On March 29, 2022 Pulmatrix, Inc. (NASDAQ: PULM), a clinical-stage biopharmaceutical company developing innovative inhaled therapies to address serious pulmonary and non-pulmonary disease using its patented iSPERSE technology, reported fourth quarter and year-end financial results for 2021 and provided a corporate update (Press release, Pulmatrix, MAR 29, 2022, View Source [SID1234611105]).

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Ted Raad, Chief Executive Officer of Pulmatrix commented, "I am excited about the future of Pulmatrix. We are well-positioned financially and operationally to advance multiple programs into the clinic as we continue to leverage our iSPERSETM technology to address significant unmet medical needs. With funding into Q1 2024, we have the opportunity to reach meaningful upcoming data milestones and we have strengthened the organization with several key hires, including the recent appointment of a new Chief Medical Offer, Dr. Margaret Wasilewski."

2021 and Recent Program and Corporate Highlights

Pulmazole

On January 27, 2021, the Company conducted a Type-C meeting with FDA to finalize its plans to commence a Phase 2b study with topline data on registrational endpoints in allergic bronchopulmonary aspergillosis (ABPA). If successful, these data may enable a Phase 3 registration study.
On November 9, 2021, the Company announced the positive resolution of a contractual dispute with Cipla, thereby allowing Pulmatrix and Cipla to progress together toward the Phase 2b study in ABPA that aims to provide data on potential registrational endpoints in ABPA. Pulmatrix expects to initiate patient dosing for the Phase 2b study in Q1 2023 and to deliver top-line data by mid-2024.
PUR3100

On April 23, 2021, the company hosted a Key Opinion Leader event on PUR3100, an orally-inhaled dihydroergotamine (DHE) therapy. Migraine experts highlighted the potential benefits that PUR3100 (iSPERSE-enabled DHE) may provide to improve the standard of care and address the unmet needs of acute migraine sufferers.
On January 25, 2022, the Company conducted a Type-C meeting with the FDA. This meeting along with pre-IND written responses has helped define the overall non-clinical and clinical program. The next trial will be a Phase 1 study in Australia allowing for generation of the most comprehensive dataset in a cost and time-efficient manner so as to have Phase 1 data in 2022. This study will form the basis for a US IND.
The Phase 1 study is intended to assess not only safety, tolerability, and pharmacokinetics of PUR3100 in humans, but also provide preliminary comparative bioavailability data to support the use of the 505(b)(2) pathway for marketing authorization. The Company anticipates initiating patient dosing for the Phase 1 study in Australia in Q3 2022, with top-line data anticipated in Q4 2022. A Phase 2 study to determine efficacy will be conducted in migraine patients evaluating two doses that were selected based on the Phase 1 study results. This study is projected to commence dosing in Q2 2023 with data by year-end 2023.
PUR1800

On August 18, 2021, the Company announced that results from 6-month and 9-month pre-clinical toxicology studies demonstrated no progression of 28-day findings, suggesting potential for chronic dosing of PUR1800 in indications beyond acute exacerbations of chronic obstructive pulmonary disease (AECOPD) including, but not limited to, steroid resistant asthma, chronic obstructive pulmonary disease (COPD) and idiopathic pulmonary fibrosis (IPF).
On March 21, 2021, the company announced that PUR1800 achieved the primary endpoint of safety and tolerability in a Phase 1b clinical study with no serious adverse events observed. Preliminary pharmacokinetic and pharmacodynamic data are supportive of continued PUR1800 development in its primary therapeutic target of AECOPD as well other chronic inflammatory airway disease such as severe asthma, COPD and IPF. The Company plans to release more detailed results of the Phase 1b clinical study results at a relevant medical conference in the future.
Corporate Highlights

On December 17, 2021, the Company announced the closing of a $6.75 million registered direct offering. Combined with $40 million raised in February 2021, the total funds raised in 2021 equaled $46.75 million in gross proceeds. The Company anticipates that its cash position is sufficient to fund operations into Q1 2024 and enable Pulmatrix to pursue clinical data milestones for PUR3100 Phase 1 and Phase 2 and Pulmazole Phase 2b trials.
On February 28, the Company completed a reverse stock-split at a ratio of 1-for-20 (the "Reverse Split") which reduced the number of outstanding shares of the Company’s common stock from approximately 65.9 million shares to 3.3 million shares. The number of authorized shares of the Company’s common stock remains at 200,000,000.
On March 1, the Company announced the hiring of Dr. Margaret Wasilewski as the Company’s Chief Medical Officer. Dr. Wasilewski leverages over 25 years of experience in pharmaceutical drug development.
On March 17, the Company announced that it regained compliance for its listing on Nasdaq, allowing continued access to capital markets and liquidity for its investors.
Fourth Quarter and Year-End Financial Results

Revenue was $5.2 million for the year ended December 31, 2021 as compared to $12.6 million for the year ended December 31, 2020, a decrease of $7.5 million. The decrease is related to $3.4 million fewer reimbursable pass-through expenses under our collaboration agreements and a $0.9 million decrease in license related revenues due to a pause in the development activities under the collaboration with Cipla during 2021, and a $3.2 million decrease in license related revenues under our prior collaboration agreement with Johnson & Johnson Enterprise Innovation.

Research and development expense was $15.4 million for the year ended December 31, 2021, as compared to $15.6 million for the year ended December 31, 2020, a decrease of $0.2 million. The decrease was primarily due to a decrease in spend of $2.5 million related to the Company’s PUR1800 program as well as a decrease in spend of $1.7 million related to the Phase 2 Pulmazole clinical trial due to its COVID-19-related termination in 2020. This amount was then partially offset by increased spend of $3.2 million on pre-clinical and manufacturing costs related to our PUR3100 program, in addition to $0.8 million of operating costs in support of our overall clinical and pre-clinical programs.

General and administrative expense was $6.4 million for the year ended December 31, 2021, as compared to $6.9 million for the year ended December 31, 2020, a decrease of $0.5 million. The decrease was primarily due to decreased employment costs of $0.5 million.

Our total cash and cash equivalents balance as of December 31, 2021 was $53.8 million.

On March 29, 2022 Delcath Systems, Inc. (Nasdaq: DCTH), an interventional oncology company focused on the treatment of primary and metastatic cancers of the liver, reported that it will be participating in the Lytham Partners Spring 2022 Investor Conference, via presentation and panel discussion (Press release, Delcath Systems, MAR 29, 2022, View Source [SID1234611104]):

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Presentation
April 4, 2022, at 11:00am ET
Webcast link: View Source

Panel Discussion – Latest Technologies to Combat Cancer

April 5, 2022, at 2:00pm ET
Webcast link: View Source
Management will be participating in virtual one-on-one meetings throughout the event. To arrange a meeting with management, please contact Lytham Partners at 1×[email protected] or register at www.lythampartners.com/spring2022invreg.

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.