hawryluk, President and Chief Executive Officer of MBX Biosciences. “The Phase 1 findings for safety, pharmacokinetics and pharmacodynamics support the potential of MBX 2109 as the first once-weekly PTH prodrug for HP, and initiation of patient dosing brings us closer to launching our potential PTH therapy. We look forward to continued enrollment and dosing of patients, with topline results anticipated in the third quarter of 2025.” The Phase 2 Avail trial is a randomized double-blind, placebo-controlled clinical trial in adult patients with HP. The Avail trial evaluates the safety, tolerability and efficacy of MBX 2109 over a 12-week period in approximately 48 patients. The primary endpoint of the Phase 2 clinical trial is the proportion of patients who can discontinue active vitamin D and reduce calcium supplements to less than or equal to 600 mg per day after 12 weeks of treatment while maintaining normal serum calcium levels. Secondary endpoints include safety and tolerability of MBX 2109 and characterization of the pharmacokinetics and pharmacodynamic activity (including urine calcium, serum phosphorus, 1,25 dihydroxyvitamin D and bone biomarkers) and the impact on quality of life using patient-reported outcome tools. In the Avail trial, patients are randomized (1:1:1:1) to weekly subcutaneous injections of placebo or 400 μg, 600 μg, and 800 μg of MBX 2109. The 12-week treatment period is comprised of a 4-week fixed dose period and an 8-week titration period. During the titration period, patients who have not been able to discontinue active vitamin D and/or reduce calcium supplements may up-titrate the study drug using a protocol-specified algorithm. Patients completing the 12-week treatment period will be eligible to participate in a 104-week long-term extension study in which all patients will receive MBX 2109. The Avail trial began in 2024 and topline results are expected in the third quarter of 2025. More information on the Phase 2 Avail™ trial can be found at www.clinicaltrials.gov, identifier NCT06465108. About Hypoparathyroidism HP is a rare endocrine disease caused by a deficiency of PTH released by the parathyroid glands that results in decreased calcium levels in the blood leading to hypocalcemia. Hypocalcemia can result in a variety of acute symptoms, such as muscle cramping or spasm, tingling, and neurological symptoms such as depression, confusion and cognitive impairment. More serious complications can occur, including seizures and cardiac arrhythmias. As a result, HP can interfere with daily activities, negatively impacting the quality of life for patients and we estimate that HP affects approximately 120,000 people in the United States and more than 250,000 in the United States and Europe. The most common cause for HP, in approximately 75% of cases, is the inadvertent removal or damage to the parathyroid glands during neck surgery. It can also be caused by certain autoimmune processes and genetic conditions. The current standard of care for HP does not address the PTH deficiency, which is the underlying cause of the disease. To avoid hypocalcemia and its symptoms due to PTH deficiency, the current standard of care consists primarily of high doses of oral calcium supplements and active vitamin D. About MBX 2109 MBX 2109 is a parathyroid hormone peptide prodrug that is designed as a potential long-acting hormone replacement therapy for the treatment of HP. Leveraging the company’s proprietary Precision Endocrine Peptide™ (PEP™) platform technology, MBX 2109 was designed to provide convenient, once-weekly administration and a continuous, infusion-like PTH exposure with lower daily peak-to-trough ratios than observed with daily PTH dosing regimens. MBX 2109 received orphan drug designation from the U.S. Food and Drug Administration for the treatment of HP. About MBX Biosciences MBX Biosciences, Inc. is a biopharmaceutical company focused on the discovery and development of novel precision peptide therapies for the treatment of endocrine and metabolic disorders. The company was founded by global leaders with a transformative approach to peptide drug design and development. Leveraging this expertise, the Company designed its proprietary Precision Endocrine Peptide™, or PEP™, platform to overcome the key limitations of unmodified and modified peptide therapies and to improve clinical outcomes and simplify disease management for patients. PEPs are selectively engineered to have optimized pharmaceutical properties, including extended time-action profiles and consistent drug concentrations with low peak-to-trough concentration ratios, consistent exposure to target tissues, and less frequent dosing. The Company is advancing a pipeline of novel candidates for endocrine and metabolic disorders with clinically validated targets, established endpoints for regulatory approval, significant unmet medical needs and large potential market opportunities. The Company’s pipeline includes its lead product candidate MBX 2109, in Phase 2 development for the treatment of chronic hypoparathyroidism (HP); MBX 1416, in Phase 1 development for the treatment of post-bariatric hypoglycemia (PBH); and an obesity portfolio that includes MBX 4291, as well as multiple discovery and pre-clinical candidates in development for the treatment of obesity. The Company is supported by leading life science investors including Deep Track Capital, Driehaus Capital Management, Frazier Life Sciences, New Enterprise Associates (NEA), Norwest Venture Partners, OrbiMed, RA Capital Management, funds and accounts advised by T. Rowe Price Associates, Inc. and Wellington Management. MBX is based in Carmel, Indiana. To learn more, please visit the company website at www.mbxbio.com and follow us on LinkedIn. Media Contact:Kate BurdickInizio Evoke Commskate.burdick@inizioevoke.com860-462-1569 Investor Contact:Irina KofflerLifeSci Advisorsikoffler@lifesciadvisors.com917-734-7387

Evaluation of the Safety and Efficacy of Eflornithine (Difluoromethylornithine, DFMO) in Patients with Gastric Premalignant Conditions in the High Incidence Areas of Latin AmericanMINNEAPOLIS, June 10, 2024 (GLOBE NEWSWIRE) -- Panbela Therapeutics, Inc. (OTCQB: PBLA), a clinical stage biopharmaceutical company developing disruptive therapeutics for the treatment of patients with cancer, today announces an oral presentation at the Digestive Disease Week (DDW) conference, which was held May 18-21, 2024. The work reflects the Company’s on-going collaboration with Vanderbilt University Medical Center. “Gastric adenocarcinoma is the fifth leading global cause of cancer mortality and leading infection-associated cancer. In the U.S., gastric adenocarcinoma represents a major cancer disparity, with incidence rates 2-3 times or more greater in all non-white populations. Patients with gastric premalignant conditions, intestinal metaplasia and atrophic gastritis, represent a high unmet need for chemopreventative approaches,” said Dr. Douglas R. Morgan, UAB Division of Gastroenterology and Hepatology, The University of Alabama at Birmingham, AL and Dr. Keith T. Wilson, Division of Gastroenterology and Hepatology, Vanderbilt University Medical Center, Nashville, TN. A National Cancer Institute funded, Phase IIa placebo-controlled Randomized Clinical Trial of eflornithine in patients with gastric premalignant conditions was conducted between September 2016 and December 2022 in rural Honduras and Puerto Rico and was an oral presentation at DDW by Dr. Wilson on May 21, 2024. H. pylori (Hp) positive and negative subjects ages 30-60 were treated with eflornithine or placebo for 18 months, with endoscopy at baseline, and 6, 18, and 24 months. At baseline, 80% of subjects were Hp positive, and 46% and 54% had global histology of atrophy and intestinal metaplasia, respectively. A total of 78, 69, and 55 patients reached the 6-month primary outcome, the end-of-treatment 18 month (EoT), and the end-of-study (EoS) 24 month time points, respectively. Eflornithine treatment was safe and well-tolerated. Overall grade 1-2 adverse events (AEs) were greater in the placebo group (108 vs 81) and grade 3 AEs were also higher in the placebo (3 vs 1). The results of the study demonstrated that eflornithine reduced DNA damage long-term in patients after completing treatment, as measured by pH2AX immunostaining, a DNA damage marker was significantly lower at the 24 month vs. 18 month time point in the eflornithine group and unchanged in the placebo group. “Panbela’s focus on the polyamine pathway has implications for both the prevention and treatment of cancer. It is established that pro-tumorigenic and cancer cells are highly dependent on polyamines for survival and the ability of eflornithine to reduce gastric epithelial cell DNA damage may have implications for prevention in those patients at high risk for developing infection-associated gastric cancer,” said Jennifer K. Simpson, PhD, MSN, CRNP, President & Chief Executive Officer of Panbela. Details of the presentation are as follows: Oral Presentation Title: Evaluation of the Safety and Efficacy of Eflornithine (Difluoromethylornithine, DFMO) in Patients with Gastric Premalignant Conditions in the High Incidence Areas of Latin America Session Type: Research Forum Session Title: Chemoprevention for GI Cancers: Drugs and/or Bugs Session Date and Time: May 21, 2024 from 2:00 PM to 3:30 PM EDT (UTC –4) Additional meeting information can be found on the DDW website: https://eppro02.ativ.me/src/EventPilot/php/express/web/planner.php?id=DDWLITE24 The abstract will also be available on the Company's website at https://panbela.com/events-presentations/ once the information has been released by DDW. About Panbela’s PipelineThe pipeline consists of assets currently in clinical trials with an initial focus on familial adenomatous polyposis (FAP), first-line metastatic pancreatic cancer, neoadjuvant pancreatic cancer, colorectal cancer prevention and ovarian cancer. The combined development programs have a steady cadence of anticipated catalysts with programs ranging from pre-clinical to registration studies. Ivospemin (SBP-101)Ivospemin is a proprietary polyamine analogue designed to induce polyamine metabolic inhibition (PMI) by exploiting an observed high affinity of the compound for pancreatic ductal adenocarcinoma and other tumors. It has shown signals of tumor growth inhibition in clinical studies of metastatic pancreatic cancer patients, demonstrating a median overall survival (OS) of 14.6 months and an objective response rate (ORR) of 48%, both exceeding what is typical for the standard of care of gemcitabine + nab-paclitaxel suggesting potential complementary activity with the existing FDA-approved standard chemotherapy regimen. In data evaluated from clinical studies to date, ivospemin has not shown exacerbation of bone marrow suppression and peripheral neuropathy, which can be chemotherapy-related adverse events. Serious visual adverse events have been evaluated and patients with a history of retinopathy or at risk of retinal detachment will be excluded from future SBP-101 studies. The safety data and PMI profile observed in the previous Panbela-sponsored clinical trials provide support for continued evaluation of ivospemin in the ASPIRE trial. Flynpovi ™Flynpovi is a combination of CPP-1X (eflornithine) and sulindac with a dual mechanism inhibiting polyamine synthesis and increasing polyamine export and catabolism. In a Phase III clinical trial in patients with sporadic large bowel polyps, the combination prevented > 90% subsequent pre-cancerous sporadic adenomas versus placebo. Focusing on FAP patients with lower gastrointestinal tract anatomy in the recent Phase III trial comparing Flynpovi to single agent eflornithine and single agent sulindac, FAP patients with lower GI anatomy (patients with an intact colon, retained rectum or surgical pouch), showed statistically significant benefit compared to both single agents (p≤0.02) in delaying surgical events in the lower GI for up to four years. The safety profile for Flynpovi did not significantly differ from the single agents and supports the continued evaluation of Flynpovi for FAP. CPP-1XCPP-1X (eflornithine) is being developed as a single agent tablet or high dose powder sachet for several indications including prevention of gastric cancer, treatment of neuroblastoma and recent onset Type 1 diabetes. Preclinical studies as well as Phase I or Phase II investigator-initiated trials suggest that CPP-1X treatment may be well-tolerated and has potential activity. About PanbelaPanbela Therapeutics, Inc. is a clinical-stage biopharmaceutical company developing disruptive therapeutics for patients with urgent unmet medical needs. Panbela’s lead assets are Ivospemin (SBP-101) and Flynpovi. Further information can be found at www.panbela.com. Panbela’s common stock is eligible for quotation on the OTCQB under the symbol “PBLA”. Cautionary Statement Regarding Forward-Looking StatementsThis press release contains “forward-looking statements,” including within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements can be identified by words such as: “anticipate,” “believe,” “can,” “design,” “expect,” “focus,” “intend,” “looking forward,” “may,” “plan,” “positioned,” “potential,” and “will.” All statements other than statements of historical fact are statements that should be deemed forward-looking statements. Forward-looking statements are neither historical facts nor assurances of future performance. Instead, they are based only on our current beliefs, expectations, and assumptions regarding the future of our business, future plans and strategies, projections, anticipated events and trends, the economy and other future conditions. Because forward-looking statements relate to the future, they are subject to inherent uncertainties, risks and changes in circumstances that are difficult to predict and many of which are outside of our control. Our actual results and financial condition may differ materially and adversely from the forward-looking statements. Therefore, you should not rely on any of these forward-looking statements. Important factors that could cause our actual results and financial condition to differ materially from those indicated in the forward-looking statements include, among others, the following: (i) our ability to obtain additional funding to execute our business and clinical development plans; (ii) progress and success of our clinical development program; (iii) the impact of the current COVID-19 pandemic on our ability to conduct our clinical trials; (iv) our ability to demonstrate the safety and effectiveness of our product candidates: ivospemin (SBP-101) and eflornithine (CPP-1X); (v) our reliance on a third party for the execution of the registration trial for our product candidate Flynpovi ; (vi) our ability to obtain regulatory approvals for our product candidates, SBP-101 and CPP-1X in the United States, the European Union or other international markets; (vii) the market acceptance and level of future sales of our product candidates, SBP-101 and CPP-1X; (viii) the cost and delays in product development that may result from changes in regulatory oversight applicable to our product candidates, SBP-101 and CPP-1X; (ix) the rate of progress in establishing reimbursement arrangements with third-party payors; (x) the effect of competing technological and market developments; (xi) the costs involved in filing and prosecuting patent applications and enforcing or defending patent claims; (xii) our ability to obtain a listing of our common stock on a national securities exchange; and (xii) such other factors as discussed in Part I, Item 1A under the caption “Risk Factors” in our most recent Annual Report on Form 10-K, any additional risks presented in our Quarterly Reports on Form 10-Q and our Current Reports on Form 8-K. Any forward-looking statement made by us in this press release is based on information currently available to us and speaks only as of the date on which it is made. We undertake no obligation to publicly update any forward-looking statement or reasons why actual results would differ from those anticipated in any such forward-looking statement, whether written or oral, whether as a result of new information, future developments or otherwise.