On August 12, 2022 Privo Technologies, Inc. ("Privo") reported that the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation (ODD) to Privo’s PRV platform delivery of cisplatin for the treatment of carcinoma in situ (CIS) of the anterior 2/3 of the oral cavity (Press release, Privo Technologies, AUG 12, 2022, View Source [SID1234618272]). This represents Privo’s third ODD for a rare disease following their approval in oral cavity cancers and anal cancers. Privo Technologies is a phase 3 clinical stage biopharmaceutical company that has designed and developed a nanoengineered drug delivery platform to safely deliver highly potent and toxic APIs locoregionally, led by founder and CEO Manijeh Goldberg, PhD.

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"Achieving this important regulatory milestone means we are one step closer to bringing new treatment options to patients diagnosed with oral CIS. The standard of care currently is surgery, resulting in severe disfigurement and poor quality of life. Our novel transmucosal delivery system with embedded cisplatin loaded nanoparticles, PRV111, allows for patients to be treated with topical chemotherapy providing an alternative to surgery that effectively treats cancer while maintaining the form and function of the oral cavity," said Dr. Goldberg.

FDA Orphan Drug Designation is granted to investigational therapies addressing rare medical diseases or conditions that affect fewer than 200,000 people in the United States. Orphan drug status provides benefits to drug developers that includes assistance in the drug development process, tax credits for clinical costs, exemption from FDA PDUFA fees, and seven years of post-approval exclusivity.

On August 12, 2022 Inhibikase Therapeutics, Inc. (Nasdaq: IKT) ("Inhibikase" or "Company"), a clinical-stage pharmaceutical company developing therapeutics with the potential to modify the course of Parkinson’s disease ("PD") and related disorders, reported financial results for the second quarter ended June 30, 2022 and highlighted recent developments (Press release, Inhibikase Therapeutics, AUG 12, 2022, View Source [SID1234618271]).

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"In the second quarter, we continued to make progress across our entire portfolio. We recently opened enrollment in our Phase 2a ‘201’ trial for IkT-148009 for the treatment of Parkinson’s disease. We have now opened nearly a quarter of all sites for the study and we are actively screening patients. In addition, we have submitted our Investigational New Drug (IND) application for IkT-001Pro for patients with chronic myelogenous leukemia to the U.S. Food and Drug Administration (FDA), and expect to initiate our ‘501’ bioequivalence study of IkT-001Pro following FDA review and concurrence," commented Milton Werner, Ph.D., President and Chief Executive Officer of Inhibikase. "Looking ahead, we also expect to share Parkinson’s assessment data from our Phase 1/1b ‘101’ clinical trial of IkT-148009 at the Movement Disorders Society Congress in September and are continuing to advance our regulatory activities in the U.S. and the EU27 as we plan a Phase 2 trial in Multiple System Atrophy. Taken together, we believe that these activities lay the groundwork for continued momentum across our business as we seek to deliver on our mission of bringing novel, disease-modifying treatments to patients suffering from devastating diseases."

Recent Developments and Upcoming Milestones:

Phase 2a ‘201’ Trial of IkT-148009 Open for Enrollment: In May 2022, Inhibikase opened enrollment for its Phase 2a study (‘201 trial’) evaluating IkT-148009 for the treatment of Parkinson’s disease. The Company has opened 11 of 40 planned sites and is actively screening patients. The 201 trial is a 1:1:1:1 randomized, double-blind, twelve-week dosing trial, designed to measure the safety, tolerability and steady-state pharmacokinetics (PK) of three doses of IkT-148009 in up to 120 untreated Parkinson’s patients. The trial will further measure the potential for IkT-148009 to impact the disease globally in the body using a hierarchy of Parkinson’s-related disease assessments in the brain and gut as secondary or exploratory endpoints.

The advancement of IkT-148009 into Phase 2a followed a review of the study protocol, as well as the safety, tolerability and PK data from the 101 clinical trial by the FDA. In the 101 trial, the clinical pharmacology of IkT-148009 in patients paralleled the clinical pharmacology of IkT-148009 in older heathy volunteers. IkT-148009 also demonstrated a favorable safety and tolerability profile up to a dose of 325 mg, with no clinically-significant adverse events observed. Inhibikase plans to present additional data from the 101 trial at the Movement Disorders Society Congress in Madrid, Spain, in September 2022.

Filed IND Application for IkT-001Pro for Stable-Phase Chronic Myelogenous Leukemia (CML): On June 29, 2022, Inhibikase filed its IND application with the FDA for IkT-001Pro, the Company’s prodrug of imatinib mesylate to treat Stable-phase Chronic Myelogenous Leukemia (SP-CML). A clerical error delayed the review of the IND, however the Division of Hematological Malignancies I confirmed that FDA review of the IND should be completed no later than August 26, 2022. Following clearance by the FDA, the Company expects to initiate bioequivalence studies in accordance with the 505(b)(2) regulatory pathway. IkT-001Pro will be evaluated in a two-part dose finding/dose equivalence study in up to 62 healthy volunteers. The study is designed to evaluate the steady-state pharmacokinetics of IkT-001Pro and determine the dose of IkT-001Pro equivalent to 400 mg imatinib mesylate, the standard-of-care dose for SP-CML. Inhibikase expects to initiate this two-part bioequivalence study in the normal course following review of the proposed clinical study by the FDA. Inhibikase will confer with the FDA once bioequivalence is established to begin the New Drug Application, or NDA, process on the proposed approval path for IkT-001Pro under the 505(b)(2) statute. The Company plans to simultaneously pursue a superiority study comparing the selected dose of IkT-001Pro to standard-of-care 400 mg imatinib mesylate in SP-CML patients using a novel two-period, wait-list-crossover-switching study.

Evaluating ongoing studies of IkT-148009 in Animal Models of Multiple System Atrophy (MSA): Inhibikase continues to advance regulatory activities IkT-148009 in both the U.S. and EU27 for the treatment of MSA. The Company is preparing regulatory filings in the US and EU27 to enable the planned Phase 2 MSA trial if IkT-148009 is validated to be active in MSA in animal model studies. Execution of this trial will require the Company to raise additional working capital. Two animal model studies are ongoing and a preliminary assessment in one of the two studies demonstrated an apparent functional benefit following treatment.

Receipt of new patent # 11,407,747 entitled Compositions and methods for inhibiting kinases: This patent was issued August 9, 2022 for the application of the Company’s c-Abl kinase inhibitor portfolio to the treatment of cancer or bacterial or viral infections. This patent is the fifth U.S. patent issued for our portfolio of compounds that inhibit Abelson Tyrosine Kinases for a therapeutic purpose.
Second Quarter 2022 Financial Results

Net Loss: Net loss for the second quarter ended June 30, 2022, was $4.6 million, or $0.18 per share, compared to a net loss of $ 2.6 million, or $0.22 per share for the second quarter in 2021.

Net loss for the six months ended June 30, 2022, was $9.3 million or $0.37 per share, compared to a net loss of $5.3 million, or $0.47 per share in the six months ended June 30, 2021.

R&D Expenses: Research and development expenses were $3.0 million for the second quarter ended June 30, 2022, compared to $2.4 million in the second quarter of 2021. The increase of $0.6 million was driven by a $2.1 million increase in non-grant related research offset by a decrease of $1.4 million in grant related research expenditures and a decrease of $0.1 million in non-cash stock compensation expense. The non-grant related research was expended primarily in connection with the Company’s PD clinical trials.

Research and development expenses were $6.0 million for the six months ended June 30, 2022 compared to $4.8 million in the comparable period in 2021. The $1.2 million increase was driven by a $4.3 million increase in non-grant related research offset by a decrease of $2.7 million in grant related research expenditures and a decrease of $0.4 million in non-cash stock compensation expense. The non-grant related research was expended primarily in connection with the Company’s PD clinical trials.

SG&A Expenses: Selling, general and administrative expenses for the quarter ended June 30, 2022 were $1.7 million compared to $1.6 million for the second quarter in 2021. The increase was primarily from increased headcount resulting in increased compensation expense of $0.1 million, increased legal fees, board fees, investor relation and consulting fees of $0.3 million and a net increase of $0.1 million for other normal operating expenses offset by decreased non-cash stock based compensation expense of $0.4 million.

Selling, general and administrative expenses for the six months ended June 30, 2022 were $3.3 million compared to $3.2 million for comparable period in 2021. The increase was primarily from increased headcount resulting in increased compensation expense of $0.3 million, increased legal fees, board fees, investor relation and consulting fees of $0.6 million and a net decrease of $0.1 million for other normal operating expenses offset by decreased non-cash stock based compensation expense of $0.7 million.

Cash Position: Cash and cash equivalents were $32.2 million as of June 30, 2022. The Company expects that existing cash and cash equivalents will be sufficient to fund its normal operations and capital expenditure requirements through December 31, 2023.

Conference Call Information
The conference call is scheduled to begin at 8:00am ET on Monday, August 15, 2022. Participants should dial 844-825-9789 (United States) or 412-317-5180 (International) with the conference code 10169366. A live webcast may be accessed using the link here, or by visiting the investors section of the Company’s website at www.inhibikase.com. After the live webcast, the event will be archived on Inhibikase’s website for approximately 90 days after the call.

On August 12, 2022 Thermo Fisher Scientific reported The U.S. Food and Drug Administration (FDA) has granted premarket approval to Thermo Fisher Scientific’s Oncomine Dx Target Test as a companion diagnostic (CDx) to identify patients whose tumors have a HER2 (ERBB2) activating mutations (SNVs & Exon 20 Insertion) in non-small cell lung cancer (NSCLC) who may be candidates for ENHERTU (fam-trastuzumab deruxtecan-nxki) (Press release, Thermo Fisher Scientific, AUG 12, 2022, View Source [SID1234618270]). ENHERTU is a specifically engineered HER2 directed antibody drug conjugate (ADC) being jointly developed and commercialized by Daiichi Sankyo and AstraZeneca.

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"To ensure patients and clinicians can readily access testing to inform care decisions, we are committed to making NGS accessible and easy to use so patients everywhere will be able to benefit from precision therapies, when indicated."

The FDA approved ENHERTU on August 11 for the treatment of adult patients with unresectable or metastatic non-small cell lung cancer (NSCLC) whose tumors have a HER2 (ERBB2) activating mutations, as detected by an FDA-approved test, and who have received a prior systemic therapy. The approval follows a Priority Review, granted by the U.S. FDA in April 2022.

Lung cancer is the leading cause of cancer death among both men and women in the United States.i For patients with metastatic NSCLC, prognosis is particularly poor, as only 8% of patients will live beyond five years after diagnosis.ii HER2-mutant NSCLC occurs in approximately 2-4% of patients with non-squamous NCSLC,iii and Enhertu is the first HER2-directed therapy for patients with NSCLC approved in the U.S.

"With care decisions increasingly made based on a tumor’s molecular profile, the FDA’s latest approval of ENHERTU in HER2 mutant metastatic non-small cell lung cancer and the additional approval of the Oncomine Dx Target Test as a companion diagnostic marks a significant step forward for precision oncology," said Garret Hampton, president, clinical next-generation sequencing and oncology at Thermo Fisher Scientific. "To ensure patients and clinicians can readily access testing to inform care decisions, we are committed to making NGS accessible and easy to use so patients everywhere will be able to benefit from precision therapies, when indicated."

The Oncomine Dx Target Test is currently the only distributed NGS CDx that has received regulatory approval and is available in 17 countries for 15 targeted therapies, covering more than 550 million lives globally. It is designed to simultaneously evaluate 23 genes associated with NSCLC. The test received its first approval by the FDA as a CDx in 2017. In the US alone, it is approved for seven targeted therapies for NSCLC and one for cholangiocarcinoma.

On August 12, 2022 Guardant Health, Inc. (Nasdaq: GH), a leading precision oncology company, reported that the U.S. Food and Drug Administration (FDA) has approved its Guardant360 CDx liquid biopsy test as a companion diagnostic (CDx) to select patients with unresectable or metastatic HER2-mutant non-small cell lung cancer (NSCLC) whose tumors have activating HER2 (ERBB2) mutations for treatment with ENHERTU (fam-trastuzumab deruxtecan-nxki), a HER2-directed antibody drug conjugate (ADC) jointly developed and commercialized by Daiichi Sankyo and AstraZeneca (Press release, Guardant Health, AUG 12, 2022, https://www.businesswire.com/news/home/20220811005816/en/Guardant-Health-Receives-FDA-Approval-for-Guardant360%C2%AE-CDx-as-Companion-Diagnostic-for-Daiichi-Sankyo-and-AstraZeneca%E2%80%99s-ENHERTU%C2%AE-for-Treatment-of-NSCLC-Patients-With-Activating-HER2-Mutations [SID1234618269]).

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The approval means that Guardant360 CDx, a next generation sequencing (NGS)-based assay that detects genomic alterations using circulating tumor DNA from blood, is validated as a CDx assay to identify NSCLC patients who have an activating HER2 mutation (SNVs and exon 20 insertions) and may benefit from treatment with ENHERTU. Mutations in the HER2 gene, also called ERBB2, drive approximately 2-4% of non-squamous NSCLC.1 Non-small cell lung cancer represents about 82% of all lung cancer,2 which is the leading cause of cancer death in the U.S.3

"This is great news for metastatic NSCLC patients with activating HER2 mutations, who now have, for the first time, an approved treatment for their cancer, but also the first blood-based companion diagnostic in Guardant360 CDx," said Helmy Eltoukhy, Guardant Health co-CEO. "We are proud to offer our Guardant360 CDx liquid biopsy as a companion diagnostic so that patients can access comprehensive genomic profiling to see if they are eligible to receive this therapy."

Guardant360 CDx

For oncologists, the FDA-approved Guardant360 CDx test provides comprehensive genomic results from a simple blood draw in seven days, helping them move beyond the limitations of tissue biopsies to rapidly obtain clinically relevant information in time to match patients to the optimal personalized treatment. Guardant360 CDx covers all genes recommended by the National Comprehensive Cancer Network, including those most relevant to clinical care and NSCLC treatment guidelines.

Since being introduced as a laboratory developed test (LDT), the Guardant360 test has become widely accepted for blood-based comprehensive genomic profiling with more than 300 peer-reviewed publications. It has been trusted by more than 12,000 oncologists, with more than 300,000 tests performed to date, and is broadly covered by Medicare and many private payers, representing over 200 million lives.

On August 12, 2022 Humanigen, Inc. (Nasdaq: HGEN) ("Humanigen"), a clinical-stage biopharmaceutical company focused on developing lenzilumab (LENZ), a first-in class antibody that neutralizes granulocyte-macrophage colony-stimulating factor (GM-CSF), reported financial results for the second quarter and six months ended June 30, 2022 (Press release, Humanigen, AUG 12, 2022, View Source [SID1234618268]).

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"We have made excellent progress on the strategic realignment announced in July. We have increased the number of sites for the PREACH-M study in Australia of lenzilumab in chronic myelomonocytic leukemia (‘CMML’), a rare blood cancer, and have interest from top oncology centers in the United States. We are on track to enroll the first patient in the RATinG study in the UK of lenzilumab in acute graft versus host disease (‘aGvHD’) and expect dosing soon," stated Cameron Durrant, Chairman and Chief Executive Officer, Humanigen. "Given the positive results from the company’s LIVE-AIR study and the survival trend observed in the ACTIV-5/BET-B study, we have interest from a global group of leading institutions and research networks to include lenzilumab in their large-scale, multinational studies of COVID-19. Tocilizumab and baricitinib demonstrated mortality benefit following inclusion in such studies despite having failed to do so in smaller studies. We are exploring the requirements for inclusion in these studies and plan to provide an update before the end of 2022. In addition, we are currently assessing requests for investigator-initiated trials (‘IIT’s’) of lenzilumab in combination with CAR-T therapies and plan to continue the development of ifabotuzumab (‘iFab’), an EpAh-3 targeted monoclonal antibody currently in Phase 1 development, as part of an antibody drug conjugate (‘ADC’), for certain solid tumors in Australia."

As recently announced, the company has strategically realigned its pipeline and resources with plans to accelerate the development of lenzilumab in CMML, for which the "PREcision Approach to Chronic Myelomonocytic Leukemia," or "PREACH-M" study, is already underway and to continue the "Risk Adapted Therapy in Acute GvHD," or " RATinG" study, in patients undergoing bone marrow transplant, that is expected to enroll its first patient in the third quarter of 2022. These studies are majority funded by the company’s partners. Under the realignment plan, the company will deemphasize the deployment of certain resources for the development of lenzilumab for COVID-19. The preliminary topline results from the Accelerating COVID-19 Therapeutic Interventions and Vaccines-5 ("ACTIV-5") and Big Effect Trial, in the "B" arm of the trial ("BET-B"), referred to as the ACTIV-5/BET-B trial did indicate that lenzilumab demonstrated a positive trend in mortality. The company continues to support National Institutes of Health’s ("NIH’s") further analysis of the data.

Second Quarter and Six Months Ended June 30, 2022 Financial Results

Net loss for the quarter ended June 30, 2022 was $30.1 million, or $0.43 per share, as compared to $70.8 million, or $1.20 per share, for the quarter ended June 30, 2021. The net loss for the six months ended June 30, 2022 was $51.4 million or $0.75 per share, as compared to $136.4 million or $2.45 per share for the six months ended June 30, 2021. The decrease in net loss for both periods was largely due to a decrease in expenses, mainly Research and Development ("R&D") expense. R&D expense decreased $36.6 million from $63.0 million for the three months ended June 30, 2021, to $26.4 million for the three months ended June 30, 2022 and decreased $79.2 million from $122.9 million for the six months ended June 30, 2021 to $43.7 million for the six months ended June 30, 2022. The decrease in R&D expense is primarily due to decreased lenzilumab manufacturing costs for the quarter ended June 30, 2022 of $34.6 million, and for the six months ended June 30, 2022 of $70.3 million.

Cash and Cash Equivalents

Net cash used in operating activities, net of balance sheet changes, was $44.8 million for the six months ended June 30, 2022. During the first half of 2022, the company sold shares of its common stock under its At-the-Market or "ATM" facility, raising net proceeds of approximately $21.8 million. As of June 30, 2022, the company had cash and cash equivalents of approximately $47.0 million. Subsequent to end of the quarter and through August 10, 2022, the company raised an additional $15.9 million under the ATM.

In July 2022, the company repaid the Term Loan with Hercules by prepaying $25.0 million of outstanding principal, together with approximately $1.7 million of accrued interest, fees and other amounts, due under the loan, terminating all obligations, liens and security interests thereunder. By retiring the Term Loan, the company reduced future cash payments for interest and enhanced its ability to generate additional liquidity from its intellectual property by removing the loan’s collateral requirements.

A summary of key financial highlights as of and for the three and six months ended June 30, 2022 and 2021 is as follows ($ in thousands):

About Lenzilumab

Lenzilumab is a proprietary Humaneered first-in-class monoclonal antibody that has been proven to neutralize GM-CSF, a cytokine of critical importance in the hyperinflammatory cascade, sometimes referred to as cytokine release syndrome, or cytokine storm. Humanigen believes that GM-CSF neutralization with lenzilumab also has the potential to treat patients with CMML and to reduce the hyper-inflammatory cascade known as cytokine release syndrome common to aGvHD. A study of lenzilumab is underway for patients with CMML exhibiting RAS pathway mutations. This study builds on evidence from a Phase 1 study, conducted by Humanigen, that showed RAS mutations are associated with hyper-proliferative features, which may be sensitive to GM-CSF neutralization. Lenzilumab will also be tested to assess its ability to prevent and/or treat aGvHD in patients undergoing allogeneic hematopoietic stem cell transplantation.