On May 20, 2021 Medivir AB (Nasdaq Stockholm: MVIR) reported that results from the investigator-initiated phase II clinical study in patients with squamous cell carcinoma (SCC) (Press release, Medivir, MAY 20, 2021, View Source [SID1234580403]). The primary objective of the study was to assess the effects of topical remetinostat on biopsy-proven SCC and SCC in situ tumors. This clinical study was conducted at the Stanford University School of Medicine in California, USA under the leadership of the principal investigator, Dr Kavita Sarin. Medivir is providing remetinostat drug supply for this study, and has full access to, and the rights to use, all clinical data after the study is complete.

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Four patients with five cutaneous SCCs were included in this case series and treated with remetinostat gel 1%. All five tumours, including a range of histological subtypes, demonstrated complete clinical and pathological resolution after up to 8 weeks of treatment. All patients experienced a localized cutaneous reaction in response to the treatment, which required one patient to discontinue therapy. No systemic adverse events were reported. Further details of the study can be found at www.clinicaltrials.gov, reference number NCT03875859.

"These very encouraging results further supports the potential of remetinostat to be used in multiple skin-associated cancers beyond cutaneous T-cell lymphoma (CTCL)", said Magnus Christensen, interim CEO of Medivir.
For more information please contact:
Magnus Christensen, interim CEO, Medivir AB, phone: +46 (0)8 5468 3100.
[email protected]

About squamous cell carcinoma

Squamous cell carcinoma (SCC) is the second most common form of cancer in humans occurring in the skin. Surgical excision is standard of care and there are currently no marketed products approved for the treatment of SCC. Other therapies for SCC exist, such as imiquimod, 5-fluorouracil and photodynamic therapy, however their use is limited to SCC in situ (SCCIS). There is a clear need for efficacious and safe treatments when surgery is impractical, e.g. multiple lesions and/or difficult treatment sites.

About remetinostat

Remetinostat is a topical histone deacetylase (HDAC) inhibitor. A clinical phase II study in mycosis fungoides-cutaneous T-cell-lymphoma (MF-CTCL) has been completed demonstrating that remetinostat reduced severity of CTCL skin lesions with an objective response rate (ORR) of 40%. The study also showed a clinically significant reduction in the severity of pruritus (itching) in 80% of the patients. In addition, two investigator-initiated phase II studies have been conducted at Stanford University in the USA, demonstrating efficacy in both cutaneous Basal Cell Carcinoma (BCC) and Squamous Cell Carcinoma (SCC). In BCC, 25 patients who were treated with remetinostat showed an ORR of 69.7%.

On May 20, 2021 Rubius Therapeutics, Inc. (Nasdaq: RUBY), a clinical-stage biopharmaceutical company that is genetically engineering red blood cells to create an entirely new class of cellular medicines called Red Cell Therapeutics, reported that Pablo J. Cagnoni, M.D., president and chief executive officer, will participate in a fireside chat at the Jefferies Virtual Healthcare Conference on June 2, 2021, at 4:00 p.m. EDT (Press release, Rubius Therapeutics, MAY 20, 2021, View Source [SID1234584707]).

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A live audio webcast will be available within the Investors & Media section of the Rubius Therapeutics website. An archived replay will be accessible for 90 days following the event.

On May 19, 2021 Syros Pharmaceuticals (NASDAQ:SYRS), a leader in the development of medicines that control the expression of genes, reported that it plans to host a three-part key opinion leader (KOL) webcast series on its portfolio of investigational targeted therapies in hematology (Press release, Syros Pharmaceuticals, MAY 19, 2021, View Source [SID1234580241]). The series will consist of presentations from Syros leaders, as well as KOLs, who will review recent progress for SY-1425 in newly diagnosed higher-risk myelodysplastic syndrome (MDS) and newly diagnosed unfit acute myeloid leukemia (AML) and for SY-2101 in acute promyelocytic leukemia (APL), and discuss the unmet need and evolving treatment landscape in these diseases. Each event will be webcast live on Investors & Media section of the Syros website at www.syros.com. An archived replay will be available for approximately 30 days following each presentation.

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The Targeted Hematology Portfolio KOL Webcast Series schedule will be as follows:

SY-1425 in Newly Diagnosed HR-MDS:

Date: Wednesday, May 26
Time: 8:30-10:00am ET
Guest Speaker: Amy DeZern, M.D., M.H.S., Director, Bone Marrow Failure and MDS Program and Associate Professor of Oncology, Johns Hopkins University
SY-1425 in Newly Diagnosed Unfit AML:

Date: Tuesday, June 22
Time: 8:30-10:00am ET
Guest Speaker: Daniel Pollyea, M.D., M.S., Associate Professor of Medicine, Clinical Director of Leukemia Services and Robert H. Allen MD Chair in Hematology Research, University of Colorado School of Medicine
SY-2101 in APL:

Date: Tuesday, July 20
Time: 8:30-10:00am ET
Guest Speaker: Farhad Ravandi, M.D., Janiece and Stephen A. Lasher Professor of Medicine, Chief of Section of Acute Myeloid Leukemia, Department of Leukemia at The University of Texas – MD Anderson Cancer Center

On May 19, 2021 Veracyte, Inc. (Nasdaq: VCYT) reported pivotal clinical validation data showing that the company’s noninvasive nasal swab test can significantly improve the early assessment of lung cancer (Press release, Veracyte, MAY 19, 2021, View Source [SID1234580257]). The new findings show that the Percepta Nasal Swab, a first-of-its-kind genomic test, accurately classifies lung cancer risk in current or former smokers with lung nodules so that those with benign nodules may safely avoid unnecessary additional procedures, while those with likely cancerous nodules may receive more timely diagnosis and treatment. The findings will be presented June 4, 2021 at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting.

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"Lung nodules are often the first sign of lung cancer and cannot be ignored, yet most of them are benign," said Carla R. Lamb, M.D., interventional pulmonologist at Lahey Hospital & Medical Center in Burlington, Mass., who was an investigator on the nasal classifier study. "Today, physicians have limited objective tools to determine which patients with lung nodules found on CT scans have cancer and which don’t. Our findings showed that the nasal swab test can determine, with a high level of accuracy, which patients are at low risk of cancer and can avoid invasive procedures. Similarly, it can identify which patients are at high risk and may be confidently directed to further work-up and, potentially, to the treatment they need. An objective tool that can accurately inform these decisions could be a game-changer for early lung cancer assessment."

For the study, researchers evaluated the performance of the nasal swab test on a blinded, independent validation set of 249 patients from multiple cohorts of prospectively collected nasal samples of current or former smokers undergoing evaluation for lung nodules found on computed tomography (CT). All were followed for up to one year or until physicians made a final, adjudicated diagnosis. The cancer prevalence in the validation cohort was 54% — higher than the 25% cancer prevalence expected in the broad population of patients with suspicious nodules, on which the test is expected to be used.

Results of the validation study demonstrate that the test identifies patients as low risk for cancer with a sensitivity of 96.3% (CI: 91.6%-98.4%) and specificity of 41.7% (CI: 33.1%-50.9%). At the same time, the test identifies patients as high risk for cancer with a specificity of 90.4% (CI: 83.68%-94.57%) and sensitivity of 58.2% (CI: 49.74%-66.22%). These findings show the test classifies more than 40% of patients with confirmed benign nodules as low risk, allowing them to avoid further procedures, and it classifies nearly 60% with confirmed malignant nodules as high risk, enabling them to be directed to more timely diagnosis and potential treatment. The remaining patients were classified as intermediate risk for cancer.

When the test’s performance was applied to a population with 25% cancer prevalence, it showed that the test’s negative predictive value (NPV) is 97.1%, which means that a patient classified as low risk has only a 2.9% risk of malignancy. Similarly, the positive predictive value (PPV) is 67%, meaning that nearly 70% of patients classified as high risk will have lung cancer. The American College of Chest Physicians’ current guidelines recommend diagnostic biopsy for patients with more than 65% cancer risk.

"What is really exciting about these data is that doctors will be able to tell their patients with suspicious lung nodules that they are low risk for cancer and can likely avoid further work-up, with very high certainty that they have not missed a cancer," said Giulia C. Kennedy, Ph.D., Veracyte’s chief scientific officer and chief medical officer. "At the same time, they can be confident in guiding patients who are high risk to further diagnostic procedures, in line with current guidelines. These findings suggest that the Percepta Nasal Swab test will be able to objectively and accurately stratify approximately half of the patients with lung nodules found on CT scans to low or high risk, while those not classified will remain a candidate for current standard of care. We are excited about the opportunity to transform the early assessment of lung nodules with a simple nasal swab test."

The Percepta Nasal Swab test uses advanced genomic and established "field of injury" technology to detect smoking-related damage associated with lung cancer in current or former smokers using a sample collected from the nasal passage. Veracyte developed the final classifier using RNA whole-transcriptome sequencing and machine learning on a rich training set of nasal samples from more than 1,100 patients representing a wide range of lung and tumor biology.

Veracyte expects to begin making the Percepta Nasal Swab test available to a select number of sites in the second half of 2021. The company aims to adapt the test on the nCounter Analysis System in 2022, enabling its expansion to physicians and their patients in global markets in 2023.

The Percepta Nasal Swab test is a key part of Veracyte’s comprehensive lung cancer portfolio, which aims to transform care at every step of the patient journey. Collectively, the company’s tests are leveraging cutting-edge genomic science and technology to provide answers and insights that enable physicians and patients to make better, faster and more confident care decisions. The lung cancer portfolio includes the Percepta Genomic Sequencing Classifier, which helps improve lung cancer diagnosis when bronchoscopy results are inconclusive, and the in-development Percepta Genomic Atlas, which is intended to detect gene alterations that may inform lung cancer treatment decisions, using the same small biopsy that was collected for diagnosis.

Development and validation of the Percepta Nasal Swab test is part of Veracyte’s ongoing collaboration with the Lung Cancer Initiative at Johnson & Johnson.*

Conference Call and Webcast Details

Veracyte will host a conference call and webcast on Thursday, May 20, at 10:00 a.m. Eastern Time to discuss the Percepta Nasal Swab test data. The conference call will be webcast live from the company’s website and will be available via the following link: View Source The webcast should be accessed 10 minutes prior to the conference call start time. A replay of the webcast will be available for one year following the conclusion of the live broadcast and will be accessible on the company’s website at View Source

The conference call can be accessed as follows:

About Lung Cancer

Lung cancer kills more than 1.8 million people worldwide each year.i Early detection is key, with a five-year survival rate of nearly 60 percent when the cancer is found early, compared to 6 percent when it is found at a later stage.ii Lung nodules are typically the first sign of lung cancer, but most lung nodules are benign. Each year in the U.S., an estimated 1.6 million lung nodules are found incidentally on CT scans and, with recently expanded recommendations from the U.S. Preventive Services Task Force, an estimated 15 million Americans are eligible for annual lung cancer CT screening. Physicians currently lack objective tools to determine which lung nodules found on CT are benign and which are cancerous.

On May 19, 2021 Oncternal Therapeutics, Inc. (Nasdaq: ONCT), a clinical-stage biopharmaceutical company focused on the development of novel oncology therapies, reported updated interim clinical data, from the ongoing Phase 1/2 CIRLL (Cirmtuzumab and Ibrutinib targeting ROR1 for Leukemia and Lymphoma) clinical trial, that will be presented in poster form at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2021 Annual Meeting (Press release, Oncternal Therapeutics, MAY 19, 2021, View Source [SID1234580274]). In the CIRLL study, cirmtuzumab, an investigational anti-ROR1 monoclonal antibody, is being evaluated in combination with ibrutinib in patients with MCL and CLL. The clinical trial is being conducted in collaboration with UC San Diego School of Medicine and is partially funded by the California Institute for Regenerative Medicine.

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The updated interim data will be presented at the Hematologic Malignancies—Lymphoma and Chronic Lymphocytic Leukemia session on June 7, 2021 as part of the ASCO (Free ASCO Whitepaper) 2021 Annual Meeting:

Abstract Title: Phase 1/2 Study of Cirmtuzumab and Ibrutinib in Mantle Cell Lymphoma (MCL) or Chronic
Lymphocytic Leukemia (CLL)
Abstract Number: 7556
Session Title: Hematologic Malignancies—Lymphoma and Chronic Lymphocytic Leukemia
Session Date and Time: June 7, 2021 at 11:30 am (Eastern Time)
"These updated clinical data with the combination of cirmtuzumab and ibrutinib remain very encouraging in heavily pre-treated patients with relapsed/refractory MCL, including the impressive 83% best ORR and the durability of complete responses. The enrolled patients also had negative prognostic features, making the results even more compelling. Deep responses to cirmtuzumab plus ibrutinib after prior ibrutinib therapy are particularly intriguing. We look forward to the continuing development of cirmtuzumab," said Hun Ju Lee, M.D., Associate Professor of Medicine in the Department of Lymphoma & Myeloma at the University of Texas MD Anderson Cancer Center, who is an investigator on the CIRLL clinical trial and the first author on the 2021 ASCO (Free ASCO Whitepaper) poster presentation.

"We are pleased that the interim results of the CIRLL study remain strong and consistent with further follow-up, including that median PFS and OS have still not been reached for these heavily pre-treated MCL and CLL patients. Adding cirmtuzumab to ibrutinib appears well tolerated, with no apparent additional toxicities noted to date," said James Breitmeyer, M.D., Ph.D., Oncternal’s President and CEO. "We are particularly pleased with our robust enrollment to date and the number and durability of CRs for the patients with MCL. We remain in active dialogue with the US FDA concerning potential pivotal study designs and the potential pathway to seeking regulatory approval."

The results that will be presented in poster form at ASCO (Free ASCO Whitepaper) 2021 include an increased population of 26 patients with relapsed/refractory MCL enrolled in the dose-finding and dose-expansion cohorts of the CIRLL clinical trial (Part 1 + Part 2), of whom 18 were evaluable for efficacy as of the April 16, 2021 data cut-off date.

Patients had high-risk factors and were heavily pre-treated at study entry, 70% with a high Ki-67 proliferative index (≥30%), 15% with intermediate/high sMIPI prognostic score, and a median of two systemic prior therapies (range 1-5).
The ORR of 83% (15 of 18 evaluable patients), which includes recently enrolled patients with relatively short follow-up time, is comparable to the 87% ORR (13 of 15 evaluable patients) previously presented at the American Society of Hematology (ASH) (Free ASH Whitepaper) 2020 Annual Meeting.
Eight of 18 (44%) evaluable patients achieved a partial response (PR) and two patients (11%) had stable disease (SD), for a total clinical best benefit rate (CR, PR, SD) of 94%.
The complete response rate was 39% (7 of 18 evaluable patients). CRs have remained durable, for 8-30+ months as of the data cutoff date.
The clinical benefit rate and median duration of response were favorable in patients with high-risk features associated with difficult to treat disease:
≥30% Ki-67: Clinical benefit rate of 89%; median duration of response of 14 months (95% CI: 8.66, NE)
>1 prior systemic therapy: Clinical benefit rate of 100%; median duration of response not reached
Four patients had received prior treatment with ibrutinib and all four achieved clinical responses, with two CRs and two PRs.
Median PFS and OS have not been reached, after a median follow-up of 18.9 months, regardless of number of prior systemic therapies, including three recently enrolled evaluable patients with a shorter follow-up time. Further, median PFS has not been reached for patients achieving a CR.
Historical data published for single agent ibrutinib for 370 patients with relapsed/refractory MCL from three clinical trials showed an ORR of 66%, CR rate of 20% and median PFS of 12.8 months (Rule et al., 2017, British Journal of Haematology).
As of the April 16, 2021 data cut-off date, 34 patients with CLL have been enrolled in the dose-finding and dose-confirming cohorts of this clinical trial (Part 1 & Part 2), of which 34 were evaluable for efficacy. The Company plans to present updated data from the randomized cohort (Part 3) in the second half of 2021.

Patients had high-risk factors, and most were heavily pre-treated at study entry, with 85% having RAI staging ≥2, 65% with lymphocytosis, and a median of two systemic prior therapies (range 1-15).
The ORR is similar, 94% (32 of 34 evaluable patients), compared to a 91% ORR presented for 31 of 34 evaluable patients at ASH (Free ASH Whitepaper) 2020.
The CR rate is 15% (5 of 34 evaluable patients), 3 CRs were unconfirmed. Twenty-seven patients (79%) achieved a PR and two patients (6%) had SD, for a total clinical benefit rate (CR, PR, SD) of 100%.
Median PFS and OS have not been reached, after a median follow up of 22.1 months, in this high risk and mostly heavily pre-treated CLL population.
The combination of cirmtuzumab plus ibrutinib has been well tolerated, with treatment emergent adverse events and hematologic abnormalities consistent with, or slightly lower than those reported for ibrutinib alone. There have been no dose-limiting toxicities and no serious adverse events attributed to cirmtuzumab alone.

About the CIRLL Clinical Trial
The CIRLL clinical trial (CIRM-0001) is a Phase 1/2 trial evaluating cirmtuzumab in combination with ibrutinib in separate groups of patients with CLL or MCL. Enrollment of the dose-finding cohorts in CLL and MCL, dose-expansion cohort in CLL and randomized Phase 2 cohort in CLL has been completed. Enrollment of the dose-expansion cohort in MCL is ongoing. Additional information about the CIRM-0001 clinical trial and other clinical trials of cirmtuzumab may be accessed at ClinicalTrials.gov.

About Cirmtuzumab
Cirmtuzumab is an investigational, potentially first-in-class monoclonal antibody targeting ROR1, or Receptor tyrosine kinase-like Orphan Receptor 1. Cirmtuzumab is currently being evaluated in a Phase 1/2 clinical trial in combination with ibrutinib for the treatment of MCL or CLL, in a collaboration with the University of California San Diego (UC San Diego) School of Medicine and the California Institute for Regenerative Medicine (CIRM). In addition, Oncternal is supporting two investigator-sponsored studies being conducted at the UC San Diego School of Medicine: (i) a Phase 1b clinical trial of cirmtuzumab in combination with paclitaxel for the treatment of women with HER2-negative metastatic or locally advanced, unresectable breast cancer, and (ii) a Phase 2 clinical trial of cirmtuzumab in combination with venetoclax, a Bcl-2 inhibitor, in patients with relapsed/refractory CLL.

ROR1 is a potentially attractive target for cancer therapy because it is an onco-embryonic antigen – not usually expressed on adult cells, and its expression confers a survival and fitness advantage when reactivated and expressed by tumor cells. Researchers at the UC San Diego School of Medicine discovered that targeting a critical epitope on ROR1 was key to specifically targeting ROR1 expressing tumors. This led to the development of cirmtuzumab, that binds this critical epitope of ROR1, which is highly expressed on many different cancers but not on normal tissues. Preclinical data showed that when cirmtuzumab bound to ROR1, it blocked Wnt5a signaling, inhibited tumor cell proliferation, migration and survival, and induced differentiation of the tumor cells. The FDA has granted Orphan Drug Designations to cirmtuzumab for the treatment of MCL and CLL/small lymphocytic lymphoma. Cirmtuzumab is in clinical development and has not been approved by the FDA for any indication.