On November 19, 2019 Moleculin Biotech, Inc., (Nasdaq: MBRX) ("Moleculin" or the "Company"), a clinical stage pharmaceutical company with a broad portfolio of drug candidates targeting highly resistant tumors, reported it now has preliminary data from its clinical trial of WP1220 for the treatment of cutaneous T-cell lymphoma ("CTCL"), which was published in Blood in conjunction with the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition to be held December 7-10, 2019 (Press release, Moleculin, NOV 19, 2019, View Source [SID1234551470]).

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"For years, p-STAT3 (the activated form of STAT3) has been considered an ‘undruggable’ target because of the difficulty of reaching and affecting this cell-signaling protein," commented Walter Klemp, Moleculin’s Chairman and CEO. "Some consider it to be a master regulator of cancer-related gene transcription, so we believe the ability to show a therapeutic effect from a p-STAT3 inhibitor could be considered a significant breakthrough in cancer research."

Results: There were 6 patients screened, and 5 patients enrolled between March and July 2019. Three are evaluable for both safety and efficacy after completing 3 months of treatment, with 2 ongoing and evaluable for safety. The only AE reported potentially related to study drug in one of the five patients was a mild contact dermatitis not requiring treatment. CAILS scores on index lesions were significantly decreased in the first 3 patients, who were stages IA, IB, and IIB, respectively, at entry. A composite score was obtained for all treated lesions for each patient, and percent changes were calculated from baseline to Day 84. There was a median reduction of 70.8% (range 62.1%-76.2%) for the 3 patients. Improvement was noted as early as 7 days after initiation of treatment, and maintenance of improvement was also shown at follow up (1 month after discontinuation, as per protocol). The fourth patient has also shown an initial reduction in the composite CAILS score after 56 days (26.7%), and is continuing on treatment. Evaluations of the biopsy samples for histopathology and status of p-STAT3 in treated lesions are in progress.

Conclusions: WP1220, an inhibitor of p-STAT3, has shown demonstrable safety and significant efficacy after at least 3 months of topical treatment in 3 patients with progressive MF, with a continuing trend towards improvement in additional patients currently in treatment. This is the first demonstration that inhibition of the STAT3 activation pathway with topical therapy has impacted the course of this disease. The trial is continuing, and updated and more comprehensive data from this study as well as assessment of STAT3 phosphorylation in treated lesions will be reported.

Dr. Sandra Silberman, Chief Medical Officer for New Projects at Moleculin, added: "this is the first topical delivery of a p-STAT3 inhibitor that we know of for CTCL, where there is a significant unmet need for improved treatment of the lesions associated with this potentially deadly skin cancer. But, we believe the significance

of this data goes well beyond CTCL, as it speaks to the targeting of p-STAT3 as a general strategy. We are excited to share these preliminary results in association with ASH (Free ASH Whitepaper), especially because we believe showing activity here could have exciting implications for the future of STAT3 inhibitors in general. Although this is a relatively small pilot study, we believe the results justify an expansion to a larger patient population in a Phase 2 clinical trial."

On November 19, 2019 Genmab A/S (Nasdaq: GMAB) reported that the European Commission (EC) has granted marketing authorization for DARZALEX (daratumumab) in combination with lenalidomide and dexamethasone (Rd) as treatment for adult patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant (ASCT) (Press release, Genmab, NOV 19, 2019, View Source [SID1234551468]). The EC approval follows a positive opinion issued for DARZALEX by the CHMP of the European Medicines Agency (EMA) in October 2019. In August 2012, Genmab granted Janssen Biotech, Inc. (Janssen) an exclusive worldwide license to develop, manufacture and commercialize daratumumab.

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"We are pleased that with this approval, patients in the European Union newly diagnosed with multiple myeloma who are not candidates for transplant will now have two potential options for treatment with DARZALEX containing regimens. We look forward to seeing the combination therapy of DARZALEX with lenalidomide and dexamethasone launched in Europe," said Jan van de Winkel, Ph.D., Chief Executive Officer of Genmab.

The approval was based on data from the Phase III MAIA (MMY3008) study of daratumumab in combination with Rd as treatment for patients with newly diagnosed multiple myeloma, who are not candidates for high dose chemotherapy and ASCT. Data from this study was published in The New England Journal of Medicine and was presented as a Late-Breaking Abstract at the 2018 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in December 2018.

About the MAIA (MMY3008) study
The Phase III study (NCT02252172) is a randomized, open-label, multicenter study that includes 737 newly diagnosed patients with multiple myeloma who are not candidates for high dose chemotherapy and ASCT. Patients were randomized to receive either treatment with daratumumab in combination with lenalidomide (an immunomodulatory drug) and dexamethasone (a corticosteroid) or treatment with lenalidomide and dexamethasone alone. In the daratumumab treatment arm, patients received 16 milligrams per kilogram (mg/kg) weekly for the first 8 weeks (Cycles 1 and 2), every other week for 16 weeks (Cycles 3 to 6) and then every 4 weeks (Cycle 7 and beyond) until progression of disease or unacceptable toxicity. Lenalidomide is administered at 25 mg orally on days 1 through 21 of each 28-day cycle, and dexamethasone is administered at 40 mg once a week for both treatment arms. Participants in both treatment arms will continue Rd until disease progression or unacceptable toxicity. The primary endpoint of the study is progression free survival.

About multiple myeloma
Multiple myeloma is an incurable blood cancer that starts in the bone marrow and is characterized by an excess proliferation of plasma cells.1 Approximately 16,830 new patients were expected to be diagnosed with multiple myeloma and approximately 10,480 people were expected to die from the disease in the Western Europe in 2018.2 Globally, it was estimated that 160,000 people were diagnosed and 106,000 died from the disease in 2018.3 While some patients with multiple myeloma have no symptoms at all, most patients are diagnosed due to symptoms which can include bone problems, low blood counts, calcium elevation, kidney problems or infections.4

About DARZALEX(daratumumab)
DARZALEX (daratumumab) intravenous infusion is indicated for the treatment of adult patients in the United States: in combination with bortezomib, thalidomide and dexamethasone as treatment for patients newly diagnosed with multiple myeloma who are eligible for autologous stem cell transplant; in combination with lenalidomide and dexamethasone for the treatment of patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant; in combination with bortezomib, melphalan and prednisone for the treatment of patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant; in combination with lenalidomide and dexamethasone, or bortezomib and dexamethasone, for the treatment of patients with multiple myeloma who have received at least one prior therapy; in combination with pomalidomide and dexamethasone for the treatment of patients with multiple myeloma who have received at least two prior therapies, including lenalidomide and a proteasome inhibitor (PI); and as a monotherapy for the treatment of patients with multiple myeloma who have received at least three prior lines of therapy, including a PI and an immunomodulatory agent, or who are double-refractory to a PI and an immunomodulatory agent.5 DARZALEX is the first monoclonal antibody (mAb) to receive U.S. Food and Drug Administration (U.S. FDA) approval to treat multiple myeloma. DARZALEX intravenous infusion is indicated for the treatment of adult patients in Europe: in combination with lenalidomide and dexamethasone for the treatment of patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant; in combination with bortezomib, melphalan and prednisone for the treatment of adult patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant; for use in combination with lenalidomide and dexamethasone, or bortezomib and dexamethasone, for the treatment of adult patients with multiple myeloma who have received at least one prior therapy; and as monotherapy for the treatment of adult patients with relapsed and refractory multiple myeloma, whose prior therapy included a PI and an immunomodulatory agent and who have demonstrated disease progression on the last therapy6. The option to split the first infusion of DARZALEX over two consecutive days has been approved in both Europe and the U.S. In Japan, DARZALEX intravenous infusion is approved for the treatment of adult patients: in combination with lenalidomide and dexamethasone, or bortezomib and dexamethasone for the treatment of relapsed or refractory multiple myeloma; in combination with bortezomib, melphalan and prednisone for the treatment of patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant. DARZALEX is the first human CD38 monoclonal antibody to reach the market in the United States, Europe and Japan. For more information, visit www.DARZALEX.com.

Daratumumab is a human IgG1k monoclonal antibody (mAb) that binds with high affinity to the CD38 molecule, which is highly expressed on the surface of multiple myeloma cells. Daratumumab triggers a person’s own immune system to attack the cancer cells, resulting in rapid tumor cell death through multiple immune-mediated mechanisms of action and through immunomodulatory effects, in addition to direct tumor cell death, via apoptosis (programmed cell death).5,6,7,8,9,10

Daratumumab is being developed by Janssen Biotech, Inc. under an exclusive worldwide license to develop, manufacture and commercialize daratumumab from Genmab. A comprehensive clinical development program for daratumumab is ongoing, including multiple Phase III studies in smoldering, relapsed and refractory and frontline multiple myeloma settings. Additional studies are ongoing or planned to assess the potential of daratumumab in other malignant and pre-malignant diseases in which CD38 is expressed, such as amyloidosis, NKT-cell lymphoma and B-cell and T-cell ALL. Daratumumab has received two Breakthrough Therapy Designations from the U.S. FDA for certain indications of multiple myeloma, including as a monotherapy for heavily pretreated multiple myeloma and in combination with certain other therapies for second-line treatment of multiple myeloma.

On November 19, 2019 Nordic Nanovector ASA (OSE: NANO) reported its results for the third quarter 2019. A presentation by the company’s senior management team will take place today in Oslo at 08:30 CET, see details below (Press release, Nordic Nanovector, NOV 19, 2019, View Source;results-for-the-third-quarter-2019-300960626.html [SID1234551505]).

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Eduardo Bravo, CEO, commented: "We are encouraged with the clinical results emerging from the different clinical trials with Betalutin in non-Hodgkin’s lymphoma (NHL). As stated by key opinion leaders in the field during the R&D Day hosted by Nordic Nanovector in September, the emerging profile of Betalutin is unique and very competitive. There are many patients who suffer from these devastating diseases who lack effective, safe and convenient treatment options. We remain fully committed to deliver on our timelines to bring this novel product to the market as quickly as possible."

Highlights Q3 `19

Updated analysis from Phase 1/2a LYMRIT 37-01 trial of Betalutin in relapsed/refractory follicular lymphoma (R/R FL) presented at R&D Day (September)
Median duration of response of 13.6 months for all responders and 32.0 months for complete responders (vs 9.0 and 20.7 months, respectively reported at ASH (Free ASH Whitepaper) 2018
Median follow-up time for responders of 30.0 months (range: 12.0 – 60.7 months)
Pivotal Phase 2b PARADIGME trial of Betalutin in advanced, recurrent and CD20 antibody treatment refractory 3L Follicular Lymphoma (FL) is progressing
87 sites in 24 countries open for enrolment as of November 18th, 2019
Patient enrolment is expected to be completed in the second half of 2020
Phase 1b Archer-1 trial of Betalutin in combination with rituximab (RTX) in patients with R/R 2nd line FL (2L FL) advanced to second cohort
100% Overall Response Rate (3/3 Complete Responses) observed in the first patient cohort
Promising preclinical results with Alpha37 for B-cell tumours presented at EANM conference by partner Orano Med
Alpha37 project received grant funding of EUR 0.6 million from Eurostars
Approximately NOK 243 million (USD 26.4m) (gross) raised 18th October in private placement of new shares providing additional funds to support the continued clinical development of Betalutin (including completion of enrolment of PARADIGME and all ongoing clinical trials), manufacturing and other activities in preparation for the commercialisation of Betalutin
Dr Gabriele Elbl appointed as VP Global Regulatory Affairs to drive the company’s Regulatory Affairs strategy
Financial Highlights Q3 `19

(Figures in brackets = same period 2018 unless otherwise stated)

Revenues for the third quarter amounted to NOK 0.0 (NOK 0.0 million). Revenues for the first nine months 2019 were NOK 0.0 (NOK 0.0 million)
Total operating expenses for the third quarter were NOK 100.2 million (NOK 76.9 million). Total operating expenses for the first nine months of 2019 amounted to NOK 301.1 million (NOK 243.7 million)
Research and development (preclinical, clinical, medical affairs, regulatory and CMC (Chemistry, Manufacturing and Controls) activities expenses accounted for 78 % of total operating expenses for the first nine months of 2019 (73 %)
Comprehensive loss for the third quarter amounted to NOK 93.6 million (loss of NOK 75.4 million). Comprehensive loss for the first nine months was NOK 295.6 million (loss of NOK 249.1 million)
Cash and cash equivalents amounted to NOK 345.9 million at the end of September 2019 (NOK 440.1 million at 31 December 2018), exclusive of new funds raised in October 2019 of NOK 243 million (gross)
Outlook

Nordic Nanovector aspires to become a leader in the field of targeted radioimmunotherapies for haematological cancers by developing, manufacturing and commercialising innovative products to address major unmet medical needs and advance cancer care.

Betalutin, the company’s most advanced radioimmunotherapy candidate, is developing a highly differentiated, competitive, clinical profile. Nordic Nanovector is confident that Betalutin could become an attractive and convenient once-only therapeutic option, which, based on detailed market research, has the potential to be commercially successful.

Betalutin is being developed for recurrent FL, based on the promising results from the LYMRIT 37-01 Phase 1/2 clinical trial. The company’s pivotal Phase 2b PARADIGME trial with Betalutin in 3L R/R FL is underway. Patient enrolment is expected to be completed in the second half of 2020. The study’s preliminary data read-out is planned a few months later. A BLA filing to gain marketing approval for Betalutin is expected in the first half of 2021. Nordic Nanovector intends to retain marketing rights and to actively participate in the commercialisation of Betalutin in core markets.

Nordic Nanovector intends to maximize the value of Betalutin across the major types of NHL (FL and DLBCL) and in earlier treatment lines in combination with standard treatments. The company is also evaluating opportunities with other CD37-targeting radioimmunotherapies across NHL and other haematological cancer indications.

Presentation and Webcast

A presentation by Nordic Nanovector’s senior management team will take place today at 8:30am CET at:

Thon Hotel Vika Atrium, Munkedamsveien 45, 0250 Oslo

Meeting Room: AKER

The presentation will be recorded as a webcast and will be available at www.nordicnanovector.com in the section: Investors & Media

The results report and the presentation will be available at www.nordicnanovector.com in the section: Investors & Media/Reports and Presentation/Interim Reports/2019.

On November 19, 2019 Mallinckrodt plc (NYSE: MNK), a global biopharmaceutical company, will present on Tuesday, Dec. 3, 2019, at the Piper Jaffray Annual Healthcare Conference at the Lotte New York Palace, 455 Madison Ave, New York (Press release, , NOV 19, 2019, View Source [SID1234551504]).

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Mark Trudeau, President and Chief Executive Officer, and Steven Romano, M.D., Executive Vice President and Chief Scientific Officer, will represent the company in a fireside chat at 8:30 a.m. Eastern.

Individuals who cannot attend the meeting in person can find webcast information at: http://www.mallinckrodt.com/investors. A replay will also be available following the meeting.

On November 19, 2019 Cellect Biotechnology Ltd. (NASDAQ: APOP), a developer of innovative technology which enables the functional selection of stem cells, reported financial and operating results for the third quarter ended September 30, 2019 and provided a corporate update (Press release, Cellect Biotechnology, NOV 19, 2019, View Source [SID1234551503]).

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Recent Highlights

Received an Investigational New Drug (IND) approval from the U.S. Food and Drug Administration (FDA) for the commencement of a clinical trial to determine the safety and tolerability of the ApoGraft technology for haploidentical bone marrow transplantations. This development represents the Company’s first-ever clinical trial approval in the U.S. using its ApoGraft stem cell selection technology, which is designed to significantly reduce acute graft-versus-host disease (aGVHD) following bone marrow transplantation.
Successfully validated the Company’s technology in collaboration with a regenerative medicine company. The study, when combined with others and internal findings increase the body of evidence supporting the Company’s technology and add further proof to support Cellect ASCs derived stem cells program. Biocompatibility with certain collagen-based matrixes successfully demonstrated that cells grown utilizing the Company’s protocol can be incorporated into matrixes for expansion, transplantation and tissue regeneration.
"Our clinical and regulatory teams remained focused during the third quarter and the more recent positive developments position us to achieve our goals, both in the U.S. and Israel," commented Dr. Shai Yarkoni, Chief Executive Officer. "In the U.S., the IND approval is a significant achievement and represents our first-ever FDA IND in the U.S., with Washington University School of Medicine. In Israel, our Phase 1/2 clinical study of ApoGraft is progressing slowly and we expect to complete the recruitment around the end of the year."

"With our prudent use of cash during the third quarter and the anticipated cash usage needs over the coming quarters, we continue to believe we have the resources to execute our clinical and regulatory plans for the foreseeable future," said Eyal Leibovitz, Chief Financial Officer.

Third Quarter 2019 Financial Results:

Research and development (R&D) expenses for the third quarter of 2019 were $0.71 million compared to $1.18 million in the third quarter of 2018. The Company remains committed to the ongoing clinical trials in Israel as well as pursuing the regulatory approval from the FDA to commence its US-based trial.

General and administrative (G&A) expenses for the third quarter of 2019 were $0.80 million compared to $1.13 million in the third quarter of 2018. The decrease reflects the cost cutting initiatives implemented by the Company during the third quarter of 2019.

Finance income for the third quarter of 2019 were $0.12 million compared to finance income of $0.36 million in the third quarter of 2018. The decrease was primarily due to changes related to fair value of the tradable and non-tradable warrants issued in prior fundraising.

Net loss for the third quarter of 2019 was $1.4 million, or $0.01 per share and $0.12 per ADS, compared to $1.9 million, or $0.014 per share and $0.29 per ADS, in the third quarter of 2018.

Cash and cash equivalents, $6.27 million as of September 30, 2019.
*For the convenience of the reader, the amounts above have been translated from NIS into U.S. dollars, at the representative rate of exchange on September 30, 2019 (U.S. $1 = NIS 3.482).

Strategic Review Progress Update

On May 16, 2019, the Company disclosed that it commenced plans to explore strategic alternatives to maximize shareholder value. Potential strategic alternatives that may be evaluated include, but are not limited to, an acquisition, merger, business combination, including in other business fields than the Company’s in-licensing, or other strategic transaction involving the Company or its assets. The Company continues to evaluate business development opportunities and will keep investors informed as they mature or warrant investor disclosure.