On April 16, 2024 VBI Vaccines Inc. (Nasdaq: VBIV) (VBI), a biopharmaceutical company driven by immunology in the pursuit of powerful prevention and treatment of disease, reported a business update and announced financial results for the year ended December 31, 2023 (Press release, VBI Vaccines, APR 16, 2024, View Source [SID1234642102]).

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"Over the last 12 months, we’ve seen continued progress across all of our lead programs – with increased use of PreHevbrio and an ever-expanding access and distribution network in place, as well as positive clinical data announcements from all lead pipeline programs, we are helping to make great strides in the fight against GBM, hepatitis B, and coronaviruses," said Jeff Baxter, VBI’s President and CEO. "In the face of a challenging financial market for biotechnology companies, we’ve taken steps to improve our financial stability, including strengthening our balance sheet through equity offerings and business development collaborations, as well as executing on a plan to significantly reduce our debt liability. With a number of near-term milestones expected, we are focused on managing our operational expenses and capital to fuel sustainable growth and value for key stakeholders – patients, healthcare providers, and shareholders."

Recent Key Program Achievements and Projected Upcoming Milestones

PreHevbrio [Hepatitis B Vaccine (Recombinant)]

Product revenue, net of $3.1 million in 2023 compared to $0.9 million in 2022, a 234% year-over-year increase
Preliminary 2024 PreHevbrio U.S. sales demonstrate substantial outpacing of 2023 on a volume basis – through early March 2024, U.S. sales volume totaled approximately 65% of 2023 full year volume
Market segment contracting platforms continue to see growth across Integrated Delivery Networks (IDNs) and large hospital systems, national and regional pharmacy networks, multiple large military and federal facilities, prisons, and independent and public health clinics:
A top-five national health system converted to PreHevbrio in early 2024 with contracting efforts underway to secure additional IDN partners
Retail usage supported by strong ordering from national chains, including Walmart, along with key regional pharmacy networks
PreHevbrio is also available for purchase through the CDC Adult Vaccine Contract
Ex-US – PreHevbri:
Exclusive licensing deal with Brii Biosciences (Brii Bio) announced for the development and commercialization of PreHevbri in the Asia Pacific region (APAC), excluding Japan
An update to the hepatitis B chapter of the Green Book, a resource for healthcare providers with the latest information on vaccines in the UK, was published in April and included detailed information about PreHevbri
PreHevbri became available in Sweden at the end of 2023, and VBI expects that PreHevbri will be made available in certain additional European Union countries in 2024 through its partnership with Valneva
VBI-1901: Cancer Vaccine Immunotherapeutic Candidate – Glioblastoma (GBM)

September 2023: First patients dosed in the randomized, controlled Phase 2b study of VBI-1901, an FDA Fast Track and Orphan Drug Designated cancer vaccine candidate, in recurrent GBM patients
April 2024: Encouraging early tumor response data from Phase 2b study in recurrent GBM presented at World Vaccine Congress 2024:
VBI-1901 Arm: 2 stable disease (SD) observations among patients eligible for evaluation at week 12 (n=2/5), achieving a 40% disease control rate, consistent with 44% disease control rate observed in the Phase 1/2a portion of the study
Control Arm (carmustine or lomustine chemotherapy): No tumor responses have been observed to date (n=0/6; 0% disease control rate) – all evaluable patients experienced a 2-8x increase in tumor size by week 6 and have been taken off study protocol
Mid-Year and Year-End 2024: Additional interim data expected from ongoing randomized Phase 2b study in recurrent GBM patients, subject to speed of enrollment
Novel mRNA-Launched eVLP (MLE) Technology Platform

October 2023:Expansion of proprietary virus-like particle (VLP) platforms announced, supported by preclinical data that demonstrated significant immunologic and manufacturing benefits
MLE technology enables the manufacture of particulate vaccines, capable of driving polyfunctional B-cell and T-cell activation, on accelerated timelines, similar to other mRNA vaccine production timelines
Multiple animal studies have assessed MLE presentation of target antigens compared to mRNA expression alone – studies conducted include target antigens for cytomegalovirus (CMV), Epstein-Barr virus (EBV), and coronaviruses
Breadth and quality of immune response expand the potential for the MLE platform across indications
April 2024: Announced expansion of strategic partnership with Canadian Government to advance the development of the MLE technology platform, supported by the CAD$28 million funding award remaining under the original agreement
Throughout 2024: MLE technology under evaluation by potential partners
VBI-2901: Multivalent Pan-Coronavirus Vaccine Candidate

September 2023: Initial data from Phase 1 study of VBI-2901 reported – the first clinical data from a pan-coronavirus vaccine candidate, demonstrating vaccine benefit represented as boosting and/or greater durability of neutralizing antibody titers
2024: Additional data expected from Phase 1 study
Corporate Updates

February 2024: Announcement of agreement to sell manufacturing capabilities, certain related assets, and enter into new license agreement with Brii Bio – VBI to receive up to $33 million in consideration, subject to achievement of certain activities, which will be used to reduce VBI’s loan obligations due to K2 HealthVentures (K2HV), for:
VBI’s manufacturing capabilities and certain related assets at Rehovot manufacturing facility
Intellectual property for VBI-2601, VBI’s hepatitis B immunotherapeutic candidate
Exclusive Asia Pacific (APAC), excluding Japan, license for development and commercialization of VBI-1901, VBI’s GBM immunotherapeutic candidate
Following completion of all transactions, VBI expects its total debt principal with K2HV to be significantly reduced from $50 million to $17 million
In connection with the transactions, certain covenants in VBI’s loan agreement with K2HV have been amended
To date in 2024, the Company has raised approximately $3.6 million in aggregate gross proceeds from the issuance of its common shares and warrants, and the exercise of previously issued warrants
Financial Results for the Twelve Months Ended December 2023

Cash Position: As of December 31, 2023, VBI had $23.7 million in cash as compared with $62.6 million in cash as of December 31, 2022. Cash position at December 31, 2023, does not include approximately $3.6 million gross proceeds from registered direct offering of common shares and warrants, warrant exercises, and sale of common shares through VBI’s at-the-market facility subsequent to December 31, 2023 and through early April 2024.
Revenues, net: Revenues, net for the full year 2023 were $8.7 million as compared to $1.1 million for the same period in 2022. The revenue increase was a result of an increase in product sales of PreHevbrio in the U.S. and of PreHevbri to VBI’s partner, Valneva, in Europe, in addition to the license revenue and R&D services revenue associated with the agreements with Brii Bio.
Cost of Revenues: Cost of revenues was $12.5 million for the full year 2023 as compared to $11.3 million in 2022. The increase in the cost of revenues was due to increased sales and increased outsourced testing costs during the year ended December 31, 2023.

Research and Development (R&D): R&D expenses for the year ended December 2023 were $9.3 million as compared to $15.5 million in 2022. R&D expenses were offset by $8.3 million for the year ended December 31, 2023, and $8.9 million for the year ended December 31, 2022, due to government grants and funding arrangements. The decrease in R&D expenses is primarily a result of decreased development expenses for VBI’s pan-coronavirus and GBM candidates, VBI-2901 and VBI-1901, due to timing of ongoing clinical studies of each candidate.
Sales, General, and Administrative (SG&A): SG&A expenses for the full year 2023 were $42.1 million as compared to $56.1 million in 2022. The decrease in SG&A expenses was mainly a result of the April 2023 organizational changes that reduced our internal workforce and operating expenses.
Net Cash Used in Operating Activities: Net cash used in operating activities for the full year 2023 was $60.9 million compared to $73.7 million for 2022. The decrease in cash outflows is largely a result of non-cash reconciling items, mainly impairment charges and unrealized foreign exchange loss, and the change in operating working capital, most notably in inventory, other current assets, accounts payable, deferred revenues, and other current liabilities. As a result of the organizational changes announced in April 2023, operating cash outflows in the second half of 2023 were $20 million as compared to $36.3 million in the second half of 2022, representing a 45% reduction.
Net Loss and Net Loss Per Share: Net loss and net loss per share for the full year 2023 were $92.8 million and $6.03, respectively, compared to a net loss and net loss per share of $113.3 million and $13.16 for the full year 2022, respectively.

Net Loss and Net Loss Per Share, Excluding Impairment Charges and Foreign Exchange Loss: Net loss and net loss per share, excluding impairment charges and foreign exchange loss, for the full year 2023 were $61.7 million and $3.96, respectively, compared to $85.8 million and $9.97 for the year ended 2022, respectively. See "Use of Non-GAAP Financial Measures" below for additional information regarding this non-GAAP financial measure, and "GAAP to Non-GAAP Reconciliation" for a reconciliation of this non-GAAP financial measure to net loss and net loss per share.
Impairment charges andforeign exchange loss for the full year 2023 were $24.6 million and $6.5 million, respectively, as compared to none and $27.5 million in 2022. Certain intercompany loans between the Company and its subsidiaries are denominated in a currency other than the functional currency of each entity. The primary driver of the increase in foreign exchange loss was the impact of the relative strengthening of the U.S. and Canadian Dollars against the New Israeli Shekel upon translation of these intercompany loans.

On April 16, 2024 TScan Therapeutics, Inc. (Nasdaq: TCRX), a clinical-stage biopharmaceutical company focused on the development of T cell receptor (TCR)-engineered T cell therapies (TCR-T) for the treatment of patients with cancer, reported an update on its solid tumor and heme malignancies clinical programs (Press release, TScan Therapeutics, APR 16, 2024, View Source [SID1234642101]).

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"We continue to make meaningful progress across both our solid tumor and heme malignancies Phase 1 clinical programs. As we rapidly approach dosing the first patients in the solid tumor program, I am pleased to share that over 40 patients have completed all biomarker testing in the screening protocol, with the majority qualifying for at least one TCR-T in our ImmunoBank and many qualifying for multiplex therapy. This should allow for rapid enrollment into the treatment protocol over the course of the year," said Gavin MacBeath, Ph.D., Chief Executive Officer. "With respect to the heme program, we are encouraged to see continued positive data with all treatment-arm patients remaining relapse-free with no detectable cancer to date, now with a median follow-up of over 10 months."

Solid Tumor Program: TScan continues to expand the ImmunoBank, a collection of therapeutic TCR-Ts that target different cancer-associated antigens presented on diverse HLA types. TScan’s strategy is to treat patients with multiple TCR-Ts to overcome tumor heterogeneity and prevent resistance that may arise from either target or HLA loss (screening protocol: NCT05812027; treatment protocol: NCT05973487).

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Phase 1 solid tumor clinical study has been initiated; first three patients expected to be dosed in early May 2024.

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More than 40 patients have completed all biomarker testing in the screening protocol across a broad array of tumor types. 60% of patients qualify for at least one TCR-T in the ImmunoBank and approximately 30% are eligible for multiplex therapy (T-Plex), potentially enabling rapid enrollment into the treatment protocol upon disease progression.

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Patients have been identified across all six TCR-T cohorts with dosing expected to commence early May.

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Initial data on patients from both singleplex and multiplex cohorts expected in the second half of 2024.

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Additional IND filings planned to continue to expand the ImmunoBank.

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Long-term duration of response data for multiplex therapy anticipated in 2025.

Heme Malignancies Program: TScan’s two lead TCR-T cell therapy candidates, TSC-100 and TSC-101, are designed to treat residual disease and prevent relapse in patients with acute myeloid leukemia (AML), acute lymphocytic leukemia (ALL), or myelodysplastic syndromes (MDS) undergoing allogeneic hematopoietic cell transplantation (HCT) (NCT05473910).

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All eight patients treated with TSC-100 or TSC-101 remain MRD negative, relapse-free with no detectable cancer to date in either bone marrow biopsies or peripheral blood (median follow-up of >10 months) and no dose limiting toxicities observed to date.

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To date, all but one patient has exhibited complete donor chimerism in all subsets of blood cells at all time-points, indicating that only donor-derived cells are present in these patients following treatment with either TSC-100 or TSC-101. One patient with T-ALL who was treated with TSC-100 at the lowest dose level exhibited minimally detectable (<0.3%) mixed donor chimerism at 10.5 months and 12 months post-transplant.

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No detectable mixed chimerism was observed in the malignant cell lineage (CD3+ T-cells) for this patient; mixed chimerism was only observed in healthy nonmalignant blood cells (CD33+ myeloid cells)

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In contrast, for the control arm (transplant alone), eight patients have now been enrolled and only one has achieved and maintained complete donor chimerism to date. Two patients relapsed approximate six months post-transplant and one of these patients died approximately three months later. A third patient required clinical intervention on day 133 because of concerns of impending relapse, and a fourth died 21 days post-transplant.

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Opening of expansion cohorts at the recommended Phase 2 dose level to further characterize safety and evaluate translational and efficacy endpoints is planned for the third quarter of 2024.

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Completion of Phase 1 enrollment and reporting of one-year clinical and translational data on initial patients is anticipated in the second half of 2024.

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Expects to initiate registration trial pending feedback from regulatory authorities and report two-year relapse data in 2025.

On April 16, 2024 Propanc Biopharma, Inc. (OTC Pink: PPCB) ("Propanc" or the "Company"), a biopharmaceutical company developing novel cancer treatments for patients suffering from recurring and metastatic cancer, reported that a certificate of grant for the Company’s "composition of proenzymes for cancer treatment," patent was received from the European Patent Office (Press release, Propanc, APR 16, 2024, View Source [SID1234642100]). The patent covers lower dosage ratios of the two proenzymes (trypsinogen and chymotrypsinogen) contained in the PRP formulation. This is the fourth European patent granted and after validation in selected countries across Europe, will result in the Company’s IP portfolio growing to 94 patents filed in major global jurisdictions.

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This PRP dosing patent is an important part of the IP portfolio as it covers possible future clinical dosage ranges for PRP as the Company advances into early-stage clinical development. Furthermore, Europe is considered a major global region which accounted for 23.4% of global pharmaceutical sales in 2021, according to the European Federation of Pharmaceutical Industries and Associations (EFPIA). This is a significant percentage when you consider that in 2022, the worldwide pharmaceutical market was valued at approximately $1.48 trillion by Statista.com.

"The PRP dosing patent at lower dosage ratios is an important part of our growing IP portfolio, as we seek to protect our novel invention and create future value for our shareholders," said Mr. James Nathanielsz, Propanc’s Chief Executive Officer. "We look forward to advancing PRP into the clinic, and together with our growing IP portfolio, establishing the Company as a pioneer in the way we treat this killer disease."

About PRP:

PRP is a mixture of two proenzymes, trypsinogen and chymotrypsinogen from bovine pancreas, administered by intravenous injection. A synergistic ratio of 1:6 inhibits growth of most tumor cells. Examples include pancreatic, ovarian, kidney, breast, brain, prostate, colorectal, lung, liver, uterine, and skin cancers. Orphan Drug Designation status of PRP has been granted from the US Food and Drug Administration (FDA) for treatment of pancreatic cancer.

To view the Company’s "Mechanism of Action" video on the Company’s lead asset, PRP, please click on the following link: View Source

On April 16, 2024 Precision BioSciences, Inc. (Nasdaq: DTIL), an advanced gene editing company, reported the anticipated return of three programs from Prevail Therapeutics Inc., a wholly owned subsidiary of Eli Lilly and Company (Press release, Precision Biosciences, APR 16, 2024, View Source [SID1234642099]). Precision exercised its option to regain rights for the programs following Prevail Therapeutics’ decision to conclude the collaboration. Precision uses its novel proprietary ARCUS platform to develop in vivo gene editing therapies for sophisticated gene edits, including gene elimination, insertion, and excision. The collaboration began in January 2021 and was amended in June 2023 to transfer certain preclinical research, manufacturing, and investigational new drug (IND)-enabling activities from Precision BioSciences to Prevail Therapeutics.

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"We enjoyed a productive gene editing collaboration with Prevail Therapeutics and appreciate their contributions to the success of these programs. Together, we advanced three programs from concept toward clinical candidates, and Precision completed its workplan for these programs to the next stage gate, taking us to an important development decision point," said Michael Amoroso, President and Chief Executive Officer of Precision BioSciences. "Our decision to regain control of the programs brings exciting development opportunities to Precision’s pipeline with a focus on benefiting people born with incurable genetic diseases."

"These in vivo gene editing programs are designed to take advantage of unique attributes of ARCUS, namely its cut, size, and simplicity. Our next steps will be to prepare for GLP toxicology studies followed by potential IND and clinical trial application (CTA) submissions," said Jeff Smith, PhD, Co-Founder and Chief Research Officer. "We are excited about the compelling in vivo proof-of-concept data generated for ARCUS gene excision of a "hot spot" region of the dystrophin gene in the DMD program. Additionally, in vivo data for the gene insertion program demonstrated up to 45% high efficiency gene insertion in non-dividing cells of non-human primates measured by total liver tissue. This is important and differentiating proof-of-concept data for ARCUS compared to CRISPR, base editors and prime editors, which have not demonstrated such high levels of gene insertion efficiency in dividing or non-dividing cells in vivo, potentially enabling broader therapeutic applicability for ARCUS."

As a result of the strong proof of concept data generated to date, Precision is exploring opportunities to develop the returned programs independently or in partnership with others. Importantly, the return of these programs does not impact the Company’s near-term clinical priorities in ornithine transcarbamylase (OTC) deficiency, HBV, and PMM or its expected cash runway to achieve these clinical data milestones.

"Turning to our fundamental story, Precision continues to make progress with our wholly owned programs for HBV and PMM as well as through partnerships with Novartis and iECURE. Most recently, iECURE has commenced regulatory and clinical activities in major markets around the world to use the ARCUS platform for gene insertion to address OTC deficiency using an ARCUS nuclease," added Mr. Amoroso.

Precision BioSciences remains focused on its most important near-term priorities and clinical data milestones with several opportunities to validate ARCUS for both wholly owned and the lead partnered program in 2024 and 2025.

The OTC deficiency program partnered with iECURE is the most advanced ARCUS in vivo gene editing program with first-in-human clinical dosing expected to commence in 2024. IND and CTAs have been approved in the United States, United Kingdom, and Australia for the Phase 1/2 OTC-HOPE study.
Following receipt of regulatory guidance in and outside of the United States, Precision’s wholly owned PBGENE-HBV viral elimination program has commenced final IND and CTA enabling studies and is rapidly progressing toward the clinic with submissions planned in 2024.
The PBGENE-PMM mutant mitochondrial DNA elimination program is on track for IND and/or CTA submission in 2025.
The cash received from our recent public offering, upfront and potential near-term cash from cell therapy transactions, along with existing cash and cash equivalents, expected operational receipts, continued fiscal and operating discipline, availability of our at-the-market facility, and available credit, are expected to provide Precision with a cash runway into the second half of 2026. The completion of the collaboration does not impact Precision’s expected cash runway as no milestones from Prevail Therapeutics were assumed in our cash runway through 2026.

Company-Hosted Webcast and Conference Call Information

Precision will host a conference call and webcast on Tuesday, April 16, 2024, at 5:00pm EDT to discuss its in vivo gene editing business. The dial-in conference call number is (800) 715-9871 and the conference ID number for the call is 2110172. Participants may access the live webcast, and accompanying presentation materials, as well as the archived webcast on Precision’s website in the Investors section under Events & Presentations: View Source

On April 16, 2024 Nascent Biotech, Inc. (OTCQB:NBIO) ("Nascent Biotech", "Nascent", or the "Company"), a clinical-stage biotechnology Company developing monoclonal antibodies targeting various cancer types, reported that it is embarking on a fresh manufacturing run to ensure Pritumumab ("PTB") supplies are at maximum potency when patient dosing begins in the upcoming Phase II clinical trial (Press release, Nascent Biotech, APR 16, 2024, View Source [SID1234642098]).

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"Taking the time to navigate this opportunity with proper care and diligence is a step that demonstrates our commitment to the best interests of our investors, partners, shareholders, and potential future patients," remarked Sean Carrick, CEO of NBIO. "No one likes delays, but patience and care at this critical stage is necessary in maximizing patient outcomes and long-term shareholder value. We want to be careful not to make hasty decisions while exploring all options.

This announcement comes as the Company prepares for a Phase II trial that follows a very successful Phase I trial-where safety at five ascending dose cohorts was observed-involving 15 patients who received PTB for various types of brain tumors. The most common and most challenging to treat tumor type in the trial was Glioblastoma. 12 of 15 patients had this diagnosis.

There were no dose-related toxicities. Overall, the study found that single agent Pritumumab is safe up to a dose of 16.2 mg/kg every 7 days in brain tumor patients. One partial response showed a 98.0% and 40.8% reduction in 2 tumor lesions for 21 months on study.

Mr. Carrick continued, "Phase I demonstrated an excellent safety profile at various doses. Phase II will be focused on clinical outcomes, and we are taking no shortcuts. We have been in discussions with potential Phase II study sites to ensure patients are being actively recruited and the most efficient process in place once we are ready to begin dosing."

Management anticipates that some research sites will open for the start of Phase II testing late this summer with actual patients involved later in 2024.