On May 14, 2024 Lumos Pharma, Inc., a clinical stage biopharmaceutical company focused on therapeutics for rare diseases, reported the outcome from its End-of-Phase 2 meeting with the FDA, provided a clinical programs update, and reported financial results for the quarter ended March 31, 2024 (Press release, Lumos Pharma, MAY 15, 2024, View Source [SID1234643324]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"We are pleased to announce that, earlier this quarter, we had a very productive End-of-Phase 2 meeting with the FDA," said Rick Hawkins, Chairman and CEO of Lumos Pharma. "In this review, the FDA recognized LUM-201’s unique mechanism as a growth hormone secretagogue and acknowledged the use of a placebo-controlled clinical trial design as an appropriate option for a LUM-201 Phase 3 trial. Based on the FDA’s feedback, we plan to move forward with a proposal for a single Phase 3 study that will be a double-blinded, placebo-controlled clinical trial with a 2:1 randomization in approximately 150 patients. We expect to finalize design details with the FDA in the third quarter and to be in position to initiate this trial before the end of this year."

"In addition to our encouraging engagement with the FDA, we are also very pleased to share updated data from our Phase 2 OraGrowtH trials. These data continue to show that LUM-201 produces a significant increase in growth from baseline in annualized height velocity (AHV) at 6 and 12 months in per protocol analysis. Combined data also suggest durable benefit out to 24 months."

"We believe these developments have positioned us to advance LUM-201 toward both a Phase 3 registrational trial and potential approval of LUM-201 as the first oral therapeutic for moderate pediatric growth hormone deficiency," Rick Hawkins concluded.

Recent Highlights

•End of Phase 2 Meeting Held with FDA
◦FDA indicated that a placebo-controlled trial design is an appropriate option for a Phase 3 trial for LUM-201. We believe this reflects FDA’s recognition of unique qualities of LUM-201’s mechanism of action as a growth hormone secretagogue.
◦Proposal for a Phase 3 trial to include a 12-month double-blinded, placebo-controlled design with 2:1 randomization, ~150 patients with the placebo-controlled portion of the study lasting six months, which we believe will improve the likelihood of success when compared to a non-inferiority study.
◦Planning is ongoing, and the Company expects to initiate a Phase 3 trial of LUM-201 in Q4 2024, subject to FDA approval.
•Updated LUM-201 Data from Combined OraGrowtH210 and OraGrowtH212 Trials

Exhibit 99.1

◦Additional data continue to show durable LUM-201 treatment effect to 12 and 24 months.
◦Full 12-month data from OraGrowtH210 demonstrated LUM-201 produces significant increase in growth from baseline with AHVs of 8.2 cm/yr (N=22) and 7.6 cm/yr (N=21) at 6 and 12 months, respectively, at the 1.6 mg/kg dose vs. 4.7 cm/yr baseline growth (N=13).*
◦Full 12-month data from OraGrowtH210 continued to show durable effect to 12 months for all LUM-201 cohorts and 1.6 mg/kg/day as optimal dose to advance to Phase 3.
◦Updated combined data from OraGrowtH210 and OraGrowtH212 trials continued to demonstrate LUM-201 AHV durable to 24 months with per protocol-24M (N=12) AHV of 8.1 cm/yr and 7.3 cm/yr at 12 and 24 months, respectively .
◦More moderate year-2 decline in AHV of 9.9% for LUM-201 compared to year-2 decline in AHV of 19.7% observed in historical rhGH benchmarks likely due to LUM-201 restoration of GH and IGF-1 to normal levels via amplification of physiologic pulsatile secretion of growth hormone within the natural endocrine feedback loop.
◦Investigational safety profile continues to be favorable.
•Data from Phase 2 OraGrowtH210 and OraGrowtH212 Trials Presented at Medical Meetings in US and Europe
◦Pediatric Endocrinology Society (PES)
◦10th International Congress of the Growth Hormone Research Society (GRS)
◦European Congress of Endocrinology (ECE)
◦Data presented at these medical conferences demonstrate that by augmenting the natural pulsatile secretion of growth hormone LUM-201 produces comparable growth to injectable rhGH with significantly less exposure to circulating growth hormone.
•Additional Data from Phase 2 OraGrowtH Trials to be Presented in Q2 2024
◦Full 12-Month OraGrowtH212 data, additional analyses of OraGrowtH210 data, and updated combined 24-month data to be presented in Q2 2024
◦Two abstracts accepted for poster presentation at the Endocrine Society (ENDO) Annual Meeting

*Baseline AHV data were not required for enrollment; baseline data available for N=13 subjects.

Financial Results for Quarter Ended March 31, 2024

Cash Position – Lumos Pharma ended the quarter on March 31, 2024, with cash, cash equivalents, and short-term investments totaling $23.2 million, as compared to $36.1 million on December 31, 2023. Cash on hand is expected to support operations through Q3 2024, which is inclusive of Phase 3 planning and preparatory activities.

R&D Expenses – Research and development expenses for the quarter ended March 31, 2024 were $7.2 million, an increase of $2.9 million compared to the same period in 2023, primarily due to increases of $2.0 million in licensing expense, $0.8 million in clinical trial expenses and $0.2 million in consulting expenses, offset by a decrease of $0.1 million in personnel-related expenses.

G&A Expenses – General and administrative expenses for the quarter ended March 31, 2024 were $3.8 million, a decrease of $0.6 million compared to the same period in 2023, primarily due to decreases of $0.4 million in licensing expenses, $0.1 million in travel expenses, $0.1 million in consulting expenses and $0.1 million in other expenses, offset by an increase of $0.1 million in personnel-related expenses.

Net Loss – The net loss for the quarter ended March 31, 2024, was $10.4 million compared to a net loss of $7.3 million for the same period in 2023.

On May 15, 2024 Vaccinex, Inc. (Nasdaq: VCNX), a clinical-stage biotechnology company pioneering a differentiated approach to treating neurodegenerative disease and cancer through the inhibition of Semaphorin 4D (SEMA4D), reported financial results for the first quarter ended March 31, 2024, and provided a corporate update on its key program for Alzheimer’s disease (Press release, Vaccinex, MAY 15, 2024, View Source [SID1234643352]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Treatment with pepinemab believed to halt or slow progression of neurodegenerative disease:

Vaccinex expects to complete the planned 12-months of treatment of the last patients enrolled in its randomized, double-blind, Phase 2a SIGNAL-AD trial of pepinemab anti-SEMA4D antibody for mild Alzheimer’s disease (NCT04381468) in early June 2024. Database lock will follow by early July to enable final analysis of the major study outcomes.

Of interest to investors:

Vaccinex’s lead product, pepinemab, is designed to block astrocyte activation that is otherwise triggered by SEMA4D upregulation on stressed or damaged neurons in the brain during progression of Alzheimer’s Disease (AD) and Huntington’s Disease (HD).
Astrocytes, which are key brain cells that support the health and function of neurons, express high affinity receptors for SEMA4D and undergo substantial changes in morphology and gene expression when SEMA4D binds to these receptors. As a result, they switch from normal supportive functions to neurotoxic inflammatory activity that is believed to accelerate and aggravate progression of neurodegenerative diseases.
The Company’s hypothesis, which is being tested in the SIGNAL-AD study, is that treating with pepinemab antibody can block signaling by SEMA4D and prevent some or all damaging consequences of astrocyte activation.
The Company has previously reported that antibody blockade of SEMA4D appears to protect and restore healthy astrocyte functions and, by some measures, also appears to slow or prevent cognitive decline in Huntington’s disease.
The Company believes that the prevalence of AD (6 million people diagnosed with AD in the US alone) and current concerns about the limitations of treatment with anti-Aβ amyloid antibodies could make pepinemab, if approved, attractive as a potential alternative treatment or possibly for use in combination with anti-Aβ to enhance the benefit to patients. Pepinemab has, to date, been well-tolerated in clinical trials that enrolled a total of more than 600 patients, with no evidence of amyloid-related imaging abnormalities (ARIA).
Of further interest to the medical and research communities:

Deposition of Aβ amyloid in the brain is recognized as the earliest event in the pathologic cascade for AD. However, the observation that many elderly, cognitively normal subjects also evidence deposition of Aβ amyloid in their brains suggests that this is not of itself sufficient for disease progression and that a sequence of subsequent events, including astrocyte activation and formation of toxic tau tangles in neurons, is required. Others have recently shown that Aβ deposition in combination with astrocyte activation is associated with increased plasma levels of phosphorylated tau peptide (p-tau 217).
Key outcomes of the SIGNAL-AD study will include impact of pepinemab treatment on brain metabolic activity, an important biomarker of clinical progression in AD, together with other biomarkers of disease progression including plasma levels of glial fibrillary acidic protein (GFAP) released by reactive astrocytes, and phosphorylated tau peptide. Exploratory evaluation of treatment effects on cognitive decline will employ several validated cognitive scales. Topline data will be presented at a major Alzheimer’s medical conference.
The SIGNAL-AD study was funded in part by two investments from the Alzheimer’s Drug Discovery Foundation (ADDF) for a total of $4.75 million, and by an $0.75 million grant from the Alzheimer’s Association.
Financial Results for the Quarter Ended March 31, 2024:

Cash and Cash Equivalents and Marketable Securities. Cash and cash equivalents and marketable securities on March 31, 2024, were $3.0 million, as compared to $1.5 million as of December 31, 2023.

On February 8, 2024, and March 28, 2024, the Company completed private placements of common stock with accompanying 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, the Company’s President and CEO, for gross proceeds of $4.94 million. On March 29, 2024 the Company raised an additional $1.50 million in a public offering and also received a $1.75 million investment from the ADDF in a private placement of preferred stock together with common warrants 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 (SEC).

Research and Development Expenses. Research and development expenses for the quarter ended March 31, 2024, were $3.4 million as compared to $3.8 million for the comparable period in 2023.

General and Administrative Expenses. General and administrative expenses for the quarter ended March 31, 2024, were $1.8 million as compared to $1.7 million for the comparable period in 2023.

Comprehensive loss/Net loss per share. The Comprehensive Loss and Net loss per share for the quarter ended March 31, 2024, were $3.9 million and $(2.94) compared to $5.0 million and $(20.89) for the comparable period in 2023.

Total Stockholders’ Equity. Stockholders’ Equity as of March 31, 2024, was $2.7 million on March 31, 2024, as compared to a deficit of $(2.3) million on December 31, 2023. The 2023 discrepancy between the stockholder’s equity balance and the Nasdaq listing requirement was largely due to a determination that the terms of warrants issued on October 3, 2023 did not meet all the requirements for classification as equity and were, therefore, classified as liabilities. The Company brought this matter to the attention of all Vaccinex warrant holders in March 2024, and the holders of 89% of all outstanding warrants agreed to modification of terms of their warrants resulting in the ability to classify the modified warrants as equity on our balance sheet as of March 31, 2024. On April 11, 2024, the Company received a letter from the Listing Qualifications staff of The Nasdaq Stock Market advising that based on the financial statements contained in its Form 10-K for the year-ended December 31, 2023, the Company no longer complied with the requirement to maintain a minimum of $2.5 million in stockholders’ equity for continued listing on the Nasdaq Capital Market (the Equity Standard). The letter from Nasdaq was not a notice of delisting and had no immediate effect on the Company’s listing on the Nasdaq Capital Market. However, Nasdaq required the Company to submit a plan by May 13, 2024, describing how it would regain compliance with the Equity Standard. The Company has submitted the required plan, and while the Company is confident that its plan is promising and feasible, the Company cannot provide assurances that Nasdaq will accept the plan or that the Company will maintain compliance with the Equity Standard.

Financial tables are included below. The Company effected a 1-for-14 reverse stock split on February 20 2024. All share and share amounts have been retroactively restated to give effect to the reverse stock split. For further details on Vaccinex’s financials and the reverse stock split, please refer to its Form 10K filed April 1, 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 other 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 with a favorable safety profile.

On May 15 2024 MaaT Pharma, a clinical-stage biotech company and a leader in the development of Microbiome Ecosystem TherapiesTM (MET) dedicated to improving survival outcomes for patients with cancer, reported the successful completion of its offering of 18.2 million euros, comprising a reserved offering of 2,161,250 new ordinary shares to institutional investors and a public offering of 112,454 new ordinary shares to retail investors via the PrimaryBid platform (the "Primary Offering"), at a price of €8 per share (the "Offering Price") (Press release, MaaT Pharma, MAY 15, 2024, View Source [SID1234643325]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The net proceeds from the Primary Offering, which are estimated to be approximately €17.3 million, will be used to fund the company’s R&D activities, covering completion of the Phase 3 trial for MaaT013 in Europe, including top-line results/primary endpoint in mid Q4 2024, continuing the pipeline development, including the initiation of Phase 3 trial activities for MaaT013 in the US and the broadening of the Phase 2b trial with MaaT033 across Europe and for working capital and other general corporate purposes, including repayment of current debts.

The Company estimates that, the total cash and cash equivalents as of March 31, 2024 of €18.2 million (unaudited), and the funds raised, will be able to finance its operations into early Q1 2025.

Concurrently with the Primary Offering, certain funds managed by Seventure Partners (together, the "Selling Shareholder"), have sold 125,000 existing shares, at the Offering Price, i.e. an amount of €1 million (the "Secondary Offering" and together with the Primary Offering, the "Global Offering"). These funds are currently in a divestment period and are progressively selling the shares they hold in the Company, but Seventure Partners will continue to support the development and growth of the Company and has committed to participate to the Reserved Offering through other funds.

Hervé Affagard, CEO and co-founder of MaaT Pharma, commented: "I extend my gratitude to both our institutional and retail investors, as well as the financial commitment from the management team. 70% of new retail investors were previously non-shareholders, showcasing a strong influx of new support, with a total investment of €0.9 million. Since our IPO in late 2021, MaaT Pharma, leader in Microbiome/Oncology, has consistently met its objectives across various fronts. Today’s investment propels us further toward in harnessing the full potential of microbiome innovations. This raise will secure our upcoming completion of Phase 3, including primary endpoint results, for MaaT013 in treating refractory acute graft-versus-host disease and contribute to positioning us to further advance our therapeutic pipeline."

Main characteristics of the Global Offering

The Global Offering, for a total amount of 19.2 million euros, was carried out by the issue of 2,273,704 new ordinary shares, in the context of the Primary Offering through (i) a reserved offering, without pre-emptive subscription rights, for the benefit of specific categories of investors of 2,161,250 new ordinary shares were subscribed for by investors in the Reserved Offering for a total amount of approximately 17.3 million euros, in accordance with the 25th resolution of the annual general meeting of June 19, 2023 (the "AGM") and pursuant to article L. 225-138 of the French Commercial Code (the "Reserved Offering"), (ii) a public offering aimed at retail investors, via the PrimaryBid platform, of 112,454 new ordinary shares were subscribed for by investors in the Reserved Offering for a total amount of approximately 0.9 million euros, in accordance with the 23rd resolution of the AGM and pursuant to article L. 225-136 of the French Commercial Code and article L. 411-2-1 1° of the French Monetary and Financial Code (the "PrimaryBid Offering") and (iii) the sale of 125,000 existing ordinary shares representing 1 Million euros held by the Selling Shareholder to the same specific categories of investors defined in the Reserved Offering.

Upon completion of the Global Offering, the share capital of the Company will be composed of 13,897,143 ordinary shares with a par value of €0.10 each. The 2,273,704 newly issued ordinary shares, represent approximately 19.6% of the Company’s share capital, on a non-diluted basis, before completion of the Global Offering and 16.3% of the Company’s share capital, on a non-diluted basis, after completion of the Global Offering. By way of illustration, a shareholder holding 1% of the share capital prior to the Global Offering and which did not participate in the Global Offering will hold 0.84% after completion of the Global Offering.

The issue price of the new ordinary shares has been set at €8 per share, representing a discount of 14.3% to the closing price of the Company’s shares on the Euronext Paris regulated market at the time of the last trading session preceding its setting (i.e. May 14, 2024) in accordance with the decisions of the Company’s Chief Executive Officer pursuant to the sub-delegations of authority granted by the Company’s Board of Directors on May 14, 2024, in accordance with the 25th, 23rd and 27th resolutions of the AGM.

To the best of the Company’s knowledge, the breakdown of shareholders before and after completion of the Global Offering is as follows:

Shareholder Pre-offer
(non-diluted basis) Post-Offer
(non-diluted basis)
Number of Ordinary Shares held Percentage of Existing Share Capital Number of Ordinary Shares held Percentage of Enlarged Share Capital
Karim Dabbagh 1 960 0,02% 1 960 0,01%
Hervé Affagard 230 776 1,99% 235 151 1,69%
Total of individual corporate officers 232 736 2,00% 237 111 1,71%
Seventure Funds 2 593 068 22,31% 2 630 568 18,93%
Crédit Mutuel Innovation SAS 1 412 364 12,15% 1 412 364 10,16%
Biocodex SAS 1 234 185 10,62% 1 859 185 13,38%
Symbiosis LLC 2 027 702 17,44% 2 027 702 14,59%
FPCI Fonds PSIM 1 802 439 15,51% 2 802 439 20,17%
Other Shareholders 1 004 141 8,64% 1 306 641 9,40%
Total Historical shareholders 10 073 899 86,67% 12 038 899 86,63%
Employees and consultants 166 471 1,43% 174 596 1,26%
Public Float 1 150 333 9,90% 1 446 537 10,41%
Total 11 623 439 100,0% 13 897 143 100,0%

Current shareholders Bpifrance Investissement, Biocodex and Seventure Partners which respectively held 15,51%, 10,62% and 22,31% of the Company’s share capital on a non-diluted basis, prior to the Global Offering, had pledged to subscribe €8 million, €5 million, and €1.3 million respectively in the Reserved Offering, subject to the Reserved Offering representing at least €17 million. The management of the Company has pledged to subscribe €100,000 in the Reserved Offering.

Members of the Company’s Board of Directors Mr. Hervé Affagard, Mr. Jean-Marie Lefèvre for Biocodex and Mrs Isabelle de Crémoux for Seventure Partners subscribed to the Global Offering for a total amount of €6.3 million. It should be noted that none of the members of the Board of Directors having subscribed to the Global Offering took part in the vote on the decision setting its terms. These investors represent approximately 32% of the Global Offering.

Admission of new ordinary shares

Settlement-delivery of the new ordinary shares and their admission to trading on the regulated market of Euronext Paris are expected to occur on May 17, 2024. The new shares will be of the same class and fungible with the existing shares, will carry all rights attached to the shares, and will be admitted to trading on the Euronext Paris market under the same ISIN code FR0012634822 – MAAT.

Undertakings to retain shares and refrain from issuing shares

In connection with the Reserved Offering, participating Directors and certain existing shareholders namely, Bpifrance Investissement, Biocodex and Seventure Partners have respectively entered into a lock-up agreement with the Placement Agents for a period of 90 days from the settlement-delivery date of the Global Offering, subject to customary exceptions.

In connection with the Reserved Offering, the Company has undertaken to refrain from issuing shares for a period of 90 days from the settlement-delivery date of the Global Offering, subject to customary exceptions.

Financial Intermediaries

Stifel Europe AG ("Stifel") is acting as Global Coordinator and Joint Bookrunner in connection with the Reserved Offering. Gilbert Dupont SNC, Groupe Société Générale, is acting as Joint Bookrunner in connection with the Reserved Offering. (together, the "Placement Agents"). The Reserved Offering is subject to a placement agreement entered into between the Company and the Placement Agents dated May 14, 2024.

Within the framework of the PrimaryBid Offering, investors subscribed via the PrimaryBid partners mentioned on the PrimaryBid website (www.PrimaryBid.fr). The PrimaryBid Offering is subject to an engagement letter entered into between the Company and PrimaryBid and is not subject to a placement agreement.

Prospectus

Since the new ordinary shares issued over a period of 12 months, including in the context of the Reserved Offering represent less than 20 % of the number of securities already admitted to trading on the same regulated market, no listing prospectus would be subject to the approval of the French Financial Markets Authority (Autorité des Marchés Financiers or the "AMF") pursuant to the Regulation (EU) 2017/1129 of the European Parliament and of the Council of 14 June 2017 (as amended, the "Prospectus Regulation").

This press release does not constitute a prospectus under Prospectus Regulation, or a public offering.

Risk factors

The public’s attention is drawn to the risk factors relating to the Company and its business, presented in chapter 3 of the universal registration document 2024 approved by the Autorité des marchés financiers on April 02, 2024, which is available free of charge on the Company’s website (www.maatpharma.com) and the website of the Autorité des marchés financiers (www.amf-france.org). The occurrence of any or all of these risks could have an adverse effect on the Company’s business, financial situation, results, development or prospects.

In addition, investors are invited to consider the following risks specific to the issue: (i) the market price of the Company’s shares could fluctuate and fall below the subscription price of the shares issued under the Global Offering, (ii) the volatility and liquidity of the Company’s shares could fluctuate significantly, (iii) sales of the Company’s shares could occur on the market and have an unfavorable impact on the Company’s share price, and (iv) the Company’s shareholders could suffer potentially significant dilution as a result of any future capital increases made necessary by the Company’s search for financing.

On May 15, 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 quarter ended March 31, 2024 (Press release, VBI Vaccines, MAY 15, 2024, View Source [SID1234643353]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"To date in 2024, our focus has centered around pipeline execution, expanding access and increased uptake of PreHevbrio in targeted market segments, and execution of strategic partnerships to drive opportunity for our portfolio assets, create shareholder value, and strengthen our balance sheet," said Jeff Baxter, VBI’s President and CEO. "We remain committed to creating opportunities for our vaccines, candidates, and technologies to meaningfully impact public health and the lives of patients, providers, and families."

Recent Key Program Achievements and Projected Upcoming Milestones

PreHevbrio [Hepatitis B Vaccine (Recombinant)]

Product revenue, net increased 105% from Q1 2023, with $1.0 million earned in Q1 2024
US:
H1 2024 PreHevbrio U.S. sales continue to demonstrate substantial growth over 2023, with over 80% of the 2023 full-year volume being sold in the first five months of 2024
Commercial execution in Q1 2024 created new demand in the large Integrated Delivery Network (IDN) and hospital system space, and saw continued national and regional pharmacy uptake
Public sector momentum building with PreHevbrio now available for purchase under the CDC Adult Vaccine Contract
Ex-US – PreHevbri:
VBI partners with Valneva SE to make PreHevbri available in certain European countries
In 2023, through this partnership, PreHevbri was launched in the UK, Sweden, Netherlands, and Belgium – in early 2024, PreHevbri also became available in Denmark and Norway
VBI-1901: Cancer Vaccine Immunotherapeutic Candidate – Glioblastoma (GBM)

VBI-1901 is being evaluated in an ongoing, randomized, controlled Phase 2b study in comparison to standard-of-care chemotherapy treatment 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 2024 and Year-End: Additional tumor response data from ongoing Phase 2b study expected mid-year 2024, with initial survival data from early-enrolled participants expected by year-end 2024, subject to speed of enrollment
Novel mRNA-Launched eVLP (MLE) Technology Platform

April 2024: Announced expansion of strategic partnership with the 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 remains under active evaluation by potential partners
Other Achievements and Upcoming Milestones

February 2024: Announced a series of agreements with Brii Biosciences ("Brii Bio"), pursuant to which, subject to achievement of certain activities, VBI would receive up to $33 million in consideration for VBI’s manufacturing capabilities and certain related assets at Rehovot manufacturing facility, the intellectual property for VBI-2601, VBI’s hepatitis B immunotherapeutic candidate, and a license for VBI-1901 in the Asia Pacific region, excluding Japan
Following completion of the full transaction, target mid-year 2024, VBI expects its total debt principal to be significantly reduced to $17 million
2024: Additional data expected from Phase 1 study of VBI-2901, VBI’s multivalent pan-coronavirus vaccine candidate – initial data from which were reported in September 2023
Recent Peer-Reviewed Publications

Langley, Gantt, et al., "An enveloped virus-like particle alum-adjuvanted cytomegalovirus vaccine is safe and immunogenic: A first-in-humans Canadian Immunization Research Network (CIRN) study" published in Vaccine – Link Here
Financial Results for the Three Months Ended March 2024

Cash Position: As of March 31, 2024 VBI had $12.6 million in cash as compared with $23.7 million in cash as of December 31, 2023. Cash position at March 31, 2024, does not include approximately $2.8 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 with Jefferies LLC, subsequent to March 31, 2024 and through early April 2024.
Revenues, net: Revenues, net for the first quarter 2024 were $1.2 million as compared to $0.5 million for the same period in 2023. The revenue increase was a result of an increase in product sales of PreHevbrio in the U.S.
Cost of Revenues: Cost of revenues was $2.7 million in the first quarter of 2024 as compared to $3.6 million in the first quarter of 2023. The decrease in the cost of revenues was a result of the April 2023 organizational changes and decreased inventory-related costs, offset by increased product sales.
Research and Development (R&D): R&D expenses for the first quarter of 2024 were $2.6 million as compared to $3.2 million for the same period in 2023. R&D expenses were offset by $0.7 million for the three months ended March 31, 2024, and $2.4 million for the three months ended March 31, 2023 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 first quarter of 2024 were $7.7 million as compared to $13.3 million in the first quarter of 2023. The decrease in SG&A expenses was mainly a result of the April 2023 organizational changes that reduced our internal workforce, commercial field teams, and operating expenses.
Net Cash Used in Operating Activities: Net cash used in operating activities for the first quarter of 2024 was $11.8 million compared to $21.7 million for the same period in 2023. The 46% decrease in cash outflows is largely due to a decrease in net loss as a result of the April 2023 organizational changes, in addition to the change in operating working capital, most notably in inventory, other current assets, accounts payable, and other current liabilities.
Net Loss and Net Loss Per Share: Net loss and net loss per share for the first quarter of 2024 were $17.9 million and $0.73, respectively, as compared to a net loss and net loss per share of $27.8 million and $3.22 for the first quarter of 2023, respectively.
Net Loss and Net Loss Per Share, Excluding Foreign Exchange Loss: Net loss and net loss per share, excluding foreign exchange loss, for the first quarter 2024 were $13.6 million and $0.55, respectively, compared to $20.9 million and $2.43 for the first quarter 2023, 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.
Foreign exchange loss for the first quarter of 2024 was $4.3 million as compared to $6.8 million for the first quarter of 2023. Certain intercompany loans between the Company and its subsidiaries are denominated in a currency other than the functional currency of each entity. The decrease in foreign exchange loss was a result of the changes in the foreign currency exchange rates (of the New Israeli Shekel and the Canadian Dollar) in which the foreign currency transactions were denominated for each of those periods, including the foreign exchange impact of intercompany loans that are translated at period end.
Use of Non-GAAP Financial Measures

Net Loss, Excluding Foreign Exchange Loss, and Net Loss Per Share, Excluding Foreign Exchange Loss, are non-GAAP financial measures and are defined as Net Loss and Net Loss Per Share excluding the foreign exchange loss in both calculations. Net Loss, Excluding Foreign Exchange Loss, and Net Loss Per Share, Excluding Foreign Exchange Loss, are not intended to replace Net Loss or Net Loss Per Share or other measures of financial performance reported in accordance with generally accepted accounting principles (GAAP). VBI’s management believes that the presentation of Net Loss, Excluding Foreign Exchange Loss, and Net Loss Per Share, Excluding Foreign Exchange Loss, are useful to investors because management does not consider foreign exchange loss, which is primarily driven by changes in exchange rates related to certain intercompany loans, and is a non-recurring item, when evaluating VBI’s operating performance. Non-GAAP financial measures are meant to supplement, and to be viewed in conjunction with, GAAP financial results. The presentation of these non-GAAP financial measures should not be considered in isolation or as a substitute for comparable GAAP financial measures and should be read only in conjunction with the Company’s financial statements prepared in accordance with GAAP. Reconciliations of the Company’s non-GAAP measures are included below.

The following represents a reconciliation of Net Loss to Net Loss, Excluding Foreign Exchange Loss, and Net Loss Per Share to Net Loss Per Share, Excluding Foreign Exchange Loss. See "Non-GAAP Financial Information" 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.

GAAP to Non-GAAP Reconciliations

The following represents a reconciliation of Net Loss to Net Loss Excluding Impairment Charges and Foreign Exchange Loss and Net Loss per Share Excluding Foreign Exchange Loss.

About PreHevbrio [Hepatitis B Vaccine (Recombinant)]

PreHevbrio is the only 3-antigen hepatitis B vaccine, comprised of the three surface antigens of the hepatitis B virus – Pre-S1, Pre-S2, and S. It is approved for use in the U.S., European Union/European Economic Area, United Kingdom, Canada, and Israel. The brand names for this vaccine are: PreHevbrio (US/Canada), PreHevbri (EU/EEA/UK), and Sci-B-Vac (Israel).

Please visit www.PreHevbrio.com for U.S. Important Safety Information for PreHevbrio [Hepatitis B Vaccine (Recombinant)], or please see U.S. Full Prescribing Information.

U.S. Indication

PreHevbrio is indicated for prevention of infection caused by all known subtypes of hepatitis B virus. PreHevbrio is approved for use in adults 18 years of age and older.

U.S. Important Safety Information (ISI)

Do not administer PreHevbrio to individuals with a history of severe allergic reaction (e.g. anaphylaxis) after a previous dose of any hepatitis B vaccine or to any component of PreHevbrio.

Appropriate medical treatment and supervision must be available to manage possible anaphylactic reactions following administration of PreHevbrio.

Immunocompromised persons, including those on immunosuppressant therapy, may have a diminished immune response to PreHevbrio.

PreHevbrio may not prevent hepatitis B infection, which has a long incubation period, in individuals who have an unrecognized hepatitis B infection at the time of vaccine administration.

The most common side effects (> 10%) in adults age 18-44, adults age 45-64, and adults age 65+ were pain and tenderness at the injection site, myalgia, fatigue, and headache.

There is a pregnancy exposure registry that monitors pregnancy outcomes in women who received PreHevbrio during pregnancy. Women who receive PreHevbrio during pregnancy are encouraged to contact 1-888-421-8808 (toll-free).

To report SUSPECTED ADVERSE REACTIONS, contact VBI Vaccines at 1-888-421-8808 (toll-free) or VAERS at 1-800-822-7967 or www.vaers.hhs.gov.

Please see Full Prescribing Information.

On May 15, 2024 Bayer reported initiation of dosing in a Phase I first-in-human clinical study with 225Ac-PSMA-Trillium (BAY 3563254), a next-generation targeted alpha therapy (Press release, Bayer, MAY 15, 2024, View Source [SID1234643373]). The investigational candidate, labeled with actinium-225 and comprising a novel PSMA (prostate-specific membrane antigen) -targeting small molecule with a customized albumin-binding moiety, is designed to potentially improve therapeutic efficacy and reduce side effects in normal organs such as salivary glands. The dose-escalation study (NCT06217822) will evaluate the safety, tolerability and efficacy of 225Ac-PSMA-Trillium in patients with advanced metastatic castration resistant prostate cancer (mCRPC).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"Despite recent advances in the treatment landscape for prostate cancer, there is still a high unmet need for novel precision therapy options to improve outcomes for patients with metastatic castration-resistant prostate cancer," said Fred Saad, MD, FRCS, Professor and Chairman of Surgery at University of Montreal and Director of Genitourinary Oncology at University of Montreal Hospital Center (CHUM), Canada."225Ac-PSMA-Trillium is a novel approach, which could provide a new treatment to address this unmet need in mCRPC."

"Targeted radionuclide therapy is a strategic pillar of precision oncology at Bayer, holding the promise to shift the treatment paradigm for patients, including those whose disease has developed resistance to other treatments," said Dominik Ruettinger, M.D., Ph.D., Head of Research and Early Development for Oncology at Bayer. "We are excited to announce initiation of the phase I and dosing of the first patient with 225Ac-PSMA-Trillium. With its unique design, we believe it could offer a meaningful benefit for patients with metastatic prostate cancer, and we look forward to advancing the program through clinical development."

Prostate cancer is the second most commonly diagnosed cancer in men1 and a key area of focus at Bayer. Despite significant advances in the last decade, mCRPC remains a deadly disease with a median survival of about 31 months.2 Bayer remains committed to advancing medical innovations for patients across all stages of prostate cancer.

In April, Bayer introduced 225Ac-PSMA-Trillium during the New Drugs on the Horizon session at the AACR (Free AACR Whitepaper) (American Association of Cancer Research) Annual Meeting.3 Along with preclinical in vitro and in vivo characterization, the results were presented for a Phase 0 clinical imaging and dosimetry study conducted in participants with prostate cancer.

About Targeted Alpha Therapy

Targeted alpha therapy is an emerging class of radionuclide therapy that can be used against a variety of tumors. It is designed to deliver alpha particle radiation directly to the tumor inside the body, either via its bone-seeking property (radium-223) or by combining alpha radionuclides, such as actinium-225, with specific targeting moieties. This localized delivery of the radioactive payload induces difficult to repair double-strand DNA breaks in tumor cells; damage that can cause cell cycle arrest or cell death. At the same time, because the energy does not travel very far, there is a potential for reduced damage to nearby normal tissues.