On March 4, 2021 NuCana plc (NASDAQ: NCNA) reported its financial results for the fourth quarter and year ended December 31, 2020 and provided an update on its broad clinical program with its transformative ProTide therapeutics (Press release, Nucana BioPharmaceuticals, MAR 4, 2021, View Source [SID1234576088]).

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As of December 31, 2020, NuCana had cash and cash equivalents of £87.4 million compared to £100.7 million at September 30, 2020 and £52.0 million at December 31, 2019. NuCana continues to advance its various clinical programs and reported a net loss of £12.3 million for the quarter ended December 31, 2020, as compared to £7.7 million for the quarter ended December 31, 2019. Net loss for the year ended December 31, 2020 was £30.7 million, compared to a net loss of £21.4 million for the year ended December 31, 2019. Basic and diluted loss per share was £0.24 for the quarter and £0.81 for the year ended December 31, 2020, as compared to £0.24 per share for the comparable quarter and £0.66 for the year ended December 31, 2019.

"2020 was a very successful year for NuCana," said Hugh S. Griffith, NuCana’s Founder and Chief Executive Officer. "Against the challenging backdrop of the COVID-19 pandemic, we continued to advance all of our clinical programs and generate encouraging clinical and non-clinical data. Throughout 2020, we made good progress in the Phase III NuTide:121 biliary tract cancer study and expect to enroll sufficient patients in 2021 to complete the first interim analysis in 2022, which may support an accelerated approval filing. In September, we provided an update on two of our lead programs at ESMO (Free ESMO Whitepaper) that further validate the use of our proprietary ProTide technology. NUC-3373 continued to demonstrate its potential to offer enhanced efficacy, an improved safety profile and a more convenient dosing regimen as compared to 5-FU in patients with colorectal cancer. We also announced the first-ever clinical data for NUC-7738, which showed promising indications of anti-cancer activity and a favorable tolerability profile."

Mr. Griffith continued: "During 2020, we completed a successful $80 million follow-on offering that significantly strengthens our balance sheet. We also appointed a new non-executive Chairman, Andrew Kay and a new non-executive director, Bali Muralidhar. Both individuals bring a wealth of experience and we look forward to their contributions as we advance NuCana’s programs."

Mr. Griffith concluded: "We were excited to begin 2021 with the announcement of additional interim data for NUC-3373 in patients with colorectal cancer that continued to demonstrate encouraging efficacy and safety at ASCO (Free ASCO Whitepaper) GI. We look forward to continuing to drive recruitment across all of our ongoing studies, announce additional clinical data, and initiate new studies in 2021. We remain focused on our mission of significantly improving treatment outcomes for patients with cancer."

Anticipated 2021 Milestones

Acelarin is a ProTide transformation of gemcitabine. In 2021, NuCana expects to:

Complete recruitment sufficient to enable the first interim analysis in 2022 of the Phase III study of Acelarin combined with cisplatin as a first-line treatment for patients with advanced biliary tract cancer.

NUC-3373 is a ProTide transformation of the active anti-cancer metabolite of 5-FU. In 2021, NuCana expects to:

Report data from the Phase Ib study (NuTide:302) of NUC-3373 in combination with other agents with which 5-FU is typically combined, such as leucovorin, oxaliplatin and irinotecan in patients with advanced colorectal cancer.
Initiate and report data from a Phase Ib expansion / Phase II study of NUC-3373 in combination with other agents for patients with colorectal cancer.
Initiate a Phase III study of NUC-3373 in combination with other agents for patients with colorectal cancer.
Report data from the Phase I study (NuTide:301) of NUC-3373 in patients with advanced solid tumors.
NUC-7738 is a ProTide transformation of a novel nucleoside analog, 3’-deoxyadenosine. In 2021, NuCana expects to:

Report data from the Phase I study (NuTide:701) of NUC-7738 in patients with advanced solid tumors.
Initiate a Phase II study of NUC-7738 in patients with solid tumors.

On March 4, 2021 Navidea Biopharmaceuticals, Inc. (NYSE American: NAVB) ("Navidea" or the "Company"), a company focused on the development of precision immunodiagnostic agents and immunotherapeutics, reported that it has executed an agreement with the Company’s largest shareholder, John K. Scott, Jr., to purchase $5 million of newly-designated Series E Redeemable Convertible Preferred Stock (Press release, Navidea Biopharmaceuticals, MAR 4, 2021, View Source [SID1234576087]). Over the next 18 months, the Series E Preferred can be converted into approximately 2.17 million shares of Navidea’s common stock.

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"We are pleased to receive continued support from our largest shareholder. Today’s financing provides additional strength to our balance sheet and will allow our company to accelerate potential value-accretive investments, including our therapeutics pipeline," commented Jed Latkin, Chief Executive Officer of Navidea. "We look forward to providing additional updates on the progress of our ongoing studies across our portfolio on our earnings call later this month."

John K. Scott, Jr. commented, "I am pleased to be given the opportunity to continue to support Navidea as it moves some of its key assets into the next phase of development. As a stockholder in Navidea since December 2003, I believe in the Company’s drug candidates and the positive contributions they can make to individuals and the scientific community."

The securities offered and to be sold by Navidea in the private placement to Mr. Scott have not been registered under the Securities Act of 1933 (the "Securities Act"), as amended, or state securities laws and may not be offered or sold in the United States absent registration with the Securities and Exchange Commission (the "SEC") or an applicable exemption from registration requirements. Navidea has agreed to file a registration statement with the SEC covering the resale of the shares of common stock to be issued to Mr. Scott.

This press release shall not constitute an offer to sell or the solicitation of an offer to buy any of Navidea’s securities. No offer, solicitation or sale will be made in any state or other jurisdiction in which such offering, solicitation or sale would be unlawful.

On March 4, 2021 Alector, Inc. (Nasdaq: ALEC), a clinical-stage biotechnology company pioneering immuno-neurology, reported that Shehnaaz Suliman, M.D., MBA, M.Phil., president and chief operating officer of Alector, will participate in a fireside chat at the Barclays Global Healthcare Conference on Thursday, March 11, 2021, at 2:25 p.m. ET.

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A live webcast of the fireside chat will be available on the "Events & Presentations" page within the Investors section of the Alector website at View Source A replay will be available on the Alector website for 30 days following the event.

On March 4, 2021 Ascendis Pharma A/S (Nasdaq: ASND), a biopharmaceutical company that utilizes its innovative TransCon technologies to create product candidates that address unmet medical needs, reported that the company will hold a conference call and live webcast on Wednesday, March 10, 2021 at 4:30 p.m. Eastern Time (ET) to review its 2020 financial results and provide a business update (Press release, Ascendis Pharma, MAR 4, 2021, View Source [SID1234576085]).

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A live webcast of the conference call will be available on the Investors and News section of the Ascendis Pharma website at www.ascendispharma.com. A webcast replay will be available on this website shortly after conclusion of the event for 30 days.

On March 4, 2021 Kadmon Holdings, Inc. (NASDAQ:KDMN) reported financial and operational results for the fourth quarter and full year ended December 31, 2020 (Press release, Kadmon, MAR 4, 2021, View Source [SID1234576084]).

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"With the acceptance of Kadmon’s new drug application for belumosudil in cGVHD in hand, we are preparing for our potential commercial launch in light of our May 30th PDUFA date," said Harlan W. Waksal, M.D., President and CEO of Kadmon. "We are leveraging our existing commercial infrastructure and have thus nearly completed the scale-up required to launch belumosudil, if approved. We continue to seek to understand the needs of cGVHD patients in key transplant centers throughout the United States and look forward to working closely with FDA to bring this much-needed therapy to cGVHD patients in the coming months."

Dr. Waksal added, "We remain enthusiastic about belumosudil’s potential in systemic sclerosis; our ongoing placebo-controlled Phase 2 trial continues to attract patients in spite of the broader COVID backdrop and our plans to initiate our open-label trial in this indication are squarely on track. Additionally, we recently completed enrollment in the first two cohorts of our Phase 1 clinical trial of KD033, our anti-PD-L1/IL-15 fusion protein, in patients with metastatic or locally advanced solid tumors. We look forward to sharing initial data from both of these programs in late 2021. By completing a transformative convertible bond financing in February 2021 that was led by fundamental, healthcare investors, we have over $300 million in capital and are well positioned to achieve our strategic goals."

2021 Anticipated Key Clinical Milestones:

Belumosudil

Continue ongoing dialogue with the U.S. Food and Drug Administration (FDA) as they review the New Drug Application (NDA) of belumosudil, the Company’s (ROCK2) inhibitor, for the treatment of patients with chronic graft-versus-host disease (cGVHD).
The FDA set a Prescription Drug User Fee Act (PDUFA) date for belumosudil of May 30, 2021. The NDA is being reviewed under the FDA’s Real-Time Oncology Review (RTOR) pilot program, which aims to explore a more efficient review process to ensure safe and effective treatments are available to patients as early as possible.
The review of the belumosudil NDA is also being conducted under Project Orbis, an initiative of the FDA Oncology Center of Excellence. Project Orbis provides a framework for concurrent submission and review of oncology drugs among participating international countries.
Continue belumosudil commercial launch readiness activities in anticipation of potential FDA approval in 1H 2021. The Company has the majority of its planned field sales and medical science liaison teams. The Company believes these focused teams can adequately address the concentrated cGVHD market, where approximately 80% of patients are treated at 70 U.S. transplant centers.
Initiate small (12-15 patient), open-label Phase 2 clinical trial of belumosudil in patients with dcSSc (Study KD025-215) in 2021. The Company plans to present initial data from this study by year-end 2021.
Continue enrollment in ongoing placebo-controlled Phase 2 clinical trial in dcSSc (KD025-209); the Company continues to work with sites and trial coordinators to facilitate patient enrollment amid the COVID-19 pandemic
KD033

Enrollment in the first two dose cohorts was successfully completed in the ongoing dose-escalation Phase 1 clinical trial of KD033, Kadmon’s anti-PD-L1/IL-15 fusion protein, in patients with metastatic or locally advanced solid tumors; enrollment is ongoing in the next dose level (cohort 3).
The Company plans to present initial data from KD033-101 in 2021.
KD045

Continue investigational new drug (IND)-enabling activities for KD045, Kadmon’s next-generation ROCK inhibitor for the treatment of fibrotic diseases.
Financial Results

Fourth Quarter 2020 Results

Loss from operations for the three months ended December 31, 2020 was $31.2 million compared to $18.3 million for the same period in 2019.

The increase in loss from operations for the three months ended December 31, 2020 as compared to 2019 was primarily attributable to increased expenses related to clinical development and regulatory costs for belumosudil, as well as costs related to preparation for the potential launch of belumosudil. Additionally, the fourth quarter of 2019 included $4.0 million in license revenues related to the BioNova strategic partnership.

Full Year 2020 Results

Loss from operations for the year ended December 31, 2020 was $102.3 million, compared to $89.1 million for the same period in 2019.

The increase in loss from operations for the year ended December 31, 2020 as compared to 2019 was primarily related to clinical development and regulatory costs for belumosudil, as well as costs related to preparation for the potential launch of belumosudil. This increase in expenses was partially offset by an increase in license revenues of $2.0 million related to the Meiji strategic partnership.

Liquidity and Capital Resources

At December 31, 2020, the Company’s cash, cash equivalents and marketable debt securities totaled $123.9 million, compared to $139.6 million at December 31, 2019. On February 16, 2021, the Company issued $240.0 million aggregate principal amount of 3.625% convertible senior notes due 2027 (the "Notes"), in a private offering conducted in accordance with Rule 144A under the Securities Act of 1933, which included the full exercise of the $40 million over-allotment option. The initial conversion price of the Notes is equivalent to $6.96 per share, a premium of approximately 30% over the $5.35 per share closing price of Kadmon’s common stock on February 10, 2021. The Company entered into capped call transactions to raise the conversion price to $10.70 per share, a premium of 100% over the $5.35 per share closing price of Kadmon’s common stock on February 10, 2021. The Company received net proceeds from the offering of approximately $200.0 million, after deducting the initial purchasers’ discount and the cost of the capped call transactions.

In addition, as of December 31, 2020, the Company held approximately 0.7 million ordinary shares of MeiraGTx Holdings plc (MeiraGTx), a clinical-stage gene therapy company, as compared to approximately 2.1 million ordinary shares held as of December 31, 2019. During fiscal year ended December 31, 2020, the Company entered into multiple transactions pursuant to which it sold approximately 1.4 million ordinary shares of MeiraGTx for total net proceeds of approximately $19.8 million.

About Belumosudil

Belumosudil (KD025) is a selective oral inhibitor of Rho-associated coiled-coil kinase 2 (ROCK2), a signaling pathway that modulates inflammatory response and pro-fibrotic processes. In November 2020, the U.S. Food and Drug Administration (FDA) accepted the NDA for belumosudil for the treatment of patients with cGVHD. The FDA granted Priority Review for the NDA for belumosudil and assigned a Prescription Drug User Fee Act (PDUFA) target action date of May 30, 2021. The NDA is being reviewed under the FDA’s Real-Time Oncology Review (RTOR) and Project Orbis pilot programs. The FDA has granted Breakthrough Therapy Designation to belumosudil for the treatment of patients with cGVHD after failure of two or more lines of systemic therapy as well as Orphan Drug Designation to belumosudil for the treatment of cGVHD.