Abeona Therapeutics Announces Participation in Upcoming Investor Conferences

On September 7, 2023 Abeona Therapeutics Inc. (Nasdaq: ABEO) reported that members of its management team will participate in upcoming investor conferences in September 2023 (Press release, Abeona Therapeutics, SEP 7, 2023, View Source [SID1234634979]):

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A company presentation at the H.C. Wainwright Annual Global Investment Conference on Wednesday, September 13, 2023 at 10:00 a.m. ET.
A fireside chat at the Cantor Global Healthcare Conference on Tuesday, September 26, 2023 at 2:10 p.m. ET.
A fireside chat at the Jefferies Cell & Genetic Medicine Summit on Wednesday, September 27, 2023 at 9:30 a.m. ET.

A live webcast of the presentation and fireside chats can be accessed on the Investors section of the Abeona website under "Events" at View Source, where a replay of the events will also be available for a limited time.

Vivoryon Therapeutics N.V. Reports H1 2023 Financial Results and Highlights Operational Progress

On September 6, 2023 Vivoryon Therapeutics N.V. (Euronext Amsterdam: VVY; NL00150002Q7) (Vivoryon), a clinical stage company focused on the discovery and development of small molecule medicines to modulate the activity and stability of pathologically altered proteins, reported financial results for the six-month period ended June 30, 2023, and provided an update on its corporate progress (Press release, Vivoryon Therapeutics, SEP 7, 2023, View Source [SID1234634951]). The report is available on the Company’s website View Source

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"We enter into the second half of 2023 with key milestones achieved and a strong cash position bringing us beyond the VIVIAD Phase 2b study read-out, an exceptionally talented, passionate team, encouraging safety results at 600 mg twice daily in the VIVIAD study of varoglutamstat and a precision recruitment strategy applied to successfully identify the right patients for VIVIAD," said Frank Weber, MD, CEO of Vivoryon. "Our achievements from the first half of this year include a successful fundraise significantly extending our cash runway and supportive independent Data Safety Monitoring Board decisions for VIVIAD and VIVA-MIND. We are laser focused on delivering VIVIAD study results in the first quarter of 2024 and monitor study progress as well as blinded safety and efficacy outcome parameters continuously. With varoglutamstat’s favorable safety profile, ease of administration, and strong signs of efficacy and synaptic improvement, we believe we are uniquely positioned to bring a highly differentiated, potentially first-in-class therapeutic option to patients with Alzheimer’s disease."

H1 2023 and Post-Period Portfolio Highlights

Varoglutamstat Clinical Program:

Varoglutamstat is a differentiated investigational small-molecule medicine in development to treat Alzheimer’s disease (AD). It is currently being investigated in two large Phase 2 studies, VIVIAD (NCT04498650) in Europe and VIVA-MIND (NCT03919162) in the U.S., where it continues to show evidence of a favorable safety profile at the therapeutic dose of 600 mg twice daily (BID), a dose demonstrated to result in a target occupancy of nearly 90%.

Varoglutamstat is designed to prevent N3pE-Abeta formation, rather than aiming to clear existing plaques, making it an intervention upstream of other approaches such as monoclonal antibodies (mAbs). Through a second mode of action, varoglutamstat also modulates neuroinflammation via the CCL2 pathway, which, in turn, has an impact on tau pathology.

VIVIAD

VIVIAD (NCT04498650) is a state-of-the-art Phase 2b study being conducted in Europe and designed to evaluate the safety, tolerability, and efficacy of varoglutamstat in 259 (final number of randomized participants) subjects with mild cognitive impairment (MCI) and mild AD.

In March 2023, Vivoryon announced an update on the clinical development of varoglutamstat, including the VIVIAD trial, at the International Conference on Alzheimer’s and Parkinson’s Diseases and related neurological disorders (AD/PD) in Gothenburg, Sweden. As of the data cut-off date of January 5, 2023, over 100 of the 259 participants randomized into the VIVIAD study had been treated for at least 48 weeks. Varoglutamstat showed, to date, no on-target toxicity and no clinical signs of brain swelling or hemorrhages (ARIA), which are a limiting class side effect of Abeta antibodies. The discontinuation rate due to adverse events in VIVIAD was considerably lower in the completed Phase 2a SAPHIR study at comparable timepoints, while retaining a similar level of target inhibition (around 90%) at the dosing in both studies.
In July 2023, Vivoryon presented a poster titled, "VIVIAD, a Phase 2b Study Investigating Varoglutamstat in Patients with MCI or Mild AD: Analysis of Baseline Cognition Data" at the Alzheimer’s Association International Conference (AAIC), in Amsterdam, the Netherlands. These data demonstrated that Vivoryon’s strategy of precisely recruiting individuals with evidence of at least minimal baseline deficits on the WAIS-IV Coding test, a well-known measure of cognitive function, successfully identifies patients with MCI, enabling a reliable assessment of potential cognitive improvement after treatment.
In July 2023, Vivoryon announced a safety update based on data from all 259 randomized patients which showed no clinical signs of varoglutamstat associated ARIA`s at the cutoff date of June 14, 2023. After carefully reviewing the updated safety data, the independent Data Safety Monitoring Board (DSMB) decided in its recent meeting on June 22, 2023, that the study should continue as planned and that no additional DSMB meeting will be required until study completion.
In July 2023, Vivoryon announced that it commenced preparations for an open-label extension (OLE) study to provide a long-term treatment option to patients after completion of treatment under the VIVIAD or VIVA-MIND protocol. The launch of the OLE study is contingent on the outcome of VIVIAD.
Vivoryon remains on track to report the final data readout from the VIVIAD study in the first quarter of 2024.
VIVA-MIND

VIVA-MIND (NCT03919162) is a complementary Phase 2 study for varoglutamstat being conducted in the U.S. which seeks to enroll 180 patients with early AD into the Phase 2a adaptive dose finding portion and to enroll a further 234 patients in the Phase 2b portion of the study.

In July 2023, Vivoryon announced that the first cohort was fully randomized into the study as planned and the study is now recruiting participants into the second cohort, with 19 sites open across the U.S. In June 2023, the study’s independent DSMB recommended to continue the study without modification, supporting the rationale for accelerated uptitration to 600 mg BID dosing.
The Company intends to provide a study update in the fourth quarter of 2023.
Corporate Development Highlights

In May 2023, Vivoryon announced the successful raise of EUR 25 million in an accelerated bookbuild offering through a private placement of 1,785,715 ordinary shares, with a nominal value of EUR 1.00 each, in the issued share capital of the Company at an issue price of EUR 14.00 per share (such shares the "New Shares"). The New Shares from the capital increase represented approximately 7.4% of Vivoryon’s existing issued share capital and were issued from the Company’s authorized capital under exclusion of the existing shareholders’ pre-emptive rights. Consequently, the Company’s issued share capital increased to EUR 25,890,993.00.
In June 2023, Vivoryon announced the appointment of Kugan Sathiyanandarajah and Professor Dr. Morten Asser Karsdal as Non-Executive members to its Board of Directors, strengthening the Board with their extensive scientific knowledge and business acumen. Both appointments were approved during Vivoryon’s Annual General Meeting which took place on June 21, 2023. All voting items were passed with a majority.
In August 2023, Vivoryon announced that the Board identified long-standing member of Vivoryon’s management team and Chief Medical Officer (CMO), Frank Weber, MD, as the optimal candidate to assume the responsibilities of CEO, effective August 14, 2023. Additionally, the Board proposed a newly created position, Chief Strategy & Investor Relations Officer (CS&IRO), which was assumed by Head of Investor Relations, Anne Doering, CFA. Vivoryon will hold an Extraordinary General Meeting (EGM) on Friday, September 15, 2023, related to their appointments as new members of Vivoryon’s Board of Directors. The appointments follow the decision of Chief Executive Officer (CEO), Ulrich Dauer, PhD, to step down from his position following his notification to the Board to not renew his contract, previously announced on June 15, 2023. Dr. Dauer will support the Company through the transition period in an advisory role through the end of 2023.
In August 2023, Vivoryon and Scenic Biotech B.V. ("Scenic") reached an agreement regarding the settlement of their patent dispute. In 2019, Vivoryon had initiated proceedings on the merits with the District Court of The Hague against Scenic, Stichting Het Nederlands Kanker Instituut-Antoni van Leeuwenhoek Ziekenhuis and Academisch Ziekenhuis Leiden h.o.d.n LUMC, in connection with certain of Vivoryon´s patents related to varoglutamstat (PQ912) and certain other QPCT inhibitors. As part of the settlement, Scenic’s affiliate, Scenic Immunology B.V., and Vivoryon have entered into a patent license agreement, under which Scenic Immunology B.V. granted to Vivoryon certain rights to certain patents controlled by Scenic Immunology B.V. in the field of oncology.
Financial Results for the First Half Year 2023

No revenues were generated in the first half year of 2023 or the first half year of 2022.

Research and development expenses of EUR 6.3 million in the six months ended June 30, 2023, decreased by EUR 4.8 million compared to the six months ended June 30, 2022. This decrease is primarily attributable to EUR 2.5 million lower expenses related to our clinical trial VIVIAD and EUR 2.2 lower manufacturing cost for study drug production.

General and administrative expenses of EUR 4.4 million for the six months ended June 30, 2023, increased by EUR 2.1 million from EUR 2.3 million in the six months ended June 30, 2022. EUR 1.3 million of the increase is attributable to higher costs for the Non-Executive Board members, including share-based payments and compensation of EUR 0.9 million and one-time severance payments of EUR 0.4 million. Furthermore, an increase of EUR 0.8 million was largely due to higher consulting and personnel costs.

Net loss of EUR 10.7 million for the six months ended June 30, 2023, compares to EUR 12.6 million for the six months ended June 30, 2022.

The Company held EUR 29.6 million in cash and cash equivalents as of June 30, 2023, compared to EUR 26.6 million as of December 31, 2022. Additionally, in the six months ended June 30, 2023, the Company entered into Euro term deposits of EUR 9.0 million resulting in a reclassification of these funds in the balance sheet into financial assets.

Cash flows used in operating activities were EUR 20.3 million for the six months ended June 30, 2023, compared to EUR 10.2 million in the six months ended June 30, 2022. The change in operating cash flow by EUR (10.0) million mainly results from the reclassification of new term deposits with a term of more than three months of EUR (9.0) million that are disclosed in the Company`s financial assets and not in cash equivalents as well as other changes in working capital.

Cash flows provided from financing activities were EUR 23.4 million for the six months ended June 30, 2023, compared to EUR 19.6 million in the six months ended June 30, 2022. The cash flows from financing mainly result from a private placement on May 26, 2023, placing 1,785,715 registered shares at an offering price of EUR 14.00 per share with gross proceeds of EUR 25 million. The Company’s issued share capital has increased to EUR 25,961,892, including the exercise of share options.

Financial Guidance

Including the proceeds from the capital raise completed in May 2023, according to current planning and estimates, Vivoryon expects that its existing cash and cash equivalents will be sufficient to fund its research and development expenses, as well the general and administrative expenses and cash flows from investing and financing activities into the second half of 2024. This guidance does not include potential milestone payments from development partnerships, potential payments from licensing agreements and/or additional financing measures, as exercise of the options granted in connection with the private placement announced September 30, 2022 (see note 8.11 of the Company’s annual financial statements for the year ended December 31, 2022).

Upcoming Investor and Analyst Events

Vivoryon will hold an Extraordinary General Meeting (EGM) Friday, September 15, 2023, at 1:00 p.m. (CEST) related to the appointments of Frank Weber, MD, and Anne Doering, CFA, as new members of Vivoryon’s Board of Directors. The full agenda and all relevant documents for the upcoming EGM are available on the Company’s website (View Source)
Vivoryon will host a virtual R&D Day with Key Opinion Leaders (KOLs) in Q4/2023 focused on, the Company’s scientific approach, varoglutamstat and study design.
Conference Call and Webcast

Vivoryon will host a conference call and webcast today, September 7, 2023, at 3:00 pm CEST (9:00 am EDT). A Q&A session will follow the presentation of the first half 2023 financial results.

A live webcast and slides will be made available at: www.vivoryon.com/investors-news/news-and-events/presentations-webcasts/

To join the conference call via phone, participants may pre-register and will receive dedicated dial-in details to easily and quickly access the call via the following website: https://register.vevent.com/register/BI37d47846ffb6452eaad13f500e1acec2

It is suggested participants dial into the conference call 15 minutes prior to the scheduled start time to avoid any delays in attendance.

Approximately one day after the call, a slide-synchronized audio replay of the conference will be available on: www.vivoryon.com/investors-news/news-and-events/presentations-webcasts/

Repare Therapeutics to Participate at the Morgan Stanley 21st Annual Global Healthcare Conference

On September 6, 2023 Repare Therapeutics Inc. ("Repare" or the "Company") (Nasdaq: RPTX), a leading clinical-stage precision oncology company, reported that members of its senior management team will participate in a fireside chat at the Morgan Stanley 21st Annual Global Healthcare Conference on Wednesday, September 13, 2023, at 11:30 a.m. Eastern Time in New York, NY (Press release, Repare Therapeutics, SEP 6, 2023, View Source [SID1234634970]).

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A live webcast of the fireside chat can be accessed in the Investor section of the Company’s website at View Source A replay of the webcast will be archived on the Company’s website for 90 days.

KSQ Therapeutics and CTMC Announce Strategic Collaboration to Accelerate the Development of Novel Engineered Tumor Infiltrating Lymphocyte (eTIL®) Therapies for the Treatment of Solid Tumors

On September 6, 2023 KSQ Therapeutics, Inc. ("KSQ"), a clinical-stage biotechnology company developing cancer therapies using its proprietary CRISPRomics discovery platform, and CTMC, a joint venture between National Resilience, Inc. and MD Anderson Cancer Center, reported a strategic collaboration to expedite the development of KSQ’s two lead engineered tumor-infiltrating lymphocyte (eTIL) programs, KSQ-001EX and KSQ-004EX, both of which are advancing toward clinical studies for the treatment of solid tumors (Press release, KSQ Therapeutics, SEP 6, 2023, View Source [SID1234634969]).

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"Our eTIL programs – which edit the SOCS1 and Regnase-1 genes – have the potential to be first- and best-in-class cell therapies for cancer treatment. As our eTIL programs move through IND-enabling studies, our partnership with CTMC will have us ready to manufacture KSQ-001EX and KSQ-004EX for clinical studies," said Qasim Rizvi, Chief Executive Officer of KSQ. "We believe our eTIL programs have the potential to address the significant unmet need in the solid tumor space."

"TIL therapies have tremendous potential for the treatment of solid tumors as they are naturally selected to target a host of tumor antigens specific to the patient. However, repeated antigen exposure in an immunosuppressive tumor environment has driven T-cells to a dysfunctional status. We believe the key to unlocking their full potential resides in the engineering of TIL therapies to enable maximal function within the tumor microenvironment," said Jason Bock, CEO of CTMC. "This is where KSQ’s approach of identifying the optimal gene targets to boost TIL function fits nicely. We’re thrilled to collaborate with them to bring these valuable therapies to patients."

KSQ-001EX and KSQ-004EX

KSQ-001EX, a single-edit eTIL that deletes the SOCS1 gene, and KSQ-004EX, a dual-edit eTIL that deletes both the SOCS1 and Regnase-1 genes, are KSQ’s lead eTIL cell therapy programs, which have the potential to revolutionize the treatment of solid tumors. These potential first-in-class cell therapy programs were identified through our CRISPRomics platform as the optimal gene targets that drive T cell function, and we believe we can supercharge TIL therapies for potency and persistence by genetically modifying and optimizing them. In preclinical models, these eTIL therapies have demonstrated: enhanced potency and anti-tumor function, including in PD-1 refractory solid tumors; enhanced persistence and memory formation of T cells; and the opportunity to reduce or eliminate the need for lymphodepletion and IL-2 conditioning.

Apollo Therapeutics Closes $226.5 Million Series C Financing Led by Patient Square Capital

On September 6, 2023 Apollo Therapeutics ("Apollo" or "the Company"), a portfolio biopharmaceutical company focused on translating fundamental medical research into medicines through a hub-and-spoke approach, reported the close of $226.5 million in a Series C financing led by lead investor Patient Square Capital (Press release, Apollo Therapeutics, SEP 6, 2023, View Source [SID1234634968]).

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$226.5m million raised.
Financing led by Patient Square Capital.
Participation by multiple new investors including M&G plc and two of the largest US public pension plans, along with existing investor Rock Springs Capital.
Financing significantly adds to the $195 million previously raised by the Company.
Apollo’s broad and diversified pipeline currently consists of over 20 programs with multiple near and medium-term value catalysts and uncorrelated risk.
Proceeds will be used to (i) advance pipeline programs through clinical development and further fund Apollo’s drug discovery and development activities based on breakthroughs in basic medical research made at its partner institutions, (ii) licence or acquire additional clinical stage programs that meet strict selection criteria.
Apollo has a scalable R&D platform for the evergreen discovery and development of new medicines. Program selection is agnostic to indication and primarily driven by quality of science and the potential to transform the standard of care in major commercial markets. This is enabled by an unprecedented level of access to world-class research institutions through unique partnership agreements.
Current partner institutions are: The University of Cambridge, Imperial College London, University College London, King’s College London, and The Institute of Cancer Research.
"We are delighted to announce the close of our latest fundraise and wish to thank our existing investors for their continued support, as well as warmly welcoming all the new investors." said Dr. Richard Mason, Chief Executive Officer of Apollo Therapeutics. "Apollo has built a broad and diversified pipeline focused on major commercial markets and this latest fund-raising enables us to take our most advanced programs to key clinical value inflection points."

"I have long believed in a business model for drug development characterized by extremely talented management, sufficient capital to pursue multiple programs and world-class science focused on unmet medical need. We are proud to support Apollo Therapeutics as it develops a number of promising medicines that we anticipate will eventually transform patient care." commented Jim Momtazee, Managing Partner of Patient Square Capital, and member of Apollo Therapeutic’s Board of Directors.