On February 6, 2020 Celsion Corporation (NASDAQ: CLSN) reported that the independent Data Safety Monitoring Board (DSMB) has completed its initial safety review of data from the first fifteen patients treated with the first four neoadjuvant doses of GEN-1 at 100 mg/m² in the ongoing Phase I/II OVATION 2 Study (Press release, Celsion, FEB 6, 2020, View Source [SID1234553952]). As requested by the U.S. Food and Drug Administration (FDA), a follow-on Phase 1 review by the DSMB will evaluate the safety of GEN-1 in up to 17 weekly doses before initiating the Phase 2 portion of the Study. The OVATION 2 Study combines GEN-1, the Company’s IL-12 gene-mediated immunotherapy, with neoadjuvant chemotherapy (NACT), a standard of care for newly diagnosed patients with Stage III and IV ovarian cancer. Following NACT, patients undergo interval debulking surgery (IDS) followed by three additional cycles of chemotherapy.

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The OVATION 2 Study is an open label, 130 patient, 1 to 1 randomized Phase II trial, 80% powered to show the equivalent of a 33% improvement in progression-free survival (PFS), the primary endpoint, when comparing the treatment arm (NACT + GEN-1) with the control arm (NACT alone). GEN-1, is a formulation of Celsion’s proprietary, synthetic, non-viral cell transfection platform TheraPlas, incorporating, DNA plasmids coded for the inflammatory protein, interleukin-12 (IL-12). Cell transfection is followed by persistent, local secretion of the IL-12 protein, expected at therapeutic levels.

The OVATION 2 Study builds on promising clinical and translational research data from the Phase IB OVATION I Study, in which enrolled patients received escalating weekly doses of GEN-1 (from 36 mg/m² to 79 mg/m²) for a total of eight treatments in combination with NACT, followed by IDS. These data from the OVATION I Study were presented at the ASCO (Free ASCO Whitepaper)-SITC Clinical-Oncology Symposium by Dr. Premal H. Thaker, M.D., M.S. on May 4, 2019 and can be reviewed here. In addition to exploring a higher dose of GEN-1 in the OVATION 2 Study, patients will continue to receive GEN-1 after their IDS in combination with adjuvant chemotherapy.

"This latest DSMB review of GEN-1 at 100 mg/m² confirmed that there were no dose limiting toxicities detected in any of the six evaluable patients (those patients who received at least four doses of GEN-1) and that intraperitoneal GEN-1 administration is well tolerated even when given with standard NACT," said Nicholas Borys, M.D., executive vice president and chief medical officer of Celsion. "Of the fifteen patients treated in the Phase I portion of the OVATION 2 Study, nine patients were treated with GEN-1 plus NACT and six patients were treated with NACT only. After the final six patients in the Phase I portion of the Study have completed their interval debulking surgery, we will be reporting surgical results and overall tumor response rates for all fifteen patients from the Phase I portion of the trial later this quarter."

Dr. Borys concluded, "We anticipate that the Phase II portion of the OVATION 2 Study will begin enrolling patients at more than 25 clinical sites in the U.S. and Canada beginning in the second quarter of 2020. As requested by the FDA, a follow-on safety review will be conducted by the DSMB after all the patients have concluded their chemotherapy dosing. The Study protocol allows for up to 17 doses of GEN-1. Our goal is to complete enrollment of all patients in the OVATION 2 Study by the first quarter of 2021."

The OVATION 2 Study is supported with promising clinical and translational data from the Company’s prior Phase Ib OVATION I Study. In addition to a 100% objective response rate (complete response + partial response) observed in all 9 patients at the two highest dose cohorts, translational research data demonstrates that GEN-1 is biologically active, producing therapeutic levels of IL-12 cytokines and positively impacting T-cell population in the tumor.

On February 6, 2020 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 that it has entered into definitive agreements with institutional investors to purchase an aggregate of up to 7,500,000 shares and warrants to purchase 5,625,000 shares of common stock at a combined public offering price of $0.80 per share and related warrant in a registered direct offering (Press release, Moleculin, FEB 6, 2020, View Source [SID1234553951]).

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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.)

Each warrant will have an exercise price of $1.05 per share and is exercisable six months and one day after issuance. The warrants will expire five years from the date they first become exercisable. The shares of common stock and the related warrants can only be purchased together in this offering but will be issued separately and will be immediately separable upon issuance. The offering is expected to close on or about February 10, 2020, subject to customary closing conditions.

The gross proceeds of the offering are expected to be $6.0 million, prior to deducting the placement agent fees and other estimated offering expenses.

Oppenheimer & Co. Inc. is acting as the sole placement agent for the offering. Roth Capital Partners and Maxim Group LLC are acting as financial advisors to the Company.

The Company intends to use the net proceeds of the offering to fund its planned clinical trials, preclinical programs, for other research and development activities and for general corporate purposes.

The securities described above are being offered pursuant to a prospectus supplement and an accompanying prospectus forming part of a shelf registration statement on Form S-3 (No. 333-219434) previously filed with and declared effective by the Securities and Exchange Commission (SEC). A final prospectus supplement and the accompanying prospectus relating to and describing the terms of the offering will be filed with the SEC and will be available on the SEC’s website at View Source Copies of the final prospectus supplement and the accompanying prospectus may be obtained, when available, from Oppenheimer & Co. Inc., Attention: Syndicate Prospectus Department, 85 Broad Street, 26th Floor, New York, New York 10004, by telephone at (212) 667-8055, or by email at [email protected].

This press release shall not constitute an offer to sell or the solicitation of an offer to buy any of the securities described herein, nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

On February 6, 2020 Magenta Therapeutics (NASDAQ: MGTA), a clinical-stage biotechnology company developing novel medicines to bring the curative power of immune reset to more patients, reported that the company is scheduled to participate in a fireside chat at the Guggenheim Healthcare Talks Oncology Day on Thursday, February 13th, 2020, at 11:00 a.m. ET at the St. Regis Hotel in New York, NY (Press release, Magenta Therapeutics, FEB 6, 2020, View Source [SID1234553950]).

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A live webcast of the fireside chat can be accessed on the Magenta Therapeutics website at View Source The webcast replay will be available for 90 days following the event.

On February 6, 2020 Oncternal Therapeutics, Inc. (Nasdaq: ONCT), a clinical-stage biopharmaceutical company focused on the development of novel oncology therapies, reported the presentation of preclinical data from its ROR1 chimeric antigen receptor T cell (CAR-T) program at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) – Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) meeting in Orlando, Florida (Press release, Oncternal Therapeutics, FEB 6, 2020, View Source [SID1234553949]). A copy of the poster presentation is available online at www.oncternal.com.

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In preclinical studies, anti-ROR1 CAR-T constructs were evaluated in an animal model of human leukemia. A single dose of anti-ROR1 CAR T-cells expanded in treated animals and the chimeric T cells trafficked to the disease sites. By week four, leukemia cells were cleared from major tissue reservoirs, including bone marrow, kidneys and spleen. CAR-T cell-treated animals survived longer than 90 days compared to 21 days for animals in control groups. The CAR-T cells were highly active and detected in mouse tissues more than two months after injection.

This research effort was led by Professor Thomas J. Kipps, M.D., Ph.D., and Charles Prussack, Pharm.D., Ph.D., at the University of California San Diego (UC San Diego) under a research grant from the California Institute of Regenerative Medicine (CIRM).

"It is exciting to see the potent preclinical activity of the ROR1 CAR-T cell therapy and its selectivity in targeting tumors," said Thomas Kipps, M.D., Ph.D., Professor of Medicine, Evelyn and Edwin Tasch Chair in Cancer Research, and Deputy Director of Research Operations at the UC San Diego Moores Cancer Center. "This CAR-T cell product utilizes the ROR1 binding domain derived from cirmtuzumab (UC-961), a clinical stage antibody that is currently being evaluated in patients with hematological malignancies and solid tumors. The challenges of CAR-T therapies include patient relapses due to the loss of target antigen and safety issues due to targeting of normal cells expressing the antigen. Harnessing cirmtuzumab’s specificity for ROR1 expressed on cancer cells has the potential to improve CAR-T efficacy and safety, and address the high unmet medical need for treating patients with aggressive cancers."

"We are encouraged by the preclinical results of this ROR1 CAR-T program and look forward to advancing it to clinical testing, initially for treating patients with hematological cancers, potentially in the fourth quarter of this year," said James Breitmeyer, M.D., Ph.D., Oncternal’s President and CEO.

About ROR1 CAR-T

Oncternal Therapeutics is developing a ROR1-targeting (Receptor tyrosine kinase-like Orphan Receptor 1) chimeric antigen receptor T cell therapy (CAR-T) as a potential treatment for patients with aggressive hematological malignancies or solid tumors, in collaboration with the UC San Diego School of Medicine and the California Institute for Regenerative Medicine (CIRM). The Company’s CAR-T program is based on the binding domain of cirmtuzumab, which is an investigational, potentially first-in-class monoclonal antibody targeting ROR1. Cirmtuzumab is currently being evaluated in a Phase 1/2 clinical trial in combination with ibrutinib for the treatment of patients with chronic lymphocytic leukemia, or CLL, or mantle cell lymphoma, or MCL, and in an investigator-initiated Phase 1 clinical trial in combination with paclitaxel for the treatment of women with metastatic or unresectable breast cancer.

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. Because the epitope of ROR1 recognized by cirmtuzumab appears to be restricted to tumor cells, a cirmtuzumab-based CAR-T may be selective in distinguishing cancer from normal tissues. Cirmtuzumab and ROR1 CAR-T cell therapy have not been approved by the U.S. Food and Drug Administration for any indication.

On February 6, 2020 Molecular Partners AG (SIX: MOLN), a clinical-stage biotech company that is developing a new class of drugs known as DARPin therapies, reported its unaudited financial results for 2019 and corporate highlights for the fourth quarter 2019 (Press release, Molecular Partners, FEB 6, 2020, View Source [SID1234553948]). The fourth quarter was marked by positive updated data on MP0250, refinement of the MP0250 development strategy, and noteworthy progress on the company’s pipeline of novel therapeutic designs.

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"We have delivered on our commitment to constructing groundbreaking therapeutic designs and advancing them rapidly to patients. Both the initiation of our phase 1 trial for MP0310 and the nomination of MP0317 as our second immuno-oncology candidate have underscored the potential of our novel therapeutic designs," said Patrick Amstutz, Ph.D., Chief Executive Officer of Molecular Partners. "As we embark on this new year, we look forward to FDA and EMA review of the regulatory submissions for abicipar, and working with our partner Allergan to deliver the first commercialized DARPin therapeutic to patients with neovascular AMD."

Abicipar: AAO Data highlight that vision gains were maintained throughout second year
In Q4 2019, two-year data from the CEDAR and SEQUOIA clinical studies of investigational Abicipar in patients with neovascular (wet) age-related macular degeneration (nAMD) were presented as a late-breaking oral presentation during Retina Subspecialty Day at the Annual Meeting of the American Academy of Ophthalmology (AAO). In the second year of these studies, four injections of Abicipar resulted in the maintenance of visual gains comparable to monthly ranibizumab.

Through week 104, patients received Abicipar 2 mg every 8-weeks or every 12-weeks or ranibizumab 0.5 mg every 4 weeks. At week 104 in the pooled Phase 3 data, the proportion of patients with stable vision was 93%, 90% and 94% in 8-week Abicipar; 12-week Abicipar and 4-week ranibizumab treatment regimens, respectively. This continuation of stable vision in year 2 further reinforces the ability of Abicipar to deliver consistent quarterly dosing for the majority of patients.

The pooled rate of new cases of intraocular inflammation in year two for patients who received Abicipar in the 8-and 12-week arms was 1.9%, which is similar to the ranibizumab arm of 1%.

The U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA) are currently reviewing regulatory applications for Abicipar in patients with nAMD. The FDA is expected to take action on the BLA in mid-2020. A decision from the European Commission is expected in the second half of 2020.

Oncology: Updated data and Orphan Drug Designation for MP0250 in multiple myeloma
Data presented at the 61st Annual Meeting of the American Society of Hematology (ASH) (Free ASH Whitepaper) in December 2019 indicate that MP0250 continues to show long-lasting and deepening responses across a variety of patients with multiple myeloma in the relapsed/refractory setting. MP0250 is a multi-DARPin candidate that targets hepatocyte growth factor (HGF) and vascular endothelial growth factor (VEGF), two prominent tumor escape pathways, and has the potential to improve sensitivity, or re-sensitize patients, to existing and emerging treatments.

The phase 2 trial for MP0250 in combination with bortezomib (Velcade) and dexamethasone in patients with multiple myeloma who have failed standard therapies is ongoing. The company announced at its R&D Day in December 2019 its intent to evaluate partnering opportunities for MP0250. In conjunction with this endeavor, the company announced it will not start the previously planned clinical trial investigating MP0250 in combination with an IMiD. This is aligned with the company’s corporate strategy to pursue combination data for the most relevant clinical combinations of MP0250, which would be more appropriately determined in collaboration with a partner. Additionally, the company announced in December 2019 that MP0250 has received Orphan Drug Designation by the U.S. Food and Drug Administration (FDA).

Immuno-oncology: Phase 1 trial of MP0310 continues dose escalation
For MP0310 (AMG 506), the phase 1 MP0310-CP101 trial is ongoing and dose escalation is underway. The trial expects to enroll up to 54 patients at three sites in France to evaluate the safety, tolerability and pharmacokinetics of MP0310 in patients with locally advanced or metastatic solid tumors. Current clinical timelines are on track; the trial is expected to expand into additional combination cohorts in H2 2020. These combination trials will be conducted by Amgen.

Immuno-oncology: MP0317 (FAP x CD40) nominated as next IND candidate stemming from the company’s immuno-oncology DARPin toolbox
In Q4 2019, Molecular Partners nominated tumor-localized immune agonist MP0317 as the second DARPin protein in the company’s immuno-oncology pipeline. MP0317 comprises localizer (FAP) and stimulator (CD40) DARPin domains. It is designed to activate immune cells specifically in the tumor and not in the rest of the body, potentially delivering greater efficacy with fewer side effects.

Preclinical data demonstrated that the company’s multi-specific FAP x CD40 DARPin molecule induced FAP-dependent activation of B cells, dendritic cells and macrophages.

Oncology: Dosing ongoing in trial for MP0274 in HER2-positive solid tumors
Recruitment for the phase 1 trial for MP0274 and the dose escalation phase continues. MP0274 is a multi-DARPin product candidate being developed for the treatment of HER2-positive solid tumors. In preclinical trials MP0274 inhibits downstream signaling pathways, and directly kills HER2-addicted tumor cells through the induction of apoptosis. This represents a new and differentiated mode of action as compared to current standard of care antibodies.

Financial highlights: Net result and cash position on previous year’s level
In the financial year 2019, Molecular Partners recognized total revenues of CHF 20.4 million (2018: CHF 10.4 million) and incurred total expenses of CHF 57.6 million (2018: CHF 47.8 million). This led to an operating loss of CHF 37.2 million for 2019 (2018: Operating loss of CHF 37.4 million). The net financial result of CHF 0.4 million recorded in 2019 remained on the same level as in 2018. This resulted in a 2019 net loss of CHF 36.8 million (2018: Net loss of CHF 37.0 million).

The net cash used for operating activities in 2019 was CHF 1.2 million (2018: net cash used of CHF 42.5 million). Including time deposits, the cash and cash equivalents position decreased by CHF 3.9 million vs. year-end 2018 to CHF 95.1 million as of December 31, 2019 (December 31, 2018: CHF 99.0 million). Total shareholders’ equity stood at CHF 53.6 million as of December 31, 2019, a decrease of CHF 38.1 million (December 31, 2018: CHF 91.7 million).

As of December 31, 2019, the company employed 135 FTE, up 15% compared to year-end 2018. Approximately 85% of the employees are employed in R&D-related functions.

Business outlook and priorities
In 2020, Molecular Partners anticipates regulatory decisions by the FDA and EMA regarding the market launch of abicipar for patients with nAMD. The FDA is expected to take action on the BLA in mid-2020, and a decision from the European Commission is expected in the second half of 2020. Molecular Partners continues to work closely with its partner Allergan in the preparation and education of the market for the expected launch.

In immuno-oncology, recruitment of patients will continue in the phase 1 trial of MP0310 (AMG 506). Molecular Partners and Amgen expect to collect initial data from this trial in H2 2020.

In oncology, the company intends to continue to advance its phase 2 trial of MP0250 in patients with multiple myeloma in combination with Velcade and will pursue partnership opportunities for the MP0250 program. The company further plans in 2020 to establish dosing and clinical strategy for MP0274, as that therapeutic candidate concludes its phase 1 dose escalation.

Additionally, Molecular Partners will continue to advance its immuno-oncology research pipeline, specifically the MP0317, the CD3 DARPin T cell-engager platform and peptide-MHC programs.

Financial outlook 2020
For the FY 2020, at constant exchange rates, the company expects total expenses of CHF 60-70 million, of which around CHF 6 million will be non-cash effective costs for share-based payments, IFRS pension accounting and depreciations. The increase versus the previous year is driven by the progress of the company’s pipeline as well as the budgeted growth of the company’s workforce. Capital expenditures in FY 2020 are expected to be approximately CHF 3 million.

This guidance is subject to the progress of the pipeline, mainly driven by manufacturing costs, the speed of enrollment of patients in clinical trials and data from research and development projects. No guidance can be provided with regard to net cash flow projections. Timelines and potential milestone payments for existing and potentially new partnerships are not disclosed.

Investor documentation of FY 2019 results
This FY 2019 press release as well as the FY 2019 results presentation are available on the investors section of the company’s website.

FY 2019 results presentation, conference call and audio webcast
Molecular Partners will hold the FY 2019 results presentation in its headquarters in Zurich-Schlieren on February 06, 2020, 2:00pm CET (1:00pm GMT, 8:00am EST). For those who are unable to participate in the live event, the company provides conference call and audio webcast facilities.

In order to register for the FY 2019 conference call, please dial the following numbers approximately 10 minutes before the start of the presentation:

Switzerland / Europe +41 (0) 58 310 5000

UK +44 (0) 203 059 5862

USA +1 (1) 631 570 5613

Participants will have the opportunity to ask questions after the presentation.

Audio webcast
The FY19 results presentation will be webcast live and will be made available on the company’s website under the Investors section. The replay will be available for 90 days following the presentation.

Financial Calendar
March 20, 2020 Expected Publication of Annual Report 2019
April 29, 2020 Annual General Meeting
May 7, 2020 Interim Management Statement Q1 2020
August 26, 2020 Publication of Half-year Results 2020 (unaudited)
October 29, 2020 Interim Management Statement Q3 2020
View Source

About the DARPin Difference
DARPin therapeutics are a new class of protein therapeutics opening an extra dimension of multi-specificity and multi-functionality. DARPin candidates can engage more than five targets, offering potential benefits over those offered by conventional monoclonal antibodies or other currently available protein therapeutics. The DARPin technology is a fast and cost-effective drug discovery engine, producing drug candidates with ideal properties for development and very high production yields.

With their low immunogenicity and long half-life in the bloodstream and the eye, DARPin therapeutics have the potential to advance modern medicine and significantly improve the treatment of serious diseases, including cancer and sight-threatening disorders. Molecular Partners is partnering with Allergan to advance clinical programs in ophthalmology and is advancing a proprietary pipeline of DARPin drug candidates in oncology and immuno-oncology. The most advanced global product candidate in partnership with Allergan is abicipar, a molecule for which phase 3 data have been filed to the respective regulators in both the US and in Europe. Several DARPin molecules for various ophthalmic indications are also in preclinical development. The most advanced DARPin therapeutic candidate wholly owned by Molecular Partners, MP0250, is in phase 2 clinical development for the treatment of hematological tumors. MP0274, the second-most advanced DARPin candidate owned by Molecular Partners, binds to Her2 and inhibits downstream signaling, which leads to induction of apoptosis. MP0274 is currently in phase 1. The company’s lead immuno-oncology product candidate MP0310 is a FAP x 4-1BB multi-DARPin therapeutic candidate designed to locally activate immune cells in the tumor by binding to FAP on tumor stromal cells (localizer) and co-stimulating T cells via 4-1BB (immune modulator). Molecular Partners has closed a collaboration agreement with Amgen for the exclusive clinical development and commercialization of MP0310. The molecule has entered in phase 1 of clinical development in H2 2019. MP0317 (FAP x CD40), the second tumor-localized immune agonist stemming from the company’s "I/O toolbox", has been nominated as next DARPin protein in Molecular Partners’ immuno-oncology pipeline. Molecular Partners is further advancing a growing preclinical and research pipeline in immuno-oncology and additional development programs such as novel therapeutic designs to target peptide-MHC complexes. DARPin is a registered trademark owned by Molecular Partners AG.