On September 15, 2022 Candel Therapeutics, Inc. (Nasdaq: CADL) (Candel or the Company), a late clinical stage biopharmaceutical company developing novel oncolytic viral immunotherapies, reported that the European Medicines Agency (EMA) Committee for Orphan Medical Products (COMP) has issued a positive opinion on the Company’s application for orphan drug designation for CAN-2409 for the treatment of glioma (Press release, Candel Therapeutics, SEP 15, 2022, View Source [SID1234619592]). CAN-2409, a genetically modified adenovirus and the Company’s most advanced investigational therapy, is currently being evaluated in multiple phase 2 and phase 3 clinical trials for lung, brain, pancreatic and prostate cancers.

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The orphan drug designation follows the U.S. Food and Drug Administration (FDA) fast track designation, which was granted in June 2021, for CAN-2409 in combination with valacyclovir following standard of care treatment in newly diagnosed high-grade glioma.

Paul Peter Tak, MD, PhD, FMedSci, President and CEO of Candel, said, "Glioma, while mercifully rare, is a devastating disease with significant morbidity and mortality. The EMA’s orphan drug designation recognizes the high unmet need and the potential of CAN-2409 in this patient population. We look forward to working with the EMA and FDA in an effort to bring our investigational medicine to patients as we initiate our phase 3 clinical trial of CAN-2409 in high-grade glioma this year."

Orphan drug designation in the European Union (EU) is granted by the European Commission based on a positive opinion issued by the EMA COMP. The EMA’s orphan drug designation is potentially available to companies developing treatments for life-threatening or chronically debilitating conditions that affect no more than five in 10,000 persons in the EU. In addition, there must be sufficient clinical or non-clinical data to suggest the product candidate may produce clinically relevant outcomes, and grounds to indicate it can provide a significant benefit over any currently authorized products. The designation can provide financial and regulatory incentives, including a 10-year period of marketing exclusivity in the EU after product approval, protocol assistance from the EMA at reduced fees during the product development phase and access to centralized marketing authorization.

About CAN-2409

CAN-2409, Candel’s most advanced oncolytic viral immunotherapy product candidate, is a genetically modified adenovirus that is designed to encode the herpes simplex virus thymidine kinase (HSV-tk) gene. HSV-tk is an enzyme that locally converts orally administered valacyclovir into a toxic metabolite that kills infected and nearby cancer cells. Intratumoral administration of CAN-2409 results in immunogenic cell death, followed by the release of tumor-specific neoantigens in the tumor microenvironment. At the same time, the adenoviral vector elicits a strong pro-inflammatory effect in the tumor microenvironment, creating the optimal conditions to induce a specific CD8+ cytotoxic T cell-mediated immune response against the injected tumor and the uninjected distant metastases. This dual mechanism of antigen unmasking and immune activation may enable CAN-2409 to generate a powerful and lasting immune response against a variety of the patient’s tumor-associated neoantigens, minimizing the possibility for immune escape and development of tolerance.

Because of its versatility, CAN-2409 may have the potential to treat a broad range of solid tumors. Encouraging activity has been shown in several preclinical and clinical settings as monotherapy as well as in combination with standard of care radiation therapy, surgery, chemotherapy, and immune checkpoint inhibitor treatment. Furthermore, more than 700 patients have been dosed to date with a favorable safety profile, supporting the potential for combination with other therapeutic strategies without inordinate concern of overlapping adverse events. Currently, Candel is evaluating the effects of treatment with CAN-2409 in high-grade glioma, non-small cell lung cancer, pancreatic cancer, and prostate cancer in ongoing clinical trials.

On September 15, 2022 Starpharma (ASX: SPL, OTCQX: SPHRY) reported that a presentation by Dr Jackie Fairley, CEO, will be broadcast on Thursday 15 September 2022 (US ET) as part of the US OTCQX’s Virtual Life Sciences Investor Forum (Press release, Starpharma, SEP 15, 2022, View Source;mc_eid=bf52dd3418 [SID1234619591]).

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The Life Sciences Investor Forum is a leading investor conference that provides a forum for companies to present to tens of thousands of retail investors as well as advisors.

Details of the forum are found via this link: Virtual Life Sciences Investor Forum.

Starpharma’s pre-recorded presentation features a company overview, including:

the latest interim clinical trial results presented at ESMO (Free ESMO Whitepaper) oncology conference that show efficacy signals in 100% of prostate cancer patients assessed following DEP cabazitaxel treatment;
an overview of Starpharma’s DEP clinical-stage and preclinical assets, and corporate partnerships for its DEP drug delivery platform, including with Merck & Co., Inc (MSD) and AstraZeneca; and
a VIRALEZE overview and recent antiviral nasal spray results, including excellent protection against the SARS-CoV-2 Omicron variant, as well as updates on Starpharma’s portfolio more broadly.

ON September 15, 2022 Oxford Biomedica plc ("Oxford Biomedica" or "the Group") (LSE: OXB), a leading gene and cell therapy group, reported that interim results for the six months ended 30 June 2022 (Press release, Oxford BioMedica, SEP 15, 2022, View Source [SID1234619589]).

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Roch Doliveux, Oxford Biomedica’s Chair and Interim Chief Executive Officer, said:
"We have made significant strategic and operational progress towards our goal of becoming a global viral vector leader. In the first half of 2022 we achieved double digit revenue growth in our core business and since the start of the year have signed numerous new or expanded partnership deals, including a new AAV deal, resulting in an over 70% increase in the number of customers with whom we work. In addition, we executed the transformational launch of Oxford Biomedica Solutions which brings innovative AAV capabilities, further capacity and a significant platform in the US, delivering on our strategic objective to become vector agnostic and provide world-leading innovative process development and manufacturing services to our clients. With a strong cash position, a robust and growing business and entry into the fast-growing AAV market, Oxford Biomedica is in an excellent position to achieve long-term future profitable growth as a leading partner of choice to deliver life-saving cell and gene therapies to patients."

H1 2022 FINANCIAL HIGHLIGHTS
– Double digit revenue growth in the core business (excluding COVID-19 vaccine manufacturing) compared to H1 2021 offset by the decrease in COVID-19 vaccine manufacturing; total revenue decreased by 21% to £64.0 million (H1 2021: £81.3 million)

– Bioprocessing and commercial development revenues decreased by 24% to £57.3 million (H1 2021: £75.6 million) largely driven by a reduction in COVID-19 vaccine manufacturing revenues but partly offset by an increase in revenues from lentiviral vector and AAV commercial development and manufacturing activities

– Licences, milestones & royalties were £6.7 million (H1 2021: £5.7 million), the increase of 18% resulting from licence fees from new partner programmes

– The launch of Oxford Biomedica Solutions, enabling entry into the fast-growing AAV market whilst also establishing a key strategic presence in the US, including one-off acquisition-related costs, drove an increase in operating expenses to £56.2 million (H1 2021: £23.6 million). Active cost control initiatives were initiated to reduce the Group’s operating cost base as the COVID-19 pandemic continues to ease

– Operating EBITDA1 loss and operating loss of £5.8 million and £19.2 million respectively (H1 2021 EBITDA1 profit and operating profit of £27.1 million and £19.7 million respectively); this included one-off acquisition-related due diligence costs of £5.1 million relating to the transaction with Homology Medicines to establish Oxford Biomedica Solutions

– Cash used in operations was £24.5 million compared to £22.2 million generated in H1 2021
– The Group’s capital expenditure of £6.0 million (H1 2021: £3.5 million) consisted mainly of purchases of equipment required for manufacturing and laboratory facilities

– Cash at 30 June 2022 was £118.5 million and £115.8 million at 31 August 2022; net cash at 30 June 2022 was £50.1 million and £42.1 million at 31 August 2022

OTHER RECENT DEVELOPMENTS AND OUTLOOK
– The Group is in the process of part-repaying and refinancing the $85 million Oaktree loan facility taken out in March 2022 and a process is underway for the sale and leaseback of the Group’s Windrush Court facility in Oxford

– The Group expects similar levels of revenues in the second half of 2022 as those achieved in the first half of 2022 and is expecting to deliver broadly break-even Operating EBITDA for the second half of the year

1Operating EBITDA (Earnings Before Net Finance Costs, Tax, Depreciation, Amortisation, fair value adjustments of assets at fair value through profit and loss, and Share Based Payments) is a non-GAAP measure often used as a surrogate for operational cash flow as it excludes from operating profit or loss all non-cash items, including the charge for share options. A reconciliation to GAAP measures is provided on page 12.

OPERATIONAL HIGHLIGHTS (including post-period events)
– Entered into fast-growing AAV market through a transformational deal with Homology Medicines, completed in March 2022, to establish Oxford Biomedica Solutions LLC ("Oxford Biomedica Solutions"), a high-performing full-scope scope AAV manufacturing and innovation business near Boston, US; new AAV partnership announced in September

– Expanded customer base by more than 70%, currently working on more than 20 programmes, with a robust new business pipeline across all key vector types

– Amended and expanded existing License and Clinical Supply Agreement with Juno Therapeutics ("Juno"), a wholly-owned subsidiary of Bristol Myers Squibb Company, to include two new viral vector programmes

– Continued strong relationship with Novartis with Kymriah available in more than 400 qualified treatment centres in 30 countries having coverage for at least one indication, and expansion into a third indication

– Signed a new three-year Master Services and Development Agreement with AstraZeneca to facilitate potential future manufacturing opportunities for the AstraZeneca COVID-19 vaccine

– Signed four new US-based customer agreements with Cabaletta Bio ("Cabaletta"), with an undisclosed private biotechnology company advancing a new generation of adoptive cell therapies, with an undisclosed late-stage cell and gene therapy company, and with an undisclosed new partner for Oxford Biomedica Solutions’ AAV platform

– Continued to strengthen the Board with the appointment of Namrata Patel as an Independent Non-Executive Director. John Dawson stepped down as CEO, with Chair Roch Doliveux assuming the role of Interim CEO in January 2022. The formal process to appoint a successor is progressing well

Analyst briefing
Management will be hosting a virtual briefing and Q&A session for analysts at 13:00 BST / 8:00 EST today, 15 September. The presentation will be available on the Group’s website at www.oxb.com

On September 15, 2022 Newron Pharmaceuticals S.p.A. ("Newron") (SIX: NWRN, XETRA: NP5), a biopharmaceutical company focused on the development of novel therapies for patients with diseases of the central and peripheral nervous system, reported its financial results and operational highlights for the half year ended June 30, 2022 and provided an update on its R&D and business activities (Press release, Newron, SEP 15, 2022, View Source [SID1234619588]).

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Highlights H1 2022:

Evenamide (Schizophrenia)

Key data presented at the 33rd CINP Hybrid World Congress of Neuropsychopharmacology, Taipei, Taiwan, including:
Encouraging interim data from the first 100 patients in study 014, a world-first, randomized, open-label trial of evenamide as an add-on to an antipsychotic in patients with chronic, treatment-resistant schizophrenia (TRS). Results showed the addition of evenamide improved symptoms of psychosis. Enrollment in this study is expected to be completed by end of 2022 and results are expected in QI 2023
Study design for an upcoming potentially pivotal, randomized, double-blind, placebo-controlled, eight-week, global study (003) assessing the safety and efficacy of evenamide as an add-on treatment in patients with TRS, which is expected to commence in 2023
Safety data from more than 400 healthy volunteers and patients with schizophrenia treated with evenamide, showing that the compound is safe and well tolerated
The first potentially pivotal study of the Phase II/III evenamide development program, study 008A with evenamide as add on therapy in patients with chronic schizophrenia experiencing inadequate response to their current antipsychotics, is continuing to enroll patients, and results are expected in first half of 2023
Newron continues to evaluate strategic commercial and development partnering options for evenamide
Xadago/Safinamide (Parkinson’s disease)

Newron and its partners Zambon and Supernus continue to work to protect intellectual property rights associated with Xadago/safinamide in the US, responding to Paragraph IV Notice Letters regarding Abbreviated New Drug Applications submitted from generic pharmaceutical manufacturers
Newron continues to plan a potentially pivotal study with safinamide in Parkinson’s disease patients with levodopa-induced dyskinesia (PD LID) in partnership with Zambon
Corporate

Reaffirmed commitment to Environmental, Social and Governance (ESG) reporting and standards through the establishment of an ESG board committee, which is undertaking a comprehensive assessment of key ESG areas to identify measurable targets for Newron
Strengthened senior leadership team with the appointment of Filippo Moriggia as Vice President Operations, who will also lead Newron’s operational ESG activities
Newron continues to explore a number of potential opportunities to expand its pipeline in central nervous system diseases
Stefan Weber, CEO of Newron, commented:

"In particular, we are very excited about the encouraging progress made in the first half of 2022 with our ongoing evenamide development program. This includes the interim results from study 014 – the first ever randomized international trial with a New Chemical Entity, evenamide, as add-on therapy in patients with treatment-resistant schizophrenia – as well as the continued progress in study 008A, the first potentially pivotal study with evenamide as add-on therapy in patients with schizophrenia who are inadequate responders to atypical antipsychotics. We also strengthened our senior leadership team and furthered our commitments to ESG standards, principles and outcomes."

Evenamide

In June 2022, Newron presented key scientific data of evenamide at the 33rd Collegium Internationale Neuro-Psychopharmacologicum (CINP) Hybrid World Congress of Neuropsychopharmacology, in Taipei, Taiwan.

Among others, the Company presented interim results from the first 100 patients in study 014, an open-label study of evenamide as an add-on antipsychotic in patients with moderate to severe treatment resistant schizophrenia (TRS), who were not responding to current antipsychotic medication. The primary objective of the study was to evaluate the safety and tolerability of evenamide given orally at three fixed doses (7.5, 15 and 30 mg bid). The vast majority of these patients were treated with 7.5mg and 15mg bid doses, as an Independent Safety Monitoring Board first reviewed the safety data from the lower doses before allowing randomization to a 30mg bid dose. The assessment of preliminary efficacy was a secondary objective and was based on changes from baseline in the Positive and Negative Syndrome Scale (PANSS); Clinical Global Impression – Change from baseline (CGI-C); Severity of illness (CGI-S); and Strauss-Carpenter Level of Functioning (LOF) scale.

The interim results from this world-first, international, six-week, open-label, randomized, rater-blinded and multi-center trial showed that the addition of evenamide improved symptoms of psychosis in patients with chronic TRS. This was reflected by an approximately 12% reduction in the PANSS score, CGI-S improvement of 0.7, and CGI-C ratings, indicating that 77% of patients were considered to have improved. The results confirm the potential of evenamide as an add-on therapy to antipsychotics to improve the lives of patients who continue to experience severe symptoms of psychosis under their current medication.

Newron sees significant progress in the enrolment of patients for study 014 and expects to complete the process by the end of 2022, with results from the study due in QI/2023. Newron is especially excited by the impressive rate (> 90%) of treatment resistant patients deciding to continue treatment with evenamide by entering into the long-term extension study 015. At the CINP congress, the Company had also outlined safety data from more than 400 healthy volunteers and patients with schizophrenia who were treated with evenamide. The results showed that evenamide was well tolerated, with no safety issues identified.

Finally, Newron presented the study design for an upcoming randomised, double-blind, placebo-controlled, eight-week, global study (003) assessing the safety and efficacy of evenamide (15/30mg bid) as an add-on treatment in patients with TRS not responding to their current atypical antipsychotics. This second potentially pivotal study 003 is expected to commence in 2023. Study 003, together with study 008A, form Newron’s promising Phase II/III evenamide development program.

The ongoing Study 008A is a four-week, randomised, double-blind and placebo-controlled study assessing the efficacy, tolerability, and safety (including electroencephalogram effects) of evenamide (30mg bid) in patients with chronic schizophrenia currently being treated with a second-generation antipsychotic, but who are not classed as having TRS. The study is enrolling patients in treatment centers in Europe, Asia and Latin America. Recently, an additional 10 study centers have been added in Europe, to arrive at a total of more than 50 centers, globally. This will help enrol patients to the study and ensure timely progress in the study. The results from study 008A are expected in the first half of 2023. If positive, the study would mark the first well-controlled, potentially pivotal study of evenamide in schizophrenic patients who do not adequately respond to treatment with atypical anti-psychotics.

If approved, evenamide would be the first add-on therapy for schizophrenia. It acts through selective attenuation of the abnormal release of glutamate, which is a novel, alternative mechanism of action to the common dopaminergic or serotonergic anti-psychotics. It would also offer a new therapeutic option for TRS patients, who make up roughly one-third of patients suffering from schizophrenia. This would represent a significant advancement in the approach to schizophrenia and TRS specifically, especially as there are currently no new drugs in development for TRS.

Newron continues to evaluate opportunities to partner on the development and commercialisation of evenamide.

Xadago/safinamide

Newron continues to further develop and market its product, Xadago/safinamide, with its partners, Zambon and Meiji Seika. The Company also continues to plan a potentially pivotal study with safinamide in Parkinson’s disease patients with levodopa-induced dyskinesia (PD LID) in partnership with Zambon.

In reference to the receipt of several Paragraph IV Notice Letters in May 2021 regarding the submission by generic manufacturers of an Abbreviated New Drug Application to the US Food and Drug Administration (FDA), Newron and its partners Zambon and Supernus continue to challenge these submissions and note that Newron’s patents on Xadago (safinamide) tablets remain protected by three patents in the FDA Approved Drugs Product List (Orange Book) until at least 2027.

Corporate

Post period, in July 2022, Newron announced the strengthening of its Senior Management team through the appointment of Filippo Moriggia to the newly created position of Vice President of Operations. Filippo joined Newron in November 2016 as IT Director and became Director of Operations in January 2022.

Newron also reaffirmed its commitment to Environmental, Social and Governance (ESG) through the initiation of a comprehensive assessment of key ESG topics that impact the company and its shareholders, in order to identify measurable targets for its sustainability activities. To effectively pursue these targets and track their progress, the Board of Directors also in July 2022 established an ESG Committee, with Filippo Moriggia, newly appointed VP of Operations, leading the ESG work on an operational level. Newron’s material ESG topics and the corresponding KPI’s and reporting framework should be communicated by the end of 2022, while annual ESG reporting will commence in spring 2023 with the publication of the 2022 Annual Report.

Newron’s Half-Report 2022 is available for download on the Company’s website:
View Source

Outlook:

"Looking ahead to the next months, we remain dedicated to our mission of developing novel treatments for diseases of the central and peripheral nervous system. We also remain on track to publish full results from study 014 in QI/2023 and from study 008A in the first half of 2023. We will continue to explore commercial partnerships as well as opportunities to in-licence drugs. The total available cash resources, in addition to royalty income and Italian R&D tax credits, will fund the planned development programs and operations of our Company well into 2024," outlined Stefan Weber, CEO of Newron.

On September 15, 2022 VBI Vaccines Inc. (Nasdaq: VBIV) (VBI), a biopharmaceutical company driven by immunology in the pursuit of powerful prevention and treatment of disease, reported it has entered into a refinanced and upsized debt facility of up to $100 million with its existing lender, K2 HealthVentures (K2HV), a healthcare-focused specialty finance company (Press release, VBI Vaccines, SEP 15, 2022, View Source [SID1234619586]).

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Under the terms of the agreement, $50 million is immediately available to VBI upon closing, comprised of $30 million to refinance the outstanding facility the Company held with K2HV, and an incremental $20 million of non-dilutive funding. Future tranches of up to $25 million are committed and will be made available to VBI upon the achievement of certain clinical and financial milestones, and a further tranche of $25 million is available at the discretion of K2HV.

"Through our successful partnership with K2HV, this refinancing and access to additional capital further enables important financial flexibility as we continue our commercial launch of PreHevbrio and advance our prophylactic and therapeutic vaccine pipeline," said Jeff Baxter, VBI’s President and CEO. "The K2HV team has proven to have a deep understanding of our business and the science driving VBI, and we are grateful for their support of and investment in VBI’s future."

"We are pleased to be able to maintain and increase our support of VBI’s commercialization and pipeline development efforts, and we look forward to continuing to work with the VBI team in their mission to address significant unmet needs in infectious disease and oncology," said Anup Arora, Founding Managing Director and Chief Investment Officer of K2HV.

About PreHevbrio [Hepatitis B Vaccine (Recombinant)]

VBI’s hepatitis B vaccine is the only 3-antigen hepatitis B vaccine, comprised of the three hepatitis B surface antigens of the hepatitis B virus – S, pre-S1, and pre-S2. It is approved for use in the United States, European Union/European Economic Area, United Kingdom, and Israel. The brand names for this vaccine are: PreHevbrio (US), 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.

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).