On June 4, 2020 Moleculin Biotech, Inc., (Nasdaq: MBRX) ("Moleculin" or the "Company"), a clinical stage pharmaceutical company with a broad portfolio of drug candidates, reported that it has submitted requests to Polish regulatory authorities for approval to open two additional clinical sites for its Phase 1/2 clinical study of Annamycin for the treatment of acute myeloid leukemia ("AML") (Press release, Moleculin, JUN 4, 2020, View Source [SID1234560825]).

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!

Moleculin Biotech, Inc. is a clinical stage pharmaceutical company focused on the development of a broad portfolio of oncology drug candidates for the treatment of highly resistant tumors. (PRNewsfoto/Moleculin Biotech, Inc.)

"We are pleased to report that the Annamycin trial in AML continues forward despite the disruptions from the COVID-19 pandemic," commented Walter Klemp, Chairman and CEO of Moleculin. "Two additional hospitals in Poland, one in Szczecin and one in Kielce, have been moving quickly to participate in this trial. Assuming these requests are granted in a timely manner, we expect both sites to begin recruiting in the third quarter."

Moleculin currently has 5 clinical sites for the Annamycin AML clinical trial operating in Europe. The addition of sites in Szczecin and Kielce would bring this total to 7.

On June 4, 2020 PharmaEssentia Corporation (TPEx: 6446), a global biopharmaceutical innovator leveraging deep expertise and proven scientific principles to deliver new biologics in hematology and oncology, reported that the U.S. Food and Drug Administration (FDA) has recently accepted its Biologics License Application (BLA) for ropeginterferon alfa-2b (P1101), a novel pegylated interferon intended for the treatment of the rare blood cancer polycythemia vera (PV) in the absence of symptomatic splenomegaly (Press release, PharmaEssentia, JUN 4, 2020, View Source [SID1234560843]). The company expects an agency decision in early 2021.

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!

PharmaEssentia has focused its efforts on therapeutic innovation in the category of myeloproliferative neoplasms (MPNs), which are caused by specific genetic mutations that lead to overproduction of blood components including white or red blood cells, or platelets. In PV, which is caused by a JAK2 V617F mutation, the bone marrow produces excessive red blood cells, causing the blood to be thicker than normal and potentially leading to a range of complications.1,2 PV is estimated to affect more than 160,000 people in the U.S. alone,1 who have progressively burdensome symptoms. Without proper management, the disease progresses into malignancies including myelofibrosis and acute myeloid leukemia.3

Ropeginterferon alfa-2b was invented by scientists at PharmaEssentia in Taiwan and is a structurally novel monopegylated proline interferon designed for administration once every two weeks. The U.S. filing is supported by robust, durable 24-36-month data from the Phase 3 PROUD/CONTI-PV clinical trial, which demonstrated that the investigational treatment offered high and durable hematologic responses and symptom control with good tolerability and low rates of depression observed, with effects on relevant MPN mutations supporting a potential disease modifying capability.4

The findings were shown among patients who received either Ropeginterferon alfa-2b (n=95) or hydroxyurea/best available therapy (HU/BAT, n=74). At 36 months of treatment, patients who received Ropeginterferon alfa-2b maintained a complete hematological response longer than those who received HU/BAT (70.5% vs. 51.4%). Response rates steadily increased in the Ropeginterferon alfa-2b arm throughout 24 months of treatment and remained constant after 36 months. Further, after 36 months, two thirds (66.0%) of patients who received Ropeginterferon alfa-2b achieved a molecular response, compared with 27% in the HU/BAT arm. Importantly, these molecular responses were closely related to complete hematological responses. There were similar rates of adverse events in both arms; the most common (>10%) treatment-related adverse events included anemia, thrombocytopenia and leukopenia, which occurred more frequently under HU.4

"Our focus is on stunting these rare malignancies, preserving patient well-being and slowing the progression into more aggressive and deadly cancers," said Meredith Manning, U.S. General Manager for PharmaEssentia. "We believe Ropeginterferon alfa-2b could become an important new therapeutic tool and look forward to engaging with the regulators in our efforts to introduce this option to the underserved PV community in the U.S."

Ms. Manning was recently appointed to the U.S. GM role to guide the expansion of the company’s U.S. presence, with near-term focus on the commercial preparations for the first target indication in PV. Ms. Manning brings PharmaEssentia dynamic expertise in commercialization and market strategy. She joined from resTORbio, where she served as Chief Commercial Officer to define the corporate strategy and the commercial launch approach for an aging-related therapeutic.

About Ropeginterferon alfa-2b

Ropeginterferon alfa-2b is a novel, long-acting, mono-pegylated proline interferon that has been engineered with an optimized profile to support improved pharmacokinetic properties and demonstrated tolerability and convenience compared with conventional interferons. It is designed for administration once every two weeks, or once every four weeks during long-term maintenance. Ropeginterferon alfa-2b has Orphan Drug designation for treatment of polycythemia vera (PV) in the United States. Marketed as Besremi in Europe, the product was approved by the European Medicines Agency (EMA) in 2019. Ropeginterferon alfa-2b was discovered and is manufactured by PharmaEssentia in its Taichung plant, which was cGMP certified by TFDA in 2017 and by EMA in January 2018.

About Polycythemia Vera

Polycythemia Vera (PV) is a cancer originating from a disease-initiating stem cell in the bone marrow resulting in a chronic increase of red blood cells, white blood cells, and platelets. This condition may result in cardiovascular complications such as thrombosis and embolism, as well as transformation to secondary 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 JAK2.3

On June 4, 2020 Alpha Tau Medical, the developer of breakthrough alpha-radiation cancer therapy Alpha DaRT, reported the closing of a Series B equity financing of $26 million (Press release, Alpha Tau Medical, JUN 4, 2020, View Source [SID1234560859]). Investors in the round included a mix of existing investors from previous rounds, such as Shavit Capital, Medison Ventures, and OurCrowd, who continue to demonstrate support for the clinical development of Alpha DaRT, as well as a number of new private and family office investors primarily from Israel and North America.

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!

During the last year, Alpha Tau successfully completed its first-in-human clinical trial of Alpha DaRT with squamous cell carcinoma patients from Italy and Israel. The impressive results from this study, published in the prestigious International Journal of Radiation Oncology, Biology, Physics (known as the "Red Journal") were subsequently applauded in this recent editorial in the same journal.

The Company is now conducting clinical trials in multiple clinical indications across the world, including its first US trial at Memorial Sloan Kettering Cancer Center in New York, a pancreatic cancer trial at CHUM in Montreal, and trials at three academic institutions in Japan. The Company is also establishing new global manufacturing facilities in Israel and elsewhere, and is in the middle of a process for CE marking for its first indication. These activities and others will be supported by the proceeds of this financing.

CEO Uzi Sofer commented, "We are humbled by the groundswell of continued support we’ve seen from both existing and new investors. This will enable us to push forward our mission to help cancer patients across the world, even during these challenging times in which COVID-19 is the focus of everyone’s health concerns."

CFO Raphi Levy added, "We have been very fortunate to continue our progress at full speed across all fronts, including R&D, clinical and operations, and now financing as well, even during a period of global turmoil. As concerns associated with systemic cancer therapies that affect the immune system have become more salient, we see strong interest in our trials from clinicians, patients, and investors who recognize the advantages of a focused and highly potent cancer therapy."

About AlphaDaRT

Alpha DaRT (Diffusing Alpha-emitters Radiation Therapy) enables highly potent and conformal alpha-irradiation of solid tumors. The treatment is delivered by intratumoral insertion of radium-224 impregnated seeds. When the radium decays, its short-lived daughters are released from the seed, and disperse while emitting high-energy alpha particles that destroy the tumor. Since the alpha-emitting atoms diffuse only a short distance, Alpha DaRT mainly affects the tumor, sparing the healthy tissue around it.

On June 4, 2020 Jounce Therapeutics, Inc. (NASDAQ: JNCE), a clinical-stage company focused on the discovery and development of novel cancer immunotherapies and predictive biomarkers, reported that it has regained the worldwide rights to JTX-8064 from Bristol Myers Squibb (Press release, Jounce Therapeutics, JUN 4, 2020, View Source [SID1234560826]). JTX-8064 is a highly-selective, potential first-in-class antibody that targets the Leukocyte Immunoglobulin Like Receptor B2 (LILRB2) on macrophages, and was licensed to Celgene in July 2019. As part of its Celgene integration process, Bristol Myers Squibb is streamlining its pipeline and addressing areas of overlap. As a result, Bristol Myers Squibb notified Jounce that the JTX-8064 License Agreement is being terminated.

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!

"We are thrilled to regain the rights to JTX-8064 and we view this as a significant opportunity for Jounce. Though we highly valued our longstanding partnership with Celgene, now a Bristol Myers Squibb company, having an additional wholly-owned program enables us to further our mission to discover new immunotherapies from a variety of important immune cell types, and develop them for patients who are not well served by today’s therapies," said Richard Murray, Ph.D., chief executive officer and president of Jounce Therapeutics. "The discovery and development of JTX-8064 showcases the strength of our Translational Science Platform in target identification, and our ability to move programs towards the clinic in a rapid manner. In particular, we believe that LILRB2 may function as an immune checkpoint for macrophages and based on our body of existing preclinical data, JTX-8064 has the potential to re-program tumor-associated macrophages within the tumor microenvironment and enhance anti-tumor immunity. We are eager to advance this program into the clinic and will make every effort to do this expeditiously.

License Agreement
In July 2019, Jounce and Celgene, which is now a Bristol Myers Squibb company, announced an exclusive License Agreement for the worldwide rights to JTX-8064. Under the terms of the agreement, Jounce received a $50.0 million non-refundable license fee from Celgene. Effective, June 3, 2020, the License Agreement is terminated. Beyond transition costs and efforts, neither Bristol Myers Squibb or Jounce have any further financial or service obligations to one another. All Jounce intellectual property rights pertaining to JTX-8064 and licensed to Celgene have been reacquired by Jounce.

About JTX-8064
JTX-8064 is an anti-LILRB2 antibody and is the first tumor-associated macrophage candidate to emerge from Jounce’s Translational Science Platform. Preclinical data presented at the 2019 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting supports the development of JTX-8064 as a novel immunotherapy to reprogram immune-suppressive macrophages and enhance anti-tumor immunity.

On June 4, 2020 At the 2020 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Virtual Annual Meeting, Noxopharm, a clinical-stage Australian oncology drug development company, has reported two sets of clinical data relating to the development of NOX66 (Veyonda) as a treatment for end-stage cancer (Press release, Noxopharm, JUN 4, 2020, View Source [SID1234560844]).

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!

The posters addressed how NOX66 may improve treatment responses in men with mCRPC and also its contribution to restoring sensitivity to apoptosis and potentially modulating the immune microenvironment of nasopharyngeal carcinoma (NPC).

"We have been unaware of any therapies under development that come close to offering an anticancer effect to anything like the same degree that we are seeing with Veyonda, and in particular, delivering this level of benefit in a well-tolerated, minimally invasive, and cost-effective manner," said Dr. Graham Kelly, Noxopharm CEO. "In the context of what has been reported at ASCO (Free ASCO Whitepaper) 2020, the high response rates we are seeing with Veyonda mark it as a major drug prospect."

An estimated 300,000 men die worldwide each year from prostate cancer after exhausting available treatment options. Add to that the generally high pain levels associated with the typical spread of prostate cancer to bone, and the need for a last-line treatment offering a meaningful effect once everything else has failed becomes compelling.

"This is an exciting outcome that supports our belief in the anticancer properties of Veyonda," Dr. Kelly said. "To our knowledge, this is the first time that anyone has been able to obtain a meaningful abscopal response rate in prostate cancer. Prostate cancer has developed a reputation as a cancer with poor immune responsiveness, but this data suggests that this isn’t the case. Today’s result positions Veyonda at the forefront of this emerging area of oncology and suggests that we have an exciting new prospective treatment for end-stage prostate cancer."