On May 14, 2020 Novartis reported that data from more than 110 abstracts, including Novartis-sponsored and investigator-initiated trials, will be presented during the upcoming American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) ASCO (Free ASCO Whitepaper)20 Virtual Scientific Program and the European Hematology Association (EHA) (Free EHA Whitepaper) EHA (Free EHA Whitepaper)25 Virtual Congress (Press release, Novartis, MAY 14, 2020, View Source [SID1234558037]). The ASCO (Free ASCO Whitepaper) and EHA (Free EHA Whitepaper) meetings will be held May 29-31, and June 11-14, respectively.

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"We are living in a world of uncertainty, but cancer won’t wait. Now, more than ever, we need to continue to be bold together. Our data at ASCO (Free ASCO Whitepaper) and EHA (Free EHA Whitepaper) highlight our unique approach to harnessing the power of multiple treatment platforms to deliver transformative medicines to people living with cancer and blood disorders," said Susanne Schaffert, PhD, President, Novartis Oncology. "We look forward to ‘seeing’ everyone virtually at the congresses and helping participants access key data and information through our dedicated congress portals."

Key highlights of data accepted by ASCO (Free ASCO Whitepaper):

Kisqali overall survival subgroup analysis from MONALEESA-3 and -7 trials, and results on Piqray plus fulvestrant in ABC patients with a PIK3CA mutation from the BYLieve study:
Overall survival in patients with ABC with visceral metastases (mets), including those with liver mets, treated with ribociclib plus endocrine therapy in the MONALEESA-3 and -7 trials [Abstract # 1054; poster 139]
Alpelisib + fulvestrant in patients with PIK3CA-mutated HR+/HER2- ABC previously treated with cyclin-dependent kinase 4/6 inhibitor + aromatase inhibitor: BYLieve Study Results [Abstract #1006; oral presentation]

Five-year Tafinlar+Mekinist data in the adjuvant treatment of BRAFV600-mutated melanoma, and updated data in combination with immune checkpoint inhibitor spartalizumab (PDR001):
Long-term benefit of adjuvant dabrafenib+trametinib in patients with resected stage III BRAF V600–mutant melanoma: 5-year analysis of COMBI-AD [Abstract #10001; oral presentation]
The anti-PD-1 antibody spartalizumab in combination with dabrafenib and trametinib in advanced BRAF V600-mutant melanoma: efficacy and safety findings from Parts 1 and 2 of the Phase III COMBI-i trial [Abstract #10028; poster 377]

Tabrecta data updates from multiple analyses from the GEOMETRY study among patients with METex14-mutated and MET-amplified advanced non-small cell lung cancer (NSCLC):
Capmatinib in patients with METex14-mutated advanced NSCLC who received prior immunotherapy: results from the Phase 2 GEOMETRY Mono-1 study [Abstract #9509; oral presentation]
Capmatinib in patients with METex14-mutated or high MET-amplified advanced NSCLC: results from Cohort 6 of the phase 2 GEOMETRY mono-1 study [Abstract #9520; poster 286]

Safety data from a US expanded access program with radioligand therapy Lutathera (lutetium Lu 177 dotatate)*** in patients with advanced neuroendocrine tumors (NETs):
Safety of 177Lu‑DOTATATE in patients with advanced NETs: data from a US expanded access program [Abstract #4604; poster 212]

Learnings from the inclusion of patient insights in the research and development process, through the Novartis Global Oncology Patient Insight Panels (GOPIPs):
Patient engagement in clinical trial design and implementation: A pragmatic approach to valued insights [Abstract #e14084; online publication]
In addition, TheraP, sponsored by the Australian & New Zealand Urogenital and Prostate (ANZUP) Cancer Trials Group, comparing investigational radioligand therapy 177Lu-PSMA-617 to cabazitaxel in patients with metastatic castration resistant prostate cancer (mCRPC) progressing after docetaxel, will be presented:

TheraP: A randomised phase II trial of LuPSMA theranostic versus cabazitaxel in mCRPC progressing after docetaxel: Initial results (ANZUP protocol 1603) [Abstract #5500; oral presentation]
Key highlights of data accepted by EHA (Free EHA Whitepaper):

Efficacy and safety data on the investigational anti-TIM-3 monoclonal antibody MBG453, which targets both immune and myeloid cells in patients with high-risk myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML):
Anti-TIM-3 antibody MBG453 in combination with hypomethylating agents in patients with high-risk MDS and AML: a Phase 1 study [Abstract #S185; oral presentation; Sunday, June 14, 8:00 AM CEST]

A longer term Phase I safety and efficacy analysis of asciminib, an investigational treatment specifically targeting the BCR-ABL myristoyl pocket (STAMP), in heavily pre-treated patients with chronic myeloid leukemia (CML):
Asciminib in heavily pretreated patients with Philadelphia chromosome-positive (Ph+) CML in chronic phase sensitive to TKI therapy [Abstract #S170; oral presentation; Friday, June 12, 8:30 AM CEST]

Efficacy results in patients with acute graft-versus-host disease (GvHD) treated with Jakavi (ruxolitinib)****:
Ruxolitinib versus best available therapy in patients with steroid-refractory acute GvHD: overall response rate by baseline characteristics in the randomized Phase 3 REACH2 trial [Abstract #S255; oral presentation; Friday, June 12, 8:00 AM CEST]
More information, including the list of Novartis-sponsored abstracts, and access to the presentations for registered participants will be available on View Source, starting on May 28, and on View Source, by June 11.

On May 14, 2020 INOVIO (NASDAQ: INO) reported that 85 percent (44 out of 52) of patients newly diagnosed with the deadly brain cancer glioblastoma multiforme (GBM) who received the company’s DNA medicine INO-5401, in combination with INO-9012 and PD-1 inhibitor Libtayo (cemiplimab), were alive for at least 12 months or more (overall survival at 12 months: OS12) following treatment (Press release, Inovio, MAY 14, 2020, View Source [SID1234558053]). These data will be featured at an oral poster presentation at the ASCO (Free ASCO Whitepaper) 2020 Virtual Scientific Program, May 29-31, 2020.

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GBM is the most common and aggressive type of brain cancer. Currently, the median overall survival with standard of care therapy, which includes radiation and chemotherapy (temozolomide: TMZ), is approximately 15 to 22 months.

The Phase 1/2 clinical trial demonstrated that 84.4% percent (27 of 32) of patients with MGMT promoter unmethylated tumors, and 85% (17 of 20) of patients with MGMT promoter methylated tumors were alive at 12 months. This promising clinical result is coupled with a robust immunological response to all three cancer antigens in INO-5401, including human telomerase (hTERT), Wilms Tumor-1 (WT-1) and prostate specific membrane antigen (PSMA). Activated, cytotoxic T cells directed towards these cancer antigens commonly expressed on GBM tumors were detected in all patients tested to date and continue to support the immunogenic potential of INOVIO’s DNA medicines. Importantly, INO-5401 + INO-9012 was safe and well-tolerated when given not only with radiation and TMZ, but also with PD-1 inhibition with Libtayo, which is being jointly developed by Regeneron and Sanofi. These results are being presented in a virtual format at the 2020 Annual ASCO (Free ASCO Whitepaper) meeting (Abstract #2514).

Dr. David Reardon, Clinical Director, Center for Neuro-Oncology of Dana-Farber Cancer Institute and coordinating principal investigator of GBM-001 said, "Although these data are preliminary, and follow-up remains early, this novel combination of a cancer antigen-specific, T cell generating DNA medicine with a PD-1 inhibitor is exciting and may overcome more than 20 years of a standard of care that has proven sub-optimal for our patients with GBM. A tolerable, new combination of medicines utilizing a novel mechanism of action, such as that provided by INO-5401 and INO-9012 with cemiplimab, is very welcome for this hard-to-treat brain cancer, especially when shown to be tolerable with standards such as radiation and chemotherapy, and when demonstrating the immunogenicity seen in the GBM-001 study."

Dr. J. Joseph Kim, INOVIO’s President & CEO, said, "While we recognize these data are early, we are very excited to see robust immunogenicity and the potential for extending survival, coupled with a clear ability to be able to combine not only with the standard of care, but with a checkpoint inhibitor, Libtayo. Where others have failed with single-agent checkpoint inhibition in GBM, our DNA medicine combined with Libtayo and standard of care has demonstrated clear immunogenicity and the potential to extend overall survival."

In a previous announcement, INOVIO reported key interim data from the 52-patient clinical trial showed that 80% (16 of 20) of MGMT gene promoter methylated patients and 75% (24 of 32) unmethylated patients were progression-free at six months (PFS6) measured from the time of their first dose, exceeding historical standard-of-care data.

This immunotherapy combination with a PD-1 checkpoint inhibitor also exhibited supportive safety, tolerability, and immunogenicity data and suggested an acceptable safety profile consistent with that of Libtayo and INOVIO’s platform technology. The majority of patients tested had a T cell immune response to one or more tumor-associated antigens encoded by INO-5401. Immune responses to all three tumor-associated antigens were demonstrated in this study. INOVIO plans to report 18-month overall survival data later this year.

Study Design

The trial was designed to evaluate safety, immunogenicity and preliminary efficacy of INO-5401 and INO-9012 in combination with Libtayo, with radiation and chemotherapy, in subjects with newly-diagnosed glioblastoma (GBM). This is a Phase 1/2, open-label, multi-center trial conducted in 52 evaluable patients with GBM. There are two cohorts in this trial. Cohort A includes 32 participants with a tumor with an unmethylated O6-methylguanine-deoxyribonucleic acid (DNA) methyltransferase (MGMT) promoter. Cohort B includes 20 participants with a tumor with a MGMT methylated promoter. Both cohorts received INO-5401 and INO-9012 and Libtayo at the same doses and on the same dosing schedule, and both cohorts received radiation and temozolomide (TMZ). Interim data presented here and at SITC (Free SITC Whitepaper) was obtained as of October 2019 and overall survival data at 18 months is expected in Q4 2020. For more information of the clinical study, see www.clinicaltrials.gov, identifier NCT03491683.

Poster Details

Abstract/Poster 2514
Poster Discussion Session: Central Nervous System Tumors
The ASCO (Free ASCO Whitepaper) 2020 Virtual Scientific Program runs from May 29 -31.

About Glioblastoma Multiforme (GBM)

GBM is the most common and aggressive type of brain cancer and remains a devastating disease for both patients and caregivers. Its prognosis is extremely poor, despite a limited number of new therapies approved over the last 10 years. The median overall survival for patients receiving standard of care therapy is approximately 15 to 22 months and the median progression-free survival is approximately 7 months. In the U.S., the estimated annual incidence of GBM is 11,362 cases or 3.21 cases per 100,000 persons and the median age at diagnosis is 65 years.

About INO-5401 and INO-9012

INO-5401 encodes for INOVIO’s SynCon antigens for hTERT, WT1, and PSMA, and has the potential to be a powerful cancer immunotherapy in combination with checkpoint inhibitors. The National Cancer Institute previously highlighted hTERT, WT1, and PSMA among a list of important cancer antigens, designating them as high priorities for cancer immunotherapy development. These three antigens were reported to be over-expressed, and often mutated, in a variety of human cancers, and targeting these antigens may prove efficacious in the treatment of patients with cancer. INO-9012 encodes for IL-12, which is a T cell immune activator.

About INOVIO’s DNA Medicines Platform

INOVIO has 15 DNA medicine clinical programs currently in development focused on HPV-associated diseases, cancer, and infectious diseases, including coronaviruses associated with MERS and COVID-19 diseases being developed under grants from the Coalition for Epidemic Preparedness Innovations (CEPI). DNA medicines are composed of optimized DNA plasmids, which are small circles of double-stranded DNA that are synthesized or reorganized by a computer sequencing technology and designed to produce a specific immune response in the body.

INOVIO’s DNA medicines deliver optimized plasmids directly into cells intramuscularly or intradermally using INOVIO’s proprietary hand-held smart device called CELLECTRA. The CELLECTRA device uses a brief electrical pulse to reversibly open small pores in the cell to allow the plasmids to enter, overcoming a key limitation of other DNA and other nucleic acid approaches, such as mRNA. Once inside the cell, the DNA plasmids enable the cell to produce the targeted antigen. The antigen is processed naturally in the cell and triggers the desired T cell and antibody-mediated immune responses. Administration with the CELLECTRA device ensures that the DNA medicine is efficiently delivered directly into the body’s cells, where it can go to work to drive an immune response. INOVIO’s DNA medicines do not interfere with or change in any way an individual’s own DNA. The advantages of INOVIO’s DNA medicine platform are how fast DNA medicines can be designed and manufactured, the stability of the products which do not require freezing in storage and transport, and the robust immune response, safety profile, and tolerability that have been demonstrated in clinical trials.

With more than 2,000 patients receiving INOVIO investigational DNA medicines in more than 6,000 applications across a range of clinical trials, INOVIO has a strong track record of rapidly generating DNA medicine candidates with potential to meet urgent global health needs.

On May 14, 2020 ALX Oncology Holdings Inc., a clinical-stage immuno-oncology company developing therapies that block the CD47 checkpoint pathway, reported that the Phase 1 study of ALX148 in patients with advanced solid tumors has been selected for presentation in the Poster Discussion Session at the Virtual 2020 ASCO (Free ASCO Whitepaper) Annual Meeting, May 29 – 31, 2020 (Press release, ALX Oncology, MAY 14, 2020, View Source [SID1234558070]).

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Virtual ASCO (Free ASCO Whitepaper) 2020 Presentation Information
Title: A Phase 1 study of ALX148, a CD47 blocker, in combination with standard anticancer antibodies and chemotherapy regimens in patients with advanced malignancy (Abstract #3056).
Poster Discussion Session: Developmental Therapeutics—Immunotherapy
Date: Friday, May 29

On May 14, 2020 Regulus Therapeutics Inc. (Nasdaq: RGLS), a biopharmaceutical company focused on the discovery and development of innovative medicines targeting microRNAs (the "Company" or "Regulus"), reported financial results for the first quarter ended March 31, 2020 and provided a corporate update (Press release, Regulus, MAY 14, 2020, View Source [SID1234558086]).

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"We have completed the second cohort and have initiated the dosing for the third and final cohort in the Phase 1 multiple ascending dose ("MAD") clinical study for RGLS4326" said Jay Hagan, CEO of Regulus. "Additionally, preparations for the Phase 1b study in patients with autosomal dominant polycystic kidney disease ("ADPKD") are well underway with plans to initiate in the second half of 2020."

Program Highlights

Initiated Dosing of the Third and Final Cohort in RGLS4326 Phase 1 for ADPKD: In April 2020, the Company initiated dosing of the third and final cohort of the MAD clinical study of RGLS4326, a novel oligonucleotide designed to inhibit miR-17 for the treatment of ADPKD. The Company expects to complete this study in mid-2020 with topline results available thereafter. The Company is planning to initiate a Phase 1b short-term dosing study in patients with ADPKD in the second half of 2020 to evaluate RGLS4326 for safety, pharmacokinetics, and biomarkers of pharmacodynamic activity.

Financial Results

Cash Position: As of March 31, 2020, Regulus had $28.1 million in cash and cash equivalents.

Research and Development (R&D) Expenses: R&D expenses were $3.1 million for the three months ended March 31, 2020, compared to $6.0 million for the same period in 2019. The aggregate decrease was driven by a $1.5 million reduction in personnel and internal expenses and a $1.0 million reduction in external development expenses, both of which were primarily attributable to a reduction in costs associated with the partial clinical hold of the RGLS4326 MAD study. In December 2019, the U.S. Food and Drug Administration ("FDA") lifted the partial clinical hold on the MAD study and it was recommenced in February 2020.

General and Administrative (G&A) Expenses: G&A expenses were $2.4 million for the three months ended March 31, 2020 compared to $3.5 million for the same period in 2019. These amounts reflect personnel-related and ongoing general business operating costs. The decrease is primarily attributable to continued cost reduction efforts subsequent to our corporate restructuring in the third quarter of 2018.

Revenue: Revenue was less than $0.1 million for the three months ended March 31, 2020. Revenue was $6.8 million for the three months ended March 31, 2019, attributable to revenue recognition of the upfront payments received under the 2018 Sanofi Amendment related to the transfer of the RG-012 program to Sanofi.

Net Loss: Net loss was $5.9 million, or $0.25 per share (basic and diluted), for the three months ended March 31, 2020, compared to $3.3 million, or $0.31 per share (basic and diluted), for the same period in 2019.

About ADPKD

ADPKD, caused by the mutations in the PKD1 or PKD2 genes, is among the most common human monogenic disorders and a leading cause of end-stage renal disease. The disease is characterized by the development of multiple fluid filled cysts primarily in the kidneys, and to a lesser extent in the liver and other organs. Excessive kidney cyst cell proliferation, a central pathological feature, ultimately leads to end-stage renal disease in approximately 50% of ADPKD patients by age 60.

About RGLS4326

RGLS4326 is a novel oligonucleotide designed to inhibit miR-17 and designed to preferentially target the kidney. Preclinical studies with RGLS4326 have demonstrated direct regulation of PKD1 and PKD2 in human ADPKD cyst cells, a reduction in kidney cyst formation, improved kidney weight/body weight ratio, decreased cyst cell proliferation, and preserved kidney function in mouse models of ADPKD. The RGLS4326 IND is currently on a Partial Clinical Hold for treatment of extended duration by the U.S. Food and Drug Administration until the second set of requirements outlined by FDA have been satisfactorily addressed. Information from the Phase 1 clinical studies, together with information from additional nonclinical studies, will be used to address the second set of requirements to support studies of extended duration.

On May 14, 2020 Oncopeptides AB (Nasdaq Stockholm: ONCO) reported that data from the pivotal phase 2 study HORIZON, and additional clinical and preclinical data that further evaluate the therapeutic peptide-drug conjugate platform, have been accepted by the European Hematology Association (EHA) (Free EHA Whitepaper) (Press release, Oncopeptides, MAY 14, 2020, View Source [SID1234557993]). The abstracts are now published online.

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Melflufen (melphalan flufenamide) is a first-in-class anticancer peptide-drug conjugate that rapidly delivers an alkylating payload into tumor cells. Melflufen is in late stage clinical development for a potential treatment of patients with relapsed refractory multiple myeloma (RRMM).

"The primary read-out of the HORIZON-data represents an important milestone for Oncopeptides. They lay the ground for the New Drug Application to the FDA, seeking accelerated approval for intravenous melflufen in combination with dexamethasone, says Klaas Bakker, CMO of Oncopeptides. "Melflufen could provide a novel treatment option with a unique mechanism of action for a group of myeloma patients with a particularly poor prognosis".

Below is a brief description of the abstracts that have been accepted by the European Hematology Association (EHA) (Free EHA Whitepaper). The full EHA (Free EHA Whitepaper) abstract book can be found on www.ehaweb.org.

HORIZON (OP-106): Melflufen plus dexamethasone in relapsed/refractory multiple myeloma (RRMM) refractory to pomalidomide and/or an anti-CD38 monoclonal antibody – primary and subgroup analysis.
Final Abstract Code: EP945. First author: Paul G Richardson.The primary read-out of the data from the pivotal, phase II study HORIZON demonstrates clinical efficacy and a manageable safety profile of the peptide-drug conjugate melflufen in combination with dexamethasone in patients with RRMM, including patients with high-risk features and triple-class–refractory disease.
HORIZON (OP-106): An exploratory analysis of time to next treatment in patients with relapsed/refractory multiple myeloma who received melflufen plus dexamethasone.
Final Abstract Code: EP1029. First author: Maria-Victoria MateosThe abstract includes a time to next treatment analysis: The sub-analysis of the HORIZON clinical study is the first to provide important insights on time to subsequent treatment in patients with advanced RRMM (medium 5 lines of previous lines).
LIGHTHOUSE (OP-108): A phase 3 study of melflufen in combination with daratumumab versus daratumumab in patients with relapsed/refractory multiple myeloma.
Final Abstract Code: PB2018. First author: Maria-Victoria MateosThe planned randomized phase 3 trial LIGHTHOUSE will study the impact of melflufen, dexamethasone and subcutaneous daratumumab compared with subcutaneous daratumumab alone. The results will be important to confirm the preliminary efficacy, safety and tolerability results from phase 1/2 ANCHOR study, combining melflufen, dexamethasone and daratumumab supporting further regulatory milestones for Oncopeptides
Adverse event and outcome patterns in patients with advanced multiple myeloma in the US
Final Abstract Code: PB2039. First author: Joshua RichterThis real-world data study provides evidence, that albeit introduction of additional treatment options for patients with advanced multiple myeloma, their prognosis remains poor and the need for additional treatment options are high
Melflufen is a highly effective anti-neoplastic agent in bortezomib-resistant multiple myeloma models.
Final Abstract Code: EP915. First author: Konstantin ByrgazovMelflufen is more efficacious in bortezomib-resistant myeloma cell lines than in their bortezomib-naive parental cells in vitro. Bortezomib-resistant myeloma cells lines overexpress Aminopeptidase B encoded by RNPEP gene, and myeloma patients with high RNPEP expression have shorter PFS on bortezomib-containing therapies.
Melflufen efficacy in multiple myeloma with TP53 aberrations.
Final Abstract Code: EP903. First author: Ana Slipicevic.Melflufen can trigger myeloma cell death regardless of cells TP53 status and overcome the p53-deficiency-mediated melphalan resistance. Melflufen response rate in the del 17p patient subpopulation from the phase 2-study HORIZON is comparable to the general RRMM population suggesting that melflufen might be a therapeutic option for these difficult-to-treat patients.
Aminopeptidase expression in multiple myeloma associates with disease progression and sensitivity to melflufen.
Final Abstract Code: EP897. First author: Juho MiettinenAminopeptidases play a role in multiple myeloma biology. Their expression levels are dysregulated during disease progression, and majority are increased in RRMM compared to NDMM patients. Aminopeptidases LAP3 and TPP2 are identified as prognostic markers in myeloma patients, and inhibition of aminopeptidases reduces myeloma cell viability in vitro. Melflufen, an aminopeptidase substrate, is a highly efficient anticancer agent in myeloma cells resistant to other alkylators, bortezomib and selinexor.

For more information, please contact:
Klaas Bakker, MD, PhD, Chief Medical Officer of Oncopeptides
E-mail: [email protected]
Cell: +44 7818 523903

Fredrik Lehmann, EVP Research and CMC at Oncopeptides
E-mail: [email protected]
Phone: +46 8 615 20 40

Rein Piir, Head of Investor Relations at Oncopeptides
E-mail: [email protected]
Cell : +46 70 853 72 92

This information was submitted for publication at 15.00 CET May 14, 2020.

About melflufen
Melflufen (melphalan flufenamide) is a first-in-class anti-cancer peptide-drug conjugate that rapidly delivers an alkylating payload into tumor cells. Melflufen is rapidly taken up by myeloma cells due to its high lipophilicity and is immediately cleaved by peptidases to deliver an entrapped hydrophilic alkylator payload. Peptidases play a key role in protein homeostasis and feature in cellular processes such as cell-cycle progression and programmed cell death. In vitro, melflufen is 50-fold more potent in myeloma cells than the alkylator payload itself due to the increased intracellular alkylator concentration. Melflufen displays cytotoxic activity against myeloma cell lines resistant to other treatments, including alkylators, and has also demonstrated inhibition of DNA repair induction and angiogenesis in preclinical studies.