On November 3, 2022 Cimeio Therapeutics, a biotechnology company developing a novel approach to cell therapies, reported the acceptance of two abstracts for the company’s pipeline programs to be presented at the American Society of Hematology (ASH) (Free ASH Whitepaper) meeting in December 2022 in New Orleans (Press release, Cimeio Therapeutics, NOV 3, 2022, View Source;utm_medium=rss&utm_campaign=cimeio-therapeutics-announces-acceptance-of-ash-abstracts-detailing-pipeline [SID1234623097]).

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In addition, the company has strengthened its board of directors with the additions of Luca Santarelli, M.D., and Michael Tomsicek.

ASH abstracts
Cimeio’s SCIP platform (Shielded Cell & Immunotherapy Pairs) uses gene editing tools to insert novel protein variants into hematopoietic stem cells or other types of cells, allowing the cells to maintain their function while making them resistant to paired immunotherapy depletion. Cimeio’s platform has effectively shielded cells from depletion mediated by antibodies, T-cell engagers, ADCs, and CAR-T cells in preclinical studies. The company is advancing its first programs towards clinical development for genetic and malignant hematologic diseases.

The first abstract to be presented at the upcoming ASH (Free ASH Whitepaper) meeting is titled "Function-preserving single amino acid substitutions shield hematopoietic stem and progenitor cells from CD117 targeted immunotherapy in vivo." This describes how CD117-shielded HSPCs could enable higher, longer and/or repetitive antibody dosing, allowing for toxin-free conditioning, post-transplant adjustment of donor chimerism, and targeted treatment of minimal residual disease in CD117+ malignancies.

The second abstract is titled "Engineered Single Amino Acid Substitutions Protect Hematopoietic Stem and Progenitor Cells From CD123 Targeted Immunotherapy." These findings detail how single amino acid substitutions in CD123 introduced to HSPCs by CRISPR/Cas9-mediated HDR enable selective shielding from various immunotherapies, while preserving biophysical and functional properties.

"With the presentation of our first preclinical data we have demonstrated that our shielding technology works in vivo, and across two of our leading programs," said Cimeio CEO Thomas Fuchs. "Our CD117 shield results in no residual binding when paired with our highly potent CD117 antibody. This pair serves as the basis for a powerful combination that could transform HSCT for patients with severe genetic and malignant hematological diseases."

Board appointments
Dr. Santarelli is the founder and CEO of VectivBio, a clinical-stage biopharmaceutical company pioneering novel transformational treatments for severe rare conditions. He has more than 20 years of experience in academic and industrial R&D, having led drug programs in the areas of neuroscience, rare diseases, ophthalmology, metabolic and G.I., spanning the value chain from discovery research to commercialization. Previously, Dr. Santarelli served as Chief Executive Officer and co-founder of Therachon AG, which was acquired by Pfizer Inc. in 2019 for $810 million.

Prior to Therachon, he spent 12 years at Roche, most recently serving as the Senior Vice President and Head of Neuroscience, Ophthalmology and Rare Diseases, as well as Small Molecule Research at Roche in Switzerland. Dr. Santarelli completed his M.D. and Psychiatry Residency at the University of Turin, Italy, and a postdoctoral fellowship at Columbia University, focusing on the molecular mechanisms of psychiatric disorders and adult brain stem cells.

Mr. Tomsicek is the former CFO of CRISPR Therapeutics, a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases. Prior to joining CRISPR, Mr. Tomsicek served as CFO of Abiomed and before that was SVP and CFO at Cubist Pharmaceuticals.

Prior to Cubist, Mr. Tomsicek spent nearly eight years at General Electric Healthcare, as finance manager in global operations, and then as CFO of its ultrasound business. He holds a bachelor of science degree in engineering and a master of business administration, both from the University of Wisconsin.

"We are very pleased to welcome Luca and Mike to the Cimeio Board. They are both seasoned biotech executives who will be instrumental as we grow the company in this next phase," said Alex Mayweg, Ph.D., Chairman of Cimeio and Managing Director at founding investor Versant Ventures.

On November 3, 2022 Immutep Limited (ASX: IMM; NASDAQ: IMMP) ("Immutep" or "the Company"), a clinical-stage biotechnology company developing novel LAG-3 immunotherapies for cancer and autoimmune disease, reported a late-breaking abstract relating to its phase II TACTI-002 trial has been accepted for an oral presentation at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Annual Meeting 2022 (Press release, Immutep, NOV 3, 2022, View Source [SID1234623096]). The oral presentation will include new clinical data for eftilagimod alpha ("efti"), its first-in-class soluble LAG-3 protein, in combination with pembrolizumab in 1st line non-small cell lung cancer (NSCLC) patients. The 37th Annual SITC (Free SITC Whitepaper) meeting will be held in Boston, MA and virtually from 8 to 12 November 2022.

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Details of the presentation are as follows:

As already announced, initial data from the INSIGHT-003 clinical trial treating patients with various solid tumours with triple combination therapy of efti, anti-PD-1 therapy, and chemotherapy, will be presented in a poster presentation at the 37th Annual SITC (Free SITC Whitepaper) meeting. A Trial in Progress poster on the randomised Phase IIb TACTI-003 study of efti in combination with pembrolizumab as 1st line treatment of patients with recurrent or metastatic head and neck squamous cell carcinoma will also be presented. Further details on these poster presentations are available here.

Abstracts for all three presentations will be available in a Journal for ImmunoTherapy of Cancer Supplement, which will be published on 7 November 2022 at 8 am EST. The Abstracts will also be subsequently made available on the Investors section of Company’s website under Presentations.

About Eftilagimod Alpha (Efti)

Efti is Immutep’s proprietary soluble LAG-3 clinical stage candidate that is a first-in-class antigen presenting cell (APC) activator for the treatment of cancer, capitalising on LAG-3’s unique characteristics to stimulate both innate and adaptive immunity. Efti binds to and activates antigen presenting cells via MHC II molecules leading to expansion and proliferation of CD8+ (cytotoxic) T cells, CD4+ (helper) T cells, dendritic cells, NK cells, and monocytes. It also upregulates the expression of key biological molecules like CXCL10 that further boost the immune system’s ability to fight cancer.

Efti is under evaluation for a variety of solid tumours including non-small cell lung cancer (NSCLC), head and neck squamous cell carcinoma (HNSCC), and HER2–/HR+ metastatic breast cancer. Its favourable safety profile enables various combinations, including with anti-PD-[L]1 immunotherapy and/or chemotherapy. Efti has received Fast Track Designation in 1st line HNSCC and in 1st line NSCLC from the United States Food and Drug Administration (FDA).

About TACTI-002

TACTI-002 (Two ACTive Immunotherapies) is a Phase II clinical trial being conducted in collaboration with Merck & Co., Inc., Rahway, NJ, USA (known as "MSD" outside the United States and Canada). The study is evaluating the combination of Immutep’s eftilagimod alpha with MSD’s KEYTRUDA (pembrolizumab) in up to 189 patients with 2nd line head and neck squamous cell carcinoma (HNSCC) or non-small cell lung cancer (NSCLC) in 1st and 2nd line.

KEYTRUDA is a registered trademark of Merck Sharp & Dohme LLC, a subsidiary of Merck & Co., Inc., Rahway, NJ, USA.

On November 3, 2022 IVERIC bio, Inc. (Nasdaq: ISEE) reported financial and operating results for the third quarter ended September 30, 2022 and provided a general business update (Press release, Ophthotech, NOV 3, 2022, View Source [SID1234623095]).

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"With the positive data from our GATHER2 clinical trial reported in the third quarter reinforcing the positive data from GATHER1, avacincaptad pegol (ACP) became the first and only investigational therapy in geographic atrophy (GA) to achieve its 12-month, prespecified, primary endpoint in two independent pivotal, Phase 3 clinical trials," stated Glenn P. Sblendorio, Chief Executive Officer of Iveric Bio. "We are excited to announce that we are ahead of schedule in preparing our new drug application (NDA) for ACP for the treatment of GA and we are moving up our submission timeline to the end of this year."

"As we close out the year and look forward to 2023, we are working diligently to make ACP available to physicians and their patients with GA as expeditiously as possible, subject to regulatory review and approval," stated Pravin U. Dugel, MD, President of Iveric Bio. "We continue to build our U.S. launch readiness plan and prepare for potential commercialization of ACP. We also continue to explore future development opportunities for ACP in earlier patient populations and to invest in lifecycle initiatives such as sustained release delivery technologies for ACP."

Avacincaptad pegol (ACP also known as Zimura): Complement C5 Inhibitor
•In September 2022, the Company announced that GATHER2, the Company’s second Phase 3 clinical trial of ACP for the treatment of GA, met its pre-specified primary endpoint at 12 months with statistical significance and a favorable safety profile.

•Results from GATHER1, the Company’s first Phase 3 clinical trial of ACP for the treatment of GA, and GATHER2, as well as the Company’s Special Protocol Assessment with the U.S. Food and Drug Administration (FDA), provide the basis for an NDA, which the Company plans to submit to the FDA by the end of this year. The Company is also planning to submit a marketing authorization application (MAA) to the European Medicines Agency in 2023, subject to feedback from planned interactions with regulatory authorities in Europe.

•The GATHER2 topline results for ACP were presented in two oral sessions as part of the Retina Subspecialty Day at the American Academy of Ophthalmology 2022 Annual Meeting on September 30, 2022.

•The Company recently initiated an open-label extension (OLE) trial for patients who completed their month 24 visits in the GATHER2 trial, with the aim of providing patients access to ACP and collecting additional safety data. Patients will be treated with ACP for 18 months or until potential regulatory approval of ACP in the applicable region, whichever is earlier.

•The Company received favorable feedback from the FDA on its development plans for intermediate AMD. The Company is continuing to engage with the FDA regarding its development plans and strategy for this important patient population.

•In July 2022, a post-hoc analysis from the GATHER1 clinical trial was presented at the Annual Meeting of the American Society of Retina Specialists. In the post-hoc analysis, ACP was observed to be associated with a reduction in GA lesion growth compared to sham across all distances from the foveal center point.

•Patient enrollment in STAR, the Company’s Phase 2b screening clinical trial of ACP for the treatment of autosomal recessive Stargardt disease (STGD1), is ongoing.

IC-500: HtrA1 (high temperature requirement A serine peptidase 1 protein) Inhibitor
•The Company plans to conduct additional preclinical studies to optimize formulation, dosage and delivery of IC-500. As a result, the Company does not expect to submit an investigational new drug application for IC-500 mid-next year, as it had previously planned. The Company remains committed to this program and will provide additional information as it becomes available.

Gene Therapy Programs in Orphan Inherited Retinal Diseases (IRDs)
•As the Company focuses its efforts and resources on the development and potential commercialization of ACP, the Company is currently seeking potential collaborators or licensees 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).

Non-Dilutive Debt Financing Facility
In July 2022, the Company entered into a term loan debt financing facility with Hercules Capital, Inc. (Hercules Capital) and Silicon Valley Bank (SVB) providing the Company with total borrowing capacity of up to $250 million in non-dilutive debt financing. In July 2022, the Company borrowed $50 million at the close of the facility. The Company believes it has satisfied the first performance milestone under that term loan facility, which would allow it to borrow an additional $50 million. The Company plans to borrow this additional amount in the fourth quarter of 2022.

Third Quarter Financial Results and 2022 Cash Guidance

•As of September 30, 2022, the Company had $321 million in cash, cash equivalents and available for sale securities, which reflects the impact of its $50 million initial borrowing under its term loan debt financing facility with Hercules and SVB.

•The Company estimates its year-end 2022 cash, cash equivalents and available for sale securities to range between $265 and $275 million. The Company estimates that its cash, cash equivalents, available for sale securities and committed loan facilities will be sufficient to fund its planned capital expenditure requirements, debt service obligations and operating expenses through at least mid-2024. These estimates are based on the Company’s current business plan, including the continuation of its ongoing clinical development programs for ACP in GA and STGD1, including the recently initiated OLE trial, evaluating ACP for intermediate AMD, preparation and submission of an NDA and an MAA for ACP in GA, continuing preparations for potential commercialization of ACP in GA in the United States, pursuing DelSiTech’s silica-based sustained release delivery technology and exploring additional sustained release delivery technologies for ACP, and the advancement of its IC-500 development program as currently planned. These estimates do not include any potential new borrowings under the term loan facility with Hercules and SVB, including the $50 million that the Company plans to borrow in the fourth quarter of this year. Also excluded from these estimates

are any potential approval or sales milestones payable to Archemix Corp. or any potential expenses for actual commercial launch of ACP, such as associated sales force expenses, any additional expenditures related to potentially studying ACP in indications outside of GA, STGD1 and intermediate AMD, or resulting from the potential in-licensing or acquisition of additional product candidates or technologies, or any associated development the Company may pursue.

2022 Q3 Financial Highlights

•R&D Expenses: Research and development expenses were $25.0 million for the quarter ended September 30, 2022, compared to $17.9 million for the same period in 2021. For the nine months ended September 30, 2022, research and development expenses were $81.2 million compared to $60.0 million for the same period in 2021. Research and development expenses increased primarily due to the continued progress of the Company’s GATHER2 trial, increased manufacturing activities for ACP, and increases in personnel costs, including share-based compensation associated with additional research and development staffing, offset by decreases in costs associated with the Company’s gene therapy programs.

•G&A Expenses: General and administrative expenses were $17.5 million for the quarter ended September 30, 2022, compared to $6.6 million for the same period in 2021. For the nine months ended September 30, 2022, general and administrative expenses were $45.8 million compared to $21.7 million for the same period in 2021. General and administrative expenses increased primarily due to 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 September 30, 2022, of $42.4 million, or ($0.35) per diluted share, compared to a net loss of $24.6 million, or $(0.23) per diluted share, for the same period in 2021. For the nine months ended September 30, 2022, the Company reported a net loss of $126.2 million or ($1.05) per diluted share, compared to a net loss of $81.5 million or ($0.84) for the same period in 2021.

Conference Call/Webcast 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 November 3, 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 8170771. 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 6056402.

On November 3, 2022 NantHealth, Inc. (NASDAQ-GS: NH), a leading provider of enterprise solutions that help businesses transform complex data into actionable insights, reported financial results for its third quarter ended September 30, 2022 (Press release, NantHealth, NOV 3, 2022, View Source [SID1234623094]).

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"Net revenue was $16.6 million for the third quarter of 2022, representing the fourth consecutive quarter of top-line growth," said Ron Louks, Chief Operating Officer, NantHealth. "Moreover, our gross profit of $9.7 million and gross margin of 58 percent for the quarter were the highest since the fourth quarter of 2020.

"We expect to continue the positive momentum generated over the last several quarters. Recently, we signed a new customer, a leading provider of technology-enabled payment integrity, eligibility and related analytics services, to a four-year agreement for our NantHealth solutions. Importantly, we are also benefiting from a broadened product portfolio, with existing customers appreciating the value of our multiple solutions and services."

Software and Services Q3 Highlights:

Clinical Decision Support and Pre-Authorization (Eviti):
In October, secured a three-year contract extension with a large national commercial insurer providing coverage to over two million of their Medicaid members across multiple states. This client has opted to continue bringing unparalleled value to their operations by using Eviti Connect, and has used this renewal period to increase their solution set to include formulary redirection, further enabling value-based care
Announced Eviti Connect platform once again earned full URAC accreditation for Health Utilization Management through 2025. This designation demonstrates the highest level of commitment to quality healthcare
Maryland Physicians Care signed a two-year renewal agreement, ensuring it continues to receive a unique and tailored solution for its members with autoimmune diseases
Added new capability to Eviti Connect, which allows payer clients to comply seamlessly with state-mandated preferred drug lists, eliminating the need for manual reviews and secondary processing when those preferred drugs are prescribed
Payer Engagement (NaviNet):
Secured an agreement with an existing customer, one of America’s leading health insurance organizations, which added 750,000 new members to NaviNet
Went live with NaviNet Open Prior Authorization for a key customer. This capability is expected to streamline workflows and data collection to enable automated decisions and reduced manual processes. The initial deployment has been completed with further roll outs planned in Q4
Added new platform functionality to speed up the prior authorization process, enabling more timely patient care. Updates include the ability for health plans to add direct-to-provider information within the request
Network Monitoring and Management (The OpenNMS Group, Inc.):
Completed shipment of small form factor OpenNMS mini appliances for user testing. Highly optimized for secure, scalable, and reliable distributed monitoring via the OpenNMS Minion component, these hardware appliances provide visibility into remote or private areas of enterprise networks
Made significant progress toward the development of a capability that will let customers monitor infrastructures built using VMware SD-WAN. This initiative marks a significant milestone in the OpenNMS Managed Service Provider (MSP) support program
Delivered several service engagements positioning OpenNMS as strategically significant within the clients’ IT organizations. Clients included a large national retail chain, an entertainment streaming service, and a top wireless communication provider
Third quarter Financial Results: 2022 vs 2021

For the 2022 third quarter:

Total net revenue was $16.6 million compared with $14.4 million.
Gross profit was $9.7 million, or 58% of total net revenue, compared with $7.5 million, or 52% of total net revenue.
Selling, general and administrative (SG&A) expenses increased to $16.6 million from $13.0 million.
Research and development (R&D) expenses increased to $6.3 million from $4.6 million.
Net loss attributable to NantHealth was $13.7 million, or $0.12 per share, compared with $10.8 million, or $0.09 per share.
On a non-GAAP basis, net loss from continuing operations was $14.0 million, or $0.12 per share, compared with $11.5 million, or $0.10 per share.
At September 30, 2022, cash and cash equivalents totaled $0.6 million.
Conference Call Information and Forward-Looking Statements

Later today, the company will host a conference call at 1:30 p.m. PT (4:30 p.m. ET) to review its results of operations for the third quarter ended September 30, 2022. The conference call will be available to interested parties by dialing 800-942-2493 from the U.S. or Canada, or 212-231-2931 from international locations. The call will be broadcast via the Internet at www.nanthealth.com. Listeners are encouraged to visit the website at least 10 minutes prior to the start of the scheduled presentation to register, download and install any necessary audio software. A playback of the call will be archived and accessible on the same website for at least three months.

Discussion during the conference call may include forward-looking statements regarding topics such as the company’s financial status and performance, regulatory and operational developments, and other comments the company may make about its future plans or prospects in response to questions from participants on the conference call.

On November 3, 2022 NextCure, Inc. (Nasdaq: NXTC), a clinical-stage biopharmaceutical company committed to discovering and developing novel, first-in-class immunomedicines to treat cancer and other immune-related diseases, reported third quarter 2022 financial results (Press release, NextCure, NOV 3, 2022, View Source [SID1234623093]).

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"Based on current efficacy data, we have decided to discontinue our clinical development of NC318. We will continue to support Yale University through its ongoing investigator initiated trial of NC318 in combination with pembrolizumab in lung cancer patients," said Michael Richman, NextCure’s president and chief executive officer. "We will focus our development efforts on the LAIR programs, including the recently initiated Phase 1b/2 combination trial of NC410 in solid tumors and the Phase 1 trial of NC525 that is expected to start in the first quarter of 2023. In addition, we have now initiated the NC762 expansion trial. These decisions are expected to add an additional 15 months to our cash runway and extend it into mid-2025."

Business Highlights

NC318 (S15 mAb)

●Based on the totality of the NC318 monotherapy data, including no responses in the amended Phase 2 portion of the trial, we have decided to discontinue development of our NC318 program.
●We will support Yale University’s ongoing NC318 investigator initiated combination trial.

NC410 (LAIR-2 fusion)

●Completed the Phase 1a dose escalation portion of the monotherapy trial and demonstrated that NC410 was safe and well tolerated.
●Initiated a Phase 1b/2 trial to evaluate NC410 in combination with pembrolizumab in patients with immune checkpoint refractory or naïve solid tumors.

NC525 (LAIR-1 mAb)

●Filed an Investigational New Drug (IND) application with the US Food and Drug Administration (FDA) and have received clearance to proceed with a Phase 1 trial.
●Plan to initiate a Phase 1 trial in the first quarter of 2023.
NC762 (B7-H4 mAb)

●Completed the Phase 1a dose escalation portion of the trial and demonstrated that NC762 was safe and well tolerated.
●Initiated the Phase 1b expansion trial.

Financial Guidance

●The decisions related to discontinuing NC318 and prioritizing NC410 in combination therapy are expected to add an additional 15 months to our cash runway.
●NextCure now expects its existing cash, cash equivalents and marketable securities will enable it to fund operating expenses and capital expenditures into mid-2025.

Financial Results for Quarter Ended September 30, 2022

●Cash, cash equivalents, and marketable securities, excluding restricted cash as of September 30, 2022, were $169.2 million as compared to $219.6 million as of December 31, 2021. The decrease of $50.4 million primarily related to cash used to fund operations, cash used to purchase fixed assets, and changes in the fair value of our marketable securities.
●Research and development expenses were $13.5 million for the quarter ended September 30, 2022, as compared to $13.6 million for the quarter ended September 30, 2021.
●General and administrative expenses were $5.7 million for the quarter ended September 30, 2022, as compared to $4.9 million for the quarter ended September 30, 2021. The increase of $0.8 million primarily related to higher personnel-related costs.
●Net loss was $18.9 million for the quarter ended September 30, 2022, as compared with a net loss of $17.9 million for the quarter ended September 30, 2021. The change in net loss from the previous year’s quarter was primarily due to higher general and administrative expenses and lower interest income.