On February 18, 2022 Merck, a leading science and technology company, reported the results of an exploratory analysis from the Phase III JAVELIN Bladder 100 trial with 19 additional months of follow-up data from the initial primary analysis (Press release, Merck & Co, FEB 18, 2022, View Source [SID1234608330]). This analysis reinforced the original results and showed that BAVENCIO (avelumab) plus best supportive care (BSC) in the first-line maintenance setting prolonged median overall survival (OS) by 8.8 months versus BSC alone for patients with locally advanced or metastatic urothelial carcinoma (UC) whose tumors had not progressed on a platinum-based chemotherapy. These results were presented at the 2022 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper)’s annual Genitourinary Cancers Symposium taking place February 17-19, 2022.

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"Since the introduction of the first-line maintenance regimen of BAVENCIO plus best supportive care, it has been recommended with the highest level of evidence in the NCCN, ESMO (Free ESMO Whitepaper), EAU and JUA guidelines, and BAVENCIO first-line maintenance has become a standard of care in the locally advanced and metastatic UC treatment setting. The results from this analysis further reinforce the benefit of first-line maintenance therapy, and of BAVENCIO as the only immunotherapy in the maintenance setting shown to improve survival in this disease," said Thomas Powles, MBBS, MRCP, MD, Professor of Genitourinary Oncology, Lead for Solid Tumor Research at Barts Cancer Institute, Queen Mary University of London, and Director of Barts Cancer Centre, London, UK.

At 38 months median follow-up, patients who received first-line maintenance BAVENCIO plus BSC showed consistent overall survival benefit over patients on BSC alone.

Median OS was 23.8 months (95% CI, 19.9 to 28.8) vs 15.0 months (95% CI, 13.5 to 18.2) (HR 0.76; 95% CI, 0.631 to 0.915).1
43.7% of patients (95% CI, 38.2% to 49.0%) in the BAVENCIO arm were alive at 30 months vs 33.5% (95% CI, 28.4% to 38.7%) of patients who received BSC alone.1
In the population of patients with PD-L1+ tumors (n=358):

Median OS was 30.9 months (95% CI, 24.0-39.8) vs 18.5 months (95% CI, 14.1-24.2) (HR 0.69; 95% CI, 0.521 to 0.901).1
More than half (51.3%; 95% CI, 43.7% to 58.4%) of patients who received BAVENCIO were alive at 30 months vs 38.5% (95% CI, 30.9% to 46.1%) in the BSC arm.1
Median overall survival was measured from the time of randomization, after completion of four to six cycles of platinum-based chemotherapy.

The safety profile for BAVENCIO was consistent with the overall JAVELIN monotherapy clinical development program, with no new safety signals. Patients continued treatment until disease progression, unacceptable toxicity or any other criteria for withdrawal occurred. In the primary population of all randomized patients, 19.5% of patients received ≥2 years of treatment, with 10.2% of patients discontinuing due to treatment-related adverse event with onset after ≥12 months of treatment.

"Locally advanced and metastatic urothelial carcinoma has a low five-year survival rate and high recurrence rate, requiring additional medicines to maintain the benefits of chemotherapy and improve survival rates. The continued improvement in survival in the JAVELIN Bladder 100 trial further supports the use of BAVENCIO as maintenance therapy in patients whose disease has not progressed on first-line platinum-containing chemotherapy and reinforces our commitment to further evaluation of BAVENCIO in advanced bladder cancer," said Victoria Zazulina, MD, Head of Development Unit Oncology, Merck.

About JAVELIN Bladder 100
JAVELIN Bladder 100 (NCT02603432) is a Phase III, multicenter, multinational, randomized, open-label, parallel-arm study investigating first-line maintenance treatment with BAVENCIO plus BSC versus BSC alone in patients with locally advanced or metastatic UC. The primary endpoint was OS in the two primary populations of all patients and patients with PD-L1+ tumors defined by the Ventana SP263 assay. Secondary endpoints included progression-free survival, anti-tumor activity, safety, pharmacokinetics, immunogenicity, predictive biomarkers and patient-reported outcomes in the co-primary populations. All primary and secondary endpoints are measured from the time of randomization.

About Urothelial Carcinoma
Bladder cancer is the tenth most common cancer worldwide.2 In 2020, there were over half a million new cases of bladder cancer diagnosed, with around 200,000 deaths from the disease globally.2 In the US, an estimated 83,730 cases of bladder cancer were diagnosed in 2021, with around 10,000 locally advanced or metastatic cases presented annually.3 UC, which accounts for about 90% of all bladder cancers,4 becomes harder to treat as it advances, spreading through the layers of the bladder wall.5 Only 25% to 55% of patients receive any second-line therapy after first-line chemotherapy.6 In the US and EU5 markets, approximately 40% to 50% of patients receive an immune checkpoint inhibitor in second-line therapy.3 For patients with advanced UC, the five-year survival rate is 6.4%.3

About BAVENCIO (avelumab)
BAVENCIO is a human anti-programmed death ligand-1 (PD-L1) antibody. BAVENCIO has been shown in preclinical models to engage both the adaptive and innate immune functions. By blocking the interaction of PD-L1 with PD-1 receptors, BAVENCIO has been shown to release the suppression of the T cell-mediated antitumor immune response in preclinical models.7-9 In November 2014, Merck and Pfizer announced a strategic alliance to co-develop and co-commercialize BAVENCIO.

BAVENCIO Approved Indications
The European Commission (EC) has authorized the use of BAVENCIO as monotherapy for the first-line maintenance treatment of adult patients with locally advanced or metastatic urothelial carcinoma (UC) who are progression-free following platinum-based chemotherapy. BAVENCIO in combination with axitinib is indicated for the first-line treatment of adult patients with advanced renal cell carcinoma (RCC). BAVENCIO is also authorized by the EC for use as a monotherapy for the treatment of adult patients with metastatic Merkel cell carcinoma (MCC).

In the US, BAVENCIO is indicated for the maintenance treatment of patients with locally advanced or metastatic urothelial carcinoma (UC) that has not progressed with first-line platinum-containing chemotherapy. BAVENCIO is also indicated for the treatment of patients with locally advanced or metastatic UC who have disease progression during or following platinum-containing chemotherapy, or have disease progression within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy.

BAVENCIO in combination with axitinib is indicated in the US for the first-line treatment of patients with advanced RCC. Additionally, the US Food and Drug Administration (FDA) granted accelerated approval for BAVENCIO for the treatment of adults and pediatric patients 12 years and older with metastatic MCC. This indication is approved under accelerated approval based on tumor response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

BAVENCIO is currently approved for at least one indication for patients in more than 50 countries.

BAVENCIO Safety Profile from the EU Summary of Product Characteristics (SmPC)
The special warnings and precautions for use for BAVENCIO monotherapy include infusion-related reactions, as well as immune-related adverse reactions that include pneumonitis and hepatitis (including fatal cases), colitis, pancreatitis (including fatal cases), myocarditis (including fatal cases), endocrinopathies, nephritis and renal dysfunction, and other immune-related adverse reactions. The special warnings and precautions for use for BAVENCIO in combination with axitinib include hepatotoxicity.

The SmPC list of the most common adverse reactions with BAVENCIO monotherapy in patients with solid tumors includes fatigue, nausea, diarrhea, decreased appetite, constipation, infusion-related reactions, weight decreased and vomiting. The list of most common adverse reactions with BAVENCIO in combination with axitinib includes diarrhea, hypertension, fatigue, nausea, dysphonia, decreased appetite, hypothyroidism, cough, headache, dyspnea, and arthralgia.

All Merck Press Releases are distributed by e-mail at the same time they become available on the Merck Website. Please go to www.merckgroup.com/subscribe to register online, change your selection or discontinue this service.

On February 18, 2022 Legend Biotech Corporation (NASDAQ: LEGN) (Legend Biotech), a global, clinical-stage biotechnology company developing and manufacturing novel therapies, reported in conjunction with an announcement to be issued by Legend Biotech’s majority shareholder, GenScript Biotech Corporation, pursuant to the rules of The Stock Exchange of Hong Kong Limited, announced preliminary, unaudited financial results for the year ended December 31, 2021 (Press release, Legend Biotech, FEB 18, 2022, View Source [SID1234608329]).

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For the year ended December 31, 2021, Legend Biotech expects to record a loss for the year of approximately US$365.3 million to US$397.4 million and an adjusted loss for the year of approximately US$335.8 million to US$364.7 million, in each case, including research and development expenses of approximately US$297.9 million to US$321.8 million, which was mainly caused by the continuous investment into its lead product candidate, ciltacabtagene autoleucel (cilta-cel), and other product candidates in Legend Biotech’s pipeline, and selling and marketing expenses of approximately US$95.3 million to US$106.2 million, which was mainly caused by the increase of costs associated with commercial preparation activities for cilta-cel. See "Use of Non-IFRS Financial Measures" below for a reconciliation of Loss for the year to Adjusted loss for the year.

In addition, Legend Biotech expects to report a non-cash fair value loss of approximately US$5.7 million to US$6.4 million caused by the changes of fair value of Legend Biotech’s warrant liability. On May 13, 2021, Legend Biotech entered into a subscription agreement with an institutional investor relating to the offer and sale of 20,809,850 ordinary shares of the Company, par value US$0.0001 per share, in a private placement at a purchase price of US$14.41625 per ordinary share (the "PIPE Offering"). Pursuant to the subscription agreement, the Company also agreed to issue and sell concurrently with the PIPE offering a warrant (the "Warrant") exercisable for up to an aggregate of 10,000,000 ordinary shares (such transaction together with the PIPE Offering, the "Transactions"). The Transactions closed on May 21, 2021(the "Closing Date"). The Warrant is exercisable, in whole or in part, at an exercise price of US$20.00 per ordinary share, until the two-year anniversary of the Closing Date. The Warrant is accounted for as a financial liability because the Warrant may be net share settleable at the holder’s option.

As of December 31, 2021, Legend Biotech had approximately US$688.9 million of cash and cash equivalents, approximately US$168.2 million of time deposits and approximately $29.9 million of financial assets measured at amortized cost.

The financial information contained in this press release is preliminary and is based on the latest estimated unaudited management accounts for the year ended December 31, 2021. Because Legend Biotech has not yet completed its financial closing procedures for the year ended December 31, 2021, Legend Biotech has provided a range for the preliminary results described above. Such information is not a comprehensive statement of Legend Biotech’s results for, and as of, this year, and are subject to the completion of management’s and Legend Biotech’s audit committee’s reviews and other financial closing processes and potential adjustments. Accordingly, Legend Biotech’s actual results as of, and for, the year ended December 31, 2021 may differ materially from the preliminary estimated data presented in this press release. As a result, it is possible that Legend Biotech’s final results will not be within the ranges presented.

The information contained in this press release has not been, and is not based on information that has been, audited, or reviewed by Legend Biotech’s independent auditor. Investors are cautioned not to place undue reliance on these preliminary estimates.

This preliminary estimated data should not be considered a substitute for the audited financial results for the year ended December 31, 2021, to be filed with the Securities and Exchange Commission (the "SEC") on Form 20-F, which Legend Biotech expects to occur before the end of March 2022.

Use of Non-IFRS Financial Measures

We report certain financial information using non-IFRS financial measures, as we believe that these measures provide information that is useful to investors in understanding our performance. These non-IFRS financial measures do not have any standardized meaning and may not be comparable to similar measures used by other companies. For certain non-IFRS financial measures, there are no directly comparable amounts under IFRS. These non-IFRS financial measures should not be viewed as alternatives to measures of financial performance determined in accordance with IFRS.

The following table provides a reconciliation of Legend Biotech’s Loss for the year to Adjusted loss for the year:

Adjusted loss for the year is a non-IFRS financial measure. Legend Biotech is reporting Adjusted loss for the year because this financial measure is to be reported as part of a Profit Warning announcement issued by Legend Biotech’s majority shareholder, GenScript Biotech Corporation, pursuant to Rule 13.09 of the Rules Governing the Listing of Securities on The Stock Exchange of Hong Kong Limited. Adjusted loss for the year has limitations in that it does not reflect all expense items that affect Legend Biotech’s results.

Non-IFRS measures are not meant to be considered in isolation or as a substitute for financial information presented in accordance with IFRS and should be viewed as supplemental and in addition to Legend Biotech’s financial information presented in accordance with IFRS.

About Cilta-cel

Cilta-cel is an investigational chimeric antigen receptor T cell (CAR-T) therapy, formerly identified as JNJ-4528 in the United States and Europe and LCAR-B38M CAR-T cells in China, that is being studied in a comprehensive clinical development program for the treatment of patients with relapsed or refractory multiple myeloma and in earlier lines of treatment. The design consists of a structurally differentiated CAR-T with two BCMA-targeting single domain antibodies. In addition to a Breakthrough Therapy Designation (BTD) granted in the United States in December 2019, cilta-cel received a Priority Medicines (PRiME) designation from the European Commission in April 2019, and a BTD in China in August 2020. In addition, Orphan Drug Designation was granted for cilta-cel by the U.S. Food and Drug Administration (FDA) in February 2019, and by the European Commission in February 2020. A Biologics License Application seeking approval of cilta-cel was submitted to the U.S. FDA and a Marketing Authorization Application was submitted to the European Medicines Agency.

On February 18, 2022 EMD Serono, the Healthcare business of Merck KGaA, Darmstadt, Germany in the US and Canada, reported that the European Commission (EC) has approved once-daily oral TEPMETKO (tepotinib) as monotherapy for the treatment of adult patients with advanced non-small cell lung cancer (NSCLC) harboring alterations leading to mesenchymal-epithelial transition factor gene exon 14 (METex14) skipping, who require systemic therapy following prior treatment with immunotherapy and/or platinum-based chemotherapy (Press release, EMD Serono, FEB 18, 2022, View Source [SID1234608328]).

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"The approval of TEPMETKO provides a much-needed targeted treatment option for patients with advanced non-small cell lung cancer with METex14 skipping alterations," said Professor Egbert Smit, a VISION study investigator at the Netherlands Cancer Institute. "TEPMETKO has demonstrated durable and consistent response rates and has the potential to help patients with this challenging cancer."

The approval is based on results from the pivotal Phase II VISION study evaluating TEPMETKO as monotherapy in patients with advanced NSCLC with METex14 skipping alterations. Data from the primary analysis of the VISION study were previously published online in The New England Journal of Medicine.1

"The approval of TEPMETKO in Europe helps to address the need for targeted treatment options for people with lung cancer who have received prior treatment and whose tumors harbor METex14 skipping alterations," said Dr. Anne-Marie Baird, President of Lung Cancer Europe. "It is vital that biomarker testing is made consistently available and utilized across Europe to ensure people with advanced lung cancer receive an accurate diagnosis and optimal treatment."

In Europe, lung cancer is estimated to be the second most common cancer and the leading cause of cancer-related mortality, responsible for 388,000 deaths in 2018.2 Alterations of the MET signaling pathway, including METex14 skipping alterations, are found in 3% to 4% of NSCLC cases and are associated with advanced disease and poor prognosis.3-7

"With the European Commission’s approval of TEPMETKO, we are now able to bring this important medicine to more patients with this hard-to-treat and aggressive form of lung cancer," said Andrew Paterson, Chief Marketing Officer for the Healthcare business of Merck KGaA, Darmstadt, Germany. "As pioneers in the targeting of the MET signaling pathway, we will now be working on ways to bring this medicine to patients in Europe who may benefit."

About the VISION Study

VISION (NCT02864992) is an ongoing pivotal Phase II, multicenter, multi-cohort, single-arm, non-randomized, open-label study investigating tepotinib as monotherapy. Based on the 01 February 2021 data cut, 275 patients with a median age of 72.6 years with advanced or metastatic NSCLC with METex14 skipping alterations have been analyzed.

About TEPMETKO (tepotinib)

TEPMETKO is a once-daily oral MET inhibitor that inhibits the oncogenic MET receptor signaling caused by MET (gene) alterations. Discovered and developed in-house at Merck KGaA, Darmstadt, Germany, TEPMETKO has a highly selective mechanism of action, with the potential to improve outcomes in aggressive tumors that have a poor prognosis and harbor these specific alterations.

TEPMETKO was the first oral MET inhibitor to receive a regulatory approval anywhere in the world for the treatment of advanced NSCLC harboring MET gene alterations, with its approval in Japan in March 2020. In February 2021, the U.S. Food and Drug Administration granted accelerated approval to TEPMETKO, making it the first and only once-daily oral MET inhibitor approved for patients in the U.S. with metastatic NSCLC with METex14 skipping alterations. Tepotinib is available in a number of countries, and under review by various other regulatory authorities globally. To meet an urgent clinical need, tepotinib is also available in a pilot zone of China in line with the government policy to drive early access for innovative medicines approved outside of China.

Merck KGaA, Darmstadt, Germany is also investigating the potential role of tepotinib in treating patients with NSCLC and acquired resistance due to MET amplification in the Phase II INSIGHT 2 study of tepotinib in combination with osimertinib in MET amplified, advanced or metastatic NSCLC harboring activating EGFR mutations that has progressed following first-line treatment with osimertinib.

Important Safety Information from the US FDA-Approved Label

TEPMETKO can cause interstitial lung disease (ILD)/pneumonitis, which can be fatal. Monitor patients for new or worsening pulmonary symptoms indicative of ILD/pneumonitis (eg, dyspnea, cough, fever). Immediately withhold TEPMETKO in patients with suspected ILD/pneumonitis and permanently discontinue if no other potential causes of ILD/pneumonitis are identified. ILD/pneumonitis occurred in 2.2% of patients treated with TEPMETKO, with one patient experiencing a Grade 3 or higher event; this event resulted in death.

TEPMETKO can cause hepatotoxicity, which can be fatal. Monitor liver function tests (including ALT, AST, and total bilirubin) prior to the start of TEPMETKO, every 2 weeks during the first 3 months of treatment, then once a month or as clinically indicated, with more frequent testing in patients who develop increased transaminases or total bilirubin. Based on the severity of the adverse reaction, withhold, dose reduce, or permanently discontinue TEPMETKO. Increased alanine aminotransferase (ALT)/increased aspartate aminotransferase (AST) occurred in 13% of patients treated with TEPMETKO. Grade 3 or 4 increased ALT/AST occurred in 4.2% of patients. A fatal adverse reaction of hepatic failure occurred in one patient (0.2%). The median time-to-onset of Grade 3 or higher increased ALT/AST was 30 days (range 1 to 178).

TEPMETKO can cause embryo-fetal toxicity. Based on findings in animal studies and its mechanism of action, TEPMETKO can cause fetal harm when administered to a pregnant woman. Advise pregnant women of the potential risk to a fetus. Advise females of reproductive potential or males with female partners of reproductive potential to use effective contraception during treatment with TEPMETKO and for one week after the final dose.

Avoid concomitant use of TEPMETKO with dual strong CYP3A inhibitors and P-gp inhibitors and strong CYP3A inducers. Avoid concomitant use of TEPMETKO with certain P-gp substrates where minimal concentration changes may lead to serious or life-threatening toxicities. If concomitant use is unavoidable, reduce the P-gp substrate dosage if recommended in its approved product labeling.

Fatal adverse reactions occurred in one patient (0.4%) due to pneumonitis, one patient (0.4%) due to hepatic failure, and one patient (0.4%) due to dyspnea from fluid overload.

Serious adverse reactions occurred in 45% of patients who received TEPMETKO. Serious adverse reactions in >2% of patients included pleural effusion (7%), pneumonia (5%), edema (3.9%), dyspnea (3.9%), general health deterioration (3.5%), pulmonary embolism (2%), and musculoskeletal pain (2%).

The most common adverse reactions (≥20%) in patients who received TEPMETKO were edema, fatigue, nausea, diarrhea, musculoskeletal pain, and dyspnea.

Clinically relevant adverse reactions in <10% of patients who received TEPMETKO included ILD/pneumonitis, rash, fever, dizziness, pruritus, and headache.

Selected laboratory abnormalities (≥20%) from baseline in patients receiving TEPMETKO in descending order were: decreased albumin (76%), increased creatinine (55%), increased alkaline phosphatase (ALP) (50%), decreased lymphocytes (48%), increased alanine aminotransferase (ALT) (44%), increased aspartate aminotransferase (AST) (35%), decreased sodium (31%), decreased hemoglobin (27%), increased potassium (25%), increased gamma-glutamyltransferase (GGT) (24%), increased amylase (23%), and decreased leukocytes (23%).

The most common Grade 3 to 4 laboratory abnormalities (≥2%) in descending order were: decreased lymphocytes (11%), decreased albumin (9%), decreased sodium (8%), increased GGT (5%), increased amylase (4.6%), increased ALT (4.1%), increased AST (2.5%), and decreased hemoglobin (2%).

A clinically relevant laboratory abnormality in <20% of patients who received TEPMETKO was increased lipase in 18% of patients, including 3.7% Grades 3 to 4.

For more information about TEPMETKO, please see full Prescribing Information, and visit www.TEPMETKO.com.

Commitment to Cancer

EMD Serono is a science-led organization dedicated to delivering transformative medicines with the goal of making a meaningful difference in the lives of people affected by cancer. Our oncology research efforts aim to leverage our synergistic portfolio in oncogenic pathways, immuno-oncology, and DNA Damage Response (DDR) to tackle challenging tumor types in gastrointestinal, genitourinary, and thoracic cancers. Our curiosity drives our pursuit of treatments for even the most complex cancers, as we work to illuminate a path to scientific breakthroughs that transform patient outcomes. Learn more at www.emdseronooncology.com. Follow us on Twitter: @EMDOncologyUS and LinkedIn: EMD Serono, Inc.

References

Paik PK, Felip E, Veillon R, et al. Tepotinib in non–small-cell lung cancer with MET exon 14 skipping mutations. N Engl J Med 2020; 383:931-943.
Ferlay J, et al. Eur J Cancer. 2018;103:356–387.
Felip E, et al. WCLC 2021. Poster 170.
Reungwetwattana T, et al. Lung Cancer 2017;103:27-37.
Wolf J, et al. EORTC/NCI/AACR 2018. Poster 403.
Schrock AB, et al. J Thorac Oncol. 2016;11:1493–1502.
Tong JH, et al. Clin Cancer Res. 2016;22:3048–3056
TEPMETKO (tepotinib) EU SmPC. View Source Accessed February 2022.
All Merck KGaA, Darmstadt, Germany, press releases are distributed by e-mail at the same time they become available on the EMD Group Website. In case you are a resident of the USA or Canada please go to www.emdgroup.com/subscribe to register again for your online subscription of this service as our newly introduced geo-targeting requires new links in the email. You may later change your selection or discontinue this service.

On February 18, 2022 Reata Pharmaceuticals, Inc. (Nasdaq: RETA) ("Reata," the "Company," "our," "us," or "we"), a clinical-stage biopharmaceutical company, reported that it will report financial results and provide an update on the Company’s business operations and clinical development programs pre-market on February 28, 2022 (Press release, Reata Pharmaceuticals, FEB 18, 2022, View Source [SID1234608327]).

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Conference Call Information

Reata’s management will host a conference call on February 28, 2022, at 8:30 a.m. ET. The conference call will be accessible by dialing (844) 200-6205 (toll-free domestic) or (929) 526-1599 (international) using access code 052919. The webcast link is View Source

Fourth quarter and full year financial results to be discussed during the call will be included in an earnings press release that will be available on the Company’s website shortly before the call at View Source and will be available for 12 months after the call. The audio recording and webcast will be accessible for at least 90 days after the event at View Source.

On February 18, 2022 Orion Biotechnology reported that Results from an Ongoing Phase I/II CYPIDES Trial of ODM-208 Presented at ASCO (Free ASCO Whitepaper)-GU Clinical phase I data on Orion Corporation’s ODM-208, a first-in-class oral, non-steroidal and selective inhibitor of CYP11A1, the first and rate-limiting enzyme of steroid biosynthesis, were presented today at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Genitourinary Cancers Symposium (ASCO GU) (Press release, Orion Biotechnology, FEB 18, 2022, View Source [SID1234608326]). CYPIDES phase 1 data shows that ODM-208 effectively blocked the production of all steroid hormones in men with metastatic castration-resistant prostate cancer (mCRPC) and showed promising anti-tumor activity in men progressing despite extensive prior treatment with both novel hormonal therapies (NHT’s) and taxanes. Treatment responses to ODM-208 especially occurred in men with activating androgen receptor (AR) mutations.

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ODM-208 is a complete blocker of steroid biosynthesis that suppresses the production of all steroid hormones and their precursors that may activate the androgen receptor (AR) signalling pathway. This is particularly relevant in patients with AR ligand binding domain (LBD) activating somatic point mutations, a mechanism of resistance to hormone-based therapies in metastatic castration-resistant prostate cancer (mCRPC). During treatment the patients receive hormone replacement therapy to ensure sufficient adrenal function.

In CYPIDES phase I, a total of 44 patients with median age of 70 years received ODM-208. Overall, 32% of the patients achieved a PSA (prostate specific antigen) decrease of ≥50%. Of patients with AR LBD mutation (17), 68% achieved a PSA decrease of ≥50%. Also, prolonged treatment responses were observed especially in patients with AR LBD mutation. Although tolerated by most patients, the main safety finding was adrenal insufficiency (AI). Overall, 14 (32%) patients experienced severe adrenal insufficiency despite the replacement therapy requiring further adrenal supplementation after which the ODM-208 treatment commonly continued. Non-adrenal adverse events were unremarkable.

Taru Blom (MD, PhD), Vice President, Global Development and CMO, Orion R&D comments:

"ODM-208 is a first-in-class CYP11A1-inhibitor and we are excited that this novel approach works in some of the patients who have only a few effective treatment options available. The first results of CYPIDES are encouraging, and more studies are needed to confirm the potential of ODM-208 as a treatment option for men with advanced prostate cancer. In addition, we are focusing on the safety findings of the study as well as are looking into the optimization of the adrenal balance of the patients through appropriate replacement therapy."

More information about the CYPIDES trial: www.clinicaltrials.gov, Identifier: NCT03436485

The Phase II dose expansion part of CYPIDES is ongoing.

Phase I/II clinical trial CYPIDES

The Phase I/II trial CYPIDES investigates the safety, pharmacokinetics and anti-tumor activity of ODM-208 in men with mCRPC. The Phase I started in 2018 and has recruited 44 patients. More information about the trial: www.clinicaltrials.gov, Identifier: NCT03436485

ODM-208 investigational drug

ODM-208 is a novel, oral, non-steroidal and selective inhibitor of CYP11A1 enzyme developed by Orion for the treatment of prostate cancer. CYP11A1 is the rate-limiting enzyme of the steroid biosynthesis. By inhibiting CYP11A1 enzyme activity, ODM-208 suppresses the production of all steroid hormones and their precursors that may activate AR signalling pathway. Orion is the first pharmaceutical company to develop a selective drug that works by this mechanism.