On July 26, 2022 Brooklyn ImmunoTherapeutics, Inc. (Nasdaq:BTX) ("Brooklyn" or the "Company") reported that results from the INSPIRE phase 2 trial of IRX-2, a multi-cytokine biologic immunotherapy, in patients with newly diagnosed stage II, III, or IVA squamous cell carcinoma of the oral cavity (Press release, Brooklyn ImmunoTherapeutics, JUL 26, 2022, View Source [SID1234616939]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

The primary endpoint of the study was to estimate 2-year Event-Free Survival (EFS) with key secondary endpoints of Overall Survival (OS) and safety of IRX-2. One hundred and fifty patients were enrolled in the study. At two years of follow-up in the intention-to-treat (ITT, n=105) population the median EFS was 48.3 months and was not reached in the control arm (Hazard Ratio 1.10 (95% Confidence Interval, 0.6-2.1; p value=0.62)).

In key pre-specified subgroups defined by stage and type of adjuvant treatment, outcomes favored IRX-2. Patients in these subgroups were less likely to experience an EFS event in the IRX-2 arm compared to control. Trends in EFS rates as defined by the Kaplan-Meier estimate at two years of follow-up in patients with later stage (III and IV) disease were 57.2 (40.3, 70.9) vs 49.4 (28.3, 67.4) in favor of IRX-2. In patients that did not receive chemotherapy (radiation only) as part of adjuvant treatment, the EFS Kaplan-Meier estimate at two years of follow-up was 76.4 (52.2, 89.4) vs 60.6 (29.4, 81.4) in favor of IRX-2. There were no new safety signals observed with IRX-2. Adverse events related to study treatment were higher in the IRX-2 arm than the control arm (55.9% vs 40%) and were driven primarily by injection site reactions and fatigue.

"We thank the patients and their families for their participation in this trial," said Roger Sidhu, M.D., Brooklyn’s Chief Medical Officer. "IRX-2 immunotherapy treatment was administered as a local subcutaneous injection and was well tolerated in this patient population with squamous cell head and neck cancer of the oral cavity in the neoadjuvant setting. We observed compelling trends in favor of IRX-2 in patients with higher stage disease and those that did not receive chemotherapy as part of adjuvant treatment, representing patient populations with high unmet need and who comprise a significant proportion of patients with head and neck cancer. The mechanism of action of IRX-2 and prior preclinical and translational studies of IRX-2 suggest potential synergy with checkpoint inhibitors and represents a novel combination immunotherapy strategy to explore in patients that may not be eligible for or require intensive adjuvant treatment."

"The INSPIRE study achieved its primary objective of identifying patient populations that may benefit from IRX-2 in the neoadjuvant setting," said Matt Angel, Ph.D., Brooklyn’s CEO. "These encouraging results are a testament to the design of the INSPIRE study and provide a clear path forward for testing in patient populations that may benefit from treatment with IRX-2 in combination with checkpoint inhibitors. The potential to offer an effective, well tolerated treatment to patients with advanced head and neck cancer who are ineligible for chemotherapy is particularly exciting."

The Company plans to present the results of the INSPIRE study at a scientific conference later this year.

About IRX-2
IRX-2 is a primary cell-derived multi-cytokine biologic immunotherapy in development for multiple solid tumor indications. IRX-2 is administered locally by subcutaneous injection and is designed to activate T cells to generate an anti-tumor response.

About the INSPIRE Study
The INSPIRE study is a Company-sponsored, randomized (2:1 IRX-2 vs control), open label, phase 2 estimation trial of neoadjuvant therapy with IRX-2 compared to standard of care in patients with newly diagnosed stage II, III, or IVA squamous cell carcinoma of the oral cavity.

On July 26, 2022 Exelixis, Inc. (Nasdaq: EXEL) reported that its second quarter 2022 financial results will be released on Tuesday, August 9, 2022 after the markets close (Press release, Exelixis, JUL 26, 2022, View Source [SID1234616938]). At 5:00 p.m. ET / 2:00 p.m. PT, Exelixis management will host a conference call and webcast to discuss the results and provide a general business update. Access to the event is available via the Internet from the company’s website.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

To access the webcast link, log onto www.exelixis.com and proceed to the News & Events / Event Calendar page under the Investors & Media heading. Please connect to the company’s website at least 15 minutes prior to the conference call to ensure adequate time for any software download that may be required to listen to the webcast. Alternatively, please call 888-338-9509 (domestic) or 412-902-4281 (international) and ask to be joined into the Exelixis conference call to participate by phone.

A telephone replay will be available until 8:00 p.m. ET on August 11, 2022. Access numbers for the telephone replay are: 877-344-7529 (domestic) and 412-317-0088 (international); the passcode is 8698613. A webcast replay will also be archived on www.exelixis.com for one year.

On July 26, 2022 Enveric Biosciences, Inc. (NASDAQ: ENVB) ("Enveric" or the "Company"), a neuroscience-focused biotechnology company developing next-generation, psychedelic-inspired mental health medicines, reported that it has closed its previously announced registered direct offering for the purchase and sale of 375,000 shares of the Company’s common stock (or pre-funded warrants in lieu thereof), priced at a premium to market under Nasdaq rules (Press release, Enveric Biosciences, JUL 26, 2022, View Source [SID1234616937]). In addition, the Company has issued to the investors in the registered direct offering unregistered preferred investment options (the "preferred investment options") to purchase up to 375,000 additional shares of the Company’s common stock. The purchase price for one share of common stock (or pre-funded warrant) and one preferred investment option to purchase one share of common stock is $8.00. The preferred investment options have an exercise price of $7.78 per share, are immediately exercisable, and will expire five and one-half years from the date of issuance.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

The Company also closed its previously announced private placement offering for the purchase and sale of 625,000 shares of common stock (or pre-funded warrants in lieu thereof) and preferred investment options to purchase up to 625,000 shares of the Company’s common stock priced at a premium to market under Nasdaq rules. The purchase price for one share of common stock (or pre-funded warrant) and one preferred investment option to purchase one share of common stock is $8.00. The preferred investment options have an exercise price of $7.78 per share, are immediately exercisable, and will expire five and one-half years from the date of issuance.

H.C. Wainwright & Co. acted as the exclusive placement agent for the offerings.

The gross proceeds to the Company from the offerings were approximately $8 million, before deducting placement agent fees and other offering expenses payable by the Company. The Company intends to use the net proceeds from the offerings for working capital and general corporate purposes.

The Company also amended certain existing warrants to purchase up to an aggregate of 122,000 shares of common stock of the Company that were previously issued to the investors, with an exercise price of $27.50 per share and expiration date of February 15, 2027, effective upon the closings of the offerings so that the amended warrants have a reduced exercise price of $7.78 per share and expire five and one-half years following the closing of the offerings.

The shares of common stock, pre-funded warrants and shares of common stock underlying the pre-funded warrants (but excluding the shares of common stock and pre-funded warrants issued in the private placement and the preferred investment options and the shares of common stock underlying the preferred investment options) offered by the Company in the registered direct offering were offered pursuant to a "shelf" registration statement on Form S-3 (File No. 333-257690) previously filed with the Securities and Exchange Commission (the "SEC") on July 2, 2021, and declared effective by the SEC on July 9, 2021. The offering of the common stock and pre-funded warrants in the registered direct offering was made only by means of a prospectus, including a prospectus supplement, forming a part of the effective registration statement. A final prospectus supplement and accompanying prospectus relating to the securities being offered was filed with the SEC. Electronic copies of the final prospectus supplement and accompanying prospectus may be obtained on the SEC’s website at View Source and may also be obtained by contacting H.C. Wainwright & Co., LLC at 430 Park Avenue, 3rd Floor, New York, New York 10022, by phone at (212) 856-5711 or e-mail at [email protected].

The preferred investment options to purchase common stock, pre-funded warrants and the shares of common stock offered under the private placement, as well as the preferred investment options issued to investors in the registered directed offering, were offered in a transaction not involving a public offering and have not been registered under the Securities Act of 1933, as amended (the "Securities Act"), or applicable state securities laws. Accordingly, those securities may not be reoffered or resold in the United States except pursuant to an effective registration statement or an applicable exemption from the registration requirements of the Securities Act and such applicable state securities laws.

Under a registration rights agreement with the investors, the Company agreed to file a registration statement with the SEC covering the resale of the shares of the common stock and shares of common stock underlying the pre-funded warrants issued in the private placement and the shares of common stock underlying the preferred investment options issued in both offerings, within 15 days and to use best efforts to have the registration statement declared effective as promptly as practical and in any event within 45 days or within 75 days in the event of a full review by the SEC.

This press release shall not constitute an offer to sell or a solicitation of an offer to buy these securities, nor shall there be any sale of these securities in any state or other jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such state or other jurisdiction.

On July 26, 2022 IVERIC bio, Inc. (Nasdaq: ISEE) reported financial and operating results for the second quarter ended June 30, 2022 and provided a general business update (Press release, Ophthotech, JUL 26, 2022, View Source [SID1234616934]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

"We are excited to share that today marks one year since we completed patient enrollment of GATHER2, our second Phase 3 clinical trial for Zimura (avacincaptad pegol), a novel complement C5 inhibitor, for the treatment of geographic atrophy (GA)," stated Glenn P. Sblendorio, Chief Executive Officer of Iveric Bio. "Our team executed well all year as we exceeded our expectations for patient retention and injection fidelity, which we believe further de-risks GATHER2 and is an integral part of the clinical trial outcome. We look forward to reporting the topline data of GATHER2 in September of this year."

Mr. Sblendorio added, "In our topline data announcement, we intend to provide the primary efficacy endpoint analysis as well as a safety analysis, including the inflammation and endophthalmitis rates, if any, as well as rates of conversion to wet age-related macular degeneration (AMD), both using the traditional definition of a choroidal neovascular membrane (CNVM) as well as using the newer definitions of exudative versus non-exudative macular neovascularization (MNV), as defined in our Form 8-K filed on April 4, 2022."

"If the results from GATHER2 are positive, our key objective and plan is to make Zimura commercially available to physicians and their patients with GA as quickly as possible, assuming regulatory approval," stated Pravin U. Dugel, MD, President of Iveric Bio. "Over the past several months, exploratory post-hoc analyses from GATHER1, our first pivotal clinical trial for Zimura in GA, were presented at major medical conferences. We believe these analyses further support the consistency of the positive data previously reported for GATHER1 and inform future potential development opportunities for Zimura in earlier indications. We continue to invest in lifecycle initiatives such as sustained release delivery technologies for Zimura."

Zimura (avacincaptad pegol): Complement C5 Inhibitor

The Company expects topline data from the GATHER2 clinical trial to be available in September 2022. Following the Company’s topline announcement, the American Academy of Ophthalmology has reserved timeslots at its Annual Meeting on Friday, September 30th for presentations of the topline efficacy and safety results from GATHER2.
Patient retention for the GATHER2 clinical trial, as measured by the injection fidelity rate, continues to exceed the Company’s expectations. The Company achieved an injection fidelity rate for GATHER2, as measured through month 12, of 92.5%. As a comparison, the 12-month injection fidelity rate for its GATHER1 clinical trial, in which it observed a statistically significant reduction in GA progression at 12 months, was 87%. Injection fidelity is calculated by dividing the total number of actual injections (drug and sham) for all patients by the total number of expected injections (drug and sham) based on the total number of patients enrolled in the trial. The Company considers injection fidelity to be the most important and stringent measure of patient retention because it reflects the timely administration of the drug or sham into the patient’s eye.
In June 2022, the Company entered into a license agreement with DelSiTech Ltd. providing the Company with a worldwide, exclusive license to develop and commercialize new formulations of Zimura using DelSiTech’s silica-based sustained release technology.
The Company plans to initiate a clinical trial studying Zimura in patients with intermediate AMD in the fourth quarter of 2022, following planned interactions with the U.S. Food and Drug Administration (FDA) and other regulatory authorities. The Company’s development strategy in this indication is subject to regulatory feedback.
In July 2022, a post-hoc analysis from the Zimura GATHER1 clinical trial was presented at the Annual Meeting of the American Society of Retina Specialists. Results of the post hoc analysis showed that Zimura reduced GA lesion growth compared to sham across all distances from the foveal center point. In this trial, 84.4% of GA lesions were within 500 microns of the foveal center at baseline and 28.3% were within 100 microns of the foveal center at baseline. These results were generally well balanced across all treatment arms and their corresponding sham control groups.
In June 2022, a post-hoc analysis from the Zimura GATHER1 clinical trial was presented at the Macula Society Meeting. Results of the post-hoc analysis showed that optical coherence tomography (OCT)-measured GA area strongly correlated with fundus autofluorescence (FAF)-measured GA area, with minimal average differences in GA area between modalities. As typically used in GA clinical trials, FAF was utilized to measure GA in the GATHER1 clinical trial and is currently being used in the GATHER2 clinical trial. In GATHER1, a 30% reduction was observed in OCT-measured GA growth with Zimura at 12 months, which is consistent with findings using FAF-measured GA growth. The post-hoc analysis illustrates the potential for eye care providers to accurately diagnose and monitor patients with GA with OCT alone, without additional equipment required.
In May 2022, a post-hoc analysis from the Zimura GATHER1 clinical trial was presented at the Retinal World Congress. The post-hoc analysis evaluated the reduction in GA lesion growth observed for patients receiving Zimura as compared to patients receiving sham in a subset of patients based on the distance of a patient’s GA lesion from the foveal center at baseline. Consistent with previously reported GATHER1 results, the results of this post-hoc analysis showed that Zimura reduced GA lesion growth compared to sham across all baseline distances from the foveal center, and that early administration with Zimura when the GA lesion is still farther away from the foveal center and is growing the fastest, may be most beneficial.
Patient enrollment in STAR, the Company’s Phase 2b screening clinical trial of Zimura for the treatment of autosomal recessive Stargardt disease (STGD1), is ongoing. The results of this clinical trial are expected after the topline results of GATHER2.
IC-500: HtrA1 (high temperature requirement A serine peptidase 1 protein) Inhibitor

The Company is planning for IND-enabling toxicology studies for IC-500. The Company expects to submit an investigational new drug application (IND) to the FDA for IC-500 during mid-2023.
Gene Therapy Programs in Orphan Inherited Retinal Diseases (IRDs)

As the Company focuses its efforts and resources on the development and potential commercialization of Zimura, the Company is currently seeking potential collaborations for the future development and potential commercialization of IC-100, the Company’s product candidate for Rhodopsin-Mediated Autosomal Dominant Retinitis Pigmentosa (RHO-adRP) and IC-200, the Company’s product candidate for BEST1-Related IRDs.
The Company is continuing its minigene programs for Leber’s Congenital Amaurosis type 10 (CEP290), autosomal recessive Stargardt Disease (ABCA4) and Usher’s syndrome (USH2A).
Corporate Update

In July 2022, the Company entered into a term loan debt financing facility with Hercules Capital, Inc. (Hercules) and Silicon Valley Bank (SVB) providing the Company with total borrowing capacity of up to $250 million in non-dilutive debt financing. The Company is borrowing $50 million under this facility in July 2022, with an additional $150 million in the aggregate being available subject to the Company’s achievement of specified performance milestones relating to development and regulatory events for Zimura and an additional $50 million being available subject to the lenders’ approval.
Second Quarter Financial Results and 2022 Cash Guidance

As of June 30, 2022, the Company had $312 million in cash, cash equivalents and available for sale securities.
Today, the Company is borrowing $50 million under its term loan facility with Hercules and SVB. Including the proceeds of this borrowing, the Company estimates its year-end 2022 cash, cash equivalents and available for sale securities to range between $260 and $270 million.
The Company plans to provide additional information regarding its financing strategy following the GATHER2 topline data announcement.
2022 Q2 Financial Highlights

R&D Expenses: Research and development expenses were $33.6 million for the quarter ended June 30, 2022, compared to $23.5 million for the same period in 2021. For the six months ended June 30, 2022, research and development expenses were $56.2 million compared to $42.0 million for the same period in 2021. Research and development expenses increased primarily due to the continued progress of the GATHER2 trial, increased manufacturing activities for Zimura, and increases in personnel costs, including share-based compensation associated with additional research and development staffing, offset by decreases in costs associated with IC-100 and IC-200.
G&A Expenses: General and administrative expenses were $16.1 million for the quarter ended June 30, 2022, compared to $6.7 million for the same period in 2021. For the six months ended June 30, 2022, general and administration expenses were $28.2 million compared to $15.0 million for the same period in 2021. General and administrative expenses increased primarily due increases in personnel costs, including share-based compensation associated with staffing for commercial preparation.
Net Loss: The Company reported a net loss for the quarter ended June 30, 2022, of $49.3 million, or ($0.41) per diluted share, compared to a net loss of $30.1 million, or $(0.32) per diluted share, for the same period in 2021. For the six months ended June 30, 2022, the Company reported a net loss of $83.8 million or ($0.70) per diluted share, compared to a net loss of $56.9 million or ($0.61) for the same period in 2021.
Conference Call/Web Cast Information

Iveric Bio will host a conference call/webcast to discuss the Company’s financial and operating results and provide a business update. The call is scheduled for July 26, 2022, at 8:00 a.m. Eastern Time. To participate in this conference call, dial 1-888-317-6003 (USA) or 1-412-317-6061 (International), passcode 0932671. A live, listen-only audio webcast of the conference call can be accessed on the Investors section of the Iveric Bio website at www.ivericbio.com. A replay will be available approximately two hours following the live call for two weeks. The replay number is 1-877-344-7529 (USA Toll Free), passcode 6585313.

On July 26, 2022 LifeArc reported Scientists have discovered an IL-17B receptor (also known as IL-25 receptor) antibody could be used to treat colorectal cancer (Press release, LifeArc, JUL 26, 2022, https://www.lifearc.org/news/2022/study-reveals-potential-new-way-to-treat-colorectal-cancer/ [SID1234616933]). These latest findings follow the recent news that the humanised version of this antibody will also enter clinical trials for asthma.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

The study was published in the journal Science Immunology in June.

Blocking rogue cells
Kovilen-Sawmynaden-2017Building upon our long-standing relationship with the MRC Laboratory of Molecular Biology (LMB), paper co-author Dr Kovilen Sawmynaden and his team supplied Andrew McKenzie’s group with large quantities of antibody.

The LMB wanted to find out what blocking the IL-25 receptor would do to immune cells called group 2 innate lymphoid cells (ILC2s). ILC2s are interesting because they can protect against parasitic infections and are involved in allergies, but we don’t yet understand how they react to cancer.

In this study the scientists discovered that during cancer, these cells allow tumours to grow by recruiting other cells which ‘turn down the dial’ on the immune response, stopping the immune system fighting the cancer. But in mice treated with the anti-IL-25 receptor antibody to block the receptor and stop ILC2 cells boosting immune-suppressing cells, the immune system was able to fight the tumour.

Manufacturing antibodies at scale
Significant quantities of both the anti-IL-25 receptor antibody and our in-house control antibody were prepared at LifeArc by in-house protein production labs. This is staffed by a specialist team of cell culture, protein purification and biophysical characterisation scientists.

All antibodies were then pyrogen (endotoxin)-tested and screened for activity at LifeArc, before being shipped to the LMB for scientists to use in the mouse models.

Kovilen said: "We’re really pleased we have been able to supply key antibodies to the MRC for this work. It is another good example of LifeArc’s translational scientists and our in-house MRC support team assisting the MRC, one of our key partners. It is particularly exciting to see they have identified a potentially new indication for their anti-IL-17B receptor antibody."

The study was funded by the Wellcome Trust, the Horizon 2020 Framework programme, and the Medical Research Council.