
Rheumatology Guideline Updates Take New Life Course Approach
MANCHESTER, England — The latest guidelines on systemic sclerosis (SSc), axial spondyloarthritis (axSpA), and systemic lupus erythematosus (SLE) from the British Society for Rheumatology (BSR) have been updated and, where appropriate, now consider the full life course of these rheumatologic diseases, experts said at BSR 2025 Annual Meeting.
BSR Clinical Guidelines Program Manager Lindsay Turner told Medscape Medical News that the approach was 'really valuable because often it's hard to get evidence in a pediatric population. The updates now mean that recommendations relevant to that population are included.'
BSR guidelines are generally updated every 5 years, unless evidence becomes available that warrants a 'mini update,' Turner said.
But that time schedule can get stretched out over longer periods, as occurred with the SSc guideline update, as Christopher Denton, MBChB, PhD, professor of experimental rheumatology at UCL Medical School in London, England, told Medscape Medical News .
'Obviously, COVID got in the way for 2 years,' he said. 'And of course, the process itself takes at least 2 years. So I think inevitably that even if you start to do the update, it's going to be about 7-8 years.'
SSc Guideline Highlights
Denton, who is also head of the Centre for Rheumatology at the Royal Free Hospital, London, England, presented highlights only of the updated SSc guideline at the conference because these have already been published.
Christopher Denton, MBChB, PhD
One of the key recommendations he highlighted is to use nailfold capillaroscopy during the diagnosis of SSc, as this is as important as antinuclear antibody testing, taking the history, and a physical examination.
Moreover, all patients diagnosed with SSc should have a baseline assessment done regardless of what treatment plan is being considered and that should include bloodwork, ECGs, echocardiograms, pulmonary function tests, and a high-resolution CT (HRCT).
Discussing the HRCT recommendation, Denton said: 'I think it does reflect the importance of knowing as early as possible whether there is interstitial lung disease present and also to help you follow patients noninvasively over time.'
Another 'cornerstone' of the updated guidance is being vigilant and looking out for potential complications, such as malignancy.
As for treatment, 'the general recommendation, or preference, was that mycophenolate mofetil is the drug that seems to be the most effective for diffuse cutaneous disease and for interstitial lung disease and limited skin involvement,' Denton said.
The guideline also tries to make it clear when autologous hematopoietic stem cell transplantation (AHSCT) may or may not be suitable based on current evidence and states that this approach must be delivered within an experienced specialized center.
As such, the recommendation is that AHSCT may be considered an option for diffuse cutaneous SSc, where the benefit is felt to outweigh any risks. However, if there is severe internal organ disease, then this approach may not be appropriate and careful evaluation is required.
Also, while AHSCT may be considered an option for children and young people who have severe or refractory disease, regardless of whether they have diffuse cutaneous or limited disease, it is not for adults who have later-stage diffuse cutaneous or limited disease because there is not enough evidence currently to support its use, Denton said.
AxSpA Guideline Highlights
As for the updated axSpA guideline on management using biologic and targeted synthetic disease-modifying antirheumatic drugs, Sizheng Steven Zhao, MBChB, PhD, clinical senior lecturer and honorary consultant in rheumatology at The University of Manchester, Manchester, England, said there were three key points.
Sizheng Steven Zhao, MBChB, PhD
First, be open to re-evaluating the diagnosis, Zhao said: 'Getting the diagnosis right can be challenging. Be humble. Be open with your patients about the uncertainty around diagnosis and be willing to revisit that. Re-look at the [MRI] images if treatment response doesn't make sense.'
Second, 'start recording the ASDAS [Ankylosing Spondylitis Disease Activity Score],' Zhao said, in addition to recording disease activity using the Bath Ankylosing Spondylitis Disease Activity Index (BASDAI).
Recording the BASDAI is a requirement of the National Institute for Health and Care Excellence, but using ASDAS is 'where the future is. That's where we're moving toward,' Zhao said. He indicated that this should not be too difficult to incorporate into routine practice given that rheumatologists are already using scores such as the DAS in 28 joints for rheumatoid arthritis.
Third, do not rule out using certain drug classes. 'Although we're blessed with three classes of drug, we only still have three classes of drug for a condition that needs many decades of treatment. Don't reflexively rule out a mechanism of action,' Zhao said.
Specifically, he mentioned not ruling out the use of interleukin 17 inhibitors in a patient who had uveitis or inflammatory bowel disease (IBD). Work with ophthalmologists and gastroenterologists on a case-by-case basis to see if that drug class could still be suitable, Zhao said.
Three overarching principles have been added to the guidance, which consider the goals for treatment, shared care decision-making, and the need for a multidisciplinary approach. Zhao urged his audience to read these and the full guideline, which was published in April. There were 'a lot of nuances,' put into the writing of overarching principles and the 15 recommendations, he said.
The recommendations have been grouped into three broad areas: General, which covers starting, monitoring, and switching treatments; extra-musculoskeletal manifestations (EMMs), which includes uveitis, psoriasis, and IBD; and treatment strategy, which encompasses the treat-to-target approach, tapering, and treatment withdrawal.
The BSR guideline is unique in its discussion of EMMs, Zhao said. This is not done in the American or European guidelines to the same extent: 'We spent that much time thinking about this because, quite frankly, all the therapies have similar efficacy across musculoskeletal features. It is the EMMs that influence which one we choose.'
Zhao emphasized that physical therapy was not to be ignored and that pharmacologic treatments were there to 'enable our patients to continue physical activity, not instead of physical therapy.'
SLE Guideline Highlights
The updated BSR guideline for the management and treatment of SLE is just a few weeks away from publication, said Md Yuzaiful Md Yusof, MBChB, PhD, consultant rheumatologist and senior research fellow at the University of Leeds and Leeds Teaching Hospitals NHS Trust, both in Leeds, England.
Md Yuzaiful Md Yusof, MBChB, PhD
The updated guideline covers a much broader scope than its previous iteration, as it now includes recommendations for the management of children and adolescents, as well as adults. Literature searches were done from inception rather than from where the last guideline left off, 'particularly for the pediatric field,' Md Yusof said.
Detailed guidance on the management of lupus nephritis has been included, and other new features of the guideline were the inclusion of cutaneous lupus, nonpharmacologic care, and the delivery of care, Md Yusof said.
Of course, he added, 'we can't do it all,' and areas not covered were neonatal lupus, contraception and reproductive health, treatment during pregnancy and breastfeeding, complications and comorbidities, and detailed management of thrombosis and antiphospholipid syndrome. However, other national guidelines should already cover these topics.
The guideline included 102 recommendations. 'I know it sounds a bit alarming, but they're quite logical and self-explanatory,' Md Yusof said. Overall, 96 of these are shown in a single infographic which is intended to act as a 'cheat code,' he added.
The recommendations concern diagnosis, assessment and monitoring, management, and the delivery of care. In terms of diagnosis, timing is key, Md Yusof said. When primary care physicians have a strong suspicion of SLE, they should be looking to refer to secondary or tertiary care within 3 weeks, he said.
Treat-to-target is one of the key recommendations regarding assessment and monitoring. The primary treatment goal is to meet the 2021 Definition of Remission in SLE criteria, Md Yusof said. And if that is not possible, the target should be to reach the Lupus Low Disease Activity State.
As for management, there is guidance on what rheumatologists could prescribe for cutaneous disease without consulting a dermatologist, such as non-facial topical glucocorticoids and non-facial topical calcineurin inhibitors.
The of use of the British Isles Lupus Assessment Group (BILAG)-2004 index and SLE Disease Activity Index 2000 to guide management choices was recommended, with the addition of Easy-BILAG, Md Yusof said.
'We recommend all people with mild lupus to be on hydroxychloroquine at a dose of 5 mg/kg of actual body weight per day,' he said. Glucocorticoids could be used as bridging therapy to settle disease flare but not for routine long-term maintenance.
For moderate to severe disease activity, methotrexate or immunosuppression with mycophenolate mofetil, azathioprine, cyclosporin, or tacrolimus is recommended to be started early if there is no organ- or life-threatening disease. Biologics and trials are then advocated for more moderate to severe disease, where there is no renal involvement or if glucocorticoids could not be withdrawn. Trials, belimumab, rituximab, or anifrolumab are recommended for more severe disease activity.
Regarding lupus nephritis, all patients should be managed jointly between rheumatology and renal services.
'Timely biopsy is really key, and also identifying poor prognostic markers from the outset,' Md Yusof said.
A key message regarding glucocorticoid use is to put an end date on the prescription and 'to make sure you have a tapering plan.' Detailed advice is provided in the guideline on how to taper appropriately.
The recommendation on induction treatment for lupus nephritis is the most up-to-date available, with combination therapy recommended over single-agent mycophenolate mofetil. 'Whichever combination that you use for remission induction, you carry on for the maintenance,' Md Yusof added.
He concluded that the British guidelines were 'definitely more directive and also more up-to-date' than other available guidelines.
Turner reported having no relevant financial relationships. Denton reported receiving research and grant funding and consultancy and speaker fees from or acting as a clinical trial investigator and serving on a steering committee for more than 20 companies. Zhao reported receiving consultancy or speaker fees from AbbVie, Alfasigma, Novartis, and UCB. He also acknowledged receiving financial support for attending conferences from Alfasigma, Eli Lilly & Company, Novartis, and UCB. Md Yusof reported acting as an advisory board member, consultant, or speaker for Alumis, Aurinia, GlaxoSmithKline, Novartis, Roche, UCB, and Vifor.
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Perspective Therapeutics Highlights Updated Interim Data from its Ongoing Phase 1/2a Clinical Trial of [212Pb]VMT-α-NET at the 2025 ASCO Annual Meeting
Updated interim results with an additional ~16 weeks of follow-up on nine patients in Cohort 1 (2.5 mCi) and Cohort 2 (5.0 mCi) and initial safety findings in an additional 33 patients treated in Cohort 2 from the ongoing Phase 1/2a study were presented Updated interim results with an additional ~16 weeks of follow-up on nine patients in Cohort 1 (2.5 mCi) and Cohort 2 (5.0 mCi) and initial safety findings in an additional 33 patients treated in Cohort 2 from the ongoing Phase 1/2a study were presented [ 212 Pb]VMT-α-NET continued to have a favorable safety profile, with no dose-limiting toxicities and no discontinuations due to adverse events observed among a total of 42 patients who received at least one treatment Pb]VMT-α-NET continued to have a favorable safety profile, with no dose-limiting toxicities and no discontinuations due to adverse events observed among a total of 42 patients who received at least one treatment Four of seven patients in Cohort 2 experienced investigator-assessed objective responses as defined by RECIST v1.1. Three of those patients experienced confirmed response and remained in response. One new first response is subject to confirmation. Seven of nine patients in Cohorts 1 and 2 remained free from disease progression after more than one year of follow-up Perspective will hold a conference call on Monday, June 2, 2025 at 8:00 am ET SEATTLE, May 30, 2025 (GLOBE NEWSWIRE) — Perspective Therapeutics, Inc. ('Perspective' or the 'Company') (NYSE AMERICAN: CATX), a radiopharmaceutical company pioneering advanced treatments for cancers throughout the body, announced that updated interim results from its ongoing Phase 1/2a clinical trial of [212Pb]VMT-α-NET were presented at the 2025 American Society of Clinical Oncology (ASCO) Annual Meeting taking place May 30-June 3, 2025 in Chicago, Illinois. Advertisement This Phase 1/2a clinical trial is a multi-center open-label dose escalation and dose expansion study ( identifier NCT05636618) of [212Pb]VMT-α-NET in patients with unresectable or metastatic somatostatin receptor type 2 (SSTR2) expressing neuroendocrine tumors (NETs) who have not received prior radiopharmaceutical therapy (RPT) and whose tumors have shown radiological evidence of disease progression in the 12 months prior to enrollment. Updated interim efficacy data were presented for two patients in Cohort 1 and seven patients in Cohort 2 with a data cut-off date of April 30, 2025. These patients were enrolled for dose limiting toxicities observations. Results with a data cut-off date of January 10, 2025 from these patients were previously presented as a poster at the ASCO Gastrointestinal Cancers Symposium (ASCO-GI) in January 2025. Cohort 2 was reopened for enrollment in August 2024, and through April 30, 2025, a further 33 patients were enrolled in Cohort 2 and had received at least one treatment. Safety data were presented at the ASCO Annual Meeting for all 42 patients who had received at least one treatment with [212Pb]VMT-α-NET. Safety findings based on 42 patients who received at least one treatment: As of the data cut-off date of April 30, 2025, no dose limiting toxicities (DLTs), no discontinuations due to adverse events, no Grade 4 or 5 treatment emergent adverse events (TEAEs), and no deaths had been reported since the start of the study. Ten patients had experienced at least one Grade 3 TEAE; the majority of Grade 3 TEAEs were deemed unrelated to [212Pb]VMT-α-NET. Two patients experienced serious adverse events (SAEs), both of which were deemed unrelated to [212Pb]VMT-α-NET. A modest number of patients experienced low grade hematologic toxicities. Observations of lymphocyte count decrease were generally low grade, with three events at Grade 3 and none at Grades 4 or 5. Increases in blood creatinine levels were all at Grade 1. No dysphagia or serious renal complications were reported. Further anti-tumor activity observed in the nine original patients with longer follow-up. As of the data cut-off date of April 30, 2025, there was one new response pending confirmation, and three confirmed responses as defined by RECIST v1.1 in Cohort 2. Seven out of nine patients in Cohorts 1 and 2 continued to experience disease control and remained in study. One patient experienced stable disease for 48 weeks after their first dose prior to experiencing progressive disease. One patient was previously reported to have progressive disease after one dose under RECIST v1.1, by unambiguous progression of non-target lesions. The first patient who experienced a confirmed objective response remained in response for over 10 months and in study. This patient received the first two [212Pb]VMT-α-NET doses at administered dose of 5.0 mCi (equivalent to 84.6 µCi/kg), then received the remaining two doses at the next lower activity level of 2.5 mCi (equivalent to 42.4 µCi/kg). Two patients experienced initial responses after the end of their treatment periods as of the data cut-off date for the previous data presentation at ASCO-GI. Those responses have since been confirmed in subsequent scans. They remained in response and in study. These patients received four doses of 5.0 mCi (equivalent to 68.7 µCi/kg and 31.7 µCi/kg) of [212Pb]VMT-α-NET. A fourth patient was observed to experience an initial (unconfirmed) response in the seventh scan at 48 weeks after their first dose, which was the third scan conducted after the end of their treatment period. This patient received four doses of 5.0 mCi (equivalent to 49.1 µCi/kg) of [212Pb]VMT-α-NET. As stated in our August 12, 2024 business update for the second quarter of 2024, the observation period was completed for DLTs in seven patients enrolled in Cohort 2 during the second quarter of 2024. With no DLTs observed and minimal toxicity overall, the Safety Monitoring Committee (SMC) recommended proceeding with dose escalation and enrolling additional patients at 5 mCi to better understand efficacy and safety. Based on interactions with the U.S. Food and Drug Administration (FDA) prior to the initiation of patient dosing in this study in late 2023, the decision to dose patients in the next cohort will follow consultation and alignment with the FDA. '[212Pb]VMT-α-NET is emerging as an exciting potential treatment option for patients with progressive NETs, with continued durability of anti-tumor activity at the dose level used in Cohort 2 and a favorable tolerability profile seen so far,' said Vikas Prasad, MD, Associate Professor of Radiology, Mallinckrodt Institute of Radiology, Siteman Cancer Center, Washington University School of Medicine. 'I am eager to find more treatments to help my patients fight hard against their disease and return to their normal lives. I look forward to continuing to evaluate [212Pb]VMT-α-NET as a new option to help my patients, by participating in this study.' Markus Puhlmann, Chief Medical Officer of Perspective, commented, 'We are encouraged by the exciting overall clinical profile already observed at the dose level used in Cohort 2. The robust participation in the re-opened Cohort 2 in this dose finding study of [212Pb]VMT-α-NET enables us to learn more about how [212Pb]VMT-α-NET can benefit patients and fit into the current treatment paradigm. We plan to submit longer safety follow-up data for all patients enrolled in the study and preliminary efficacy data for a subgroup for presentation at a scientific congress in the second half of this year. In keeping with the commitment we made to the FDA prior to the start of dosing in this study, we are engaging with the FDA as we wish to continue to pursue dose finding for [212Pb]VMT-α-NET. An update will be provided once alignment is reached with the agency.' Thijs Spoor, Chief Executive Officer of Perspective, commented, '[212Pb]VMT-α-NET is progressing expeditiously as one of three potential new medicines in clinical development based on our next generation targeted radiopharmaceutical technology platform. We continue to evaluate opportunities to enhance our operations and infrastructure so we can support patient demand for all of our programs.' Perspective will webcast a conference call on Monday, June 2, 2025 at 8:00 am ET to discuss the data presented at the ASCO Annual Meeting. Webcast details are available on the Events page of the Company's website. Dr. Prasad will participate, along with members of Perspective's management team. A live question and answer session will follow the formal presentation. About [212Pb]VMT-α-NET Perspective designed [212Pb]VMT-α-NET to target and deliver 212Pb to tumor sites expressing SSTR2. The Company is conducting a multi-center, open-label dose escalation, dose expansion study ( identifier NCT05636618) of [212Pb]VMT-α-NET in patients with unresectable or metastatic SSTR2-positive neuroendocrine tumors who have not received prior radiopharmaceutical therapies (RPT). Results with a data cut-off date of January 10, 2025 from the first nine patients enrolled into Cohorts 1 and 2 of the study were previously presented as a poster at the American Society of Clinical Oncology Gastrointestinal Cancers Symposium (ASCO-GI) in January 2025. Cohort 2 was reopened in August 2024. During 2H 2025, some of the 33 patients enrolled after the cohort reopened and through April 30, 2025, will have had the opportunity for at least 32 weeks of follow-up after their initial doses, sufficient time to receive at least one scan after their full treatment (up to four doses every eight weeks), if they receive all four doses of treatment per protocol. About Neuroendocrine Tumors Neuroendocrine tumors form in cells that interact with the nervous system or in glands that produce hormones. They can originate in various parts of the body, most often in the gut or the lungs and can be benign or malignant. Neuroendocrine tumors are typically classified as pancreatic neuroendocrine tumors or non-pancreatic neuroendocrine tumors. According to it is estimated that more than 12,000 people in the United States are diagnosed with a NET each year. Importantly, neuroendocrine tumors are associated with a relatively long duration of survival compared to other tumors and as a result, there are over 170,000 people living with this diagnosis.1 About Perspective Therapeutics, Inc. Perspective Therapeutics, Inc. is a radiopharmaceutical development company that is pioneering advanced treatments for cancers throughout the body. The Company has proprietary technology that utilizes the alpha-emitting isotope 212Pb to deliver powerful radiation specifically to cancer cells via specialized targeting moieties. The Company is also developing complementary imaging diagnostics that incorporate the same targeting moieties, which provides the opportunity to personalize treatment and optimize patient outcomes. This 'theranostic' approach enables the ability to see the specific tumor and then treat it to potentially improve efficacy and minimize toxicity. The Company's melanoma (VMT01), neuroendocrine tumor (VMT-α-NET) and solid tumor (PSV359) programs are in Phase 1/2a imaging and therapy trials in the U.S. The Company is growing its regional network of drug product candidate finishing facilities, enabled by its proprietary 212Pb generator, to deliver patient-ready product candidates for clinical trials and commercial operations. For more information, please visit the Company's website at Safe Harbor Statement This press release contains forward-looking statements within the meaning of the United States Private Securities Litigation Reform Act of 1995. Statements in this press release that are not statements of historical fact are forward-looking statements. Words such as 'may,' 'will,' 'should,' 'expect,' 'plan,' 'anticipate,' 'could,' 'intend,' 'target,' 'project,' 'estimate,' 'believe,' 'predict,' 'potential,' or 'continue' or the negative of these terms or other similar expressions are intended to identify forward-looking statements, though not all forward-looking statements contain these identifying words. Forward-looking statements in this press release include statements concerning, among other things, the Company's ability to pioneer advanced treatments for cancers throughout the body; the potential for [212Pb]VMT-α-NET to be a treatment option for and benefit patients with progressive NETs with durable anti-tumor activity and a favorable tolerability profile, including at the dose level administered to patients in Cohort 2 of the Company's [212Pb]VMT-α-NET study; the Company's belief that robust participation in the re-opened Cohort 2 in this dose finding study of [212Pb]VMT-α-NET will enable it to learn more about how [212Pb]VMT-α-NET can benefit patients and fit into the current treatment paradigm; the potential for treatments such as [212Pb]VMT-α-NET to help patients fight hard against their disease and return to their normal lives; the Company's clinical development plans and the expected timing thereof; the expected timing for availability and release of additional data from the Company's clinical trials; the Company's anticipated timing and expectations regarding regulatory communications, requests, interactions, submissions, alignment, and approvals, including the Company's expectation that it will reach an alignment with the FDA on dose-finding for [212Pb]VMT-α-NET; the Company's activities and plans to pursue dose escalation for its Phase 1/2a clinical trial of [212Pb]VMT-α-NET; the Company's plans to enhance its operations and infrastructure to support patient demand for all of its programs; the ability of the Company's proprietary technology utilizing the alpha emitting isotope 212Pb to deliver powerful radiation specifically to cancer cells via specialized targeting moieties; the Company's prediction that complementary imaging diagnostics that incorporate certain targeting moieties provide the opportunity to personalize treatment and optimize patient outcomes; the Company's belief that its 'theranostic' approach enables the ability to see a specific tumor and then treat it to potentially improve efficacy and minimize toxicity; the Company's ability to grow its regional network of drug product finishing facilities, enabled by its proprietary 212Pb generator, to deliver patient-ready products for clinical trials and commercial operations; and other statements that are not historical fact. The Company may not actually achieve the plans, intentions, or expectations disclosed in the forward-looking statements, and you should not place undue reliance on the forward-looking statements. These forward-looking statements involve risks and uncertainties that could cause the Company's actual results to differ materially from the results described in or implied by the forward-looking statements. Certain factors that may cause the Company's actual results to differ materially from those expressed or implied in the forward-looking statements in this press release are described under the heading 'Risk Factors' in the Company's most recent Annual Report on Form 10-K filed with the Securities and Exchange Commission (the 'SEC'), in the Company's other filings with the SEC, and in the Company's future reports to be filed with the SEC and available at Forward-looking statements contained in this news release are made as of this date. Unless required to do so by law, we undertake no obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events, or otherwise. Media and Investor Relations Contacts: Perspective Therapeutics IR: Annie J. Cheng, CFA [email protected] Russo Partners, LLC Nic Johnson [email protected] 1 Wu P, He D, Chang H, Zhang X. Epidemiologic trends of and factors associated with overall survival in patients with neuroendocrine tumors over the last two decades in the USA. Endocr Connect. 2023;12(12):e230331. Published 2023 Nov 23. doi:10.1530/EC-23-0331. Disclaimer: The above press release comes to you under an arrangement with GlobeNewswire. Business Upturn takes no editorial responsibility for the same.
Yahoo
8 hours ago
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Perspective Therapeutics Highlights Updated Interim Data from its Ongoing Phase 1/2a Clinical Trial of [212Pb]VMT-α-NET at the 2025 ASCO Annual Meeting
Updated interim results with an additional ~16 weeks of follow-up on nine patients in Cohort 1 (2.5 mCi) and Cohort 2 (5.0 mCi) and initial safety findings in an additional 33 patients treated in Cohort 2 from the ongoing Phase 1/2a study were presented [212Pb]VMT-α-NET continued to have a favorable safety profile, with no dose-limiting toxicities and no discontinuations due to adverse events observed among a total of 42 patients who received at least one treatment Four of seven patients in Cohort 2 experienced investigator-assessed objective responses as defined by RECIST v1.1. Three of those patients experienced confirmed response and remained in response. One new first response is subject to confirmation. Seven of nine patients in Cohorts 1 and 2 remained free from disease progression after more than one year of follow-up Perspective will hold a conference call on Monday, June 2, 2025 at 8:00 am ET SEATTLE, May 30, 2025 (GLOBE NEWSWIRE) -- Perspective Therapeutics, Inc. ('Perspective' or the 'Company') (NYSE AMERICAN: CATX), a radiopharmaceutical company pioneering advanced treatments for cancers throughout the body, announced that updated interim results from its ongoing Phase 1/2a clinical trial of [212Pb]VMT-α-NET were presented at the 2025 American Society of Clinical Oncology (ASCO) Annual Meeting taking place May 30-June 3, 2025 in Chicago, Illinois. This Phase 1/2a clinical trial is a multi-center open-label dose escalation and dose expansion study ( identifier NCT05636618) of [212Pb]VMT-α-NET in patients with unresectable or metastatic somatostatin receptor type 2 (SSTR2) expressing neuroendocrine tumors (NETs) who have not received prior radiopharmaceutical therapy (RPT) and whose tumors have shown radiological evidence of disease progression in the 12 months prior to enrollment. Updated interim efficacy data were presented for two patients in Cohort 1 and seven patients in Cohort 2 with a data cut-off date of April 30, 2025. These patients were enrolled for dose limiting toxicities observations. Results with a data cut-off date of January 10, 2025 from these patients were previously presented as a poster at the ASCO Gastrointestinal Cancers Symposium (ASCO-GI) in January 2025. Cohort 2 was reopened for enrollment in August 2024, and through April 30, 2025, a further 33 patients were enrolled in Cohort 2 and had received at least one treatment. Safety data were presented at the ASCO Annual Meeting for all 42 patients who had received at least one treatment with [212Pb]VMT-α-NET. Safety findings based on 42 patients who received at least one treatment: As of the data cut-off date of April 30, 2025, no dose limiting toxicities (DLTs), no discontinuations due to adverse events, no Grade 4 or 5 treatment emergent adverse events (TEAEs), and no deaths had been reported since the start of the study. Ten patients had experienced at least one Grade 3 TEAE; the majority of Grade 3 TEAEs were deemed unrelated to [212Pb]VMT-α-NET. Two patients experienced serious adverse events (SAEs), both of which were deemed unrelated to [212Pb]VMT-α-NET. A modest number of patients experienced low grade hematologic toxicities. Observations of lymphocyte count decrease were generally low grade, with three events at Grade 3 and none at Grades 4 or 5. Increases in blood creatinine levels were all at Grade 1. No dysphagia or serious renal complications were reported. Further anti-tumor activity observed in the nine original patients with longer follow-up. As of the data cut-off date of April 30, 2025, there was one new response pending confirmation, and three confirmed responses as defined by RECIST v1.1 in Cohort 2. Seven out of nine patients in Cohorts 1 and 2 continued to experience disease control and remained in study. One patient experienced stable disease for 48 weeks after their first dose prior to experiencing progressive disease. One patient was previously reported to have progressive disease after one dose under RECIST v1.1, by unambiguous progression of non-target lesions. The first patient who experienced a confirmed objective response remained in response for over 10 months and in study. This patient received the first two [212Pb]VMT-α-NET doses at administered dose of 5.0 mCi (equivalent to 84.6 µCi/kg), then received the remaining two doses at the next lower activity level of 2.5 mCi (equivalent to 42.4 µCi/kg). Two patients experienced initial responses after the end of their treatment periods as of the data cut-off date for the previous data presentation at ASCO-GI. Those responses have since been confirmed in subsequent scans. They remained in response and in study. These patients received four doses of 5.0 mCi (equivalent to 68.7 µCi/kg and 31.7 µCi/kg) of [212Pb]VMT-α-NET. A fourth patient was observed to experience an initial (unconfirmed) response in the seventh scan at 48 weeks after their first dose, which was the third scan conducted after the end of their treatment period. This patient received four doses of 5.0 mCi (equivalent to 49.1 µCi/kg) of [212Pb]VMT-α-NET. As stated in our August 12, 2024 business update for the second quarter of 2024, the observation period was completed for DLTs in seven patients enrolled in Cohort 2 during the second quarter of 2024. With no DLTs observed and minimal toxicity overall, the Safety Monitoring Committee (SMC) recommended proceeding with dose escalation and enrolling additional patients at 5 mCi to better understand efficacy and safety. Based on interactions with the U.S. Food and Drug Administration (FDA) prior to the initiation of patient dosing in this study in late 2023, the decision to dose patients in the next cohort will follow consultation and alignment with the FDA. "[212Pb]VMT-α-NET is emerging as an exciting potential treatment option for patients with progressive NETs, with continued durability of anti-tumor activity at the dose level used in Cohort 2 and a favorable tolerability profile seen so far," said Vikas Prasad, MD, Associate Professor of Radiology, Mallinckrodt Institute of Radiology, Siteman Cancer Center, Washington University School of Medicine. 'I am eager to find more treatments to help my patients fight hard against their disease and return to their normal lives. I look forward to continuing to evaluate [212Pb]VMT-α-NET as a new option to help my patients, by participating in this study.' Markus Puhlmann, Chief Medical Officer of Perspective, commented, 'We are encouraged by the exciting overall clinical profile already observed at the dose level used in Cohort 2. The robust participation in the re-opened Cohort 2 in this dose finding study of [212Pb]VMT-α-NET enables us to learn more about how [212Pb]VMT-α-NET can benefit patients and fit into the current treatment paradigm. We plan to submit longer safety follow-up data for all patients enrolled in the study and preliminary efficacy data for a subgroup for presentation at a scientific congress in the second half of this year. In keeping with the commitment we made to the FDA prior to the start of dosing in this study, we are engaging with the FDA as we wish to continue to pursue dose finding for [212Pb]VMT-α-NET. An update will be provided once alignment is reached with the agency.' Thijs Spoor, Chief Executive Officer of Perspective, commented, '[212Pb]VMT-α-NET is progressing expeditiously as one of three potential new medicines in clinical development based on our next generation targeted radiopharmaceutical technology platform. We continue to evaluate opportunities to enhance our operations and infrastructure so we can support patient demand for all of our programs.' Perspective will webcast a conference call on Monday, June 2, 2025 at 8:00 am ET to discuss the data presented at the ASCO Annual Meeting. Webcast details are available on the Events page of the Company's website. Dr. Prasad will participate, along with members of Perspective's management team. A live question and answer session will follow the formal presentation. About [212Pb]VMT-α-NETPerspective designed [212Pb]VMT-α-NET to target and deliver 212Pb to tumor sites expressing SSTR2. The Company is conducting a multi-center, open-label dose escalation, dose expansion study ( identifier NCT05636618) of [212Pb]VMT-α-NET in patients with unresectable or metastatic SSTR2-positive neuroendocrine tumors who have not received prior radiopharmaceutical therapies (RPT). Results with a data cut-off date of January 10, 2025 from the first nine patients enrolled into Cohorts 1 and 2 of the study were previously presented as a poster at the American Society of Clinical Oncology Gastrointestinal Cancers Symposium (ASCO-GI) in January 2025. Cohort 2 was reopened in August 2024. During 2H 2025, some of the 33 patients enrolled after the cohort reopened and through April 30, 2025, will have had the opportunity for at least 32 weeks of follow-up after their initial doses, sufficient time to receive at least one scan after their full treatment (up to four doses every eight weeks), if they receive all four doses of treatment per protocol. About Neuroendocrine TumorsNeuroendocrine tumors form in cells that interact with the nervous system or in glands that produce hormones. They can originate in various parts of the body, most often in the gut or the lungs and can be benign or malignant. Neuroendocrine tumors are typically classified as pancreatic neuroendocrine tumors or non-pancreatic neuroendocrine tumors. According to it is estimated that more than 12,000 people in the United States are diagnosed with a NET each year. Importantly, neuroendocrine tumors are associated with a relatively long duration of survival compared to other tumors and as a result, there are over 170,000 people living with this diagnosis.1 About Perspective Therapeutics, Inc. Perspective Therapeutics, Inc. is a radiopharmaceutical development company that is pioneering advanced treatments for cancers throughout the body. The Company has proprietary technology that utilizes the alpha-emitting isotope 212Pb to deliver powerful radiation specifically to cancer cells via specialized targeting moieties. The Company is also developing complementary imaging diagnostics that incorporate the same targeting moieties, which provides the opportunity to personalize treatment and optimize patient outcomes. This "theranostic" approach enables the ability to see the specific tumor and then treat it to potentially improve efficacy and minimize toxicity. The Company's melanoma (VMT01), neuroendocrine tumor (VMT-α-NET) and solid tumor (PSV359) programs are in Phase 1/2a imaging and therapy trials in the U.S. The Company is growing its regional network of drug product candidate finishing facilities, enabled by its proprietary 212Pb generator, to deliver patient-ready product candidates for clinical trials and commercial operations. For more information, please visit the Company's website at Safe Harbor Statement This press release contains forward-looking statements within the meaning of the United States Private Securities Litigation Reform Act of 1995. Statements in this press release that are not statements of historical fact are forward-looking statements. Words such as "may," "will," "should," "expect," "plan," "anticipate," "could," "intend," "target," "project," "estimate," "believe," "predict," "potential," or "continue" or the negative of these terms or other similar expressions are intended to identify forward-looking statements, though not all forward-looking statements contain these identifying words. Forward-looking statements in this press release include statements concerning, among other things, the Company's ability to pioneer advanced treatments for cancers throughout the body; the potential for [212Pb]VMT-α-NET to be a treatment option for and benefit patients with progressive NETs with durable anti-tumor activity and a favorable tolerability profile, including at the dose level administered to patients in Cohort 2 of the Company's [212Pb]VMT-α-NET study; the Company's belief that robust participation in the re-opened Cohort 2 in this dose finding study of [212Pb]VMT-α-NET will enable it to learn more about how [212Pb]VMT-α-NET can benefit patients and fit into the current treatment paradigm; the potential for treatments such as [212Pb]VMT-α-NET to help patients fight hard against their disease and return to their normal lives; the Company's clinical development plans and the expected timing thereof; the expected timing for availability and release of additional data from the Company's clinical trials; the Company's anticipated timing and expectations regarding regulatory communications, requests, interactions, submissions, alignment, and approvals, including the Company's expectation that it will reach an alignment with the FDA on dose-finding for [212Pb]VMT-α-NET; the Company's activities and plans to pursue dose escalation for its Phase 1/2a clinical trial of [212Pb]VMT-α-NET; the Company's plans to enhance its operations and infrastructure to support patient demand for all of its programs; the ability of the Company's proprietary technology utilizing the alpha emitting isotope 212Pb to deliver powerful radiation specifically to cancer cells via specialized targeting moieties; the Company's prediction that complementary imaging diagnostics that incorporate certain targeting moieties provide the opportunity to personalize treatment and optimize patient outcomes; the Company's belief that its "theranostic" approach enables the ability to see a specific tumor and then treat it to potentially improve efficacy and minimize toxicity; the Company's ability to grow its regional network of drug product finishing facilities, enabled by its proprietary 212Pb generator, to deliver patient-ready products for clinical trials and commercial operations; and other statements that are not historical fact. The Company may not actually achieve the plans, intentions, or expectations disclosed in the forward-looking statements, and you should not place undue reliance on the forward-looking statements. These forward-looking statements involve risks and uncertainties that could cause the Company's actual results to differ materially from the results described in or implied by the forward-looking statements. Certain factors that may cause the Company's actual results to differ materially from those expressed or implied in the forward-looking statements in this press release are described under the heading "Risk Factors" in the Company's most recent Annual Report on Form 10-K filed with the Securities and Exchange Commission (the "SEC"), in the Company's other filings with the SEC, and in the Company's future reports to be filed with the SEC and available at Forward-looking statements contained in this news release are made as of this date. Unless required to do so by law, we undertake no obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events, or otherwise. Media and Investor Relations Contacts: Perspective Therapeutics IR:Annie J. Cheng, CFAir@ Russo Partners, LLCNic JohnsonPerspectiveIR@ 1 Wu P, He D, Chang H, Zhang X. Epidemiologic trends of and factors associated with overall survival in patients with neuroendocrine tumors over the last two decades in the USA. Endocr Connect. 2023;12(12):e230331. Published 2023 Nov 23. doi:10.1530/EC-23-0331.
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Perspective Therapeutics Highlights Updated Interim Data from its Ongoing Phase 1/2a Clinical Trial of [212Pb]VMT-α-NET at the 2025 ASCO Annual Meeting
Updated interim results with an additional ~16 weeks of follow-up on nine patients in Cohort 1 (2.5 mCi) and Cohort 2 (5.0 mCi) and initial safety findings in an additional 33 patients treated in Cohort 2 from the ongoing Phase 1/2a study were presented [212Pb]VMT-α-NET continued to have a favorable safety profile, with no dose-limiting toxicities and no discontinuations due to adverse events observed among a total of 42 patients who received at least one treatment Four of seven patients in Cohort 2 experienced investigator-assessed objective responses as defined by RECIST v1.1. Three of those patients experienced confirmed response and remained in response. One new first response is subject to confirmation. Seven of nine patients in Cohorts 1 and 2 remained free from disease progression after more than one year of follow-up Perspective will hold a conference call on Monday, June 2, 2025 at 8:00 am ET SEATTLE, May 30, 2025 (GLOBE NEWSWIRE) -- Perspective Therapeutics, Inc. ('Perspective' or the 'Company') (NYSE AMERICAN: CATX), a radiopharmaceutical company pioneering advanced treatments for cancers throughout the body, announced that updated interim results from its ongoing Phase 1/2a clinical trial of [212Pb]VMT-α-NET were presented at the 2025 American Society of Clinical Oncology (ASCO) Annual Meeting taking place May 30-June 3, 2025 in Chicago, Illinois. This Phase 1/2a clinical trial is a multi-center open-label dose escalation and dose expansion study ( identifier NCT05636618) of [212Pb]VMT-α-NET in patients with unresectable or metastatic somatostatin receptor type 2 (SSTR2) expressing neuroendocrine tumors (NETs) who have not received prior radiopharmaceutical therapy (RPT) and whose tumors have shown radiological evidence of disease progression in the 12 months prior to enrollment. Updated interim efficacy data were presented for two patients in Cohort 1 and seven patients in Cohort 2 with a data cut-off date of April 30, 2025. These patients were enrolled for dose limiting toxicities observations. Results with a data cut-off date of January 10, 2025 from these patients were previously presented as a poster at the ASCO Gastrointestinal Cancers Symposium (ASCO-GI) in January 2025. Cohort 2 was reopened for enrollment in August 2024, and through April 30, 2025, a further 33 patients were enrolled in Cohort 2 and had received at least one treatment. Safety data were presented at the ASCO Annual Meeting for all 42 patients who had received at least one treatment with [212Pb]VMT-α-NET. Safety findings based on 42 patients who received at least one treatment: As of the data cut-off date of April 30, 2025, no dose limiting toxicities (DLTs), no discontinuations due to adverse events, no Grade 4 or 5 treatment emergent adverse events (TEAEs), and no deaths had been reported since the start of the study. Ten patients had experienced at least one Grade 3 TEAE; the majority of Grade 3 TEAEs were deemed unrelated to [212Pb]VMT-α-NET. Two patients experienced serious adverse events (SAEs), both of which were deemed unrelated to [212Pb]VMT-α-NET. A modest number of patients experienced low grade hematologic toxicities. Observations of lymphocyte count decrease were generally low grade, with three events at Grade 3 and none at Grades 4 or 5. Increases in blood creatinine levels were all at Grade 1. No dysphagia or serious renal complications were reported. Further anti-tumor activity observed in the nine original patients with longer follow-up. As of the data cut-off date of April 30, 2025, there was one new response pending confirmation, and three confirmed responses as defined by RECIST v1.1 in Cohort 2. Seven out of nine patients in Cohorts 1 and 2 continued to experience disease control and remained in study. One patient experienced stable disease for 48 weeks after their first dose prior to experiencing progressive disease. One patient was previously reported to have progressive disease after one dose under RECIST v1.1, by unambiguous progression of non-target lesions. The first patient who experienced a confirmed objective response remained in response for over 10 months and in study. This patient received the first two [212Pb]VMT-α-NET doses at administered dose of 5.0 mCi (equivalent to 84.6 µCi/kg), then received the remaining two doses at the next lower activity level of 2.5 mCi (equivalent to 42.4 µCi/kg). Two patients experienced initial responses after the end of their treatment periods as of the data cut-off date for the previous data presentation at ASCO-GI. Those responses have since been confirmed in subsequent scans. They remained in response and in study. These patients received four doses of 5.0 mCi (equivalent to 68.7 µCi/kg and 31.7 µCi/kg) of [212Pb]VMT-α-NET. A fourth patient was observed to experience an initial (unconfirmed) response in the seventh scan at 48 weeks after their first dose, which was the third scan conducted after the end of their treatment period. This patient received four doses of 5.0 mCi (equivalent to 49.1 µCi/kg) of [212Pb]VMT-α-NET. As stated in our August 12, 2024 business update for the second quarter of 2024, the observation period was completed for DLTs in seven patients enrolled in Cohort 2 during the second quarter of 2024. With no DLTs observed and minimal toxicity overall, the Safety Monitoring Committee (SMC) recommended proceeding with dose escalation and enrolling additional patients at 5 mCi to better understand efficacy and safety. Based on interactions with the U.S. Food and Drug Administration (FDA) prior to the initiation of patient dosing in this study in late 2023, the decision to dose patients in the next cohort will follow consultation and alignment with the FDA. "[212Pb]VMT-α-NET is emerging as an exciting potential treatment option for patients with progressive NETs, with continued durability of anti-tumor activity at the dose level used in Cohort 2 and a favorable tolerability profile seen so far," said Vikas Prasad, MD, Associate Professor of Radiology, Mallinckrodt Institute of Radiology, Siteman Cancer Center, Washington University School of Medicine. 'I am eager to find more treatments to help my patients fight hard against their disease and return to their normal lives. I look forward to continuing to evaluate [212Pb]VMT-α-NET as a new option to help my patients, by participating in this study.' Markus Puhlmann, Chief Medical Officer of Perspective, commented, 'We are encouraged by the exciting overall clinical profile already observed at the dose level used in Cohort 2. The robust participation in the re-opened Cohort 2 in this dose finding study of [212Pb]VMT-α-NET enables us to learn more about how [212Pb]VMT-α-NET can benefit patients and fit into the current treatment paradigm. We plan to submit longer safety follow-up data for all patients enrolled in the study and preliminary efficacy data for a subgroup for presentation at a scientific congress in the second half of this year. In keeping with the commitment we made to the FDA prior to the start of dosing in this study, we are engaging with the FDA as we wish to continue to pursue dose finding for [212Pb]VMT-α-NET. An update will be provided once alignment is reached with the agency.' Thijs Spoor, Chief Executive Officer of Perspective, commented, '[212Pb]VMT-α-NET is progressing expeditiously as one of three potential new medicines in clinical development based on our next generation targeted radiopharmaceutical technology platform. We continue to evaluate opportunities to enhance our operations and infrastructure so we can support patient demand for all of our programs.' Perspective will webcast a conference call on Monday, June 2, 2025 at 8:00 am ET to discuss the data presented at the ASCO Annual Meeting. Webcast details are available on the Events page of the Company's website. Dr. Prasad will participate, along with members of Perspective's management team. A live question and answer session will follow the formal presentation. About [212Pb]VMT-α-NETPerspective designed [212Pb]VMT-α-NET to target and deliver 212Pb to tumor sites expressing SSTR2. The Company is conducting a multi-center, open-label dose escalation, dose expansion study ( identifier NCT05636618) of [212Pb]VMT-α-NET in patients with unresectable or metastatic SSTR2-positive neuroendocrine tumors who have not received prior radiopharmaceutical therapies (RPT). Results with a data cut-off date of January 10, 2025 from the first nine patients enrolled into Cohorts 1 and 2 of the study were previously presented as a poster at the American Society of Clinical Oncology Gastrointestinal Cancers Symposium (ASCO-GI) in January 2025. Cohort 2 was reopened in August 2024. During 2H 2025, some of the 33 patients enrolled after the cohort reopened and through April 30, 2025, will have had the opportunity for at least 32 weeks of follow-up after their initial doses, sufficient time to receive at least one scan after their full treatment (up to four doses every eight weeks), if they receive all four doses of treatment per protocol. About Neuroendocrine TumorsNeuroendocrine tumors form in cells that interact with the nervous system or in glands that produce hormones. They can originate in various parts of the body, most often in the gut or the lungs and can be benign or malignant. Neuroendocrine tumors are typically classified as pancreatic neuroendocrine tumors or non-pancreatic neuroendocrine tumors. According to it is estimated that more than 12,000 people in the United States are diagnosed with a NET each year. Importantly, neuroendocrine tumors are associated with a relatively long duration of survival compared to other tumors and as a result, there are over 170,000 people living with this diagnosis.1 About Perspective Therapeutics, Inc. Perspective Therapeutics, Inc. is a radiopharmaceutical development company that is pioneering advanced treatments for cancers throughout the body. The Company has proprietary technology that utilizes the alpha-emitting isotope 212Pb to deliver powerful radiation specifically to cancer cells via specialized targeting moieties. The Company is also developing complementary imaging diagnostics that incorporate the same targeting moieties, which provides the opportunity to personalize treatment and optimize patient outcomes. This "theranostic" approach enables the ability to see the specific tumor and then treat it to potentially improve efficacy and minimize toxicity. The Company's melanoma (VMT01), neuroendocrine tumor (VMT-α-NET) and solid tumor (PSV359) programs are in Phase 1/2a imaging and therapy trials in the U.S. The Company is growing its regional network of drug product candidate finishing facilities, enabled by its proprietary 212Pb generator, to deliver patient-ready product candidates for clinical trials and commercial operations. For more information, please visit the Company's website at Safe Harbor Statement This press release contains forward-looking statements within the meaning of the United States Private Securities Litigation Reform Act of 1995. Statements in this press release that are not statements of historical fact are forward-looking statements. Words such as "may," "will," "should," "expect," "plan," "anticipate," "could," "intend," "target," "project," "estimate," "believe," "predict," "potential," or "continue" or the negative of these terms or other similar expressions are intended to identify forward-looking statements, though not all forward-looking statements contain these identifying words. Forward-looking statements in this press release include statements concerning, among other things, the Company's ability to pioneer advanced treatments for cancers throughout the body; the potential for [212Pb]VMT-α-NET to be a treatment option for and benefit patients with progressive NETs with durable anti-tumor activity and a favorable tolerability profile, including at the dose level administered to patients in Cohort 2 of the Company's [212Pb]VMT-α-NET study; the Company's belief that robust participation in the re-opened Cohort 2 in this dose finding study of [212Pb]VMT-α-NET will enable it to learn more about how [212Pb]VMT-α-NET can benefit patients and fit into the current treatment paradigm; the potential for treatments such as [212Pb]VMT-α-NET to help patients fight hard against their disease and return to their normal lives; the Company's clinical development plans and the expected timing thereof; the expected timing for availability and release of additional data from the Company's clinical trials; the Company's anticipated timing and expectations regarding regulatory communications, requests, interactions, submissions, alignment, and approvals, including the Company's expectation that it will reach an alignment with the FDA on dose-finding for [212Pb]VMT-α-NET; the Company's activities and plans to pursue dose escalation for its Phase 1/2a clinical trial of [212Pb]VMT-α-NET; the Company's plans to enhance its operations and infrastructure to support patient demand for all of its programs; the ability of the Company's proprietary technology utilizing the alpha emitting isotope 212Pb to deliver powerful radiation specifically to cancer cells via specialized targeting moieties; the Company's prediction that complementary imaging diagnostics that incorporate certain targeting moieties provide the opportunity to personalize treatment and optimize patient outcomes; the Company's belief that its "theranostic" approach enables the ability to see a specific tumor and then treat it to potentially improve efficacy and minimize toxicity; the Company's ability to grow its regional network of drug product finishing facilities, enabled by its proprietary 212Pb generator, to deliver patient-ready products for clinical trials and commercial operations; and other statements that are not historical fact. The Company may not actually achieve the plans, intentions, or expectations disclosed in the forward-looking statements, and you should not place undue reliance on the forward-looking statements. These forward-looking statements involve risks and uncertainties that could cause the Company's actual results to differ materially from the results described in or implied by the forward-looking statements. Certain factors that may cause the Company's actual results to differ materially from those expressed or implied in the forward-looking statements in this press release are described under the heading "Risk Factors" in the Company's most recent Annual Report on Form 10-K filed with the Securities and Exchange Commission (the "SEC"), in the Company's other filings with the SEC, and in the Company's future reports to be filed with the SEC and available at Forward-looking statements contained in this news release are made as of this date. Unless required to do so by law, we undertake no obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events, or otherwise. Media and Investor Relations Contacts: Perspective Therapeutics IR:Annie J. Cheng, CFAir@ Russo Partners, LLCNic JohnsonPerspectiveIR@ 1 Wu P, He D, Chang H, Zhang X. Epidemiologic trends of and factors associated with overall survival in patients with neuroendocrine tumors over the last two decades in the USA. Endocr Connect. 2023;12(12):e230331. Published 2023 Nov 23. doi:10.1530/ in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data