Imdusiran

OXFORD, United Kingdom, Aug. 08, 2024 (GLOBE NEWSWIRE) -- Barinthus Biotherapeutics plc (NASDAQ: BRNS), a clinical-stage biopharmaceutical company developing novel T cell immunotherapeutic candidates, provided an overview of the Company’s progress and announced its financial results for the second quarter of 2024. “Following the positive VTP-300 Phase 2 data in hepatitis B virus (HBV) in the second quarter, we made the strategic decision to prioritize our HBV and celiac disease programs. These two programs have the greatest probability for success and value creation, and represent significant opportunities in therapeutic areas with substantial unmet patient need,” said Bill Enright, Chief Executive Officer of Barinthus Bio. "We are very excited as we plan to initiate our first in human clinical trial of VTP-1000 in celiac disease in the third quarter, utilizing our novel SNAP-TI platform. Celiac disease is an increasingly common autoimmune disease with no FDA or EMA-approved treatments. Towards the end of the year we look forward to sharing updated data from our HBV program as our two Phase 2 trials utilizing VTP-300, which are evaluating a potential functional cure regimen, mature further." Second Quarter 2024 and Recent Corporate Developments Clinical developments: VTP-300 (HBV) In June 2024, we announced updated data from two ongoing Phase 2 clinical trials in people with chronic hepatitis B (CHB) at the European Association for the Study of the Liver (EASL) Congress 2024. The presentations included updated interim data from the Phase 2b clinical trial (HBV003), as well as updated interim data from the Phase 2a clinical trial (IM-PROVE II, AB-729-202) in partnership with Arbutus Biopharma, both in people with CHB receiving ongoing standard of care nucleos(t)ide analogue (NUC) therapy. Interim HBV003 data: VTP-300 and Low-dose Nivolumab Interim data from the HBV003 trial showed; Nearly 20% of participants across the groups had undetectable HBsAg and this was maintained for ≥16 weeks in the two cases who had reached that time point. 76% of participants were eligible for NUC discontinuation.71% of those who did discontinue remained off NUCs at time of data cutoff on April 15, 2024.67% of participants across all groups assessed for NUC discontinuation had HBsAg <10 IU/mL at Week 24 or later.Robust T cell responses were observed to all VTP-300 encoded antigens.There were no Serious Adverse Events (SAEs), Grade 3 or 4 Adverse Events (AEs) related to treatment. Interim IM-PROVE II data: imdusiran and VTP-300 Interim data from the IM-PROVE II clinical trial showed; 20% of participants had undetectable HBsAg at Week 72 in the VTP-300 treatment group, compared to none in the placebo group.84% of participants in the VTP-300 treatment group were eligible for NUC discontinuation, compared to 52% in the placebo group.88% of those who did discontinue remained off NUCs, compared to 80% in the placebo group remaining off NUCs at time of data cutoff on April 12, 2024.Robust reductions of HBsAg were observed during the imdusiran lead-in period with 95% of participants achieving HBsAg <100 IU/mL before undergoing dosing in the VTP-300 treatment or placebo groups at week 24. A statistically significant difference (p<0.05) in HBsAg levels between the VTP-300 treatment and placebo groups was recorded at Week 72.Treatment with imdusiran and VTP-300 was generally well-tolerated, with no SAEs or treatment discontinuations reported. Corporate Updates On June 3, 2024, Dr. Leon Hooftman joined the Company as Chief Medical Officer. He brings significant drug development expertise across a broad array of therapeutic areas including immunology, autoimmunity, hematology, oncology and infectious diseases. In June 2024, we announced a strategic pipeline prioritization following the positive interim data from VTP-300 in CHB presented at EASL. This announcement included the prioritization of the development of VTP-300 in CHB and VTP-1000 in celiac disease, and consequently a reduction in workforce. Upcoming Milestones In the third quarter of 2024, the Company expects to: VTP-1000 (Celiac Disease): Dose the first patient in GLU001, a randomized, placebo-controlled Phase 1 trial including a controlled gluten challenge to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of VTP-1000 in adults with celiac disease. In the fourth quarter of 2024, the Company expects to: VTP-300 (HBV): Announce updated interim data from HBV003, our Phase 2b trial evaluating additional dosing of VTP-300 and timing of PD-1 inhibition, in people with CHB on NUC therapy.Announce updated interim data from the Phase 2a AB-729-202 clinical trial evaluating the combination of VTP-300 and Arbutus’ imdusiran, in people with CHB on NUC therapy. Pipeline These are estimated timelines only and our pipeline may be subject to change. Second Quarter 2024 Financial Highlights Cash position: As of June 30, 2024, cash, cash equivalents and restricted cash were $117.8 million, compared to $130.0 million as of March 31, 2024. The net cash used in operating activities was $12.0 million in the second quarter of 2024, primarily resulting from development of our pipeline and ongoing clinical trials offset by receipt of an R&D tax credit of $1.4 million. Based on current research and development plans, the Company expects its available resources to fund its operating expenses and capital expenditure requirements into the second quarter of 2026.Research and development expenses: Research and development expenses were $11.7 million in the second quarter of 2024 compared to $11.1 million in the first quarter of 2024, with the increase mainly attributable to the progression of VTP-300 (HBV) through the two ongoing Phase 2 trials, partially offset by a reduction in expense from VTP-200 (HPV) which completed in the first quarter of 2024 and for which data was presented in April 2024. The quarter-on-quarter R&D expense per program is outlined in the following table. Period ended Three monthsended June30, 2024 Three monthsended March31, 2024 Change $000 $000 $000 Direct research and development expenses by program: VTP-200 HPV $383 $1,253 $(870)VTP-300 HBV 3,034 1,913 1,121 VTP-500 MERS1 304 172 132 VTP-600 NSCLC2 24 164 (140)VTP-850 Prostate cancer 414 178 236 VTP-1000 Celiac 1,371 1,374 (3)Other and earlier stage programs 908 784 124 Total direct research and development expenses $6,438 $5,838 $600 Indirect research and development expenses: Personnel-related (including share-based compensation) 4,763 4,335 428 Facility related 342 390 (48)Other indirect costs 119 562 (443)Total indirect research and development expenses 5,224 5,287 (63)Total research and development expense $11,662 $11,125 $537 1 The development of VTP-500 is funded pursuant to an agreement with the Coalition for Epidemic Preparedness Innovations (CEPI). 2 The VTP-600 NSCLC Phase 1/2a trial is sponsored by Cancer Research UK. General and administrative expenses: General and administrative expenses were $7.2 million in the second quarter of 2024, compared to $6.0 million in the first quarter of 2024. The increase of $1.2 million relates primarily to a loss of $0.1 million on foreign exchange in the second quarter of 2024, compared to a gain of $1.2 million in the first quarter of 2024.Net loss: For the second quarter of 2024, the Company generated a net loss attributable to its shareholders of $16.9 million, or $(0.43) per share on both basic and fully diluted bases, compared to a net loss attributable to its shareholders of $15.5 million, or $(0.40) per share on both basic and fully diluted bases in the first quarter of 2024. About Barinthus Bio Barinthus Bio is a clinical-stage biopharmaceutical company developing novel T cell immunotherapeutic candidates designed to guide the immune system to overcome chronic infectious diseases and autoimmunity. Helping people living with serious diseases and their families is the guiding principle at the heart of Barinthus Bio. With a focused pipeline built around our proprietary platform technologies, Barinthus Bio is advancing product candidates in infectious disease and autoimmunity, including: VTP-300, an immunotherapeutic candidate utilizing our ChAdOx/MVA platform designed as a potential component of a functional cure for chronic HBV infection and VTP-1000, an autoimmune candidate designed to utilize the SNAP-Tolerance Immunotherapy (TI) platform to treat patients with celiac disease. Barinthus Bio also has a Phase 1 clinical trial for VTP-850, a second-generation immunotherapeutic candidate designed to treat recurrent prostate cancer. Barinthus Bio’s proven scientific expertise, diverse portfolio and focus on pipeline development uniquely positions the company to navigate towards delivering treatments for people with infectious diseases and autoimmunity that have a significant impact on their everyday lives. For more information, visit www.barinthusbio.com. Forward Looking Statements This press release contains forward-looking statements regarding Barinthus Bio within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, which can generally be identified as such by use of the words “may,” “will,” “plan,” “forward,” “encouraging,” “believe,” “potential,” "expect", and similar expressions, although not all forward-looking statements contain these identifying words. These forward-looking statements include, without limitation, express or implied statements regarding our future expectations, plans and prospects, including our product development activities and clinical trials, including timing for readouts of any preliminary, interim or final data for any of our programs, the timing for initiation of any clinical trials, including dosing of the first patient in GLU001 for VTP-1000, our anticipated regulatory filings and approvals, our cash runway, and our ability to develop and advance our current and future product candidates and programs. Any forward-looking statements in this press release are based on our management’s current expectations and beliefs and are subject to numerous risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, risks and uncertainties related to the success, cost and timing of our pipeline development activities and planned and ongoing clinical trials, including the risk that the timing for preliminary, interim or final data or initiation of our clinical trials may be delayed, the risk that interim or topline data may not reflect final data or results, our ability to execute on our strategy, regulatory developments, the risk that we may not achieve the anticipated benefits of our pipeline prioritization and corporate restructuring, our ability to fund our operations and access capital, our cash runway, including the risk that our estimate of our cash runway may be incorrect, global economic uncertainty, including disruptions in the banking industry, the conflicts in Ukraine, Israel and Gaza, and other risks identified in our filings with the Securities and Exchange Commission, including our Annual Report on Form 10-K for the year ended December 31, 2023, our Quarterly Reports on Form 10-Q and Current Reports on Form 8-K. We caution you not to place undue reliance on any forward-looking statements, which speak only as of the date they are made. We expressly disclaim any obligation to publicly update or revise any such statements to reflect any change in expectations or in events, conditions or circumstances on which any such statements may be based, or that may affect the likelihood that actual results will differ from those set forth in the forward-looking statements. BARINTHUS BIOTHERAPEUTICS PLC CONSOLIDATED BALANCE SHEETS (IN THOUSANDS, EXCEPT NUMBER OF SHARES AND PER SHARE AMOUNTS) (UNAUDITED) June 30,2024 December 31,2023ASSETS Cash, cash equivalents and restricted cash$117,774 $142,090 Research and development incentives receivable 4,523 4,908 Prepaid expenses and other current assets 9,582 9,907 Total current assets 131,879 156,905 Goodwill 12,209 12,209 Property and equipment, net 10,962 11,821 Intangible assets, net 23,527 25,108 Right of use assets, net 7,286 7,581 Other assets 886 882 Total assets$186,749 $214,506 LIABILITIES AND STOCKHOLDERS’ EQUITY Current liabilities: Accounts payable 2,556 1,601 Accrued expenses and other current liabilities 10,278 9,212 Deferred income 839 — Operating lease liability - current 1,916 1,785 Total current liabilities 15,589 12,598 Non-current liabilities: Operating lease liability - non-current 10,654 11,191 Contingent consideration 1,888 1,823 Other non-current liabilities 1,338 1,325 Deferred tax liability, net 490 574 Total liabilities$29,959 $27,511 Commitments and contingencies (Note 15) Stockholders’ equity: Ordinary shares, £0.000025 nominal value; 39,184,338 shares authorized, issued and outstanding (December 31, 2023: authorized, issued and outstanding:38,643,540) 1 1 Deferred A shares, £1 nominal value; 63,443 shares authorized, issued and outstanding (December 31, 2023: authorized, issued and outstanding:63,443) 86 86 Additional paid-in capital 390,273 386,602 Accumulated deficit (209,010) (176,590)Accumulated other comprehensive loss – foreign currency translation adjustments (24,732) (23,315)Total stockholders’ equity attributable to Barinthus Biotherapeutics plc shareholders 156,618 186,784 Noncontrolling interest 172 211 Total stockholders’ equity$156,790 $186,995 Total liabilities and stockholders’ equity$186,749 $214,506 BARINTHUS BIOTHERAPEUTICS PLC CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS (IN THOUSANDS, EXCEPT NUMBER OF SHARES AND PER SHARE AMOUNTS) (UNAUDITED) Three months ended Six months ended June 30, 2024 June 30, 2023 June 30, 2024 June 30, 2023License revenue 1$— $334 $— $802 Total revenue — 334 — 802 Operating expenses Research and development 11,662 13,543 22,787 23,357 General and administrative 7,201 13,128 13,195 25,266 Total operating expenses 18,863 26,671 35,982 48,623 Other operating income 577 — 782 — Loss from operations (18,286) (26,337) (35,200) (47,821)Other income/(expense): Interest income 635 522 1,410 2,110 Interest expense (12) (14) (24) (14)Research and development incentives 693 559 1,287 1,716 Other income 20 310 20 310 Total other income, net 1,336 1,377 2,693 4,122 Loss before income tax (16,950) (24,960) (32,507) (43,699)Tax benefit 7 1,136 44 1,652 Net loss (16,943) (23,824) (32,463) (42,047)Net loss attributable to noncontrolling interest 12 22 43 65 Net loss attributable to Barinthus Biotherapeutics plc shareholders (16,931) (23,802) (32,420) (41,982) Weighted-average ordinary shares outstanding, basic 39,041,111 38,407,672 38,907,296 38,211,625 Weighted-average ordinary shares outstanding, diluted 39,041,111 38,407,672 38,907,296 38,211,625 Net loss per share attributable to ordinary shareholders, basic$(0.43) $(0.62) $(0.83) $(1.10)Net loss per share attributable to ordinary shareholders, diluted$(0.43) $(0.62) $(0.83) $(1.10) Net loss$(16,943) $(23,824) $(32,463) $(42,047)Other comprehensive gain/(loss) – foreign currency translation adjustments 164 5,604 (1,413) 10,184 Comprehensive loss (16,779) (18,220) (33,876) (31,863)Comprehensive loss attributable to noncontrolling interest 11 15 39 52 Comprehensive loss attributable to Barinthus Biotherapeutics plc shareholders$(16,768) $(18,205) $(33,837) $(31,811) 1 Includes license revenue from related parties for the three and six months ended June 30, 2024 of nil (three and six months ended June 30, 2023: $0.3 million and $0.8 million, respectively). IR contacts:Christopher M. Calabrese Managing Director LifeSci Advisors+1 917-680-5608ccalabrese@lifesciadvisors.com Kevin GardnerManaging DirectorLifeSci Advisors+1 617-283-2856kgardner@lifesciadvisors.com Media contact:Audra FriisSam Brown, Inc.+1 917-519-9577audrafriis@sambrown.com Company contact:Jonothan BlackbournIR & PR ManagerBarinthus Bioir@barinthusbio.com A photo accompanying this announcement is available at https://www.globenewswire.com/NewsRoom/AttachmentNg/c0677b65-29bb-4869-b045-22325fa94f17

The industry heat has forced three biotechs to slim down on staff. As biotechs attempt to turn a fresh page in August, at least three companies have shed staff in attempts to forge on.  First up is gene therapy company UniQure. The business is undergoing an organizational restructuring designed to streamline operations, according to an Aug. 1 release on the company's second-quarter financial results.The streamlining strategy is expected to slash global headcount by 65%, or 300 roles—cuts that include the recent sale of a Massachusetts manufacturing facility to Genezen. That sale is also tied to the departure of Chief Operating Officer Pierre Caloz, who helped UniQure establish commercial manufacturing capabilities for Hemgenix, an approved hemophilia B gene therapy.Amin Abujoub, Ph.D., who previously served as the biopharma’s chief quality officer, is taking on the new role of chief technical operations officer, which will include global oversight of contract manufacturers. The restructure is expected to be complete in the fourth quarter of this year and slash recurring costs by 40%, or $75 million annually. As of June 30, UniQure had $524 million on hand, money expected to stretch to the end of 2027, according to the company release.“These actions are designed to ensure we have the funding required to achieve key milestones and drive shareholder value, as we endeavor to deliver transformative medicines to patients in need,” uniQure CEO Matt Kapusta said in the release.Some of those milestones center around AMT-130, the company’s investigational gene therapy for Huntington’s disease. The asset is currently being studied in a phase 1/2 trial, with a recent interim data drop revealing a slowing in disease progression.The FDA also granted the candidate regenerative medicine advanced therapy designation, and UniQure expects to meet with the FDA in the second half of this year about potentially pursuing an expedited clinical development path, according to Kapusta. Clinical-stage Arbutus Biopharma follows suit, with a “decision to streamline the organization” to advance two clinical programs, leaving a mid-stage trial and 40% of staff in its wake.The layoffs will mainly impact discovery roles, plus general and administrative functions, according to an Aug. 1 release on second-quarter financial results.Arbutus will zero in on phase 2b development of imdusiran, an RNA interference therapeutic made to reduce hepatitis B virus (HBV) viral proteins and antigens, as well as AB-101, an oral PD-L1 inhibitor being evaluated in an early-stage trial for chronic HBV.Meanwhile, the company will discontinue HBV research efforts and a phase 2a clinical trial dubbed IM-PROVE III. The study was assessing imdusiran in combination with anti-PD-L1 durvalumab, among patients with chronic HBV. The study had an estimated enrollment of 30 patients and expected readout slated for 2027, according to ClinicalTrials.gov. This decision to terminate the trial was “based on a prioritization of resources and the projected availability of clinical data from this trial,” according to the company's release. The reorganization is expected to extend the company’s cash runway into the final quarter of 2026.It’s the second layoff round within a year for the biotech. In November 2023, the company let go of 24% of its team after redirecting money for research programs toward clinical efforts. Last up today is Takeda spinout HilleVax. The vaccine-focused biotech revealed a 40% workforce reduction after market close yesterday. The layoffs will impact about 41 employees and is designed to reduce operating costs.The move is made so HilleVax can explore “the potential for continued development of its HIL-214 and HIL-216 norovirus vaccine candidates, as well as business development-related activities for these vaccine candidates,” according to the July 31 release. In early July, HIL-214—the company’s sole clinical-stage candidate—failed a phase 2b trial in norovirus-related acute gastroenteritis.The virus-like particle-based vaccine missed all primary and secondary endpoints of the study which included more than 2,800 infants, showing an efficacy of just 5%.