MediciNova Announces Abstract Regarding Results of a Clinical Trial of MN-166 (ibudilast) in Glioblastoma Accepted for Presentation at the 2024 American Society of Clinical Oncology Annual Meeting (2024 ASCO)

On April 2, 2024 MediciNova, Inc., a biopharmaceutical company traded on the NASDAQ Global Market (NASDAQ:MNOV) and the Standard Market of the Tokyo Stock Exchange (Code Number: 4875), reported that an abstract regarding results of a clinical trial of MN-166 (ibudilast) in glioblastoma (GBM) has been selected for an oral presentation at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) (2024 ASCO (Free ASCO Whitepaper)) Annual Meeting to be held May 31 – June 4, 2024 in Chicago (Press release, MediciNova, APR 2, 2024, View Source [SID1234641708]). The oral presentation will be presented by one of the investigators of this clinical trial, Gilbert Youssef, M.D., Attending Physician at Harvard Medical School, Center for Neuro-Oncology at Dana-Farber Cancer Institute and Brigham and Women’s Hospital.

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The presentation details are as follows:

Abstract Number: 2106

Title: Phase 1b/2a study evaluating the combination of MN-166 (ibudilast) and temozolomide in patients with newly diagnosed and recurrent glioblastoma (GBM)

Session Title: Rapid Oral Abstract – Central Nervous System Tumors
Session Date: June 2, 2024
Session Time: 11:30 AM – 1:00 PM

About MN-166 (ibudilast)

MN-166 (ibudilast) is a small molecule compound that inhibits phosphodiesterase type-4 (PDE4) and inflammatory cytokines, including macrophage migration inhibitory factor (MIF). It is in late-stage clinical development for the treatment of neurodegenerative diseases such as ALS (amyotrophic lateral sclerosis), progressive MS (multiple sclerosis), and DCM (degenerative cervical myelopathy); and is also in development for glioblastoma, Long COVID, CIPN (chemotherapy-induced peripheral neuropathy), and substance use disorder. In addition, MN-166 (ibudilast) was evaluated in patients that are at risk for developing acute respiratory distress syndrome (ARDS).

Autolus Therapeutics announces acceptance of Marketing Authorization Application (MAA) by the European Medicines Agency (EMA) for obecabtagene autoleucel (obe-cel) for Patients with Relapsed/refractory (r/r) Adult B-Cell Acute Lymphoblastic Leukemia (B-ALL)

On April 2, 2024 Autolus Therapeutics plc (Nasdaq: AUTL), a clinical-stage biopharmaceutical company developing next-generation programmed T cell therapies, reported that the European Medicines Agency (EMA) has accepted the Company’s Marketing Authorization Application (MAA) for obecabtagene autoleucel (obe-cel) (Press release, Autolus, APR 2, 2024, View Source [SID1234641707]). Obe-cel is Autolus’ lead investigational chimeric antigen receptor (CAR) T cell therapy, for the treatment of patients with relapsed/refractory (r/r) adult B-cell Acute Lymphoblastic Leukemia (ALL). The MAA submission was based on data from the pivotal Phase 2 FELIX study of obe-cel in adult r/r B-ALL.

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Autolus’ Nucleus site has recently received the formal certification from the MHRA following a full inspection of the site in February 2024. The MHRA issued two new GMP certificates to cover both clinical and commercial manufacture from the site.

"Along with the recent acceptance of the BLA by the FDA, the acceptance of our EU marketing application is an important milestone in expanding into the European market and delivering this potentially transformative therapy to B-ALL patients," commented Dr. Christian Itin, Chief Executive Officer of Autolus. "We look forward to working with the EMA throughout the evaluation process of obe-cel, and thank the internal team at Autolus for their work on the submission and Nucleus site inspection."

Obe-cel has been granted Orphan Drug Designation by the FDA, Orphan Medical Product Designation by the EMA, Regenerative Medicine Advanced Therapy (RMAT) designation by the FDA and PRIority MEdicines (PRIME) designation by the EMA for adult r/r B-ALL.

Vaccinex Reports 2023 Financial Results and Provides Corporate Update

On April 2, 2024 Vaccinex, Inc. (Nasdaq: VCNX), a clinical-stage biotechnology company pioneering a differentiated approach to treating neurodegenerative disease and cancer through the inhibition of SEMA4D, reported financial results for the fourth quarter ended December 31, 2023 and provided a corporate update on key programs (Press release, Vaccinex, APR 2, 2024, View Source [SID1234641706]).

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Vaccinex achieved several important clinical milestones for pepinemab in both Alzheimer’s disease and Head and Neck Cancer.

Alzheimer’s Disease (AD):

In June 2024, anticipate completing planned 12-months treatment of patients with mild Alzheimer’s disease in the randomized, double-blind, Phase 1b/2a SIGNAL-AD trial of pepinemab vs placebo (NCT04381468). This study was funded in part by the Alzheimer’s Drug Discovery Foundation and by a grant from the Alzheimer’s Association. Topline data is expected in Q3 2024.
Following last patient last visit we will evaluate the impact of treatment on brain metabolic activity, a key biomarker of clinical progression in AD, together with other biomarkers of disease progression and initial assessment of treatment effects on cognition employing several validated, clinically meaningful cognitive scales for AD.
An improving AD-drug development environment, based on FDA’s recent full approval of LEQEMBI, enables the pathway to reimbursement and supports partnering and further investment in Alzheimer’s Disease drug development.
As previously reported, pepinemab has a differentiated mechanism of action, blocking SEMA4D, which is upregulated in neurons during stress of Alzheimer’s and Huntington’s disease and triggers the transformation of astrocytes and microglia from normal homeostatic functions to neuroinflammatory activity. Blockade of SEMA4D is believed to reduce neuroinflammation and to protect and restore healthy astrocyte and neuronal functions (Nature Medicine 2022, View Source).
We believe that the prevalence of AD (6 million people diagnosed with AD in the US alone) and current concerns about the limitations of anti-Aβ amyloid antibodies would make pepinemab attractive as a potential alternative to anti-Aβ antibodies or possibly for use in combination with anti-Aβ for greater efficacy.
Head and Neck Cancer:

As previously reported, analysis of interim data from the first 36 patients in the single-arm, Phase 2 KEYNOTE-B84 study (NCT04815720) evaluating pepinemab in combination with KEYTRUDA in patients with recurrent or metastatic head and neck squamous cell carcinoma (HNSCC) suggests that the combination treatment resulted in an approximately 2X increase in objective responses (ORR) and progression free survival (PFS) in the subset of patients with hard-to-treat PD-L1-low tumors compared to historical response rates for checkpoint monotherapy in this population.
Biomarker data indicate that treatment induced the formation of highly organized lymphoid aggregates in tumor that correlate with disease control and have previously been shown to be predictive of positive response to checkpoint inhibitors.
Further research has suggested strategies to exploit this unique feature of pepinemab treatment in combination with KEYTRUDA so as to further enhance and expand treatment benefit. This will be the focus of continuing development.
Financial Results for the Year Ended December 31, 2023:

Cash and Cash Equivalents and Marketable Securities. Cash and cash equivalents and marketable securities on December 31, 2023 were $1.5 million, as compared to $6.4 million as of December 31, 2022.

In October 2023, the Company raised gross proceeds of $9.6 million from the sale of common stock and warrants to purchase common stock to certain investors including entities controlled by Albert D. Friedberg, the chairman of the Company’s board of directors and Maurice Zauderer, President and CEO of Vaccinex. Subsequently, on February 8, 2024, the Company completed a private placement of common stock and warrants to purchase common stock for gross proceeds of $3.7 million and on March 28, 2024 raised $1.5 million in a public offering and an additional $1.24 million on similar terms in a parallel private placement of common stock and warrants to purchase common stock. The Company was very pleased to also receive a $1.75 million investment from the Alzheimer’s Drug Discovery Foundation (ADDF) on March 29, 2024 in a private placement of preferred stock together with a common warrant to purchase common stock. ADDF has been a leading and visionary supporter of research in AD for 25 years and this was the second such award received by Vaccinex from this distinguished foundation. Details of all these transactions are available in 8-K and other periodic reports filed with the Securities and Exchange Commission (S.E.C.).

Research and Development Expenses. Research and development expenses for the year ended December 31, 2023, were $16.6 million as compared to $14.0 million for the comparable period in 2022.

General and Administrative Expenses. General and administrative expenses for the year ended December 31, 2023 were $6.9 million as compared to $6.2 million for the comparable period in 2022.

Comprehensive loss/Net loss per share. The Comprehensive Loss and Net loss per share for the year ended December 31, 2023, was $20.3 million and $(43.68) compared to $19.8 million and $(98.05) for the comparable period in 2022.

Full financial tables are included below. The Company effected a 1-for-15 reverse stock split in Q3 2023 and 1-for-14 reverse stock split in Q1 2024. All share and share amounts have been retro-actively restated to give effect to the reverse stock splits. For further details on Vaccinex’s financials and the reverse stock splits, refer to its Form 10-K filed April 2, 2024, with the SEC.

About Pepinemab
Pepinemab is a humanized IgG4 monoclonal antibody designed to block SEMA4D, which can trigger collapse of the actin cytoskeleton and loss of homeostatic functions of astrocytes and glial cells in the brain and dendritic cells in immune tissue. Over 600 patients have been treated or enrolled in clinical trials of pepinemab in different indications and pepinemab appears to be well-tolerated and to have a favorable safety profile.

Sutro Biopharma Announces Pricing of $75 Million Underwritten Offering

On April 2, 2024 Sutro Biopharma, Inc. (Sutro or the Company) (NASDAQ: STRO), a clinical-stage oncology company pioneering site-specific and novel-format antibody drug conjugates (ADCs), reported the pricing of an underwritten offering of 14,478,764 shares of its common stock at a price of $5.18 per share (Press release, Sutro Biopharma, APR 2, 2024, View Source [SID1234641705]). The gross proceeds from this offering are expected to be approximately $75.0 million, before deducting underwriting discounts and commissions and other offering expenses payable by Sutro. All of the shares of common stock are being offered by Sutro. The offering is expected to close on or about April 4, 2024, subject to the satisfaction of customary closing conditions.

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The offering was led by a high quality group of new and existing healthcare focused institutional investors.

Sutro intends to use the net proceeds of this offering, together with its existing cash, cash equivalents and marketable securities, primarily for general corporate purposes, which may include funding research, clinical and process development and manufacturing of its product candidates, increasing its working capital, developing itscommercialization infrastructure, expanding its manufacturing capabilities, acquisitions or investments in businesses, products or technologies that are complementary to its own, capital expenditures and other general corporate purposes.

BofA Securities is acting as sole book-running manager in the offering.

The shares are being offered by Sutro pursuant to a registration statement previously filed and declared effective by the Securities and Exchange Commission (SEC). A prospectus supplement and 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 www.sec.gov. Copies of the prospectus supplement and accompanying prospectus may also be obtained, when available, from: BofA Securities, NC1-0220-02-25, Attention: Prospectus Department, 201 North Tryon Street, Charlotte, North Carolina, 28255-0001, 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 securities of Sutro, 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.

Phio Pharmaceuticals Reports 2023 Year End Financial Results and Provides Business Update

On April 2, 2024 Phio Pharmaceuticals Corp. (Nasdaq: PHIO), a clinical stage biotechnology company whose proprietary INTASYL RNAi platform technology is designed to make immune cells more effective in killing tumor cells, reported its financial results for the year ended December 31, 2023 and provided a business update (Press release, Phio Pharmaceuticals, APR 2, 2024, View Source [SID1234641704]).

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Recent Corporate Updates

PH-762

In 2023, we made substantial progress as a focused drug development organization. In April, we submitted our first IND to the U.S. Food and Drug Administration (FDA), addressing an immuno-oncology disorder and seeking clearance to commence a Phase 1B dose escalation trial in various forms of skin cancer with our lead INTASYL compound PH-762. One month later we received clearance from the FDA to begin our trial in stage IV melanoma, Merkel cell and cutaneous squamous cell carcinoma (cSCC), including the early stages I and II in cSCC. The latter clearance was significant since there are no drugs specifically approved to treat early stages I and II of cSCC. The current standard of care for these diseases is surgical intervention, which may not always be an ideal medical option when tumor size or placement occur on certain areas of the face and scalp. Our INTASYL compound PH-762 may offer an alternative which could shrink tumor size, if not eliminate the lesion, as well as reducing the extent of surgical intervention to allow for tissue sparing and facilitating a faster patient recovery.

As of February, the first two patients in our first cohort have completed treatment with PH-762 with no reported adverse events.

We now have four investigation sites under contract to participate in the trial. The sites consist of George Washington University, Banner MD Anderson, Centricity and Integrity Research.

AgonOx Study Development

In February 2021, we entered into a clinical co-development collaboration agreement (the "Clinical Co-Development Agreement") with AgonOx, a private company developing a pipeline of novel immunotherapy drugs targeting key regulators of the immune response to cancer. Under the Clinical Co-Development Agreement, we and AgonOx are working to develop a T cell-based therapy using PH-762, and AgonOx’s "double positive" tumor infiltrating lymphocytes ("DP TIL") technology. AgonOx is conducting a Phase 1 clinical trial of PH-762 treated DP TIL in patients with advanced melanoma and other advanced solid tumors. In August and September, AgonOx infused the first two patients with their DP TIL. In November, a third patient was infused with a combination of TIL and our PH-762 product candidate. Further patient infusions have been delayed due to a facility renovation at the Providence Cancer Research Center, which is expected to reopen in May 2024.

Cost Rationalization

In 2023 we implemented a cost rationalization program driven by our transition from discovery research to product development. This resulted in a decision not to renew our building lease in Marlborough, MA., which lease expires on March 31, 2024. A smaller research footprint has been established in the Massachusetts Biomedical Initiatives in Worcester, MA, where we occupy 321 sq. ft of laboratory space. Additionally, we rationalized discovery research personnel resulting in a headcount reduction of 36%. Expense reductions have been redirected to funding the Phase 1B clinical trial of PH-762.

Patent Portfolio Enhancement

Two new patent applications were filed covering the intratumoral administration of PH-762 for the treatment of various skin cancers, and the synergistic combination of an INTASYL compound and a systemic antibody. In addition, five new patents covering multiple INTASYL compounds were granted in the US (2), Japan (1), South Korea (1) and Hong Kong (1).

Scientific News

We presented new data on the immunotherapeutic activity of INTASYL compounds alone and as Adoptive Cell Therapy at conferences, including American Academy of Cancer Research (AACR) (Free AACR Whitepaper), Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper), American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper), and at the triple meeting of AACR (Free AACR Whitepaper)-NCI-EORTC AACR-NCI-EORTC (Free AACR-NCI-EORTC Whitepaper) International Conference on Molecular Targets and Cancer Therapeutics (EORTC-NCI-AACR) (Free ASGCT Whitepaper) (Free EORTC-NCI-AACR Whitepaper). New data was presented on INTASYL PH-894 showing that local treatment with PH-894 presents a strategy to decrease BRD4 expression and upregulate MART-1 expression to increase immune response to cancer cells while reducing toxicities associated with systemic therapies. This further supports development of PH-894 for injectable solid tumor indications such as melanoma.

In addition, data was presented that demonstrates the effectiveness of PH-894 as an antitumor cytotoxic agent (directly killing tumor cells). The addition of PH-894 to cells in vitro elicited concentration-associated apoptosis of all human cancer cell lines tested, including head and neck squamous cell carcinoma (HNSCC), breast cancer, lung cancer, glioblastoma, melanoma, colon cancer, ovarian cancer, and cervical cancer.

As previously disclosed, we have elected to defer further work on our PH-894 product candidate in order to prioritize and advance our PH-762 clinical trials.

Financial Results

Cash Position

At December 31, 2023 we had cash of $8.5 million as compared with $11.8 million at December 31, 2022.

Research and Development Expenses

Research and development expenses were $6.3 million for the year ended December 31, 2023 as compared with $7.0 million for the year ended December 31, 2022, a decrease of 10%. The decrease was primarily due to decreased costs related to the completion of our IND-enabling preclinical studies for PH-894 and reduced lab supplies as a result of a decrease in lab personnel and a shift in focus on clinical development, partially offset by an increase in clinical-related costs for the two U.S. PH-762 Phase 1 clinical trials as compared to the prior year period.

General and Administrative Expenses

General and administrative expenses were $4.4 million for the year ended December 31, 2023 as compared with $4.5 million for the year ended December 31, 2022, a decrease of 2%. The decrease was primarily due to decreases in personnel-related expenses related to organizational department changes, one-time executive search-related fees for our President and CEO and D&O insurance premiums, partially offset by increased professional fees for legal services as compared to the prior year period.

Net Loss

Net loss was $10.8 million, or $5.20 per share, for the year ended December 31, 2023 as compared with $11.5 million, or $10.10 per share, for the year ended December 31, 2022. The decrease in net loss was primarily due to the changes in research and development expenses, as described above.