On August 8, 2024 Biohaven Ltd. (NYSE: BHVN) (Biohaven or the Company), a global clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of life-changing therapies to treat a broad range of rare and common diseases, reported financial results for the second quarter ended June 30, 2024, and provided a review of recent accomplishments and anticipated upcoming developments (Press release, Biohaven Pharmaceutical, AUG 8, 2024, View Source [SID1234645645]).

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Vlad Coric, M.D., Chairman and Chief Executive Officer of Biohaven, commented, "Last quarter, we showcased an array of exciting updates across our pipeline spanning immunology, neuroscience, obesity, and oncology at our annual R&D Day. First, it has been incredible working with Yale Professor David Spiegel and my colleagues at Biohaven to drive the advancement of groundbreaking degrader science represented by BHV-1300 using our Molecular Degrader of Extracellular Protein (MoDE) technology — we consider our initial Phase 1 data to be paradigm-shifting and we are accelerating our entire platform based upon these early results. BHV-1300 has been well-tolerated with no significant adverse effects and no clinically significant lab abnormalities or ECG changes observed to date and emerging subcutaneous data from our Phase 1 study highlights the ease of use of this technology. The platform represents a brand-new technology that has potential to treat diseases like Type 1 diabetes, autoimmune dilated cardiomyopathy, IgA nephropathy, and many others. Our differentiation continues to focus on convenient SC patient self-administration, rapid onset of action, potential for deeper IgG reduction and concurrent use with Fc containing standard of care biologics. We are excited that we remain on timelines with multiple INDs from our MoDE platform for later this year, including drug candidates that target autoantibodies against β-1AR for the potential treatment of dilated cardiomyopathy, galactose deficient IgA for IgA nephropathy and a further optimized IgG degrader for use in rare diseases."

Dr. Coric continued, "While our degrader program continues to garner significant focus, our broader pipeline is increasingly derisked with several potentially paradigm-shifting treatments for conditions including epilepsy, mood disorders, pain, immunology, rare diseases, obesity and neurodegenerative disorders. Starting with our ion channel platform; five studies with Kv7 activator BHV-7000 (three in epilepsy and two in major depressive disorder and bipolar) are underway with brisk patient enrollment. In addition, our TRPM3 antagonist BHV-2100 has completed its initial Phase 1 SAD/MAD study, attaining an exceptional PK/PD profile for the treatment of migraine and other pain disorders. All doses being advanced have reached the targeted EC90 for this molecule with excellent preliminary safety. We are excited about being back in the migraine space with this novel target and will initiate a Phase 2 study in migraine before year end, as well as initiate a POC study as a non-opiate pain treatment. With BHV-8000, our oral and brain penetrant TYK2/JAK1 inhibitor, we have aligned with the FDA on two novel study designs: one for the prevention of ARIA associated with amyloid lowering agents in Alzheimer’s disease and another with the goal of slowing disease progression in early Parkinson’s disease. The Phase 1 profile of BHV-8000 is invigorating our translational team as we have data confirming both CNS penetration and biomarker engagement of inflammatory targets in humans. Regarding our anti-myostatin taldefgrobep alfa, we shared new preclinical data demonstrating improved, durable weight reduction and lean mass preservation in combo with GLP-1 semaglutide, which was particularly noteworthy, given the muscle wasting limitations that have plagued the GLP-1 class. We also showed that taldefgrobep has direct effects on modulating fat in addition to increasing muscle mass. Our oncology platform has also emerged into the clinic with our lead Trop-2 ADC, BHV-1510, enrolling cancer patients and our efforts advancing a portfolio of several Biohaven ADCs. We are excited to get preliminary safety and efficacy data from cancer patients suffering with select advanced or metastatic epithelial tumors in the Phase 1/2 study of BHV-1510 and will be evaluating the combination of BHV-1510 with Regeneron’s anti-PD1 Libtayo — combination dosing with BHV-1510 and Libtayo is anticipated to begin in 2H 2024. Finally, with our glutamate platform represented by troriluzole, topline data from an interim analysis of the second OCD trial is expected in 2H 2024 and our first OCD trial is expected to read out in 1H 2025. Separately, we continue to have constructive dialogue with the FDA regarding our SCA development program and our European Medicines Agency application for SCA3 remains under review. We now expect topline data from a new RWE protocol assessing the efficacy of troriluzole in SCA patients treated for up to 3 years— this includes new patient data that has not previously been available. I never cease to be amazed by the power of our small but mighty Biohaven R&D team that has innovated across each of these important medical areas. We are driven to fulfill a promise to patients and our investors that we will diligently evaluate and advance potentially transformative therapies to those suffering from disease."

Second Quarter 2024 and Recent Business Highlights

Additional data supports the paradigm-shifting potential of the MoDE Platform for the treatment of immunological and inflammatory disorders – In May 2024, the Company disclosed positive new data from its ongoing Phase 1 SAD study in healthy subjects with BHV-1300, a first-in-human IgG degrader from its innovative MoDE platform. Results from the initial dose cohorts in the SAD study confirmed that BHV-1300 rapidly and selectively lowers IgG in a dose-dependent manner. Emerging Phase 1 data also showed that our novel SC formulation of BHV-1300 delivered exposures higher than the intravenous formulation, enabling the profile of a convenient patient administered auto-injector to attain targeted reduction of IgG. The Company expects to provide an update from our SAD and MAD studies of BHV-1300 in the second half of 2024 and is on schedule to file multiple new MoDE INDs this year. FDA granted INTERACT meeting request for BHV-1600, β1AR autoantibody MoDE, development program.
Ion Channel Platform: Kv7 activator and TRPM3 antagonist candidates represent transformational targets in neurology and neuropsychiatry – The Company is enrolling participants in five ongoing pivotal clinical trials with Kv7 activator, BHV-7000, targeting focal epilepsy, generalized epilepsy, bipolar disorder and major depressive disorder; separately, Phase 2 and POC studies are planned in migraine and pain, respectively, with the TRPM3 antagonist, BHV-2100, in 2H 2024.
Committed to improving muscle health with Myostatin Platform – In May 2024, the Company presented new preclinical data showing that administration of taldefgrobep alfa directly reduced the increased adipose fat storage caused by myostatin; the Company also presented new preclinical data from a diet-induced obesity mouse model showing treatment with taldefgrobep alfa together with a GLP-1 agonist produced greater reductions in body weight and fat mass, and a larger increase in lean muscle mass, compared to treatment with GLP-1 alone. The Company plans to initiate a Phase 2 study in obesity in 2H2024. Separately, the Company continues to expect Phase 3 study topline results in SMA in 2H 2024.
Selectively targeting the immune system to treat neurodegenerative diseases with neuroinflammation platform – In May 2024, the Company reported positive results from the Phase 1 SAD and MAD study with our brain-penetrant TYK2/JAK1 inhibitor, BHV-8000 in healthy subjects, including demonstration of CSF target exposures and biomarker engagement along with a well-tolerated safety profile. The Company also announced key regulatory updates, including the successful completion of two FDA meetings with favorable feedback enabling registrational programs for Parkinson’s disease and for the prevention of ARIA, a novel indication.
Oncology Platform: Antibody Drug Conjugate portfolio: positioned to achieve enhanced stability and improved efficacy – In May 2024, the Company announced that its novel Trop-2 ADC, BHV-1510, entered into the clinic for patients with advanced or metastatic epithelial tumors; the first patient was dosed in a Phase 1/2 clinical trial as monotherapy. Biohaven also entered into a clinical supply agreement with Regeneron to study the combination of BHV-1510 with Regeneron’s anti-PD1 Libtayo (cemiplimab-rwlc) in the clinical study.
Expected Upcoming Milestones:

We believe Biohaven is well positioned to achieve significant milestones in 2024 and 2025 across numerous programs:

Selective Kv7 Activator:

Continue 5 ongoing Phase 2/3 trials with BHV-7000 in focal epilepsy, idiopathic generalized epilepsy, MDD, and bipolar disorder
Troriluzole:

Topline results from a new RWE protocol of 3-year data from troriluzole treated patients in SCA in 2H 2024
Two Phase 3 trials with troriluzole in OCD: conduct interim analysis of the second Phase 3 OCD trial in 2H 2024 and report topline data from first Phase 3 OCD trial in 1H 2025
Taldefgrobep alfa:

Initiate Phase 2 trial with taldefgrobep in obesity in 2H 2024
Report topline data from Phase 3 trial with taldefgrobep in SMA in 2H 2024
First-in-class TRPM3 Antagonist:

Initiate Phase 2 trial with BHV-2100 in acute migraine in 2H 2024
Initiate POC study with BHV-2100 for pain in 2H 2024
TYK2/JAK1 Inhibitor:

Complete SAD/MAD studies with BHV-8000 and advance to Phase 2 in the coming months
MoDE Platform

Submit a total of 4 INDs in 2024
Continue to advance Phase 1 SAD and MAD studies with BHV-1300, with a further study update in 2H 2024
INTERACT meeting with FDA regarding BHV-1600, β1AR autoantibody degrader, in 2H 2024
Next Generation ADC Platform:

Advance Phase 1 Trop-2 directed program BHV-1510 into a Phase 1 study in multiple tumor types
Capital Position:

Cash, cash equivalents, marketable securities and restricted cash totaled approximately $440 million on June 30, 2024.

Second Quarter 2024 Financial Highlights:

Research and Development (R&D) Expenses: R&D expenses, including non-cash share-based compensation costs, were $314.8 million for the three months ended June 30, 2024, compared to $79.5 million for the three months ended June 30, 2023. The increase was primarily due to a one-time, non-cash expense of $171.9 million paid to Knopp Biosciences, LLC (Knopp) for a milestone and royalty buyback related to our BHV-7000 and broader Kv7 platform that was recognized during the three months ended June 30, 2024 (the buyback reduced our potential future milestone payments by $867.5 million, and replaced the scaled high single digit to low teens royalty payment obligations with a flat royalty payment in the mid-single digits for the Kv7 programs). The increase in R&D expenses was also due to advancing our 6 clinical platforms including 5 Phase 3 starts for BHV-7000, follow-on Kv7 assets, preclinical research programs, and increases in direct program spend for additional and advancing multiple clinical development programs. Non-cash share-based compensation expense was $7.1 million for the three months ended June 30, 2024, an increase of $4.6 million as compared to the same period in 2023. Non-cash share-based compensation expense was higher in the second quarter of 2024, primarily due to our annual equity incentive awards granted in the fourth quarter of 2023 and the first quarter of 2024.

General and Administrative (G&A) Expenses: General and administrative expenses were $19.0 million for the three months ended June 30, 2024, compared to $14.5 million for the three months ended June 30, 2023. The increase of $4.4 million was primarily due to increased non-cash share-based compensation expense, which was $5.2 million for the three months ended June 30, 2024, an increase of $2.9 million as compared to the same period in 2023. Non-cash share-based compensation expense was higher in the second quarter of 2024 primarily due to our annual equity incentive awards granted in the fourth quarter of 2023 and the first quarter of 2024.

Other Income, Net: Other income, net was a net other income of $14.2 million for the three months ended June 30, 2024, compared to a net other income of $5.8 million for the three months ended June 30, 2023. The increase of $8.3 million was primarily due to a $9.2 million gain recorded upon the settlement of our forward contract liability for share consideration issued under the amendment entered into with Knopp in May 2024, and increased investment income, partially offset by a decrease in other income recognized during the three months ended June 30, 2024, as compared to the same period in 2023 related to the Transition Services Agreement entered into with Biohaven Pharmaceutical Holding Company Ltd. (the Former Parent).

Net Loss: Biohaven reported a net loss for the three months ended June 30, 2024 of $319.8 million, or $3.64 per share, compared to $90.3 million, or $1.32 per share, for the same period in 2023. Non-GAAP adjusted net loss for the three months ended June 30, 2024 was $307.4 million, or $3.50 per share, compared to $85.7 million, or $1.25 per share for the same period in 2023. These non-GAAP adjusted net loss and non-GAAP adjusted net loss per share measures, more fully described below under "Non-GAAP Financial Measures," exclude non-cash share-based compensation charges and losses from the change in fair value of derivatives. A reconciliation of the GAAP financial results to non-GAAP financial results is included in the tables below.

On August 8, 2024 Arcus Biosciences, Inc. (NYSE:RCUS), a clinical-stage, global biopharmaceutical company focused on developing differentiated molecules and combination therapies for people with cancer, reported financial results for the second quarter ended June 30, 2024, and provided a pipeline update on its clinical-stage investigational molecules – targeting TIGIT, the adenosine axis (CD73 and A2a/A2b receptors), HIF-2a, AXL and PD-1 – across multiple common cancers (Press release, Arcus Biosciences, AUG 8, 2024, View Source [SID1234645586]).

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"Our upcoming presentation of efficacy and safety data for casdatifan will demonstrate that it has the potential to be the best-in-class HIF-2a inhibitor," said Terry Rosen, Ph.D., chief executive officer of Arcus. "We are pursuing a broad development program in both first- and second-line settings, as well as differentiated combinations, to maximize the opportunity for casdatifan in ccRCC. Meanwhile, the accumulating data continue to enhance our confidence that our Fc-silent anti-TIGIT antibody, domvanalimab, has the potential for an improved safety profile over that of Fc-enabled antibodies, particularly when combined with chemotherapy, which may also result in an efficacy advantage for domvanalimab. With STAR-221 enrollment completed, we are looking forward to our first Phase 3 data readout."
Corporate Updates:
•In July 2024, Taiho Pharmaceutical (Taiho) exercised its option for quemliclustat, an investigational small molecule CD73 inhibitor, in Japan and certain other territories in Asia (excluding mainland China). As a result of this option exercise, Taiho will operationalize the Phase 3 PRISM-1 study evaluating quemliclustat in pancreatic cancer in Japan, and Arcus will receive an opt-in payment and is eligible to receive near-term milestone payments.
Pipeline Highlights:
Casdatifan (HIF-2a inhibitor)
•Multiple expansion cohorts evaluating casdatifan in clear cell renal cell carcinoma (ccRCC) are underway, with several data presentations expected in the next 18 months. Each cohort is enrolling approximately 30 patients.
◦ARC-20: Phase 1/1b study evaluating casdatifan as a monotherapy and in combination with other agents:
▪100 mg daily expansion cohort in 2L+ ccRCC: ORR data are expected to be presented in the fourth quarter of 2024.
▪50 mg and 150 mg expansion cohorts in 2L+ ccRCC: Enrollment has been completed for both cohorts and data are expected to be presented in 2025.
▪An expansion cohort evaluating casdatifan in combination with cabozantinib in 2L+ ccRCC is also enrolling.
•Following FDA feedback later this year, Arcus plans to initiate its first Phase 3 study, PEAK-1, evaluating casdatifan in combination with cabozantinib versus cabozantinib monotherapy in patients with metastatic ccRCC who have previously received anti-PD-1 therapy, in the first half of 2025.

•Arcus is in advanced stages of planning with a clinical collaboration partner to evaluate casdatifan in a potential first-in-class combination regimen for first-line metastatic ccRCC.
Domvanalimab (Fc-silent anti-TIGIT antibody) plus Zimberelimab (anti-PD-1 antibody)
Domvanalimab-Zimberelimab Updates:
•Updated data presented at the ASCO (Free ASCO Whitepaper) Annual Meeting from Arm A1 of the Phase 2 EDGE-Gastric study showed 12.9 months median progression-free survival (PFS) for domvanalimab plus zimberelimab and chemotherapy in first-line upper GI adenocarcinomas, which exceeded historical benchmarks for anti-PD-1 plus chemotherapy.
◦The EDGE-Gastric study is evaluating the same regimen in the same setting as the STAR-221 Phase 3 study.
•STAR-221, a Phase 3 study evaluating domvanalimab plus zimberelimab and chemotherapy in PD-L1 all-comer first-line metastatic upper GI adenocarcinomas, completed enrollment in June.
•STAR-121, a Phase 3 study evaluating domvanalimab plus zimberelimab and chemotherapy in PD-L1 all-comer first-line metastatic non-small cell lung cancer (NSCLC), is expected to complete enrollment in 2024.
Upcoming Domvanalimab-Zimberelimab Milestones:
•Overall survival (OS) and PFS data from previously enrolled patients in Part 1 of the Phase 3 ARC-10 study, evaluating domvanalimab plus zimberelimab versus zimberelimab versus chemotherapy in first-line PD-L1-high NSCLC, are expected to be presented by the end of 2024.
•OS data from the Phase 2 EDGE-Gastric study, evaluating domvanalimab plus zimberelimab and chemotherapy in upper GI adenocarcinomas, are expected to be presented in 2025.
CD73-Adenosine Axis: Etrumadenant (A2a/A2b receptor antagonist) and Quemliclustat (small-molecule CD73 inhibitor)
Etrumadenant
•Cohort B data from ARC-9, a randomized Phase 1b/2 study evaluating etrumadenant plus zimberelimab, FOLFOX chemotherapy and bevacizumab (EZFB) versus regorafenib in third-line metastatic colorectal cancer (mCRC), were presented at ASCO (Free ASCO Whitepaper) in June.
◦Results showed 19.7 months median OS for the EZFB arm, and EZFB significantly reduced the risk of death by 63% and risk of disease progression by 73% compared to regorafenib. This is the longest median OS reported in third-line mCRC to date in a randomized trial.
◦Biomarker data from this study are expected to be presented at a scientific conference in the second half of 2024.
•Based on these encouraging results, Arcus and Gilead are determining next steps for the development of etrumadenant in mCRC.
Quemliclustat
•Initiation of a Phase 3 trial, PRISM-1, of quemliclustat combined with gemcitabine/nab-paclitaxel versus gemcitabine/nab-paclitaxel in pancreatic cancer is expected to begin by early 2025.
•Taiho exercised its option for an exclusive license to quemliclustat in Japan and certain territories in Asia and will operationalize PRISM-1 in Japan.
Early Clinical Programs
•Dose escalation for AB801, a potent and highly selective small-molecule AXL inhibitor, continues. Arcus anticipates advancing this molecule into expansion cohorts in NSCLC in early 2025.
Financial Results for Second Quarter 2024:
•Cash, Cash Equivalents and Marketable Securities were $1.0 billion as of June 30, 2024, compared to $866 million as of December 31, 2023. The increase during the period is primarily due to the receipt of $320 million in cash from Gilead for their January 2024 equity investment, partially offset by the use of cash in research and development activities. We believe our cash, cash equivalents and marketable securities on-hand will be sufficient to fund operations into 2027. Cash, cash equivalents and marketable securities are expected to be between $885 million and $925 million at the end of 2024.

•Revenues were $39 million for the second quarter 2024, compared to $29 million for the same period in 2023. In the second quarter 2024, Arcus recognized $28 million in license and development services revenue related to the advancement of programs, as well as $11 million in other collaboration revenue primarily related to Gilead’s ongoing rights to access Arcus’s research and development pipeline in accordance with the Gilead collaboration agreement.
•Research and Development (R&D) Expenses were $115 million for the second quarter 2024, compared to $84 million for the same period in 2023. The net increase of $31 million was primarily driven by higher clinical trial and headcount-related costs associated with our late-stage development program activities. Non-cash stock-based compensation expense was $10 million for the second quarter 2024, compared to $9 million for the same period in 2023. For the second quarter 2024 and 2023, Arcus recognized gross reimbursements of $40 million and $44 million, respectively, for shared expenses from its collaborations, primarily the Gilead collaboration. R&D expense by quarter may fluctuate due to the timing of clinical manufacturing and standard-of-care therapeutic purchases with a corresponding impact on reimbursements.
•General and Administrative (G&A) Expenses were $30 million for the second quarter 2024, compared to $28 million for the same period in 2023. The increase was primarily driven by higher headcount and costs incurred to obtain the Third Gilead Agreement Amendment. Non-cash stock-based compensation expense was $10 million for the second quarter 2024, compared to $9 million for the same period in 2023.
•Net Loss was $93 million for the second quarter 2024, compared to $75 million for the same period in 2023.

Conference Call Information:
Arcus will host a conference call and webcast today, August 8, at 2:00 PM PT / 5:00 PM ET to discuss its second-quarter 2024 financial results and pipeline updates. To access the call, please dial (404) 975-4839 (local) or (833) 470-1428 (toll-free), using Access Code: 287576. To access the live webcast and accompanying slide presentation, please visit the "Investors & Media" section of the Arcus Biosciences website at www.arcusbio.com. A replay of the webcast will be available following the live event.

Arcus Ongoing and Announced Clinical Studies:
Trial Name Arms Setting Status NCT No.
Lung Cancer
STAR-121
dom + zim + chemo vs. pembro + chemo 1L NSCLC (PD-L1 all-comers) Ongoing Registrational Phase 3 NCT05502237
PACIFIC-8
dom + durva vs. durva Unresectable Stage 3 NSCLC Ongoing Registrational Phase 3 NCT05211895
STAR-131 dom + zim + chemo; dom + zim Perioperative NSCLC Registrational Phase 3 In Planning TBD
ARC-7 zim vs. dom + zim vs. etruma + dom + zim 1L NSCLC (PD-L1 ≥ 50%) Ongoing Randomized Phase 2 NCT04262856
EDGE-Lung dom +/- zim +/- quemli +/- chemo 1L/2L NSCLC (lung cancer platform study) Ongoing Randomized Phase 2 NCT05676931
VELOCITY-Lung
dom +/- zim +/- etruma +/- sacituzumab govitecan-hziy or other combos 1L/2L NSCLC (lung cancer platform study) Ongoing Randomized Phase 2 NCT05633667
Upper Gastrointestinal Cancers
STAR-221 dom + zim + chemo vs. nivo + chemo 1L Gastric, GEJ and EAC Ongoing Registrational Phase 3 NCT05568095
EDGE-Gastric (ARC-21) dom +/- zim +/- quemli +/- chemo 1L/2L Upper GI Malignancies
Ongoing
Randomized Phase 2
NCT05329766
Colorectal Cancer
ARC-9 etruma + zim + mFOLFOX vs. SOC 2L/3L/3L+ CRC Ongoing
Randomized Phase 2
NCT04660812
Pancreatic Cancer
PRISM-1 quemli + gem/nab-pac vs. gem/nab-pac 1L PDAC Planned Phase 3 TBD
ARC-8 quemli + zim + gem/nab-pac vs. quemli + gem/nab-pac 1L, 2L PDAC Ongoing Randomized Phase 1/1b NCT04104672
Kidney Cancer
PEAK-1 cas + cabo vs. cabo Post-IO ccRCC Planned Phase 3 TBD
STELLAR-009 cas + zanza ccRCC Ongoing Phase 1b/2 NCT06191796
ARC-20 cas, cas + cabo Cancer Patients / ccRCC Ongoing Phase 1/1b NCT05536141
Other
ARC-25 AB598 Advanced Malignancies Ongoing NCT05891171
ARC-27 AB801 Advanced Malignancies Ongoing NCT06120075

cabo: cabozantinib; cas: casdatifan; dom: domvanalimab; durva: durvalumab; etruma: etrumadenant; gem/nab-pac: gemcitabine/nab-paclitaxel; nivo: nivolumab; pembro: pembrolizumab; quemli: quemliclustat; SOC: standard of care; zanza: zanzalintinib; zim: zimberelimab; ccRCC: clear cell renal cell carcinoma; CRC: colorectal cancer; EAC: esophageal adenocarcinoma; GEJ: gastroesophageal junction; GI: gastrointestinal; NSCLC: non-small cell lung cancer; PDAC: pancreatic ductal adenocarcinoma

On August 8, 2024 HOOKIPA Pharma Inc. (NASDAQ: HOOK, ‘HOOKIPA’), a company developing a new class of immunotherapeutics based on its proprietary arenavirus platform, reported financial results and recent business highlights for the second quarter of 2024 (Press release, Hookipa Biotech, AUG 8, 2024, View Source [SID1234645602]).

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"It is an honor to be appointed CEO of HOOKIPA at such an important time for the Company. I am optimistic about the Company’s prospects, based on the strength of the pipeline, early clinical data, and the experience and dedication of the accomplished team we have in place," said Malte Peters, Chief Executive Officer of HOOKIPA. "Our best-in-class Phase 2 data that were presented at ASCO (Free ASCO Whitepaper) has generated significant momentum among investigators and this has enhanced the pace of enrollment in our ongoing Phase 2 study. In the meantime, preparations are well underway for our AVALON-1 pivotal adaptive Phase 2/3 trial of eseba-vec, expected to be initiated in the fourth quarter of this year."

"We have important work ahead of us in the second half of this year," added Terry Coelho, Executive Vice President and Chief Financial Officer of HOOKIPA. "We are keenly focused on clinical execution and operational excellence, and I look forward to working closely with Malte to explore opportunities to ensure that we are sufficiently capitalized to reach these goals."

Anticipated Catalysts

Oncology

Eseba-vec (HPV16+ OPSCC): AVALON-1 pivotal study start (Q4 2024)
HB-700 (KRAS): Partnering and collaborations under evaluation
Infectious Disease: Partnered with Gilead

HB-400 (HBV): Complete Phase 1b enrollment and initiate Phase 2 study (timing to be determined by Gilead)
HBV-500 (HIV): Actively enrolling Phase 1b trial
Business Highlights and Recent Developments

Oncology

Eseba-vec: HOOKIPA is on track to start a seamless pivotal Phase 2/3 trial of eseba-vec in combination with pembrolizumab for the treatment of patients with Human Papillomavirus 16-positive (HPV16+) recurrent/metastatic PD-L1 CPS ≥ 20 OPSCC in the first line setting.
The AVALON-1 Phase 2/3 trial design and protocol has been aligned with the FDA with a path to potential accelerated approval.
The Company anticipates the trial will start in the fourth quarter of 2024.
Updated data from a Phase 2 trial of eseba-vec in combination with pembrolizumab were reported at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2024 Annual Meeting. The data strongly validate the Company’s clinical development plan.
HB-700: The HB-700 program is a novel arenaviral immunotherapy for KRAS-mutated cancers, including the five mutations that are the primary causes of lung, pancreatic and colon cancers. The Company received clearance from the FDA for its IND application for HB-700 for the treatment of KRAS-mutated cancers. Effective April 25, 2024, HOOKIPA regained full control of the associated intellectual property portfolio and has full collaboration and licensing rights for this program.
Preclinical data published at the ASCO (Free ASCO Whitepaper) 2024 Annual Meeting demonstrated that HB-700 was well tolerated and induced KRAS mutation specific T cell responses in HLA transgenic mice.
Infectious Disease

HB-400: HB-400 is an investigational therapeutic vaccine for the treatment of chronic hepatitis B (HBV) and is currently being evaluated in a Phase 1 trial. HB-400 is one of two independent development programs in HOOKIPA’s collaboration and license agreement with Gilead. Gilead is solely responsible for further development and commercialization of the HBV product candidate.
HB-500: HB-500 is an investigational therapeutic vaccine for the treatment of HIV, also partnered with Gilead. On July 1, 2024, HOOKIPA dosed the first eligible person living with HIV in the Phase 1b clinical trial of HB-500.
Under the collaboration agreement with Gilead, HOOKIPA received a $5 million milestone payment associated with the first dosing for this trial.
HOOKIPA and Gilead also published two peer-reviewed preclinical research papers on HB-500 during the quarter:
In articles published in the Journal of Virology, and Vaccines, research demonstrated robust immunogenicity in non-human primates driven by administration of HB-500. The research also demonstrated the potential of immunogenicity of HB-500 to be enhanced when combined with a Flt3L-Fc fusion protein.
These findings show the potential that HB-500 can be a critical component of a curative treatment for HIV.
Corporate and Financial Updates

Corporate Highlights

Reverse Split: On July 9, 2024, the Company effected a reverse stock split of the outstanding shares of its common stock on a one-for-ten (1:10) basis. The reverse stock split is part of the Company’s plan to regain compliance with the minimum bid price requirement for continued listing on the Nasdaq Capital Market.
New Leadership Team: On July 22, 2024, the Board of Directors appointed Malte Peters, M.D., as Chief Executive Officer and Terry Coelho as Executive Vice President and Chief Financial Officer to lead the Company through its next phase of development and realize the significant opportunity eseba-vec represents.
Board Appointment: Sean A. Cassidy was appointed to the Board of Directors on July 22, 2024, and will serve as the chair of the Audit Committee and member of the Compensation Committee.
Financial Highlights

Roche: In April, HOOKIPA received a final $10.0 million milestone payment under its now-terminated HB-700 collaboration agreement with Roche. The success-based milestone payment was achieved in connection with HOOKIPA’s submission of an IND application for HB-700 for the treatment of KRAS mutated tumors.
Gilead: In July, HOOKIPA received a $5.0 million milestone payment under its collaboration and license agreement with Gilead. The success-based milestone payment was achieved in connection with the dosing of the first person in a Phase 1b clinical trial of HB-500 for the treatment of HIV.
Second Quarter 2024 Financial Results

Cash Position: HOOKIPA’s cash, cash equivalents and restricted cash as of June 30, 2024 was $77.4 million compared to $117.5 million as of December 31, 2023. The decrease was primarily attributable to cash used in operating activities.

Revenue: Revenue was $1.3 million for the three months ended June 30, 2024, compared to $2.7 million for the same period in 2023. The decrease was primarily due to lower partial recognition of the upfront and milestone payments under the Roche collaboration as a result of the termination of the collaboration agreement with Roche.

Research and Development Expenses: HOOKIPA’s research and development expenses were $19.7 million for the three months ended June 30, 2024, and June 30, 2023, respectively. The primary changes in research and development expenses were lower personnel-related and laboratory-related expenses as well as lower manufacturing expenses, offset by higher clinical study expenses for the eseba-vec program.

General and Administrative Expenses: General and administrative expenses amounted to $3.9 million for the three months ended June 30, 2024, compared to $4.4 million for the same period in 2023. The primary driver of the decrease in general and administrative expenses was a decrease in personnel-related expenses and in professional and consulting fees.

Restructuring Expenses: Restructuring expenses amounted to $0.1 million for the three months ended June 30, 2024, and resulted from severance and other personnel costs as well as consulting costs associated with the Company’s restructuring plan announced in January 2024. The restructuring plan was completed as of June 30, 2024.

Net Loss: HOOKIPA’s net loss was $19.1 million for the three months ended June 30, 2024, compared to a net loss of $18.0 million for the same period in 2023. This increase was primarily due to lower revenues resulting from lower partial recognition of the upfront and milestone payments under the terminated Roche Collaboration.

On August 8, 2024 Oncternal Therapeutics, Inc. (Nasdaq: ONCT), a clinical-stage biopharmaceutical company focused on the development of novel oncology therapies, reported a business update and announced second quarter 2024 financial results (Press release, Oncternal Therapeutics, AUG 8, 2024, View Source [SID1234645619]).

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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

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"Our Phase 1/2 study of ONCT-534 in patients with R/R mCRPC continues to advance through dose escalation cohorts without dose-limiting toxicities or concerning side effects. We are encouraged by the high pace of enrollment in the study and look forward to sharing initial clinical and biomarker data later in the third quarter," said James Breitmeyer, M.D., Ph.D., Oncternal’s President and CEO. "Our Phase 1/2 study of ONCT-808, our autologous ROR1 CAR T, is open and enrolling patients. We remain encouraged we will find the optimal dose to address the high unmet need of patients with relapsed or refractory aggressive B cell lymphoma, including those who have relapsed after CD19 CAR T treatment."

Recent Highlights

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In July 2024, we announced that enrollment was complete and three patients dosed in the sixth cohort of our Phase 1/2 study of ONCT-534 for the treatment of patients with advanced prostate cancer who are relapsed or refractory to approved androgen receptor pathway inhibitors (ARPI). Patients in the sixth dosing cohort are being given 1200 mg of ONCT-534 orally once per day.
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Our dose escalation/dose expansion Phase 1/2 Study ONCT-808-101, evaluating our ROR1-targeting autologous CAR T cell therapy, ONCT-808, for the treatment of patients with relapsed or refractory aggressive B-cell lymphoma, including patients that have failed prior CD19 CAR T treatment, is open and enrolling patients. Protocol changes that include modified eligibility criteria, increased monitoring for early infection, and evaluating lower doses of ONCT-808 have now been implemented.

Expected Upcoming Milestones

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ONCT-534, our dual-action androgen receptor inhibitor
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Initial clinical data in the third quarter of 2024
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Additional clinical data readouts in the fourth quarter of 2024
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ONCT-808, our autologous ROR1-targeted CAR T cell therapy
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Clinical data update in the fourth quarter of 2024

Second Quarter 2024 Financial Results

Our grant revenue was $0.8 million for the second quarter ended June 30, 2024. Our total operating expenses for the second quarter ended June 30, 2024 were $9.7 million, including $1.4 million in non-cash stock-based compensation expense. Research and development expenses for the quarter totaled $6.6 million, and general and administrative expenses for the quarter totaled $3.1 million. Net loss for the first quarter was $8.6 million, or a net loss of $2.89 per share, basic and diluted. As of June 30, 2024, we had approximately 3.0 million shares of common stock outstanding, $21.4 million in cash, cash equivalents and short-term investments and no debt. These funds are expected to be sufficient to fund our operations into the first quarter of 2025.

On August 8, 2024 Virpax Pharmaceuticals, Inc. ("Virpax" or the "Company") (NASDAQ: VRPX), a company specializing in developing non-addictive products for pain management, post-traumatic stress disorder, central nervous system (CNS) disorders and anti-viral barrier indications, reported that Gerald W. Bruce, CEO, will present and host one-on-one meetings with investors at the Sidoti August Virtual Investor Conference, taking place on August 14-15, 2024 (Press release, Virpax Pharmaceuticals, AUG 8, 2024, View Source [SID1234645646]). Joining him will be Vinay Shah, CFO.

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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

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The presentation will begin at 2:30 PM (ET) on Wednesday, August 14th, and can be accessed live here: View Source Virpax will also host virtual one-on-ones with investors on Wednesday and Thursday, August 14-15, 2024. To register for the presentation or one-on-ones, visit www.sidoti.com/events. Registration is free and you don’t need to be a Sidoti client.