On March 24, 2022 InflaRx N.V. (Nasdaq: IFRX), a clinical-stage biopharmaceutical company developing anti-inflammatory therapeutics by targeting the complement system, reported financial and operating results for the year ended December 31, 2021 (Press release, InflaRx, MAR 24, 2022, View Source [SID1234610907]).

Prof. Niels C. Riedemann, Chief Executive Officer and Founder of InflaRx, commented: "We have made important progress in broadening and advancing our development activities over the course of 2021 and into 2022. This includes introducing a new program, the oral small molecule C5aR inhibitor, INF904, and moving vilobelimab into a new indication, cutaneous small cell carcinoma. In addition, we reported promising results with vilobelimab in pyoderma gangrenosum and ANCA-associated vasculitis, and we now await the Phase III topline results in our COVID-19 trial. We are hopeful for a positive outcome but, either way, we believe the results will help to further the overall understanding of this devastating disease."

He continued, "We are thankful for the corrected advice letter from the FDA related to vilobelimab development in hidradenitis suppurativa and will clarify our path forward in this debilitating disease in the coming months. We expect a busy year ahead as we work towards our goal of developing treatments to control inflammation and improve the lives of patients suffering from neutrophil-driven diseases."

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Recent Highlights and R&D Update

Vilobelimab for Hidradenitis Suppurativa (HS)

In February 2022, the Company received an advice letter from the FDA related to its Phase III program and its IND. The feedback to the clinical protocol indicated that the FDA recommends using the Hidradenitis Suppurativa Clinical Response Score ("HiSCR") as the primary endpoint in the Phase III trial. This FDA advice contrasted with the FDA advice provided to the Company in a Type A meeting held in the third quarter of 2021.

In the minutes from that Type A meeting, FDA provided advice on how to implement, name and validate the meaningfulness of the modified HiSCR, a new primary endpoint suggested by the Company, which would measure the reduction of all three types of inflammatory lesions in HS–inflammatory nodules, abscesses and draining tunnels. A reduction in draining tunnels is not captured by the HiSCR. In these minutes, the FDA did not recommend the traditional HiSCR as the primary endpoint measure. Based on the advice received in the Type A meeting, InflaRx announced in January 2022, the initiation of a Phase III program designed to study patients with moderate to severe HS suffering from actively draining tunnels. Because of the letter, InflaRx paused activities related to the Phase III trial.

In March 2022, the Company received a corrected advice letter from the FDA. The corrected letter states that the FDA advice received by the Company in February 2022 "contains errors." In this corrected letter, the FDA no longer recommends that the Company use the HiSCR as the primary endpoint for the chosen patient population but gives recommendations related to implementation of the modified HiSCR.

In light of this corrected advice from FDA, InflaRx believes that further development in HS is feasible. InflaRx is now evaluating next steps for the program, as the Phase III trial activities remain on hold while InflaRx evalutes next steps for the program.

Vilobelimab in Pyoderma Gangrenosum (PG)

InflaRx previously initiated an open label, multi-center Phase IIa exploratory study enrolling patients with moderate to severe PG in Canada, the United States and Poland. The study evaluated the safety and efficacy of vilobelimab in patients with PG.

In October 2021, InflaRx announced preliminary results from the third dosing cohort. At 2400mg biweekly, six of the seven patients achieved clinical remission with a PGA score of ≤1, which reflects a closure of the target ulcer. All patients in the third dosing cohort had elevated C5a levels at baseline that were continuously suppressed after initiation of vilobelimab.

In August 2021, InflaRx also reported data for ten evaluable patients in the first two dose cohorts at day 99. The patient in the second dosing cohort demonstrating complete target ulcer closure had been increased from the 1600mg dose group to the highest dose of 2400mg dose on day 57 of the study, and the ulcer closed after the dose escalation.

Final data will be presented as a late-breaker oral presentation on March 26th at the American Academy of Dermatology Association (AAD) Annual Meeting.

InflaRx plans to meet with the FDA this year to discuss the design for a pivotal study.

Vilobelimab for Severe COVID-19

In October 2021, InflaRx announced full enrollment in the Phase III part of the global Phase II/III trial evaluating vilobelimab in mechanically ventilated patients with COVID-19. A total of 369 patients across several countries, including in Europe, South America and other regions, were enrolled. Topline data for the 28-day mortality primary endpoint is expected to be available by the end of March 2022. The results from this Phase III trial will heavily influence our decision with respect to any future development of vilobelimab in COVID-19 and the larger strategic focus of the Company.

In October 2021, InflaRx announced that it had received a grant of up to €43.7 million from the German Ministry of Education and Research and the German Ministry of Health to support the Company’s development of vilobelimab for the treatment of severe COVID-19 patients. The initial tranche amounts to €25.8 million (approximately $29.9 million) and is structured as reimbursement of 80% of certain pre-specified expenses related to the clinical development and manufacturing of vilobelimab. The remainder of the grant will be awarded in three additional subsequent tranches, each conditional on reaching agreed-upon development and manufacturing-related milestones for the preceding tranche and structured as reimbursement for Company expenses. Individual tranches will not be paid if the preceding milestone of a tranche is not met. As of December 31, 2021, InflaRx had received €8.3 million of this grant funding.

Vilobelimab for ANCA-associated Vasculitis (AAV)

In May 2021, InflaRx reported topline data from the U.S. IXPLORE Phase II study of vilobelimab in AAV. The results indicated that vilobelimab, when added to the current standard of care, was well tolerated.

In November 2021, InflaRx reported topline data from the European Phase II IXCHANGE study of vilobelimab in AAV. The study achieved its principal objective, demonstrating comparable clinical response of vilobelimab to standard of care, while significantly reducing the need for glucocorticoid treatment in this life-threatening indication.

The Company plans to discuss the data from the U.S. and EU studies with regulatory authorities to determine next steps with this program.

Vilobelimab for Cutaneous Squamous Cell Carcinoma (cSCC)

InflaRx is developing vilobelimab for the treatment of PD-1/PD-L1 inhibitor resistant/refractory locally advanced or metastatic cSCC. InflaRx previously initiated an open label, non-comparative, two-stage, Phase II trial (NCT04812535) at sites in Europe, the United States and elsewhere. The study is investigating two independent arms: vilobelimab alone (Arm A) and vilobelimab in combination with pembrolizumab (Arm B). The trial is expected to enroll a total of approximately 70 patients.

In February 2022, the Company announced the start of the second dosing cohort of Arm B. The interim analysis in this arm, which is required to move to the second stage of the Phase II trial, is expected after ten patients have been treated and are evaluable for response assessment at the recommended Phase II dose level, which will be selected based on data from the safety run-in phase of the study. These data are expected to be available in the first quarter of 2023.

In parallel, enrollment continues in the monotherapy Arm A. Six patients are now enrolled in this arm. The interim analysis in Arm A required to proceed to the second stage is expected to be available after ten patients are evaluable for response assessment. These data are expected to be available in the third quarter of 2022.

INF904

InflaRx announced in January 2022 a new pipeline program, INF904, an oral small molecule inhibitor of C5aR. InflaRx has been granted a composition of matter patent for INF904 and associated compounds by the U.S. Patent and Trademark Office and has completed IND-enabling (preclinical) studies that demonstrated no obvious toxicological findings even in the highest dose groups in required GLP toxicity analyses.

InflaRx expects to initiate a Phase I program in the second half of 2022 and plans to study INF904 in complement-mediated, chronic autoimmune and inflammatory diseases where oral administration is the preferred choice for patients.

2021 Financial Highlights

Research and Development Expenses

InflaRx’s research and development expenses increased by €10.0 million in the year ended December 31, 2021 compared to the year ended December 31, 2020.

This increase is attributable to higher contract research organization (CRO) and contract manufacturing organization (CMO) costs from clinical trials in the amount of €8.4 million. This increase was primarily due to higher expense for the Phase III part of our COVID-19 trial and other running trials like Phase II clinical program in patients with AAV, the Phase II clinical program in patients with PG, the preparation of a Phase II clinical program in patients cSCC and ongoing manufacturing activities for clinical trial related materials.

In addition, a €1.5 million increase in employee-related costs was mainly caused by a €1.0 million increase in expenses from share-based compensation.

General and Administrative Expenses

InflaRx’s general and administrative expenses increased by €3.5 million to €12.0 million for the year ended December 31, 2021, from €8.5 million for the year ended December 31, 2020. This increase is primarily attributable to a €2.2 million increase in expenses from share-based compensation. Legal, consulting and audit fees and other expenses increased by €0.5 million to €2.1 million for the year ended December 31, 2021, mainly due to higher consulting and legal costs, mainly triggered by SOX implementation. The increase of other expenses by €0.4 million is primarily related to higher D&O insurance cost.

Net Financial Result

InflaRx’s net financial result increased by €2.0 million in the year ended December 31, 2021 compared to the year ended December 31, 2020. This net increase is mainly attributable to higher foreign exchange income, which increased by €1.9 million and lower foreign exchange expense, which decreased by €0.8 million. This effect was offset by lower interest income on marketable securities, which decreased by €0.8 million.

Net Loss

InflaRx incurred a net loss of €45.6 million, or €1.10 per common share, in the year ended December 31, 2021 compared to €34.0 million, or €1.3 per common share, in the year ended December 31, 2020. As of December 31, 2021, the Company’s total funds available were approximately €110.6 million, composed of €26.2 million of cash and cash equivalents and €84.4 million of financial assets.

Net Cash Used in Operating Activities

InflaRx’s net cash used in operating activities increased to €39.9 million in the year ended December 31, 2021, from €36.5 million in the year ended December 31, 2020, mainly due to the increase of research and development expenditures and higher personnel costs.

Additional information regarding these results and other relevant information is included in the notes to the financial statements as of December 31, 2021 in "Item 18. Financial Statements," which are included in InflaRx’s most recent annual report on Form 20-F as filed with the U.S. Securities and Exchange Commission.

About Vilobelimab (IFX-1):

Vilobelimab is a first-in-class monoclonal anti-human complement factor C5a antibody, which highly and effectively blocks the biological activity of C5a and demonstrates high selectivity towards its target in human blood. Thus, vilobelimab leaves the formation of the membrane attack complex (C5b-9) intact as an important defense mechanism, which is not the case for molecules blocking the cleavage of C5. Vilobelimab has been demonstrated to control the inflammatory response driven tissue and organ damage by specifically blocking C5a as a key "amplifier" of this response in pre-clinical studies. Vilobelimab is believed to be the first monoclonal anti-C5a antibody introduced into clinical development. Over 300 people have been treated with vilobelimab in clinical trials, and the antibody has been shown to be well tolerated. Vilobelimab is being developed for various indications, including hidradenitis suppurativa, ANCA-associated vasculitis and pyoderma gangrenosum, as well as severe COVID-19 and cutaneous squamous cell carcinoma (cSCC).

On March 24, 2022 Ocuphire Pharma, Inc. (Nasdaq: OCUP), a clinical-stage ophthalmic biopharmaceutical company focused on developing and commercializing therapies for the treatment of refractive and retinal eye disorders, reported financial results for the fourth quarter and year ended December 31, 2021 and provided a corporate update (Press release, Ocuphire Pharma, MAR 24, 2022, View Source [SID1234610904]).

"2021 proved to be a highly productive year for Ocuphire with 2022 setting up to be a more transformative year given our series of late-stage data read-outs in MIRA, LYNX and ZETA trials throughout the year, ending with our first planned NDA filing. We have an ambitious vision in ophthalmology targeting highly prevalent refractive and diabetic retinal diseases with our 2 lead small molecule drug candidates," said Mina Sooch, MBA, Founder and CEO of Ocuphire Pharma. "We are pleased to rapidly exceed enrollment in and complete 4 clinical trials across Nyxol and APX3330 in the first months of 2022. At our R&D Day in January, we reported for the first time positive Phase 2 results in presbyopia for Nyxol as a single-agent. With this new chronic opportunity for Nyxol alone as a pupil modulation agent, we can potentially realize synergies in presbyopia and NVD patients. We recently held a FDA Type-C meeting and gained clear guidance for the VEGA Phase 3 presbyopia program, for which we plan to initiate in mid-2022. With the successful enrollment of the 24-week study for our retinal candidate APX3330 and the continued favorable systemic and ocular safety profile that we shared at our recent R&D Day, we are also excited to lead the retinal landscape with an oral option for diabetic retinopathy patients and report our topline data from our placebo-controlled, double-masked, Phase 2b ZETA-1 trial in the second half of 2022."

Jay Pepose, MD, PhD, Ocuphire’s Medical Advisor and Board Member stated, "Ocuphire’s product candidates, if approved, would give eye care practitioners the ability to enhance their patients’ vision and overall experience. As a refractive surgeon, I am particularly excited about Nyxol for RM because there is currently no FDA approved commercially available product to treat this major clinical need and patient complaint. For presbyopia, I am impressed by Nyxol’s favorable tolerability profile and durable near vision improvements for at least 12 hours across a broad age of patients (40 to 64 years old) in the recent VEGA-1 Phase 2 trial. Nyxol is differentiated from the other miotics in the presbyopia landscape by its mechanism of action inhibiting the iris dilator muscle to achieve an optimal pupil size. Since Nyxol does not engage the iris sphincter or ciliary muscle, as a single drop, this may become a viable treatment option for presbyopia patients with high myopia, for whom miotics are contraindicated because of the risk of retinal detachment."

Cam Gallagher, Chairman of the Board for Ocuphire added, "In the past year, Ocuphire has elevated its profile within the ophthalmology and optometry medical community and we are delighted to have expanded our prestigious Medical Advisory Board to over 15 refractive and retinal KOLs. The team led by Mina has executed and delivered on several key clinical development milestones and set the momentum for a catalyst-rich 2022 that has the potential to build significant value for our company and shareholders."

Key Anticipated Future Milestones

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•Reversal of Mydriasis (RM):

•MIRA-3: Report topline results from the Phase 3 MIRA-3 registration trial at the end of 1Q 2022

•MIRA-4: Report topline results from pediatric safety trial in 2Q 2022

•New Drug Application (NDA): If the results are positive from the ongoing MIRA trials, expect to file an NDA with the FDA for Nyxol in RM indication in late 2022 with potential launch as first dilation reversal drop in 2H 2023

•Presbyopia: Initiate VEGA Phase 3 program in mid-2022 investigating Nyxol alone and Nyxol with 0.4% LDP as adjunctive treatment; and, if successful, expect to file an NDA in 2023

•Night Vision Disturbances (NVD): Report top-line results in 2Q 2022 from the Nyxol Phase 3 LYNX-1 trial

•Diabetic Retinopathy (DR) and Diabetic Macular Edema (DME): Report top-line results from the APX3330 Phase 2b ZETA-1 trial in 2H 2022

Fourth Quarter and Recent Business Highlights

Corporate

•In January 2022, the Company held an Investor R&D Day webinar that featured six ophthalmic Key Opinion Leaders (KOLs): Jay Pepose, MD, PhD, James Katz, MD and Mitchell Jackson, MD from refractive surgery, Paul Karpecki, OD from optometry, and David Boyer, MD and Peter Kaiser from retina practice areas who discussed the unmet needs in RM, presbyopia and DR being addressed by Ocuphire’s two late-stage clinical drug assets, Nyxol and APX3300. A replay of the event can be found on the company’s corporate website here.

•In December 2021, the Company strengthened its Medical Advisory Board with the addition of six world-class KOLs: David Brown, MD, FACS; David Lally, MD; Y. Ralph Chu, MD; James Katz, MD; Mitchell Jackson, MD; and Douglas Devries, OD.

Clinical Development

•In March 2022, the Company completed enrollment of 103 (target of 80-100) diabetic retinopathy patients in the ZETA-1 Phase 2b trial of first-in-class oral APX3330. Masked safety data from the trial, announced during the R&D Day event in January 2022, demonstrated a favorable safety profile, consistent with prior studies with additional exposure data in diabetic patients with retinal disease.

•In March 2022, the Company completed enrollment in MIRA-4 Trial for Nyxol in RM by enrolling 23 healthy (target of 20) pediatric subjects ages 3-11 years.

•In February 2022, the Company completed enrollment in MIRA-3 Pivotal Phase 3 Trial for Nyxol in RM, surpassing its enrollment target of 330 with 368 patients ages 12 years and over.

•In February 2022, Ocuphire held a Type-C meeting with the FDA from which it obtained guidance regarding the design of pivotal studies and clarification of the CMC and other data requirements for filing an NDA to seek approvals of Nyxol for the treatment of presbyopia, both as a single agent and with LDP as adjunct eye drops. This represents our third Type-C or Type-B End of Phase 2 meeting with FDA for the Nyxol program across indications.

•In January 2022, the Company completed enrollment of LYNX-1 Phase 3 Trial investigating Nyxol for the treatment of night vision disturbances (NVD) in 145 patients (target of 140).

•In January 2022, the Company announced new positive data from the VEGA-1 Phase 2 trial for Nyxol as a single agent in presbyopia, showing that one drop of Nyxol had statistically significant improvement in efficacy and long durability compared to placebo at 12 hours post-dosing. The Company plans to proceed with the Phase 3 VEGA program to potentially support 2 NDAs: Nyxol as a single drop and Nyxol with low-dose pilocarpine (LDP) as adjunctive treatment.

Presentations, Publications, and Conferences

•In February 2022, David Boyer, MD, presented at the Angiogenesis, Exudation, and Degeneration Conference, highlighting the favorable safety data from the ongoing ZETA-1 Phase 2 trial of APX3330 in DR.

•In February 2022, Inder Paul Singh, MD, presented at the Cataract Surgery: Telling It Like It Is Conference in Orlando. Dr. Singh presented the positive results from the completed VEGA-1 Phase 2 trial of Nyxol in presbyopia as a single agent and in combination with adjunctive LDP.

•In January 2022, Mina Sooch, Founder and CEO participated in the panel discussion titled "The Role of Gender Equality in Changing the Life Sciences Investment and Innovation Landscape" at the 11th LifeSci Partners Corporate Access Event.

•In November 2021, clinical data on Nyxol and APX3330 were presented at poster sessions at the American Academy of Ophthalmology (AAO) 2021 annual meeting held in New Orleans. In addition, Ocuphire presented new data on improvement in intermediate vision and Snellen equivalent near vision at the Eyecelerator@AAO 2021 conference. Ocuphire was one of two companies presenting clinical data for presbyopia at this meeting.

•In October 2021, the Company announced the publication of a review article titled "Inhibition of APE1/Ref-1 for Neovascular Eye Disease: From Biology to Therapy" in the Special Issue "Advances in Molecular Activity of Potential Drugs" of the International Journal of Molecular Sciences. The article underscores the role of the APE1/Ref-1 protein in pro-angiogenic pathways associated with neovascular eye disease including diabetic retinal diseases and age-related macular degeneration.

•In October 2021, the Company announced the publication of a review article in Cells titled "Potential Therapeutic Candidates for Age-Related Macular Degeneration" noting the potential of APX3330 (referred to as "E3330"). The authors conclude that APE1/Ref-1 inhibitors such as APX3330 could inhibit the abnormal blood vessel formation seen in AMD by reducing retinal endothelial cell proliferation, migration, and tube formation.

•In October 2021, Michael J. Allingham, MD, PhD presented at the 39th Annual Scientific Meeting of the American Society of Retina Specialists (ASRS) (Diabetic Retinopathy 1 Symposium) held in San Antonio, highlighting the favorable safety and tolerability data for APX3330 in over 300 healthy volunteers and cancer/hepatitis patients across 11 Phase 1 and Phase 2 studies. In addition, Mina Sooch, CEO, presented APX3330 history and the design of the ongoing Phase 2 trial in DR at the OIS Retina Innovation Summit@ASRS on October 7, 2021 in San Antonio, TX.

Fourth Quarter and Year Ended December 31, 2021 Financial Highlights

As of December 31, 2021, the Company had cash and cash equivalents of approximately $24.5 million. Based on current projections, management believes the current cash on hand will be sufficient to fund operations into the second quarter of 2023. Net cash used in operating activities for the quarter and year ended December 31, 2021 was $5.6 million and $19.4 million, respectively.

No collaboration revenue was recorded in the fourth quarter. Collaborative revenue was $0.6 million for the year ended December 31, 2021. Revenue was derived from the collaboration and license agreements with Processa and Biosense related to certain Rexhan products and technology transfers. There was no collaboration revenue recognized during the comparable prior year periods.

General and administrative expenses for the quarter and year ended December 31, 2021 were $1.4 million and $8.1 million, respectively, compared to $1.3 million and $2.8 million for the quarter and year ended December 31, 2020, respectively. The $5.3 million increase for the year over year periods was primarily attributable to administrative employee headcount, stock-based compensation, insurance, legal and settlement costs, costs associated with operating as a public company subsequent to the reverse merger, and professional services and other operating costs. General and administrative expenses included $0.3 million and $0.2 million in non-cash stock-based compensation expense during the quarters ended December 31, 2021 and 2020, respectively, and $1.1 million and $0.7 million in non-cash stock-based compensation expense during the years ended December 31, 2021 and 2020, respectively.

Research and development expenses for the quarter and year ended December 31, 2021 were $4.7 million and $15.2 million, respectively, compared to $4.3 million and $6.6 million for the quarter and year ended December 31, 2020, respectively. The $8.5 million increase for the year over year periods was primarily attributable to clinical trial expense, manufacturing activities to support clinical advancement of Nyxol and APX3330, consulting services as well as regulatory and other research and development efforts. Research and development expenses also included $0.2 million and $0.3 million in non-cash stock-based compensation expense during the quarters ended December 31, 2021 and 2020, respectively, and $0.8 million in non-cash stock-based compensation expense during each of the years ended December 31, 2021 and 2020.

The loss from operations for the quarter ended December 31, 2021 was $6.2 million, compared to $14.0 million for the quarter ended December 31, 2020. The loss from operations for the year ended December 31, 2021 was $22.7 million, compared to $20.0 million for the year ended December 31, 2020. Net loss for the quarter ended December 31, 2021 was $6.3 million, compared to $18.7 million for the quarter ended December 31, 2020. Net loss for the year ended December 31, 2021 was $56.7 million, compared to $24.6 million for the year ended December 31, 2020. Net loss per share for the quarter ended December 31, 2021 was $0.35, compared to $2.46 for the quarter ended December 31, 2020. Net loss per share for the year ended December 31, 2021 was $3.82, compared to $5.28 for the year ended December 31, 2020.

The fair value change in derivative and warrant liabilities was a non-cash expense of zero for the quarter ended December 31, 2021 compared to a non-cash expense of $1.6 million for the quarter ended December 31, 2020. The fair value change in derivative and warrant liabilities was a non-cash expense of $33.8 million for the year ended December 31, 2021 compared to a non-cash expense of $1.5 million for the year ended December 31, 2020.

For further details on Ocuphire’s financial results, refer to the Company’s Annual Report on Form 10-K for the year ended December 31, 2021 to be filed with the Securities and Exchange Commission.

On March 24, 2022 Lineage Cell Therapeutics, Inc. (NYSE American and TASE: LCTX), a clinical-stage biotechnology company developing allogeneic cell therapies for unmet medical needs, reported that Brian M. Culley, the Company’s Chief Executive Officer, will be presenting at the 2022 Virtual Growth Conference, presented by Maxim Group LLC and hosted by M-Vest (Press release, Lineage Cell Therapeutics, MAR 24, 2022, View Source [SID1234610901]). Mr. Culley will be participating in an "Ophthalmology Panel" hosted by Jason McCarthy, Ph.D., Senior Managing Director, Biotechnology, on March 28th, 2022 at 10am ET / 7am PT. Mr. Culley will also provide a corporate overview which will be available to investors on demand, starting on Monday March 28th, 2022.

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The live panel and on-demand presentation will be available to registered users directly through the M-Vest platform: https://m-vest.com/events/2022-virtual-growth-conference. Registration is required for conference participation. An archived webcast of the corporate presentation will also be available on the Events and Presentations page of the Lineage website. Additional videos are available on the Media page of the Lineage website.

On March 24, 2022 Protara Therapeutics, Inc. (Nasdaq: TARA), a clinical-stage company developing transformative therapies for the treatment of cancer and rare diseases, reported that the Company has dosed the first patient in ADVANCED-1, its Phase 1 clinical trial evaluating TARA-002, an investigational cell-based immunopotentiator, for the treatment of non-muscle invasive bladder cancer (NMIBC) (Press release, Protara Therapeutics, MAR 24, 2022, View Source [SID1234610899]).

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"NMIBC is one of the most recurrent and difficult to treat cancers with very limited treatment options," said Jesse Shefferman, Chief Executive Officer of Protara Therapeutics. "We are thrilled to have dosed the first patient in our Phase 1 study in NMIBC and look forward to exploring TARA-002’s full potential in this pressing area of high unmet need."

ADVANCED-1 is a Phase 1 dose-finding, open-label trial (NCT05085977) evaluating TARA-002 in treatment-naïve and treatment-experienced NMIBC patients with high-grade carcinoma in situ (CIS) and high-grade papillary tumors (Ta). In the initial dose escalation phase of the trial, patients will receive six weekly intravesical doses of TARA-002. The primary objective of the trial is to evaluate the safety, tolerability and preliminary signs of anti-tumor activity of TARA-002, with the goal of establishing a recommended dose for a planned Phase 2 clinical trial.

"While bladder cancer is the 6th most common cancer in the United States today, with NMIBC representing approximately 80% of diagnoses, treatment options for these patients remain limited," said Edward M. Messing, M.D., Professor of Urology, Oncology and Pathology at the University of Rochester, and a principal investigator for the study. "There is an urgent need for new therapeutic interventions for these patients, as there continues to be an increase in recurrence, progression and an escalated number of patients needing cystectomies."

About TARA-002

TARA-002 is an investigational cell therapy in development for the treatment of NMIBC and LMs for which it has been granted Rare Pediatric Disease Designation by the U.S. Food and Drug Administration. TARA-002 was developed from the same master cell bank of genetically distinct group A Streptococcus pyogenes as OK-432, a broad immunopotentiator marketed as Picibanil in Japan and Taiwan by Chugai Pharmaceutical Co., Ltd. Protara has successfully demonstrated manufacturing comparability between TARA-002 and OK-432.

When TARA-002 is administered, it is hypothesized that innate and adaptive immune cells within the cyst or tumor are activated and produce a strong immune cascade. Neutrophils, monocytes and lymphocytes infiltrate the abnormal cells and various cytokines, including interleukins IL-2, IL-6, IL-8, IL-10, IL-12, interferon (IFN)-gamma, tumor necrosis factor (TNF)-alpha, granulocyte colony-stimulating factor, and granulocyte-macrophage colony-stimulating factor are secreted by immune cells to induce a strong local inflammatory reaction and destroy the abnormal cells.

About Non-Muscle Invasive Bladder Cancer

Bladder cancer is the 6th most common cancer in the United States, with NMIBC representing approximately 80% of bladder cancer diagnoses. Approximately 65,000 patients are diagnosed with NMIBC in the United States each year. NMIBC is cancer found in the tissue that lines the inner surface of the bladder that has not spread into the bladder muscle.

On March 24, 2022 Wugen, Inc., a clinical-stage biotechnology company developing a pipeline of off-the-shelf cell therapies to treat a broad range of hematological and solid tumor malignancies, reported that management will participate in a fireside chat and presentation at the Innate Killer Summit 2022 being held in San Diego, CA from Wednesday, March 30 – Friday, April 1, 2022 (Press release, Wugen, MAR 24, 2022, View Source [SID1234610898]).

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The details of Wugen’s fireside chat and presentation are as follows:

• Format: Industry Leader’s Fireside Chat

Presenter: Dan Kemp, Ph.D., President and Chief Executive Officer, Wugen
Date & Time: Thursday, March 31, 2022 at 8:15 a.m. PT

• Format: Presentation titled "Characterizing an Ideal NK Cell Phenotype Leveraging Assays to Indicate Therapeutic Benefit"

Presenter: Ayman Kabakibi, Ph.D., Chief Operating Officer & Executive Vice President, Research & Development, Wugen
Date & Time: Thursday, March 31, 2022 at 11:30 a.m. PT

About WU-NK-101

WU-NK-101 is a novel immunotherapy harnessing the power of memory natural killer (NK) cells to treat liquid and solid tumors. Memory NK cells are hyper-functional, long-lasting immune cells that exhibit enhanced anti-tumor activity. This rare cell population has a superior phenotype, proliferation capacity, and metabolic fitness that makes it better suited for cancer therapy than other NK cell therapies. Wugen is applying its proprietary MonetaTM platform to advance WU-NK-101 as a commercially scalable, off-the-shelf cell therapy for cancer. WU-NK-101 is currently in development for acute myelogenous leukemia (AML) and solid tumors.