AOP Orphan announces three-year results on Ropeginterferon alfa-2b in Polycythemia Vera at the American Society of Hematology (ASH) Annual Meeting 2018

On December 3, 2018 AOP Orphan Pharmaceuticals AG (AOP Orphan) reported latest follow-up results on Ropeginterferon alfa-2b in patients with Polycythemia Vera (PV) from CONTINUATION-PV presented at ASH (Free ASH Whitepaper) 2018 (Press release, AOP Orphan Pharmaceuticals, DEC 3, 2018, View Source [SID1234531862]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

CONTINUATION-PV is an open-label, multicenter, phase IIIb study assessing the long-term efficacy and safety of Ropeginterferon alfa-2b versus hydroxyurea (HU) or best available treatment (BAT) in patients with PV who previously participated in the PROUD-PV study.

Ropeginterferon alfa-2b is a novel, long-acting, mono-pegylated proline interferon (ATC L03AB15). It is administered once every 2 weeks initially, or monthly after stabilization of hematological parameters. It is expected to be the first interferon approved for PV worldwide. AOP Orphan´s submission for marketing authorization in the EU is in the final stage of EMA review.

Clinical evidence:

Previously, at 12 months of treatment, Ropeginterferon alfa-2b was shown to be non-inferior to hydroxyurea (HU) in Complete Hematologic Response (CHR) and to have a significantly better safety and tolerability profile. . At 24 months Ropeginterferon alfa-2b achieved a significantly higher CHR of 70.5% versus 49.3% compared with HU/BAT (p=0.0101).

After 36 months of treatment Ropeginterferon alfa-2b sustained higher CHR response rates compared to HU/BAT (70.5% vs. 51.4%; p=0.0122). Further, the composite endpoint, CHR including disease symptom improvement was higher in patients treated with Ropeginterferon alfa-2b compared to HU/BAT (52.6% vs. 37.8%; p=0.0437).

Disease modification evidence:


66.0% of PV patients treated with Ropeginterferon alfa-2b, but only 27.0% having received HU/BAT showed a mutant JAK2 molecular response (p<0.0001) after 36 months. Importantly, molecular response strongly correlated with CHR, emphasizing the clinical relevance of mutant JAK2 allele burden reduction.

Analysis of additional non-JAK2 mutations, which are believed to contribute to disease progression revealed that Ropeginterferon alfa-2b, but not HU was able to reduce their respective mutant allele burden. This suggests that only Ropeginterferon alfa-2b but not HU has the ability to suppress additional clones with different mutations and modify the disease at the molecular level.

In line with molecular findings, disease or treatment related secondary malignancies occurred only in patients receiving HU/BAT, including 2 cases of acute myeloid leukemia, 1 melanoma and 2 basaliomas, whereas 3 malignancies (glioblastoma, seminoma, adrenal neoplasm) most likely unrelated to treatment were reported for patients treated with Ropeginterferon alfa-2b.

A similar number of patients experienced adverse events (89.8% for Ropeginterferon alfa-2b, 90.6% for HU) and treatment-related adverse events (74.8% for Ropeginterferon alfa-2b, 78.7% for HU). The most common (>10%) treatment-related adverse events anemia, thrombocytopenia and leukopenia occurred more frequently with HU, whereas liver enzyme increase was mainly observed with Ropeginterferon alfa-2b.

No new safety signals appeared in the third year of treatment.

Professor Heinz Gisslinger from the Medical University of Vienna, Austria presenting the results at ASH (Free ASH Whitepaper) stated, "The observed superior efficacy of Ropeginterferon alfa-2b over hydroxyurea/best-available-therapy after 36 months, is a clear proof of the long-term value of this treatment modality. Thus, Ropeginterferon alfa-2b will provide a valuable and safe new first line therapy for PV patients".

Professor Jean-Jacques Kiladjian from the Saint-Louis Hospital & Paris Diderot University in France, concluded, "The disease modification capability of Ropeginterferon alfa-2b suggested by a significant reduction of not only mutant JAK2, but also non-JAK2 allele burden and the specific targeting of the malignant clone, holds promise for improvement of progression-free survival and long-term patient benefit."

About Ropeginterferon alfa-2b


Ropeginterferon alfa-2b is a novel, long-acting, mono-pegylated proline interferon (ATC L03AB15) with improved pharmacokinetic properties offering improved tolerability and convenience. It is administered once every 2 weeks, or monthly during long-term maintenance, and is expected to be the first interferon approved for PV worldwide. AOP Orphan´s submission for marketing authorization in the EU is currently under EMA review.

Ropeginterferon alfa-2b was discovered by PharmaEssentia, a long-term partner of AOP Orphan. Ropeginterferon alfa-2b has Orphan Drug designation in the European Union, Switzerland and the United States of America.


In 2009, AOP Orphan has in-licensed from PharmaEssentia Corporation the exclusive rights to develop and commercialize Ropeginterferon alfa-2b in PV, other MPNs and CML for European, Commonwealth of Independent States (CIS), and Middle Eastern markets.

About Polycythemia Vera


Polycythemia Vera (PV) is a cancer of the blood-building cells in the bone marrow resulting in a chronic increase of red blood cells, white blood cells and platelets. This condition may result in circulatory disorders such as thrombosis and embolism, as well as malignant transformation to myelofibrosis or leukemia. While the molecular mechanism underlying PV is still subject of intense research, current results point to a set of acquired mutations, the most important being a mutant form of JAK2 that make the malignant clone.

Progenics Doses First Patient in Pivotal Phase 3 Study of PyL, PSMA PET Imaging Agent, for the Detection of Prostate Cancer

On December 3, 2018 Progenics Pharmaceuticals, Inc. (NASDAQ:PGNX), an oncology company developing innovative medicines and imaging analysis technology for targeting and treating cancer, reported that the first patient has been dosed in the Company’s Phase 3 CONDOR study evaluating the diagnostic performance and clinical impact of PyL (18F-DCFPyL) in men with biochemical recurrence of prostate cancer (Press release, Progenics Pharmaceuticals, DEC 3, 2018, View Source [SID1234531895]). PyL is the Company’s PSMA-targeted small molecule PET/CT imaging agent designed to visualize prostate cancer.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Dr. Lawrence Saperstein, Assistant Professor of Radiology and Biomedical Imaging and Chief of Nuclear Medicine at Yale Smilow Cancer Hospital, and a principle investigator of the trial said, "PyL has demonstrated excellent positive and negative predictive values in detecting the presence or absence of prostate cancer. This valuable information has the potential to provide earlier diagnoses, more informed treatment decisions, the ability to monitor responses, and ultimately improve patient outcomes."

The Phase 3 CONDOR trial is a multi-center, open label study and will enroll approximately 200 patients with biochemical recurrence of prostate cancer in 14 sites in the United States and Canada. The primary endpoint is based on positive predictive value and will assess the correct localization rate (CLR), defined as a percentage of subjects with a one-to-one correspondence between localization of at least one lesion identified by PyL and the composite truth standard. Secondary measures include the percentage of subjects with a change in intended prostate cancer treatment plans due to PyL PET/CT imaging.

"The initiation of this landmark trial marks an important milestone in the development of PSMA-targeted imaging agents which have the potential to transform the treatment landscape for recurrent prostate cancer," stated Mark Baker, CEO of Progenics. "With PyL, physicians may be able to detect very small lesions that are currently missed with conventional imaging methods, which in turn could change the treatment path. We look forward to reporting data from this trial in early 2020."

Progenics Initiates Phase 3 Trial of PyL PSMA PET Imaging Agent Page 2

In prior studies, PyL has shown strong positive predictive value to detect prostate cancer, with high sensitivity (93-99%) in reliably detecting metastatic prostate cancer lesions and high specificity (96-99%) in confirming the absence of pelvic lymph node disease.

About PyL for PET Imaging of Prostate Cancer

PyL (also known as [18F]DCFPyL) is a fluorinated PSMA-targeted Positron Emission Topography ("PET") imaging agent that enables visualization of both bone and soft tissue metastases to determine the presence or absence of recurrent and/or metastatic prostate cancer.

About Prostate Cancer

Prostate cancer is the second most common form of cancer affecting men in the United States: an estimated one in seven men will be diagnosed with prostate cancer in his lifetime. The American Cancer Society estimates that each year approximately 164,690 new cases of prostate cancer will be diagnosed and about 29,430 men will die of the disease. Approximately 2.9 million men in the U.S. currently count themselves among prostate cancer survivors

Biotecnol Limited and Chiome Bioscience Inc of Japan have entered into an asset purchase agreement.

On December 3, 2018 Under this agreement, Biotecnol reported that divested from it’s immuno-oncology pipeline and respective technologies (including Tb535H, and Trisoma (Press release, Biotecnol, DEC 3, 2018, View Source [SID1234570279]). Chiome will continue research and clinical development for Tb535H. In addition, Chiome will continue generating new immune-oncology products using Biotecnol’s Trisoma technology. Financial details were not disclosed.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

About Tb535H

Tb535H is a T-cell engager, trispecific antibody, directed against the 5T4/WAIF1 tumour antigen, a protein found on many different solid tumours and is thought to contribute to the spread of cancer cells. Tb535H recruits the patient’s T-cells –killer cells of the immune-system – and directs them to attack tumours. This highly targeted approach uses the patient’s own immune system to fight cancer. The WAIF1 antigen was discovered by scientists at the Cancer Research UK Manchester Institute. It could be a valuable target in many different cancer types, but the initial focus in this trial will be to treat cancers with high unmet-need. This includes thoracic cancers such as mesothelioma, small-cell lung carcinoma (SCLC) and non-small cell lung cancer (NSCLC), for which survival remains very low, and renal cell carcinoma.

About Trisoma technology

The Trisoma technology enables the generation of multi-specific antibody products. This unique technology overcomes the key shortcomings of conventional mono- as well as of currently developed bi-specific antibody formats. With the Trisoma technology, Biotecnol can engineer and assemble recombinant antibodies in a rational manner in order to design a chosen mechanism of action to address the killing of a specific tumour type.

OHSU and Aptose Present New CG-806 Preclinical Data at ASH 60th Annual Meeting

On December 3, 2018 Oregon Health & Science University (OHSU) and Aptose Biosciences Inc. (NASDAQ: APTO, TSX: APS) reported the presentation of preclinical data demonstrating that CG-806, a first-in-class pan-FLT3/pan-BTK inhibitor, exhibits broad ex vivo potency on bone marrow cells from patients with diverse hematologic malignancies and superior potency relative to ibrutinib on those bone marrow cells from patients with CLL or other B-cell cancers (Press release, Aptose Biosciences, DEC 3, 2018, View Source [SID1234531814]). In addition, bioinformatic analyses revealed an unexpected ex vivo potency of CG-806 on bone marrow cells from AML patients with IDH1 mutations or with FLT3-ITD mutations. Plus, CG-806 demonstrated a favorable safety profile in GLP toxicology and safety studies. The data were highlighted in a poster presentation on Sunday, December 2, 2018 at the 60th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition being held December 1-4, 2018 in San Diego, CA. Separately, Aptose and The University of Texas MD Anderson Cancer Center researchers also presented new data on CG-806 at ASH (Free ASH Whitepaper) (see press release here).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Studies have shown that more than 50% of patients with CLL and mantle cell lymphoma (MCL) discontinue ibrutinib treatment due to intolerance or emergence of resistant disease. "Our data indicate that CG-806 clearly addresses ibrutinib’s shortcomings, inhibiting driver and rescue pathways to directly and potently kill a broad range of malignant B-cells. CG-806 has potential to be an important part of our future armamentarium against hematologic malignancies that are resistant, refractory or intolerant to other therapies," said Jeffrey W. Tyner, Ph.D., Associate Professor in the OHSU School of Medicine department of Cell, Developmental & Cancer Biology.

The poster, CG-806, a First-in-Class Pan-FLT3/Pan-BTK Inhibitor, Exhibits Broader and Greater Potency than Ibrutinib Against Primary and Cultured Malignant B Cells, evaluated the activity of CG-806 on various hematologic malignancy cell lines and patient primary bone marrow specimens. CG-806 inhibited cell proliferation and induced apoptosis with a potency that was 50-6,000 times greater than that of ibrutinib when tested against 14 established malignant B-cell lines in vitro. When tested against 124 samples freshly isolated from the bone marrow of chronic lymphocytic leukemia (CLL) patients, the median IC50 for CG-806 was 0.11 µM and the median for ibrutinib was 4.09 µM, respectively, p<0.001. Since stromal mediated signaling plays an important role in malignant B-cell survival and chemoresistance, the apoptotic effect of CG-806 was further analyzed on cultured and primary malignant B-cells in the presence of stromal cells, and its potency was not impaired. CG-806 was shown to be significantly more potent than Ibrutinib at inhibiting malignant B-cell colony formation, migration, and inducing apoptosis in the presence of stromal cells.

Primary cells from patients with diverse hematologic malignancies are highly sensitive to CG-806, including cells with the FLT3-ITD mutation, found in approximately 30% of acute myeloid leukemia (AML) patients. The data presented at ASH (Free ASH Whitepaper) showed that primary cells from AML patients with the IDH-1 mutation similarly demonstrated high sensitivity to CG-806. Aptose also reported new results from the 28-day GLP toxicity and toxicokinetic studies of CG-806, which continue to demonstrate a highly favorable safety profile with no adverse findings to date.

"CG-806’s activity against IDH-1 mutant AML patient bone marrow cells is an unexpected new finding that further broadens its potential use and potential paths for rapid development," said William G. Rice, Ph.D., Chairman and Chief Executive Officer of Aptose. "In addition, CG-806 has continued to perform well in all toxicology and safety studies, and we look forward to filing an IND in early 2019."

About CG-806
CG-806 is a preclinical stage oral, first-in-class pan-FLT3/pan-BTK multi-cluster kinase inhibitor. This small molecule demonstrates potent inhibition of wild type and all mutant forms of FLT3 (including internal tandem duplication, or ITD, and mutations of the receptor tyrosine kinase domain and gatekeeper region), eliminates acute myeloid leukemia (AML) tumors in the absence of toxicity in murine xenograft models, and represents a potential best-in-class therapeutic for patients with AML. Likewise, CG-806 demonstrates potent, non-covalent inhibition of the wild type and Cys481Ser mutant forms of the BTK enzyme, as well as other oncogenic kinase pathways operative in B cell malignancies, suggesting CG-806 may be developed for various B-cell malignancy patients (including CLL, MCL, DLBCL and others) that are resistant/refractory/intolerant to covalent BTK inhibitors. It is in development for acute myeloid leukemia (AML) and B cell lymphoma.

TRACON Pharmaceuticals And I-Mab Biopharma To Host Conference Call To Discuss Strategic Partnerships For Multiple Immuno-Oncology Programs

On December 3, 2018 TRACON Pharmaceuticals (Nasdaq: TCON), a clinical stage biopharmaceutical company focused on the development and commercialization of novel targeted therapeutics for cancer, and I-Mab Biopharma ("I-Mab"), a China-based clinical stage biopharmaceutical company exclusively focused on the development of innovative biologics in immuno-oncology and autoimmune diseases, will host a conference call and webcast on Wednesday, December 5, 2018, to discuss the recently announced series of strategic collaborative partnerships for developing multiple immuno-oncology programs (Press release, Tracon Pharmaceuticals, DEC 3, 2018, http://ir.traconpharma.com/news-releases/news-release-details/tracon-pharmaceuticals-and-i-mab-biopharma-host-conference-call [SID1234531830]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Wednesday, December 5th @ 4:30pm Eastern Time
US Investors: 877-407-9039
International: 201-689-8470
Passcode: 13685586
Webcast: View Source

Replays, Available through December 19th:
Domestic: 844-512-2921
International: 412-317-6671
Replay PIN: 13685586
About TRACON
TRACON develops targeted therapies for cancer and ophthalmic diseases. The Company’s clinical-stage pipeline includes: TRC105, an endoglin antibody that is being developed for the treatment of multiple cancers; DE-122, the ophthalmic formulation of TRC105 that is being developed in wet AMD by corporate partner Santen Pharmaceutical Company Ltd.; TRC102, a small molecule being developed for the treatment of lung cancer and solid tumors; and TRC253, a small molecule being developed for the treatment of prostate cancer. To learn more about TRACON and its product candidates, visit TRACON’s website at www.traconpharma.com.

About I-Mab
I-Mab is a dynamic and fast-growing global company exclusively focused on developing first-in-class and best-in-class biologics in the areas of immuno-oncology and autoimmune diseases through internal R&D capabilities and global partnerships. I-Mab’s pipeline is driven by the company’s development strategy to address unmet needs in China and to bring innovative assets to the world. The company is prepared to submit additional INDs in order to initiate clinical trials in China and the U.S., including multiple Phase II and Phase III studies. I-Mab is on a fast track towards becoming an end-to-end fully integrated biopharma company. The company has been well-recognized by capital markets with the recent $220 million Series C financing representing one of the largest amounts ever raised by an innovative biotech company in China. www.i-mabbiopharma.com