On August 8, 2024 Orion reported half-year financial report (Presentation, Orion, AUG 8, 2024, View Source [SID1234646716]).

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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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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.

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 OmniAb, Inc. (NASDAQ: OABI) reported financial results for the three and six months ended June 30, 2024, and provided operating and partner program updates (Press release, OmniAb, AUG 8, 2024, View Source [SID1234645644]).

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"Second quarter results were in-line with our expectations and support our view of the full year. We advanced our business by adding new partnerships and programs as existing partners made important clinical progress in their respective programs," said Matt Foehr, Chief Executive Officer of OmniAb. "With recent momentum in business development, 2024 has the potential to be a record year in terms of deal flow, which we believe is driven by the quality and differentiation of our technology platforms and by the superior execution of our team."

Second Quarter 2024 Financial Results

Revenue for the second quarter of 2024 was $7.6 million, compared with $6.9 million for the same period in 2023, with the increase primarily due to the accelerated recognition of deferred service revenue related to the discontinuation of a GSK ion channel program.

Research and development expense was $13.9 million for the second quarter of 2024, compared with $14.1 million for the same period in 2023. General and administrative expense was $8.0 million for the second quarter of 2024, compared with $8.7 million for the same period in 2023, with the decrease primarily due to lower share-based compensation expense as well as nonrecurring prior-year costs associated with ERP system implementation. Amortization of intangible assets increased in the second quarter of 2024 due to a $1.2 million impairment related to assets associated with two legacy unpartnered Ab Initio programs. Other operating income for the second quarter of 2024 includes a $2.6 million reduction in contingent liabilities primarily attributed to changes in partner ion channel programs.

Net loss for the second quarter of 2024 was $13.6 million, or $0.13 per share, compared with a net loss of $14.7 million, or $0.15 per share, for the same period in 2023.

Year-to-Date Financial Results

Revenue for the six months ended June 30, 2024 was $11.4 million, compared with $23.9 million for the same period in 2023, with the decrease primarily due to the recognition in the 2023 period of various milestones, including a $10 million milestone related to the first commercial sale of TECVAYLI (teclistamab) in the European Union.

Research and development expense was $28.5 million for the six months ended June 30, 2024, compared with $27.9 million for the same period in 2023, with the increase primarily due to higher contract research costs partially offset by lower share-based compensation. General and administrative expense was $16.3 million for the six months ended June 30, 2024, compared with $16.9 million for the same period in 2023, with the decrease primarily due to non-recurring consulting and other outside service expenses incurred in 2023 related to the Company’s spin-off. Amortization of intangible assets increased for the six months ended June 30, 2024 due to a $1.2 million impairment related to assets associated with two legacy unpartnered Ab Initio programs. Other operating income for the six months ended June 30, 2024 includes a $2.6 million reduction in contingent liabilities attributed to changes in partner ion channel programs.

Net loss for the six months ended June 30, 2024 was $32.6 million, or $0.32 per share, compared with a net loss of $20.8 million, or $0.21 per share, for the same period in 2023.

As of June 30, 2024, OmniAb had cash, cash equivalents and short-term investments of $57.2 million.

2024 Financial Guidance

OmniAb now expects total operating expenses in 2024 to be slightly less than total operating expenses in 2023.

OmniAb continues to expect its cash use in 2024 to be relatively similar to its cash use in 2023, excluding the $35 million TECVAYLI milestone payment received in 2023. Given the expected progression of the existing partnered pipeline, OmniAb expects its cash use in 2025 to be substantially lower than in 2024. OmniAb’s current cash balance and cash from operations are expected to provide sufficient capital to fund operations for the foreseeable future.

Second Quarter 2024 and Recent Business Highlights

During the second quarter of 2024, OmniAb entered into new platform license agreements with DAAN Bio and Topaz Therapeutics, and expanded its technology access license relationship with HanAll Biopharma. As of June 30, 2024, the Company had 83 active partners and 333 active programs, including 32 OmniAb-derived programs in clinical development or being commercialized. OmniAb also recently entered into new platform license agreements with 92Bio, Inc. and Memorial Sloan Kettering Cancer Center. Additionally, OmniAb recently entered into a joint antibody drug conjugate asset discovery and material transfer agreement with Redwood Bioscience, Inc., a wholly owned subsidiary of Catalent, Inc.

Second quarter 2024 and recent partner and business highlights include the following:

IMVT-1402

In the second quarter, Immunovant disclosed that it expects to advance IMVT-1402 in further studies for indications such as myasthenia gravis, Graves’ disease and chronic inflammatory demyelinating polyneuropathy, all with ongoing studies using batoclimab. The decision of whether to advance IMVT-1402 to a registrational study for thyroid eye disease (TED) will be made after disclosing topline data for batoclimab in TED in the first half of 2025.
Batoclimab

Harbour Biomed announced that it has resubmitted the Biologics License Application of batoclimab (HBM9161) to the National Medical Products Administration of China for the treatment of generalized myasthenia gravis.
Acasunlimab

Genmab announced initial data from the Phase 2 GCT1046-04 trial evaluating acasunlimab (DuoBody-PD-L1x4-1BB), an investigational bispecific antibody also known as GEN1046/BNT311, as monotherapy and in combination with pembrolizumab in patients with PD-L1-positive metastatic non-small cell lung cancer who had disease progression following one or more prior lines of anti-PD-L1-containing treatment. The results showed a 12-month overall survival rate of 69%, a median overall survival of 17.5 months and a 30% overall response rate (confirmed ORR 17%) at the time of data cut-off in patients treated with the combination of acasunlimab and pembrolizumab every six weeks. Data from this ongoing Phase 2 study will inform the planned pivotal Phase 3 trial, which is expected to start before year-end 2024.
Sugemalimab

CStone recently announced European approval for sugemalimab, an anti-PD-L1 monoclonal antibody (mAb) for first-line treatment of both squamous and non-squamous non-small cell lung cancer. CStone is actively preparing to submit additional Marketing Authorization Applications to the European Medicines Agency for new indications, including Stage III non-small cell lung cancer, first-line gastric cancer, first-line esophageal cancer, and relapsed/refractory extranodal natural killer/T-cell lymphoma.
CStone announced that it entered into a strategic commercial collaboration with Ewopharma. Under the licensing and commercialization agreement, Ewopharma gained the commercial rights for sugemalimab in Switzerland and 18 Central Eastern European countries. CStone also disclosed that discussions for commercial partnerships in Western Europe, Latin America, the Middle East, Southeast Asia, etc., are progressing well and are expected to conclude soon.
Zimberelimab

Arcus announced the completion of patient enrollment for STAR-221, a Phase 3 study in collaboration with Gilead Sciences evaluating the combination of the Fc-silent anti-TIGIT antibody domvanalimab plus the anti-PD-1 mAb zimberelimab and chemotherapy in patients with locally advanced unresectable or metastatic gastric, gastroesophageal junction or esophageal adenocarcinoma.
ALTA-002

Tallac Therapeutics disclosed U.S. Food and Drug Administration clearance of its investigational new drug application for ALTA-002, a SIRPα targeting toll-like receptor agonist antibody conjugates (TRAAC), in patients with advanced solid tumors.
M9140

At the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting, Merck KGaA presented data on M9140, a novel antibody-drug conjugate with topoisomerase 1 inhibitor payload targeting carcinoembryonic antigen-related cell adhesion molecule 5 (CEACAM5) expressing tumors. M9140 demonstrated high potency, strong antitumor activity and bystander effect in preclinical models.
A first-in-human Phase 1 study to evaluate the safety, tolerability, pharmacokinetics and preliminary clinical activity of M9140 in patients with advanced solid tumors is ongoing.
CSX-1004

Cessation announced the presentation of preliminary data from its Phase 1a first-in-human study of CSX-1004, an investigational mAb for prophylaxis against fentanyl-related overdose, showing that CSX-1004 is safe and well-tolerated under the conditions tested. The exposure data were also predictive of efficacy for blocking fentanyl-induced respiratory depression.
Small Molecule Ion Channel Program Updates

In the second quarter, GSK discontinued its work on a small molecule Nav1.1 sodium channel modulator program. OmniAb retains co-ownership of all intellectual property for the program. GSK and OmniAb maintain a collaboration on a small molecule ion channel preclinical-stage program for neurological disease.
Subsequent to the second quarter, OmniAb was notified that Roche is returning its rights to a pre-clinically differentiated small molecule targeting Kv7.2. The termination is not due to scientific reasons. Roche and OmniAb maintain collaborations on two additional small molecule ion channel programs for potential treatment of central nervous system disorders.
xPloration Technology Platform Recent Events

OmniAb presented an overview of xPloration, its high-throughput single B-cell screening microcapillary platform that leverages machine learning and AI, in a presentation titled "Deep Screening in Harmony with Artificial Intelligence for Bispecific Antibody Discovery" at the 20 th Annual PEGS Boston Conference and Expo. This presentation highlighted the technical synergies between xPloration, OmniFlic , OmniClic and OmniDeep to enable new bispecific antibody discovery workflows for OmniAb’s current and future partners.
OmniAb disclosed the issuance of U.S. Patent number 12,024,705 B2 entitled "Methods and Systems for Screening Using Microcapillary Arrays." The patent was issued by the United States Patent and Trademark Office on July 2, 2024 and has an expected expiry of December 12, 2036. Additionally, a patent titled "Lateral Loading of Microcapillary Arrays" was issued on May 1, 2024 in Europe as EP3890876 with an expected expiry of December 5, 2039.
In June, OmniAb announced the publication of a peer-reviewed paper titled "Chickens with a Truncated Light Chain Transgene Express Single-Domain H Chain-Only Antibodies" in the Journal of Immunology demonstrating that chickens can be genetically engineered to produce functional heavy chain-only single-domain antibodies. sdAbs are produced naturally by camelids and have gained popularity as small, robust and highly modular building blocks for antibody discovery.

Conference Call and Webcast

OmniAb management will host a conference call with accompanying slides today beginning at 4:30 p.m. Eastern time (1:30 p.m. Pacific time) to discuss this announcement and answer questions. To participate via telephone, please dial (800) 549 8228 using the conference ID 01718. Slides, as well as the live and replay webcast of the call, are available at View Source .

On August 8, 2024 Verastem Oncology (Nasdaq: VSTM), a biopharmaceutical company committed to advancing new medicines for patients with cancer, reported business updates and reported financial results for the second quarter ended June 30, 2024 (Press release, Verastem, AUG 8, 2024, View Source [SID1234645643]).

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"We have made tremendous progress in the second quarter as we initiated our rolling NDA submission for avutometinib and defactinib combination in recurrent KRAS mutant low-grade serous ovarian cancer, shared updated topline results from our RAMP 201 trial with a minimum of five months of follow-up, and presented positive initial interim results from our ongoing first-line metastatic pancreatic cancer trial," said Dan Paterson, president and chief executive officer of Verastem Oncology. "In the second half of the year, we plan to present mature data from RAMP 201, complete our rolling NDA submission with the mature data from RAMP 201 trial, and share interim topline data from our lung cancer programs."

Second Quarter 2024 and Recent Updates

Avutometinib and Defactinib Combination in Low-Grade Serous Ovarian Cancer (LGSOC)

Announced the initiation of a rolling New Drug Application (NDA) submission seeking accelerated approval of the combination of avutometinib and defactinib for adult patients with recurrent KRAS mutant LGSOC who received at least one prior systemic therapy. Read the press release here.
Announced topline interim data from the RAMP 201 trial, with a February 2024 data cutoff, that continued to show robust overall response rates and durable responses with low discontinuation rates due to adverse events in patients with KRAS mutant or KRAS wild-type LGSOC who had a minimum follow-up of five months, in May 2024.
Expect to complete the rolling NDA submission after obtaining and submitting to the FDA the mature safety and efficacy data from the RAMP 201 trial, including 12 months of follow-up, to inform the final indication in the second half of 2024.
Enrollment and site activations are underway in the U.S., Australia, Europe, and the UK, for the international confirmatory Phase 3 RAMP 301 trial evaluating the avutometinib and defactinib combination versus standard of care chemotherapy or hormonal therapy for the treatment of recurrent LGSOC.
Preparations for a potential U.S. commercial launch in 2025 are ongoing and plans to initiate discussions with European and Japanese regulatory authorities to address patient needs outside the U.S. continue to advance.
Avutometinib in Combination with KRAS G12C Inhibitors in Non-Small Cell Lung Cancer (NSCLC)

In the RAMP 203 trial, patients who previously progressed on KRAS G12C inhibitors were initiated on the triplet combination of avutometinib, defactinib and sotorasib.
Expect to report updated interim data from patients with KRAS G12C-mutant NSCLC in the Phase 1/2 RAMP 203 trial evaluating avutometinib plus defactinib and sotorasib are planned for H2 2024.
Expect to report initial interim data from patients with KRAS G12C-mutant NSCLC in the Phase 1/2 RAMP 204 trial evaluating avutometinib and adagrasib are planned for H2 2024.
Avutometinib and Defactinib Combination in First-Line Metastatic Pancreatic Cancer

Presented initial interim safety and efficacy results from the ongoing RAMP 205 trial of avutometinib and defactinib in combination with current standard of care gemcitabine and nab-paclitaxel in first-line metastatic pancreatic cancer on June 1, 2024, at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting. As of May 14, 2024, 41 patients had been treated in one of four dose cohort regimens and only patients in dose cohort 1 had a minimum follow up of six months. In the dose level 1 cohort, 83% (5/6) of patients achieved a confirmed partial response with more than six months of follow up at the time of data cutoff. One dose-limiting toxicity (DLT) was observed in the dose level 1 cohort, and the dose level was subsequently cleared after additional patients were enrolled. Of the 26 patients in all cohorts who have had the opportunity to have their first scan while on treatment, 21 have experienced a reduction of the change in target lesion sum of diameters. Read the press release here.
FDA granted Orphan Drug Designation to avutometinib and defactinib combination for the treatment of pancreatic cancer in July 2024.
Expect to report updated data from the ongoing RAMP 205 trial in Q1 2025.
GFH375 (VS-7375): Oral KRAS G12D (ON/OFF) Inhibitor

GenFleet Therapeutics investigational new drug (IND) application for GFH375 (VS-7375) in China was cleared in June 2024.
GenFleet dosed the first patient in the Phase 1/2 trial for GFH375 (VS-7375) in China in patients with KRAS G12D-mutated advanced solid tumors in July 2024.
Plan to initiate development studies outside of China after evaluating initial dose escalation data from the Phase 1 study of GFH375 (VS-7375) in China to accelerate a path forward in the U.S. and rest of world.
Discovery/lead optimization continues for second and third programs with GenFleet collaboration.
Corporate Updates

Verastem strengthened its executive team with the appointment of John Hayslip, M.D., as chief medical officer in April 2024 and the promotion of Nate Sanburn to chief business officer in June 2024.
In July 2024, Verastem strengthened its balance sheet by raising net proceeds of approximately $51.1 million in a public offering of 13.3 million shares of its common stock and accompanying warrants to purchase up to 13.3 million shares of its common stock, and pre-funded warrants to purchase up to 5.0 million shares of its common and accompany warrants to purchase up to 5.0 million shares of its common stock.
Secura Bio, Inc. ("Secura") achieved $100 million of cumulative worldwide net sales of COPIKTRA during Q2 2024, entitling Verastem to a $10 million milestone payment. Verastem received the $10 million milestone payment in July 2024.
Second Quarter 2024 Financial Results

Verastem Oncology ended the second quarter of 2024 with cash, cash equivalents and investments of $83.4 million. On a pro forma basis, inclusive of the $51.1 million net proceeds raised through issuance of common stock, pre-funded warrants, and warrants in July 2024, and the $10 million net sales milestone from Secura, cash, cash equivalents and investments were $144.5 million as of June 30, 2024. These additional sources of capital along with the existing cash, cash equivalents, and investments through the second quarter of 2024 provides an expected cash runway through the potential approval of avutometinib and defactinib for recurrent LGSOC in the first half of 2025.

Total revenue for the three months ended June 30, 2024 (the "2024 Quarter") was $10.0 million, compared to $0.0 million for the three months ended June 30, 2023 (the "2023 Quarter"). Revenue for the 2024 Quarter was comprised of one sales milestone payment of $10.0 million due upon Secura achieving total worldwide net sales of COPIKTRA exceeding $100.0 million during the 2024 Quarter. The $10.0 million milestone payment was received by Verastem in July 2024.

Total operating expenses for the 2024 Quarter were $28.3 million, compared to $20.3 million for the three months ended June 30, 2023.

Research & development expenses for the 2024 Quarter were $18.1 million, compared to $12.9 million for the 2023 Quarter. The increase of $5.2 million, or 40.3%, was primarily related to increased contract research organization costs, increased consulting costs, and increased investigator fees.

Selling, general & administrative expenses for the 2024 Quarter were $10.2 million, compared to $7.4 million for the 2023 Quarter. The increase of $2.8 million, or 37.8%, was primarily related to additional costs in anticipation of a potential launch of avutometinib and defactinib in LGSOC, and increased personnel costs, including non-cash stock compensation.

Net loss for the 2024 Quarter was $8.3 million, or $0.31 per share (basic and diluted), compared to $24.3 million, or $1.37 per share (basic and diluted, each as adjusted for the Company’s reverse stock split) for the 2023 Quarter.

For the 2024 Quarter, non-GAAP adjusted net loss was $16.5 million, or $0.61 per share (diluted) compared to non-GAAP adjusted net loss of $18.8 million, or $1.06 per share (diluted, as adjusted for the Company’s reverse stock split), for the 2023 Quarter. Please refer to the GAAP to non-GAAP Reconciliation attached to this press release.