On August 11, 2022 Abeona Therapeutics Inc. (Nasdaq: ABEO), a fully-integrated leader in cell and gene therapy, reported financial results for the second quarter of 2022 (Press release, Abeona Therapeutics, AUG 11, 2022, View Source [SID1234618149]). The Company will host a conference call and webcast today, August 11, 2022, at 8:30 a.m. ET, to discuss its financial results and business update.

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"We have taken decisive action to focus our resources on our lead asset EB-101, for which we expect pivotal Phase 3 VIITAL topline results in the coming months," said Vish Seshadri, Chief Executive Officer of Abeona. "With Ultragenyx assuming all financial responsibility for the continued development of ABO-102, we were able to extend our cash runway well beyond the VIITAL data readout. The additional runway puts us in a strong position to forge the optimal commercial partnership that fully appreciates the value of EB-101 post-data readout."

Second Quarter and Recent Operating Highlights

Ultragenyx Pharmaceutical Inc. and Abeona entered into an exclusive license agreement for ABO-102 (now UX111) for Sanfilippo syndrome type A (MPS IIIA), under which Ultragenyx assumes responsibility for the ABO-102 program and in return Abeona is eligible to receive tiered royalties and commercial milestone payments following potential regulatory approval.
Topline data from the Phase 3 VIITAL study of EB-101 in RDEB is on track for late third quarter to early fourth quarter of 2022 upon completion of the last patient monitoring visit, which is expected by mid-September. Baseline wound characteristics underscore the large size and severe pain associated with wounds included in VIITAL. Treated randomized wounds had mean per patient body surface area of 156 cm2, the largest reported for pivotal studies in RDEB. Baseline pain reported for randomized wounds using the Wong-Baker FACES Pain Rating Scale of 0-10 further highlights the severity of these wounds, with eight of the 11 patients reporting a minimum pain score of 6 in at least one randomized wound and four of the 11 patients reporting a maximum baseline pain score of 10 for certain randomized wounds.
Additional long-term follow up data up to eight years and quality of life data from a completed Phase 1/2 study evaluating EB-101 for RDEB were presented at the Society of Investigative Dermatology (SID) Annual Meeting. The data showed EB-101 treatment of large chronic RDEB wounds resulted in considerable wound healing with mean 5.9 years of follow-up. In addition, reduced wound burden was associated with long-term symptomatic relief, including reduction in pain.
Reported non-human primate data for AAV204, a novel adeno-associated virus (AAV) capsid from Abeona’s AIM capsid library, highlighting its ability to produce more robust transduction in the macula area of the eye following para-retinal administration, which unlike subretinal administration does not create a retinal detachment. The data was featured at the Association for Research and Vision in Ophthalmology (ARVO) 2022 Annual Meeting.
On July 19, 2022, Abeona received notice from Nasdaq that the Company has regained compliance with the minimum bid price requirement under Nasdaq Listing Rule 5550(a)(2) for continued listing on the Nasdaq Capital Market.
Second Quarter Financial Results

Cash, cash equivalents, restricted cash and short-term investments totaled $26.0 million as of June 30, 2022. Net cash used in operating activities was $9.0 million for the second quarter of 2022, compared to $13.7 million in the first quarter of 2022.

License and other revenues in the second quarter of 2022 were $1.0 million, compared to nil in the second quarter of 2021. The revenue in the second quarter of 2022 resulted from a clinical milestone achieved under a sublicense agreement with Taysha Gene Therapies relating to an investigational AAV-based gene therapy for Rett syndrome, including certain intellectual property relating to MECP2 gene constructs and regulation of their expression.

Research and development (R&D) expenses for the three months ended June 30, 2022 were $6.7 million, compared to $8.5 million for the same period of 2021. General and administrative (G&A) expenses were $3.5 million for the three months ended June 30, 2022, compared to $5.2 million for the same period of 2021.

Net loss attributable to common shareholders for the second quarter of 2022 was $12.1 million, or $2.08 loss per common share as compared to $15.2 million, or $3.93 loss per common share, in the second quarter of 2021.

Conference Call Details

Abeona Therapeutics will host a conference call and webcast today, August 11, 2022, at 8:30 a.m. ET, to discuss its financial results and business update. To access the call, dial 877-545-0523 (U.S. toll-free) or 973-528-0016 (international) and Entry Code: 857476 five minutes prior to the start of the call. A live, listen-only webcast and archived replay of the call can be accessed on the Investors & Media section of Abeona’s website at www.abeonatherapeutics.com. The archived webcast replay will be available for 30 days following the call.

On August 11, 2022 Theseus Pharmaceuticals, Inc. (NASDAQ: THRX) (Theseus or the Company), a clinical-stage biopharmaceutical company focused on improving the lives of cancer patients through the discovery, development, and commercialization of transformative targeted therapies, reported business and pipeline highlights and reported financial results for the second quarter ended June 30, 2022 (Press release, Theseus Pharmaceuticals, AUG 11, 2022, View Source [SID1234618148]).

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"At Theseus, we are developing next-generation tyrosine kinase inhibitors (TKIs) to outsmart cancer resistance—a key challenge facing existing therapies in targeted oncology. 2022 is a critical year of execution and we have continued to make significant progress across our programs," said Tim Clackson, Ph.D., President and Chief Executive Officer of Theseus. "Enrollment in the dose escalation portion of the Phase 1/2 trial of THE-630 in patients with advanced GIST is on track and we look forward to the expected announcement of initial clinical data in the first half of 2023. We are on track to nominate a development candidate for our EGFR inhibitor program for the treatment of non-small cell lung cancer in the third quarter of 2022 and plan to present preclinical data supporting the promising profile for this candidate in the fourth quarter of 2022. We expect to expand our pipeline with the introduction of a third TKI program later this year."

Recent Pipeline Highlights and Upcoming Expected Milestones:

THE-630: a single molecule, pan-variant inhibitor of the receptor tyrosine kinase KIT. Designed for patients whose gastrointestinal stromal tumors (GIST), a disease that remains largely KIT-dependent following multiple lines of treatment, has developed resistance to earlier lines of therapy.

A Phase 1/2 dose-escalation and expansion clinical trial evaluating THE-630 in patients with advanced GIST is ongoing, as the Company completes site activations and continues to enroll patients in the dose-escalation portion of the trial.
Initial data from the Phase 1 dose-escalation portion of the clinical trial are expected to be presented at a scientific conference in the second quarter of 2023.
EGFR inhibitor for non-small cell lung cancer (NSCLC): a fourth-generation, single molecule, selective EGFR inhibitor, designed to inhibit all major single-, double-, and triple-mutant EGFR variants found in the tumors of patients with EGFR-mutant NSCLC that have developed resistance to first- or later-line osimertinib treatment, including the C797S and T790M mutations, with central nervous system (CNS) activity.

Theseus expects to nominate a development candidate for this program in the third quarter of 2022 and to introduce this candidate along with a presentation of new preclinical data at a scientific conference in the fourth quarter of 2022.
Theseus expects to submit an Investigational New Drug (IND) application to the U.S. Food and Drug Administration in 2023.
At the 2022 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting in April 2022, the Company presented preclinical data demonstrating that pan-variant inhibition of all major EGFR mutations consisting of single-, double-, and triple-EGFR mutants, including T790M and C797S, with selectivity over wild-type and CNS activity, can be achieved with a single molecule.
R&D Portfolio: Theseus’ discovery platform incorporates its structure-guided drug design and predictive resistance assay (PRA) to develop next-generation, pan-variant inhibitor molecules targeting all major cancer-causing and treatment-resistance mutations of tyrosine kinase targets.

The Company continues advancing its preclinical discovery pipeline and expects to introduce a new kinase target program by the end of 2022.
Business Highlights:

In May 2022, Theseus announced the appointment of Don Hayden to its Board of Directors. Mr. Hayden brings over 40 years of industry experience to the Board, including previously serving as President of Oncology and Immunology and as President of Global Pharmaceuticals at Bristol Myers-Squibb and as Chair at numerous biotech companies.
In June 2022, Theseus appointed Steve Tuller as Vice President, Quality Assurance. Mr. Tuller has 24 years of experience in the pharmaceutical and biotechnology industries. Before joining Theseus, he served as Vice President, Head of Quality Assurance at Epizyme, Inc., where he initiated the GxP Quality Assurance organization.
Second Quarter Financial Results:

Cash Position: As of June 30, 2022, Theseus had cash, cash equivalents and short-term and long-term investments of $228.6 million. Theseus expects its cash, cash equivalents, and investments to fund operations and capital expenditures into the fourth quarter of 2024 based on its current operating plan.

R&D Expenses: Research and development expenses were $7.3 million for the second quarter of 2022, as compared to $4.5 million for the same period in 2021. This increase was primarily due to $2.0 million of increased employee-related costs, and $0.6 million of increased expenses for clinical and preclinical studies.

G&A Expenses: General and administrative expenses were $4.7 million for the second quarter of 2022, as compared to $2.3 million for the same period in 2021. This increase was primarily due to $1.6 million of increased employee-related costs, as well as $0.5 million of increased professional fees.

Net Loss: Net loss was $11.6 million for the second quarter of 2022, as compared to a net loss of $6.8 million for the same period in 2021.

On August 11, 2022 Werewolf Therapeutics, Inc. (the "Company" or "Werewolf") (Nasdaq: HOWL), an innovative biopharmaceutical company pioneering the development of conditionally activated therapeutics engineered to stimulate the body’s immune system for the treatment of cancer, reported financial results for the second quarter ended June 30, 2022 (Press release, Werewolf Therapeutics, AUG 11, 2022, View Source [SID1234618147]).

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"Werewolf continues to advance our conditionally activated INDUKINE therapies into clinical development," said Daniel J. Hicklin, Ph.D., President and Chief Executive Officer of Werewolf. "Most notably, we received FDA clearance to launch our first-in-human study of WTX-124, an IL-2 INDUKINE, for treatment of advanced solid tumors, marking our transition to a clinical-stage company. Additionally, we are planning to submit an IND for our IL-12 INDUKINE, WTX-330, in the second half of 2022. We believe that these initial candidates offer compelling opportunities not just to advance patient outcomes in their respective indications, but also to validate the distinct advantages of our platform. Importantly, we believe that we remain well-capitalized to advance clinical development for both programs as we proceed with novel discovery efforts in parallel."

Recent Highlights and Upcoming Milestones

WTX-124: a systemically delivered, conditionally activated Interleukin-2 (IL-2) INDUKINE molecule in development as monotherapy or in combination with checkpoint inhibitors in multiple solid tumor types.

During the second quarter of 2022, Werewolf received clearance from the U.S. Food and Drug Administration (FDA) on its Investigational New Drug (IND) application for WTX-124. This Phase 1/1b clinical trial will evaluate WTX-124 as a monotherapy and in combination with KEYTRUDA (pembrolizumab), Merck’s anti-PD-1 (programmed death receptor-1) therapy, in patients with advanced or metastatic solid tumors.
The Company plans to provide an update after dosing the first patient in the WTX-124 Phase 1/1b clinical trial.
WTX-330: a systemically delivered, conditionally activated Interleukin-12 (IL-12) INDUKINE molecule in development as monotherapy or in combination with checkpoint inhibitors in refractory and/or immunologically unresponsive tumors.

Werewolf is concluding IND-enabling work for WTX-330 and is planning to submit an IND application for this program in the second half of 2022.
Corporate:

In April 2022, Jazz Pharmaceuticals (Jazz) and Werewolf entered into a collaboration and license agreement under which Werewolf granted Jazz certain licenses to develop and commercialize WTX-613 (now known as JZP898), an Interferon alpha (IFNα) molecule. During the second quarter of 2022, Werewolf received an upfront payment of $15.0 million from Jazz and is eligible to receive up to $520.0 million in development and regulatory milestones and up to $740.0 million in commercial milestone payments, as well as a tiered, mid-single-digit percentage royalty on net sales, pending approval.
Second Quarter 2022 Financial Highlights

Cash position: As of June 30, 2022, cash and cash equivalents were $145.7 million, compared to $157.5 million as of December 31, 2021. The Company expects that its existing cash and cash equivalents will be sufficient to enable funding of its operating expenses and capital expenditure requirements through at least the fourth quarter of 2023.
Collaboration revenue: Collaboration revenue was $4.1 million for the second quarter of 2022, compared to zero for the same period in 2021. Collaboration revenue is related to amortization of the $15.0 million upfront payment received in April 2022 upon the execution of Werewolf’s licensing agreement with Jazz and costs incurred for research services to be reimbursed by Jazz.
Research and development expenses: Research and development expenses were $13.9 million for the second quarter of 2022, compared to $7.3 million for the same period in 2021. The increase in research and development expenses was primarily due to manufacturing expenses incurred to support the production of preclinical and future clinical trial materials associated with the Company’s product candidates WTX-124, WTX-330 and WTX-613, increased employee compensation costs related to increased headcount and increased contract research organization expenses incurred to support IND-enabling studies and clinical start-up activities for WTX-124 and WTX-330.
General and administrative expenses: General and administrative expenses were $5.2 million for the second quarter of 2022, compared to $3.7 million for the same period in 2021. The increase in general and administrative expenses was primarily due to increased personnel and other costs attributable to operating as a public company.
Net loss: Net loss was $14.6 million for the second quarter of 2022, compared to $10.9 million for the same period in 2021.

On August 11, 2022 ALX Oncology Holdings Inc., ("ALX Oncology") (Nasdaq: ALXO), a clinical-stage immuno-oncology company developing therapies that block the CD47 checkpoint pathway, reported the initiation of a Phase 2 investigator-sponsored study of evorpacept, a next generation CD47 blocker, in combination with ERBITUX (cetuximab) and KEYTRUDA (pembrolizumab), Merck’s anti-PD-1 therapy, in patients with refractory microsatellite stable metastatic colorectal cancer ("mCRC") who have progressed on at least two lines of systemic therapy (Press release, ALX Oncology, AUG 11, 2022, View Source [SID1234618146]).

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This is an open-label, multi-center, single-arm phase 2 clinical trial (NCT05167409). Eli Lilly and Company and Merck, known as MSD outside the United States and Canada, will respectively provide ERBITUX and KEYTRUDA to support this study. This trial is being co-led by Wells Messersmith, M.D., Professor and Head of Medical Oncology at the University of Colorado Cancer Center and Director of the Academic GI Cancer Consortium ("AGICC"), and Robert Lentz, M.D., Assistant Professor of Medical Oncology at the University of Colorado Cancer Center. AGICC brings together a world class team of institutions and investigators to design and rapidly complete clinical trials in gastrointestinal cancers. The study is being managed by Criterium, Inc., a full-service contract research organization dedicated to providing efficiency and expertise in clinical trial services.

"In conjunction with AGICC, we are excited to initiate this study that builds upon the promising clinical activity and tolerability observed in patients with advanced solid tumors observed in ASPEN-01, ALX Oncology’s phase 1b study," said Dr. Messersmith. "mCRC is a difficult-to-treat cancer and patients, particularly in the relapsed/refractory setting, will benefit from novel therapeutic options to help improve disease outcomes. From a mechanistic perspective, the combination of a CD47 blocker with cetuximab and pembrolizumab acts through different but complementary mechanisms, and we aim to see evorpacept in this triplet combination positively impact efficacy without increasing toxicity."

ALX Oncology owns worldwide commercial rights to evorpacept.

About Colorectal Cancer

Colorectal cancer ("CRC") starts in the colon or the rectum. These cancers can also be called colon cancer or rectal cancer, depending on where they start. Most CRCs start as polyps on the inner lining of the colon or rectum and grow into the wall of the colon or rectum over time. According to the National Cancer Institute, CRC is the fourth most common cancer diagnosed in the United States. In 2019, there were an estimated 1,369,000 Americans living with the disease, and people with metastatic CRC have a 5-year survival rate of just 15.1%.

On August 11, 2022 Aravive, Inc. (Nasdaq: ARAV, "the Company"), a late clinical-stage oncology company developing targeted therapeutics to treat metastatic disease, reported financial results for the second quarter ended June 30, 2022 and provided corporate updates (Press release, Aravive, AUG 11, 2022, View Source [SID1234618145]).

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"We continued to achieve great progress for Aravive in the second quarter of 2022. All of our batiraxcept clinical trials for the treatment of ovarian, kidney and pancreatic cancers continued to advance at a strong pace," said Gail McIntyre, Ph.D., DABT, Chief Executive Officer of Aravive. "We remain on track with our lead Phase 3 registrational study in ovarian cancer to complete enrollment around year-end 2022, which would enable us to read out topline data mid-2023 and potentially file a BLA with the FDA by year-end 2023."

"We continue to successfully enroll patients in our Phase 2 trial of batiraxcept in clear cell renal cancer and will have updates throughout the remainder of this year. Our Phase 1b trial in pancreatic cancer has completed enrollment and updated results are anticipated later this year. We remain confident in the execution of our clinical trials and encouraged by the continued signals of the potential therapeutic success of batiraxcept."

"Finally, we hired two industry seasoned veterans to round out our senior management team. Rudy Howard and Dr. Robert Geller will not only add tremendous support as we continue our trials, but also as we potentially move towards and beyond regulatory approval of a commercially viable portfolio."

Recent Corporate Highlights

Batiraxcept in Platinum Resistant Ovarian Cancer (PROC): The registration-directed Phase 3 program of batiraxcept in combination with paclitaxel in PROC remains on track to complete enrollment around year-end 2022. The Company expects to report topline data from the trial by mid-2023. CMC work remains on track with the goal of filing a BLA by year-end 2023. The global, randomized, double-blind, placebo-controlled Phase 3 trial is evaluating efficacy and tolerability of batiraxcept at a dose of 15 mg/kg in combination with paclitaxel versus placebo in combination with paclitaxel. The trial aims to enroll 350 patients with platinum resistant, high-grade serous ovarian cancer who have received 1-4 prior lines of therapy.
Batiraxcept in Clear Cell Renal Cell Carcinoma (ccRCC) and Serum-Based Biomarker: As of April 30, 2022 and as presented during the KOL symposium, 26 previously treated (2L+) patients with ccRCC were treated with batiraxcept in the Phase 1b portion of the trial at doses of 15 mg/kg (n=16) and 20 mg/kg (n=10), plus cabozantinib 60 mg daily. There were no dose limiting toxicities observed at either dose and 14 of the 26 patients remained on study. The best overall response rate (ORR, confirmed + unconfirmed) in the ITT population was 46% and 50% in patients dosed with 15 mg/kg (the recommended Phase 2 dose). One of the objectives of the ongoing Phase 1b/2 ccRCC trial is to evaluate the correlation of baseline serum soluble AXL (sAXL)/GAS6 (biomarker) with radiographic response in patients with ccRCC treated with batiraxcept plus cabozantinib. The best ORR in the biomarker high population was 60%, and 67% in the biomarker high population dosed at 15 mg/kg. The 7-month progression-free survival (PFS) rate was 71% in the ITT population, 83% in the biomarker high population, and 91% in the 15 mg/kg biomarker high group. Eight patients experienced resolution of one or more target lesions. The Company has discussed a registrational path with the US FDA that includes use of the sAXL/Gas6 ratio as a basis for an accelerated approval. The Company is on track to report updated results from the Phase 1b and Phase 2 portions of the trial in the second half of 2022.
Batiraxcept in Pancreatic Adenocarcinoma: As of May 3, 2022 and as discussed at the KOL symposium, 21 patients with advanced or metastatic pancreatic adenocarcinoma have been treated with 15 mg/kg batiraxcept in combination with gemcitabine and nab-paclitaxel as a first-line treatment. Batiraxcept has been generally well-tolerated with no unexpected safety signals. The best ORR (confirmed + unconfirmed) was 29%. As noted with the other programs, an observable correlation of baseline levels of serum soluble AXL (sAXL)/GAS6 (biomarker) to clinical activity was noted in this trial and the best ORR in the biomarker high population was 40%. Five patients experienced resolution of one or more target lesions; however, two of these patients have since progressed. The Company is on track to report additional updated data from the Phase 1b portion of the trial in the second half of 2022.
Second Quarter 2022 Financial Results
Revenues for the three months ended June 30, 2022 were $1.6 million, compared to $1.1 million for the three months ended March 31, 2022. Revenues were derived solely from the Company’s collaboration and license agreement with 3D Medicines, executed in November 2020 to develop and commercialize batiraxcept in oncology indications in Greater China. Revenues represent 1) a portion of initial signing and milestone payments received from 3D Medicines that is recognized at the time of the receipt and 2) a portion of the payments that is deferred and recognized over the PROC trial period. The increase in revenues is attributable to increased expenditures related to the PROC trial which drives the recognition of deferred payments over the PROC trial period.

Total operating expenses for the three months ended June 30, 2022 were $21.0 million, compared to $16.1 million for the three months ended March 31, 2022. Research and development expense for the three months ended June 30, 2022 was $17.3 million, compared to $13.0 million for the three months ended March 31, 2022. The increase in research and development expense is attributable to the continued progression of the Company’s PROC and ccRCC clinical trials, as well as increases in related CMC costs. General and administrative expense for the three months ended June 30, 2022 was $3.7 million, compared to $3.1 million for the three months ended March 31, 2022. The increase in general and administrative expense is attributable to increased stock-based compensation, increased consulting fees, and severance expense.

Aravive reported a net loss of $18.5 million, or $0.61 per share, for the three months ended June 30, 2022, compared to a net loss of $13.1 million, or $0.62 per share, for the three months ended March 31, 2022.

Cash Position
As of June 30, 2022, cash and cash equivalents were $46.8 million, compared to $65.8 million as of March 31, 2022 and $59.4 million as of December 31, 2021. The Company anticipates that its current cash and cash equivalents will fund its operating plans into the first quarter of 2023.