On March 29, 2022 Vincerx Pharma, Inc. (Nasdaq: VINC), a biopharmaceutical company aspiring to address the unmet medical needs of patients with cancer through paradigm-shifting therapeutics, reported financial results for the fourth quarter and full year ended December 31, 2021, and provided a corporate update (Press release, Vincerx Pharma, MAR 29, 2022, View Source [SID1234611094]).

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"We are excited to report the recent dosing of the first patient in the VIP152 and pembrolizumab combination arm of our ongoing Phase 1b trial. Additionally, in December, we dosed the first patient in our Phase 1b dose-escalation study of VIP152 in relapsed or refractory chronic lymphocytic leukemia or Richter Syndrome, marking the initiation of the second Vincerx-sponsored clinical trial in 2021," said Ahmed Hamdy M.D., Chief Executive Officer of Vincerx. "While we have experienced challenges to patient enrollment in our two ongoing Phase 1b trials similar to those faced by many of our peers due to the effect of the ongoing pandemic, we are proactively taking steps to address this with the addition of multiple new trial sites globally and are encouraged by an acceleration in recruitment to start the year. Moreover, on a strategic level, we are aligning our clinical trial design with the FDA’s Project Optimus initiative (View Source), which strives to reform the dose optimization and dose selection paradigm in oncology drug development. While we believe we have identified the recommended Phase 2 dose of VIP152, we are planning a dose optimization cohort to position VIP152 for success as we move forward with our clinical trials. We remain on track to initiate Phase 2 studies of VIP152 in the second half of this year."

"In parallel, we continue to advance our preclinical bioconjugation pipeline, comprising our diverse, modular platform of linkers and payloads that can be conjugated with typical antibodies, bispecifics, and small molecules, creating the potential for novel drugs with improved efficacy and safety compared with current antibody-drug conjugates. We recently announced the publication of key preclinical data for our small molecule-drug conjugate, VIP236, in the journal Cancers, which highlighted VIP236’s potential to direct a potent cancer chemotherapy to tumors while sparing healthy tissues. We are on-target to file an IND for VIP236 in the second half of the year," continued Dr. Hamdy.

"Our balance sheet, strengthened by the proceeds from our successfully completed private placement last September, positions us to continue to execute on our upcoming clinical and regulatory milestones," concluded Dr. Hamdy.

Recent Corporate Highlights

Received Investigational New Drug (IND) Application approval for Phase 1/2 study of VIP152, venetoclax, and prednisone (VVIP) in relapsed or refractory lymphoid malignancies in collaboration with the NIH

Dosed first patient in the VIP152 and pembrolizumab combination arm of the Company’s ongoing Phase 1b study (VNC-152-101)
Announced dosing of first patient in the Company’s Phase 1 dose-escalation study of VIP152 in relapsed or refractory (R/R) chronic lymphocytic leukemia (CLL) or Richter Syndrome (RS) (VNC-152-102)

Received Orphan Drug Designation from European Commission for VIP152 for the Treatment of Diffuse Large B-Cell Lymphoma (DLBCL)

Announced poster presentation, "VIP152, a selective CDK9 inhibitor, demonstrates sensitivity in gynecologic cell lines that are cisplatin sensitive or resistant and delivers in vivo antitumor efficacy," at upcoming American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting in April 2022

Hosted key opinion leader webinar and presented data on VIP152 in high-grade B-cell lymphoma (HGBL) and CLL at the 63rd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting 2021

Completed GLP toxicity studies of VIP236 in rats; initiating additional GLP toxicity studies in dogs to continue preclinical advancement of VIP236 towards IND filing in 2H22

Approved for research grant program from the German government providing for tax refunds/reimbursements of up to EUR 1 million per year over six years
Several key publications:
Publication of a peer-reviewed article titled, "A Small Molecule–Drug Conjugate (SMDC) Consisting of a Modified Camptothecin Payload Linked to an αVß3 Binder for the Treatment of Multiple Cancer Types," in the special issue, "The Role of Tumor Microenvironment in Solid Tumors: The New Frontier of Cancer Research" of the journal Cancers

Publication of a peer-reviewed article titled, "First-in-human dose escalation study of cyclin-dependent kinase-9 inhibitor VIP152 in patients with advanced malignancies shows early signs of clinical efficacy," in the journal Clinical Cancer Research

Publication of article titled "New frontiers in ADCs and SMDCs" in the journal Nature

Publication of book chapter titled, "Protease-sensitive linkers," co-authored by Vincerx’s Chief Scientific Officer, Hans-Georg Lerchen, Ph.D., in the e-Book, Chemical Linkers in Antibody-Drug Conjugates (ADCs)

Publication of book chapter titled, "IL3RA-Targeting Antibody-Drug Conjugate BAY-943 with a Kinesin Spindle Protein Inhibitor Payload Shows Efficacy in Preclinical Models of Hematologic Malignancies," co-authored by Vincerx’s Chief Scientific Officer, Hans-Georg Lerchen, Ph.D. and Chief Development Officer, Beatrix Stelte-Ludwig, Ph.D., in the e-Book, Therapeutic Monoclonal Antibodies and Antibody Products, Their Optimization and Drug Design in Cancers

Fourth Quarter and Full Year 2021 Financial Results

Vincerx Pharma had $111.5 million in cash as of December 31, 2021, as compared to $61.8 million as of December 31, 2020. Based on its current business plans and assumptions, Vincerx believes its available cash will be sufficient to meet its operating requirements through 2023.

Research and development (R&D) expenses for the fourth quarter and full year 2021 were $12.3 million and $40.1 million, respectively, as compared to $2.1 million for each of the same periods in 2020. The quarterly and annual increases were primarily driven by increases in stock-based compensation expense, third party preclinical, clinical and manufacturing services in connection with our preclinical studies and clinical trials and new employee salaries.

General and administrative (G&A) expenses for the fourth quarter and full year 2021 were $5.4 million and $22.6 million, respectively, as compared to $3.3 million and $3.6 million for the same periods in 2020. The quarterly and annual increases were primarily driven by increases in legal (both general and patent protection and filings), insurance and accounting and other professional services in support of our operations as a public company, stock-based compensation expense and new employee salaries.

For the fourth quarter and full year 2021, Vincerx reported a net loss of $6.5 million, or $0.31 per share, and a net loss of $39.3 million, or $2.29 per share, respectively. For the fourth quarter and full year 2020, Vincerx reported a net loss of $16.3 million, or $2.74 per share, and a net loss of $16.6 million, or $3.16 per share, respectively.

On March 29, 2022 Soligenix, Inc. (Nasdaq: SNGX) (Soligenix or the Company), a late-stage biopharmaceutical company focused on developing and commercializing products to treat rare diseases where there is an unmet medical need, reported its recent accomplishments and financial results for the year ended December 31, 2021 (Press release, Soligenix, MAR 29, 2022, View Source [SID1234611093]).

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"This year will be a pivotal year for the Company as we anticipate achieving a number of transformational milestones," stated Christopher J. Schaber, PhD, President and Chief Executive Officer of Soligenix. "Most importantly, these milestones include submission of the new drug application (NDA) to the United States (U.S.) Food and Drug Administration (FDA) for marketing authorization of HyBryte (SGX301 or synthetic hypericin) in the treatment of cutaneous T-cell lymphoma (CTCL), a rare form of skin cancer. Additionally, we anticipate initiating a Phase 2a clinical trial in mild-to-moderate psoriasis with SGX302 (synthetic hypericin), where we have already demonstrated positive proof of concept in a small Phase 1/2 pilot study. Under our Public Health Solutions business segment, we continue to advance our heat stable vaccine platform technology, ThermoVax, including through development of filovirus vaccine candidates (targeting Ebola, Sudan, and Marburg viruses), a novel heat stable COVID-19 vaccine candidate, CiVax, and a ricin toxin vaccine, RiVax, where non-human primate (NHP) data for all three vaccine programs has demonstrated significant efficacy."

Dr. Schaber continued, "With approximately $23.3 million in cash, not including our non-dilutive government funding, we expect to have the capital required to accomplish our near-term milestones, including NDA filing and expansion into psoriasis with the conduct of the Phase 2a clinical trial. We continue to evaluate various strategic options, including but not limited to, partnership and merger and acquisition opportunities."

Soligenix Recent Accomplishments

On March 17, 2022, the Company announced the results of a booster vaccination study using CiVax (heat stable COVID-19 subunit vaccine program) in NHPs demonstrating rapid enhancement of neutralizing antibody responses to SARS-CoV-2, including against Delta and Omicron variants. To view this press release, please click here.
On January 10, 2022, the Company issued an update letter from its President and Chief Executive Officer, Dr. Christopher J. Schaber. To view this press release, please click here.
On January 4, 2022, the Company announced that dusquetide is effective at reducing tumor size in nonclinical xenograft models. To view this press release, please click here.
On December 2, 2021, the Company announced 100% protection of NHPs against lethal Sudan ebolavirus challenge using a bivalent, thermostabilized vaccine formulated in a single vial, reconstituted only with water immediately prior to use. To view this press release, please click here.
Financial Results – Year Ended December 31, 2021

Soligenix’s revenues for the year ended December 31, 2021 were $0.8 million as compared to $2.4 million for the year ended December 31, 2020. Revenues primarily included payments on grants received to support the development of: SGX943 for treatment of emerging and/or antibiotic-resistant infectious diseases; ThermoVax, our thermostabilization platform technology; and CiVax, our vaccine candidate for the prevention of COVID-19.

Soligenix’s basic net loss was $12.6 million, or ($0.31) per share, for the year ended December 31, 2021, as compared to $17.7 million, or ($0.64) per share, for the year ended December 31, 2020. The decrease in net loss is primarily due to the additional costs in 2020 relating to the issuance of $5.0M worth of fully vested shares of common stock to Hy Biopharma, Inc. ("Hy Biopharma") in connection with the achievement of a development milestone.

Research and development expenses were $8.4 million as compared to $9.8 million for the years ended December 31, 2021 and 2020, respectively. The decrease in research and development spending for the year ended December 31, 2021 was related to the conclusion of the CTCL and oral mucositis Phase 3 studies.

General and administrative expenses were $4.8 million and $4.3 million for the years ended December 31, 2021 and 2020, respectively. This increase in general and administrative expenses is primarily due to an increase in legal fees associated with the Emergent arbitration partially offset by a decrease in company headcount.

As of December 31, 2021, the Company’s cash position was approximately $26.0 million.

On March 29, 2022 Ribon Therapeutics, a clinical stage biotechnology company developing therapeutics targeting stress support pathways, reported the first patient has been dosed in the Phase 1b/2 study of RBN-2397 in combination with the anti-PD-1 checkpoint inhibitor therapy, pembrolizumab, in patients with squamous cell carcinoma of the lung (SCCL) (Press release, Ribon Therapeutics, MAR 29, 2022, View Source [SID1234611092]). RBN-2397 is a small molecule inhibitor of PARP7 being evaluated in multiple clinical trials for the treatment of cancer.

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"RBN-2397 is a selective PARP7 inhibitor designed to activate the Type I interferon response in tumor cells and overcome a major limitation of immune checkpoint inhibitors (ICI). Combining RBN-2397 with an anti-PD-1 ICI is expected to treat a variety of tumor types including SCCL, a devastating disease representing the second most common form of non-small cell lung cancer," said Prakash Raman, Ph.D., President and Chief Executive Officer, Ribon Therapeutics. "The initiation of the Phase 1b/2 study of RBN-2397 with pembrolizumab will enable us to further understand the potential utility of this combination therapy."

"PARP7 is amplified and highly expressed in SCCL and certain other solid tumors. We have seen encouraging results from our ongoing Phase 1 trial of RBN-2397 as a monotherapy, which is currently evaluating a number of defined expansion cohorts, including patients with SCCL," said Sudha Parasuraman, M.D., Chief Medical Officer, Ribon Therapeutics. "The RBN-2397- pembrolizumab combination is anticipated to drive activated T cells into tumors with the potential to overcome resistance to ICIs. We look forward to evaluating this biology-driven combination in the clinical setting for patients with SCCL who are in need of new therapeutic options."

About RBN-2397

RBN-2397 is an orally available small molecule inhibitor of PARP7 being developed for the treatment cancer. PARP7 is upregulated in response to cellular stress, including genomic instability in cancers, and acts as a brake on the cellular stress response by negatively regulating the Type I interferon response. By inhibiting PARP7 in tumor cells, RBN-2397 has been shown to directly inhibit cellular proliferation and restore interferon signaling to stimulate an innate and adaptive antitumor immune response. RBN-2397 is currently in a Phase 1 clinical trial as a monotherapy in patients with advanced solid tumors and in a Phase 1b/2 clinical trial in combination with pembrolizumab. PARP7 is overexpressed in a number of tumors, including squamous cell carcinoma of the lung, or SCCL, which represents approximately 30% of all non-small cell lung cancers.

On March 29, 2022 Nuvalent, Inc. (Nasdaq: NUVL), a clinical-stage biopharmaceutical company focused on creating precisely targeted therapies for clinically proven kinase targets in cancer, reported pipeline and business progress and fourth quarter and full year 2021 financial results (Press release, Nuvalent, MAR 29, 2022, View Source [SID1234611091]).

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As part of today’s update, Nuvalent is announcing that its Investigational New Drug (IND) application for NVL-655 for the treatment of ALK-positive NSCLC and other solid tumors was cleared by the U.S. Food and Drug Administration (FDA). NVL-655 is a novel ALK-selective inhibitor designed with the aim to address the clinical challenges of emergent treatment resistance, central nervous system (CNS)-related adverse events, and brain metastases that may limit the use of currently available ALK inhibitors. Nuvalent plans to initiate the ALKOVE-1 Phase 1/2 study of NVL-655 for patients with advanced ALK-positive non-small cell lung cancer (NSCLC) and other solid tumors in the second quarter of 2022.

"The Nuvalent team has continued to demonstrate the ability to discover novel molecules with preclinical profiles suggesting best-in-class potential and to progress them efficiently into clinical development. With the clearance of our IND for NVL-655, Nuvalent is on track to have two parallel lead compounds in clinical trials by mid-year," said James Porter, Ph.D., Chief Executive Officer at Nuvalent. "We believe we are well positioned with two clinical-stage, novel product candidates in areas of significant medical need, and sustainable internal discovery efforts with the goal of delivering multiple additional product candidates. I am incredibly proud of all that this team has accomplished and their continued dedication to advancing new potential therapeutic options for patients in need."

Recent Pipeline Highlights

Enrollment Ongoing in ARROS-1 Trial of NVL-520 for Patients with Advanced ROS1-positive NSCLC: Nuvalent is actively enrolling patients in the Phase 1 portion of its ARROS-1 clinical trial, a Phase 1/2 study evaluating NVL-520 in patients with advanced ROS1-positive NSCLC and other solid tumors. NVL-520, Nuvalent’s lead product candidate, is a novel ROS1-selective inhibitor designed with the aim to address the clinical challenges of emergent treatment resistance, CNS adverse events, and brain metastases that may limit the use of currently available ROS1 kinase inhibitors.
Discovery Pipeline Advancing toward Selection of Next Development Candidates: Nuvalent continues to advance its early pipeline efforts with multiple discovery-stage research programs. The company expects to nominate product candidates in 2022 for its discovery programs directed toward ALK IXDN compound resistance mutations and HER2 exon 20 insertions.
Recent Business Highlights

Strengthened Board of Directors with Appointment of Emily Drabant Conley, Ph.D.: In February 2022, Nuvalent appointed Emily Drabant Conley, Ph.D., Chief Executive Officer of Federation Bio, to its Board of Directors. Dr. Conley’s work has contributed to industry-shaping advances in genomics that empower patients and clinicians with actionable health data. Prior to Federation Bio, she spent over a decade at 23andMe where, as Vice President of Business Development, she was instrumental in powering the company’s growth from 30 employees into a household name.
Fourth Quarter and Full Year 2021 Financial Results

Cash Position: Cash, cash equivalents and marketable securities were $288.1 million as of December 31, 2021. Nuvalent expects that its existing cash, cash equivalents and marketable securities will be sufficient to fund its planned operations into 2024.
R&D Expenses: Research and development (R&D) expenses were $13.2 million for the quarter ended December 31, 2021, and $35.6 million for the year ended December 31, 2021.
G&A Expenses: General and administrative (G&A) expenses were $4.2 million for the quarter ended December 31, 2021, and $10.3 million for the year ended December 31, 2021.
Net Loss: Net loss was $17.3 million for the quarter ended December 31, 2021, and $46.3 million for the year ended December 31, 2021.

On March 29, 2022 Monte Rosa Therapeutics, Inc. (NASDAQ: GLUE), a biotechnology company developing novel molecular glue degrader (MGD) medicines, reported business highlights and financial results for the fourth quarter and full year, ended December 31, 2021 (Press release, Monte Rosa Therapeutics, MAR 29, 2022, View Source [SID1234611090]).

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"Last year was transformational for Monte Rosa as we continued to build our world-class leadership team, expanded operations across our two sites in Boston and Basel, named MRT-2359 – our development candidate for the GSPT1 program – and secured $377.6 million in funding," said Markus Warmuth, M.D., CEO of Monte Rosa. "As we look to 2022, we anticipate significant pipeline progress, including the submission of our IND for MRT-2359, as well as continued lead optimization on our CDK2 and NEK7 programs. We are also seeing exciting advancements across our proprietary QuEEN platform, including the expansion of our understanding of structural characteristics of degradable proteins driven by both our experimental and AI platforms. We believe we are well-positioned both organizationally and financially to execute on our vision of tackling historically undruggable targets and identifying potent, highly selective therapies for patients with few or no treatment options."

FOURTH QUARTER 2021 & RECENT HIGHLIGHTS

•Presented preclinical data for MRT-2359 demonstrating anti-tumor activity in L- and N-Myc-positive non-small cell lung cancer patient-derived xenograft (PDX) models. The data were featured as part of the company’s presentation at the 40th Annual J.P. Morgan Healthcare Conference. MRT-2359 is a potent, selective and orally bioavailable GSPT1-directed molecular glue degrader. MRT-2359 has been shown to induce tumor regression in multiple Myc-driven preclinical models, including models of non-small cell lung cancer and small cell lung cancer

•Advanced CDK2 degrader program into lead optimization. Leveraging the company’s QuEEN platform, Monte Rosa has identified selective MGDs for CDK2, a highly validated oncogenic driver of breast, gynecological and other cancers

•Announced license and research collaboration agreement with Dr. Nir London and the Yeda Research and Development Company Ltd., the commercial arm of the Weizmann Institute of Science. The goal of the collaboration is to leverage innovative covalent chemistry to further expand the target space for molecular glue degradation

•Strengthened executive leadership team with promotions of Jullian Jones, Ph.D., J.D., MBA, to Chief Business Officer and Phil Nickson, Ph.D., J.D., to General Counsel

•Presented at recent scientific and medical conferences, including:
•3rd Annual Protein Degradation and Targeting Undruggables Congress, March 8-9
•ESMO Targeted Anticancer Therapies Congress 2022, March 7-8
•2nd Annual Targeted Protein Degradation Europe, March 15-17

UPCOMING MILESTONES & DATA PRESENTATIONS

•Submission of Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) for MRT-2359 expected in mid-2022

•Initiation of at least one additional lead optimization program expected in 2022

•Share new preclinical data supporting MRT-2359 program in a poster presentation at the upcoming American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting, hosted April 8-13, 2022, in New Orleans; presentation details, as follows:
Title: Identification of MRT-2359 a potent, selective and orally bioavailable GSPT1-directed molecular glue degrader (MGD) for the treatment of cancers with Myc-induced translational addiction

Abstract: 3929

Presenter: Gerald Gavory, Ph.D.

Date and Time: April 13, 2022, 9:00 AM – 12:30 PM

UPCOMING INVESTOR EVENTS

Monte Rosa will be participating in the following upcoming investor conferences:

•Wells Fargo Biotech Forum, April 11-13
•Piper Sandler Boston Biotech Bus Tour, May 4-5
•UBS Global Healthcare Conference, May 23-25
•Jefferies Global Healthcare Conference, June 8-10

FOURTH QUARTER AND FULL YEAR 2021 FINANCIAL RESULTS

Research and Development (R&D) Expenses: R&D expenses were $18.1 million for the fourth quarter of 2021 and $57.2 million for the year ended December 31, 2021, compared to $9.9 million and $24.0 million, respectively for the same periods of 2020. These increases were due to the expansion of research and development activities, including the development of our QuEEN

ACTIVE/115879056.5

platform and discovery, lead optimization efforts of our GSPT1 program, and the advancement of development candidate MRT-2359, as well as increases in headcount and laboratory-related expenses due to our continued growth as a research and development organization. R&D expenses included non-cash stock-based compensation of $1.0 million for the fourth quarter of 2021 and $2.6 million for the year ended December 31, 2021, compared to $0.1 million and $0.2 million, respectively, for the same periods in 2020.

General and Administrative (G&A) Expenses: G&A expenses for the fourth quarter of 2021 were $5.3 million compared to $2.1 million for the fourth quarter of 2020, and $15.7 million for the year ended December 31, 2021, compared to $4.0 million for the year ended December 31, 2020. The increase in G&A expenses were a result of increased headcount and expenses in support of the company’s growth and operations as a public company and director and officer liability insurance. G&A expenses included non-cash stock-based compensation of $1.0 million for the fourth quarter of 2021 and $2.6 million for the year ended December 31, 2021, compared to $0.1 million and $0.2 million, respectively, for the same periods in 2020.

Net Loss: Net loss for the fourth quarter of 2021 was $23.4 million compared to $19.7 million for the fourth quarter of 2020, and $74.0 million for the year ended December 31, 2021, compared to $35.9 million for the year ended December 21, 2020.

Cash Position and Financial Guidance: Cash, cash equivalents and restricted cash as of December 31, 2021, were $351.4 million, compared to $42.9 million as of December 31, 2020. The company expects that its cash and cash equivalents, including the aggregate net proceeds from the initial public offering, will be sufficient to fund planned operations and capital expenditures into late 2024.