On May 6, 2021 Galapagos NV (Euronext & NASDAQ: GLPG) reported its unaudited Q1 results and operational highlights, which are further detailed in the Q1 2021 report available on the Galapagos website, www.glpg.com (Press release, Galapagos, MAY 6, 2021, View Source [SID1234579437]).

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"These last months, we completed a review of our portfolio and development plans with the goal to select a more risk-balanced pipeline. We decided to retain our focus on novel targets to address unmet medical needs in inflammation, fibrosis, and kidney diseases. We also remain fully committed to the launch of Jyseleca in Europe. Moving forward with confidence, we decided to:

Refocus our clinical pipeline by critically examining its risk profile and breadth;
Cut significant cost in the organization to support this re-sized pipeline development;
Task our business development team to identify and execute on a transformative opportunity.
We believe that our strong cash position, expert teams, and solid scientific foundation position us well for future growth," said Onno van de Stolpe, CEO of Galapagos.

Refocused pipeline
In the revision exercise, Galapagos set goals to focus and adjust the overall risk profile of its clinical pipeline. Consequently, we prioritized those assets with what we believe have enhanced chances of clinical success in our core therapeutic areas. As such, we announce:

We are testing our lead Toledo program ‘3970, a SIK2/3 inhibitor, in five Proof of Concept studies in different indications, and pending the outcome of the studies, we plan to roll out our further development plans in the second half of the year;
We selected an additional molecule from our Toledo program, SIK2/3 inhibitor ‘4876, as a candidate to accelerate from preclinical phase into clinical development;
We aim to progress our TYK2 inhibitor ‘3667 into Phase 2b;
We selected chitinase inhibitor ’4617 to progress to Phase 2 in IPF and decided to stop development of our other IPF molecule ’1205;
We stopped further work on ‘4059 for metabolic disease, given that this is not a core therapeutic area;
We discontinued our early research efforts in metabolic diseases and osteoarthritis; and
We challenged and fine-tuned our stage-gating process to advance compounds.

Commercial progress

We remain well on track in launching filgotinib in Europe. In the first quarter, we successfully completed the transitions of commercial and medical teams from Gilead in Germany, the UK, Spain, and Italy. We believe everything is in place to complete the final transitions from Gilead to us by year-end. Q1 also saw progress on access and reimbursement for filgotinib in rheumatoid arthritis (RA). Gilead submitted the new drug application in Japan for the treatment of ulcerative colitis (UC). We are encouraged by the primary endpoint outcome with the MANTA/RAy semen parameter studies as we await the Committee for Medicinal Products for Human Use (CHMP) opinion in UC.

Bart Filius, President and COO, added, "In line with our review, we decided to discontinue or cancel certain studies and consequently identified opportunities to reduce operational costs, for a total potential savings of €150M on a full-year basis. Roughly half of these savings will be realized in 2021, resulting in a 2021 cash burni guidance of between €580 million and €620 million. We are working towards a right-sized, refocused version of Galapagos, setting us on a path towards success with our first commercial product, new R&D opportunities, substantial clinical news flow, and a lengthened cash runway for validation of our early pipeline assets."

(*) The 2020 comparatives have been restated to consider the impact of classifying the Fidelta business as discontinued operations in 2020.

Details of the financial results
Due to the sale of our fee-for-service business (Fidelta) to Selvita on 4 January 2021 for a total consideration of €37.1 million (including customary adjustments for net cash and working capital), the results of Fidelta are presented as "Net profit from discontinued operations" in our consolidated income statements for the three months ended 31 March 2021 and 31 March 2020.

Revenues and other income from continuing operations
Our revenues and other income from continuing operations for the first three months of 2021 increased to €124.2 million compared to €103.6 million in the first three months of 2020. Our revenues from the Gilead collaboration in the first three months of 2021 (€113.7 million) related to (i) the exclusive access to our drug discovery platform (€57.8 million), (ii) the filgotinib revenue recognition (€55.3 million) and (iii) royalties (€0.7 million).

Our deferred income balance on 31 March 2021 includes €1.9 billion allocated to our drug discovery platform that is recognized linearly over 10 years, and €0.8 billion allocated for the filgotinib development (including considerations for the previous and the renegotiated collaboration combined) that is recognized over time until the end of the development period.

Results from continuing operations
We realized a net loss from continuing operations of €12.8 million for the first three months of 2021, compared to a net loss of €52.3 million for the first three months of 2020.

We reported an operating loss amounting to €50.8 million for the first three months of 2021, compared to an operating loss of €46.2 million for the same period last year.

Our R&D expenditure in the first three months of 2021 amounted to €130.0 million, compared to €115.5 million for the first three months of 2020. This increase was due to an increase in subcontracting costs primarily related to our filgotinib program, our Toledo program and other clinical programs, compensated by a decrease for ziritaxestat, the OA program with GLPG1972 and the program in atopic dermatitis (AtD) with MOR106. Furthermore, the increase in personnel costs is explained by a planned headcount increase following the growth in our activities, and increased cost of the subscription right plans. This factor, and the increased cost of the commercial launch of filgotinib in Europe, contributed to the increase in our S&M and G&A expenses, which were respectively €14.6 million and €30.4 million in the first three months of 2021, compared to respectively €9.8 million and €24.5 million in the first three months of 2020.

We reported a non-cash fair value gain from the re-measurement of initial warrant B issued to Gilead, amounting to €2.0 million, mainly due to the decreased implied volatility of the Galapagos share price and its evolution between 31 December 2020 and 31 March 2021.

Net other financial income in the first three months of 2021 amounted to €36.2 million, compared to net other financial income of €14.8 million for the first three months of 2020, which was primarily attributable to €45.5 million of currency exchange gain on our cash and cash equivalents and current financial investments in U.S. dollars, and to €6.5 million of negative changes in (fair) value of current financial investments and financial assets.

Results from discontinued operations
The net profit from discontinued operations for the three months ended 31March 2021 consisted of the gain on the sale of Fidelta, our fee-for-services business, for €22.2 million.

Group net results
We reported a group net profit for the first three months of 2021 of €9.4 million, compared to a group net loss of €50.6 million for the first three months of 2020.

Cash position
Current financial investments and cash and cash equivalents totaled €5,114.7 million on 31 March 2021, as compared to €5,169.3 million on 31 December 2020.

Total net decrease in cash and cash equivalents and current financial investments amounted to €54.6 million during the first three months of 2021, compared to a net decrease of €58.4 million during the first three months of 2020. This net decrease was composed of (i) €127.7 million of operational cash burn, (ii) offset by €2.3 million of cash proceeds from capital and share premium increase from exercise of subscription rights in the first three months of 2021, (iii) €3.6 million negative changes in (fair) value of current financial investments and €45.7 million of mainly positive exchange rate differences, (iv) €28.7 million cash in from disposal of subsidiaries, net of cash disposed.

Finally, our balance sheet on 31 March 2021 held a receivable from the French government (Crédit d’Impôt Rechercheiv) and a receivable from the Belgian Government for R&D incentives, for a total of both receivables of €142.3 million.

Outlook 2021
We anticipate several regulatory announcements on filgotinib as well as progress in our differentiated pipeline of novel target-based candidates.

We expect reimbursement decisions in most key European markets for filgotinib in RA this year, as we complete the transition to a full European commercial operation by year end. We anticipate a CHMP opinion and a European Commission decision for filgotinib in UC. We expect that our collaboration partner Gilead will complete recruitment for the global DIVERSITY Phase 3 trial in Crohn’s disease this year.

Within our broader inflammation portfolio, we expect to report topline results from several trials this year, including a Phase 1b trial with TYK2 inhibitor ‘3667 in psoriasis, and three Proof of Concept studies with lead Toledo candidate SIK2/3 inhibitor ‘3970 in psoriasis, UC, and RA.

Within our fibrosis portfolio, we expect to progress early clinical compounds with novel mechanisms of action, with the aim to develop novel treatments to help patients suffering from this debilitating condition.

Following the review of our plans for 2021, we give guidance for full year 2021 operational cash burn of €580 to €620 million.

First quarter report 2021

Galapagos’ financial report for the first three months ended 31 March 2021, including details of the unaudited consolidated results, is accessible via www.glpg.com/financial-reports.

Results of annual ordinary shareholders’ meeting

On 28 April 2021, Galapagos held its annual ordinary shareholders’ meeting. All agenda items were approved, including the re-appointments of Ms. Katrine Bosley and Dr. Raj Parekh as members of the supervisory board, and approval of the remuneration report. All documents relating to the shareholders’ meeting are posted on our website at View Source

Conference call and webcast presentation

Galapagos will conduct a conference call open to the public tomorrow, 07 May 2021, at 14:00 CET / 8 AM ET, which will also be webcasted. To participate in the conference call, please call one of the following numbers ten minutes prior to commencement:

A question and answer session will follow the presentation of the results. Go to www.glpg.com to access the live audio webcast. The archived webcast will also be available for replay shortly after the close of the call.

On May 6, 2021 Checkpoint Therapeutics, Inc. ("Checkpoint") (NASDAQ: CKPT), a clinical-stage immunotherapy and targeted oncology company, reported financial results for the first quarter ended March 31, 2021, and recent corporate highlights (Press release, Checkpoint Therapeutics, MAY 6, 2021, View Source [SID1234579436]).

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James F. Oliviero, President and Chief Executive Officer of Checkpoint, stated, "During the first quarter of 2021, we continued to advance the development of our two lead drug candidates while also enhancing our cash position. Enrollment in our registration-enabling study for cosibelimab in metastatic cutaneous squamous cell carcinoma ("mCSCC") is nearly complete and the study remains on track to report top-line results by year-end. With a successful study, we anticipate submitting our first application for marketing approval for cosibelimab next year."

Mr. Oliviero continued, "Additionally, our collaboration partner in Asia for olafertinib (CK-101), Neupharma Inc., continues to enroll patients as planned into a Phase 3, registration-enabling study in first-line, EGFR mutation-positive locally advanced or metastatic non-small cell lung cancer ("NSCLC"). We intend to meet with the FDA to discuss the ongoing Phase 3 study design and its potential use to support a New Drug Application submission in the United States."

Recent Corporate Highlights:

In March 2021, Checkpoint announced the formation of a Scientific Advisory Board comprised of industry thought leaders. Members include Wayne A. Marasco, M.D., Ph.D., Roy S. Herbst, M.D., Ph.D., F. Stephen Hodi, Jr., M.D., Bruce E. Johnson, M.D., David Miller, M.D., Ph.D., and Emily Ruiz, M.D., M.P.H.
During the first quarter of 2021, Checkpoint raised $23.9 million of net proceeds from the utilization of the Company’s At-the-Market Issuance Sales Agreement at an average price of $3.50.
Financial Results:

Cash Position: As of March 31, 2021, Checkpoint’s cash and cash equivalents totaled $60.0 million, compared to $40.8 million at December 31, 2020, an increase of $19.2 million.
R&D Expenses: Research and development expenses for the first quarter of 2021 were $4.2 million, compared to $2.6 million for the first quarter of 2020, an increase of $1.6 million. Research and development expenses for the first quarter of 2021 included $0.2 million of non-cash stock expenses, compared to $0.1 million in the first quarter of 2020.
G&A Expenses: General and administrative expenses for the first quarter of 2021 were $2.4 million, compared to $1.7 million for the first quarter of 2020, an increase of $0.7 million. General and administrative expenses for the first quarter of 2021 included $1.2 million of non-cash stock expenses, compared to $0.5 million for the first quarter of 2020.
Net Loss: Net loss attributable to common stockholders for the first quarter of 2021 was $6.5 million, or $0.09 per share, compared to a net loss of $3.3 million, or $0.06 per share, in the first quarter of 2020. Net loss for the first quarter of 2021 included $1.4 million of non-cash stock expenses, compared to $0.6 million for the first quarter of 2020.

On May 6, 2021 Celldex Therapeutics, Inc. (NASDAQ:CLDX) reported financial results for the first quarter ended March 31, 2021 and provided a corporate update (Press release, Celldex Therapeutics, MAY 6, 2021, View Source [SID1234579435]).

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"We are very encouraged by the recently reported positive interim results from our ongoing Phase 1b study of CDX-0159 in chronic inducible urticaria. These data demonstrated an 80% complete response rate and a well-tolerated safety profile, which we believe is a significant accomplishment in this complex disease setting with limited treatment options," said Anthony Marucci, Co-founder, President and Chief Executive Officer of Celldex Therapeutics. "Based on these exciting results, we recently amended the protocol to add a third cohort in cholinergic urticaria, a debilitating disease affecting mainly young adults, where passive or active sweating induces hives and can severely impair their quality of life. We are nearing completion of patient dosing across both the cold induced and symptomatic dermographism cohorts from this study and look forward to presenting updated results from these cohorts this summer."

Mr. Marucci continued, "We are also making progress advancing our bispecific platform, presenting promising preclinical data at AACR (Free AACR Whitepaper) that supports the development of clinical bispecific candidates that co-target ILT4 and PD-(L)1. We look forward to continuing these efforts while also exploring important targets controlling inflammation and auto-immune pathways."

Recent Pipeline Highlights

CDX-0159 – KIT Inhibitor Program

CDX-0159 is a humanized monoclonal antibody developed by Celldex that binds the KIT receptor with high specificity and potently inhibits its activity. The KIT receptor tyrosine kinase is expressed in a variety of cells, including mast cells, which mediate inflammatory responses such as hypersensitivity and allergic reactions. KIT signaling controls the differentiation, tissue recruitment, survival and activity of mast cells.

Celldex initiated a Phase 1b open label study designed to evaluate the safety of a single dose (3.0 mg/kg) of CDX-0159 administered intravenously in December of 2020. Up to 20 patients with cold contact urticaria (ColdU; n=10) or symptomatic dermographism (SD; n=10) who are refractory to antihistamines are being enrolled. Patients’ symptoms are induced via provocation testing that resembles real life triggering situations. Secondary and exploratory objectives include pharmacokinetic and pharmacodynamic assessments, including changes from baseline provocation thresholds, measurement of tryptase and stem cell factor levels, clinical activity outcomes (impact on urticaria symptoms, disease control, clinical response), quality of life assessments and measurement of tissue mast cells through skin biopsies. The study is being conducted by Dr. Marcus Maurer, Professor of Dermatology and Allergy at Charité – Universitätsmedizin in Berlin.

Interim data from this study were reported in late March. Fifteen out of 20 planned patients with antihistamine refractory CIndU had received a single intravenous infusion of CDX-0159 at 3 mg/kg, including nine patients with ColdU and six patients with SD. Safety results were reported for all 15 patients; activity results were reported for all patients assessed for at least 15 days/2 weeks after treatment (n=10; 7 ColdU and 3 SD). Patients had high disease activity as assessed by provocation threshold testing.

Eight of 10 patients, including all patients with ColdU, (7 ColdU; 1 SD) experienced a complete response (CR) as assessed by provocation threshold testing. One patient experienced a partial response (PR). All patients will continue to be assessed for response through week 12. CDX-0159 was generally well tolerated.
Enrollment is nearing completion in the ColdU and SD cohorts. Based on these compelling results, the study has been expanded to also include 10 patients with cholinergic urticaria and patient screening is expected to begin this month.
Updated results from additional patients with cold induced urticaria and symptomatic dermographism and long term follow up that continues to characterize magnitude and duration of treatment effect and their link to changes in tryptase levels are expected this summer.
Celldex initiated dosing in a Phase 1b multi-center study of CDX-0159 in chronic spontaneous urticaria (CSU) in October. This study is a randomized, double-blind, placebo-controlled clinical trial designed to assess the safety of multiple ascending doses of CDX-0159 in up to 40 patients with CSU who remain symptomatic despite treatment with antihistamines. Secondary and exploratory objectives include pharmacokinetic and pharmacodynamic assessments, including measurement of tryptase and stem cell factor levels and clinical activity outcomes (impact on urticaria symptoms, disease control, clinical response) as well as quality of life assessments. Results from the study are expected by the end of 2021.

CDX-0159 development is being expanded into prurigo nodularis (PN), a chronic skin disease characterized by the development of hard, intensely itchy (pruritic) nodules on the skin. Initiation of this study is planned for the fourth quarter of 2021.

Manufacturing activities are also progressing as planned to support the introduction of the CDX-0159 subcutaneous formulation into the clinical program in the third quarter of 2021.
CDX-1140 – CD40 Agonist Program

CDX-1140 is a potent CD40 human agonist antibody developed by Celldex that the Company believes has the potential to successfully balance systemic doses for good tissue and tumor penetration with an acceptable safety profile.

In the Phase 1 study of CDX-1140 in patients with recurrent, locally advanced or metastatic solid tumors and B cell lymphomas, both the monotherapy and combination with CDX-301 portions of the trial are complete. Expansion cohorts are actively recruiting including:

CDX-1140 with KEYTRUDA (pembrolizumab) in patients with squamous cell head and neck cancer and non small cell lung cancer who have progressed on checkpoint therapy; and,
CDX-1140 with standard of care chemotherapy in first line metastatic pancreatic cancer.

Updated data from this program are expected to be presented later this year.
CDX-527 – Bispecific Antibody Program

CDX-527 is the first candidate developed by Celldex from its bispecific platform which utilizes the Company’s proprietary highly active anti-PD-L1 and CD27 human antibodies to couple CD27 co-stimulation with blockade of the PD-L1/PD-1 pathway.

In August 2020, Celldex initiated a Phase 1 dose-escalation study in up to ~40 patients with advanced or metastatic solid tumors that have progressed during or after standard of care therapy to be followed by tumor-specific expansion cohorts. The study is designed to determine the MTD during a dose-escalation phase and to recommend a dose level for further study in the subsequent expansion phase. The expansion is designed to further evaluate the tolerability, and biologic and anti-tumor effects of selected dose level(s) of CDX-527 in specific tumor types. Initial data from the Phase 1 study focused on pharmacokinetic and pharmacodynamic properties have been accepted for presentation at the 2021 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting to be held June 4th-8th.
Preclinical Programs

In April 2021, Celldex presented promising data from the Company’s growing bispecific platform at the American Association of Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting. The Company described the discovery and characterization of ILT4 inhibitory monoclonal antibodies (mAbs) for engineering bispecific antibodies (bsAbs) that revert myeloid cell suppression by antagonizing ILT4 and activate T-cell responses through PD-(L)1 inhibition. Based on the results, Celldex is developing clinical bispecific candidates that co-target ILT4 and PD-(L)1. Celldex is also exploring important targets controlling inflammation and auto-immune pathways.

In direct support of our bispecific platform, Celldex has entered into a research and collaboration agreement with Biosion, Inc., a global biotechnology company focused on the discovery and clinical development of innovative biologicals for unmet medical needs, to construct and develop bispecific antibodies that combine undisclosed components from both Celldex and Biosion discoveries.
While Celldex’s clinical development programs have not been significantly, negatively impacted by COVID-19 to date, the Company continues to carefully monitor the evolving situation closely across all development programs and work to minimize potential impact/disruptions.

First Quarter 2021 Financial Highlights and 2021 Guidance

Cash Position: Cash, cash equivalents and marketable securities as of March 31, 2021 were $176.1 million compared to $194.4 million as of December 31, 2020. The decrease was primarily driven by first quarter cash used in operating activities of $18.1 million. At March 31, 2021, Celldex had 39.6 million shares outstanding.

Revenues: Total revenue was $0.7 million in the first quarter of 2021 compared to $2.7 million for the comparable period in 2020. The decrease in revenue was primarily due to the $1.8 million milestone payment received from Rockefeller University in the first quarter of 2020 related to Celldex’s manufacturing and development services agreement.

R&D Expenses: Research and development (R&D) expenses were $12.7 million in the first quarter of 2021 compared to $11.7 million for the comparable period in 2020. The increase in R&D expenses was primarily due to an increase in contract research and clinical trial expenses.

G&A Expenses: General and administrative (G&A) expenses were $4.1 million in the first quarter of 2021 compared to $3.7 million for the comparable period in 2020. The increase in G&A expenses was primarily due to higher stock-based compensation expense.

Changes in Fair Value Remeasurement of Contingent Consideration: During the quarter ended March 31, 2021, the Company recorded a $0.5 million loss on fair value remeasurement of contingent consideration primarily due to changes in discount rates and the passage of time.

Net Loss: Net loss was $16.5 million, or ($0.42) per share, for the first quarter of 2021 compared to a net loss of $12.6 million, or ($0.73) per share, for the comparable period in 2020.

Financial Guidance: Celldex believes that the cash, cash equivalents and marketable securities at March 31, 2021 are sufficient to meet estimated working capital requirements and fund planned operations through 2023.

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

On May 6, 2021 Aprea Therapeutics, Inc. (Nasdaq: APRE), a biopharmaceutical company focused on developing and commercializing novel cancer therapeutics that reactivate the mutant tumor suppressor protein, p53, reported financial results for the three months ended March 31, 2021 and provided a business update (Press release, Aprea, MAY 6, 2021, View Source [SID1234579431]).

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"During our recent R&D Day presentation, we reported an analysis of available data from its Phase 3 MDS trial. We identified an imbalance in dose modifications in the experimental arm which we believe negatively impacted efficacy, particularly the primary CR endpoint," said Christian S. Schade, Chairman and Chief Executive Officer of Aprea. "We remain confident that eprenetapopt and our next-generation oral agent, APR-548, represent important potential therapeutic options for cancer patients and are encouraged by emerging data from our ongoing clinical trials. We look forward to sharing data updates from these clinical trials as well as our continued progress in expanding the opportunity for our therapies in new indications."

Business Operations Update:

The Company is conducting, supporting, and planning multiple clinical trials of eprenetapopt (APR-246) and APR-548:

Phase 3 Frontline MDS Trial — In June 2020, the Company completed full enrollment of 154 patients in a pivotal Phase 3 trial of eprenetapopt with azacitidine for frontline treatment of patients with TP53 mutant MDS. The pivotal Phase 3 trial is supported by data from two Phase 1b/2 investigator-initiated trials, one in the U.S. and one in France, testing eprenetapopt with azacitidine as frontline treatment in TP53 mutant MDS and AML patients. The data from the U.S. and French Phase 1b/2 trials were published in The Journal of Clinical Oncology in January 2021 and February 2021, respectively. In December 2020, the Company announced that its pivotal Phase 3 trial failed to meet its predefined primary endpoint of complete remission (CR) rate. Analysis of the primary endpoint at this data cut demonstrated a higher CR rate (53% more patients achieving a CR) in the experimental arm receiving eprenetapopt with azacitidine versus the control arm receiving azacitidine alone but did not reach statistical significance. Based on a thorough analysis of the current Phase 3 trial data and comparisons to the U.S. and French Phase 1b/2 trials the Company believes that despite similar types and frequency of adverse events observed in the Phase 3 experimental arm and the Phase 1b/2 trials, patients in the Phase 3 experimental arm experienced substantially more study treatment dose modifications compared to the experience in the U.S. and French Phase 1b/2 trials. The Company believes that dose modifications of eprenetapopt and azacitidine led to undertreatment in the Phase 3 experimental arm that negatively impacted efficacy, particularly the primary endpoint of CR rate. The Company continues to follow patients who remain on-study and anticipates discussing with FDA the Phase 3 data and future possible regulatory pathways in the second half of 2021.
Phase 2 MDS/AML Post-Transplant Trial – The Company has completed enrollment of 33 patients in a single-arm, open-label Phase 2 clinical trial evaluating eprenetapopt with azacitidine as post-transplant maintenance therapy in TP53 mutant MDS and AML patients who have received an allogeneic stem cell transplant. The primary endpoint of the trial is the rate of relapse-free survival (RFS) at 12 months, with a published benchmark of ~30%. An interim analysis in April 2021 showed a 62% rate of RFS at 12 months, with a median RFS of 462 days. An interim analysis of overall survival (OS) showed a 77% OS at 1 year, with a median number of events not yet reached. The Company anticipates initial results from the primary endpoint of RFS at 12 months in the second quarter of 2021.
Phase 1/2 AML Trial – The Company is currently enrolling a Phase 1/2 clinical trial evaluating the safety, tolerability, and preliminary efficacy of eprenetapopt therapy in TP53 mutant AML patients. The lead-in portion of the trial evaluated the tolerability of eprenetapopt with venetoclax, with or without azacitidine, and no dose-limiting toxicities were observed in 12 patients receiving either regimen. Based on these results, the Company has expanded the trial to treat 33 additional frontline TP53 mutant AML patients with the combination of eprenetapopt, venetoclax and azacitidine. In the 19 frontline AML patients who are evaluable for efficacy with the triplet regimen, the Company has observed a 63% CR + CRi composite response rate and a 31% CR rate. The Company anticipates completion of enrollment in the triplet regimen expansion cohort during the second quarter of 2021 and availability of preliminary response rate data from the cohort also in the second quarter of 2021.
Phase 1 NHL Trial – The Company is currently enrolling a Phase 1 clinical trial in relapsed/refractory TP53 mutant chronic lymphoid leukemia (CLL) assessing eprenetapopt with venetoclax and rituximab and eprenetapopt with ibrutinib in order to further assess eprenetapopt in hematological malignancies. The first patient was enrolled in the first quarter of 2021. The Company is also planning to evaluate the combination of eprenetapopt with venetoclax in relapsed/refractory mantle cell lymphoma.
Phase 1/2 Solid Tumor Trial – The Company is currently enrolling a Phase 1/2 clinical trial in relapsed/refractory gastric, bladder and non-small cell lung cancers assessing eprenetapopt with anti-PD-1 therapy. The dose-escalation phase of the trial enrolled 6 patients with advanced solid tumors and no dose-limiting toxicities were observed. Based on these results, the Company is enrolling expansion cohorts for patients with advanced gastric, bladder and non-small cell lung cancers and has currently enrolled 15 patients across these expansion arms. A poster presentation for this trial has been accepted for presentation at the 2021 ASCO (Free ASCO Whitepaper) Annual Meeting (abstract TPS3161).
APR-548 Phase 1 Trial — The Company’s second product candidate, APR-548, is a next-generation p53 reactivator that is being developed in an oral dosage form. The Company has planned a Phase 1 dose-escalation clinical trial evaluating the safety, tolerability, and preliminary efficacy of APR-548 with azacitidine in frontline and relapsed/refractory MDS patients. The Company anticipates the first patient to be enrolled in the second quarter of 2021.
First Quarter Financial Results

Cash and cash equivalents: As of March 31, 2021, the Company had $77.6 million of cash and cash equivalents compared to $89.0 million of cash and cash equivalents as of December 31, 2020. The Company expects cash burn for the full year 2021 to be between $30.0 million $35.0 million. The Company believes its cash and cash equivalents as of March 31, 2021 will be sufficient to meet its current projected operating requirements into 2023.
Research and Development (R&D) expenses: R&D expenses were $6.8 million for the quarter ended March 31, 2021, compared to $9.1 million for the comparable period in 2020. The decrease in R&D expenses was primarily due to decreases in clinical trial costs for (i) our pivotal Phase 3 clinical trial of eprenetapopt with azacitidine for the frontline treatment of TP53 mutant MDS which completed enrollment in Q2 2020 and (ii) our Phase 2 post-transplant MDS/AML clinical trial. These decreases were partially offset by increases in clinical trial costs for our Phase 1/2 clinical trial for the treatment of TP53 mutant AML with venetoclax and azacitidine, our Phase 1/2 clinical trial in relapsed/refractory gastric, bladder and non-small cell lung cancers assessing eprenetapopt with anti-PD-1 therapy, and our Phase 1 clinical trial in relapsed/refractory TP53 mutant chronic lymphoid leukemia (CLL) assessing eprenetapopt with venetoclax and rituximab, and eprenetapopt with ibrutinib.
General and Administrative (G&A) expenses: G&A expenses were $3.4 million for the quarter ended March 31, 2021, compared to $2.8 million for the comparable period in 2020. The increase in G&A expenses was primarily due to increases in non-cash stock-based compensation and insurance expense.
Net loss: Net loss was $9.7 million, or $0.46 per share for the quarter ended March 31, 2021, compared to a net loss of $9.4 million, or $0.45 per share for the quarter ended March 31, 2020. The Company had 21,186,827 shares of common stock outstanding as of March 31, 2021.

On May 6, 2021 Aldeyra Therapeutics, Inc. (Nasdaq: ALDX) (Aldeyra), a clinical-stage biotechnology company focused on the development of novel therapies with the potential to improve the lives of patients with immune-mediated diseases, reported financial results for the quarter ended March 31, 2021 and provided recent business highlights (Press release, Aldeyra Therapeutics, MAY 6, 2021, View Source [SID1234579428]).

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"We expect 2021 to be a catalyst-rich year for Aldeyra as we continue to advance reproxalap, our lead program, toward potential commercialization in anterior ocular inflammatory disease," stated President and CEO Todd C. Brady, M.D., Ph.D. "We recently completed the Phase 3 INVIGORATE Trial of reproxalap, achieving statistically significant superiority over vehicle across all assessed signs and symptoms of allergic conjunctivitis, including ocular itching and redness. We look forward to meeting with the U.S. Food and Drug Administration in the second half of this year to discuss the INVIGORATE results and the potential submission of a New Drug Application (NDA). In addition, we remain on track to report top-line results in the second half of this year from the Phase 3 TRANQUILITY and TRANQUILITY-2 clinical trials of reproxalap in dry eye disease.

"We believe we continue to operate from a position of financial strength," Dr. Brady stated. "With the recent follow-on public offering, we expect to have sufficient capital to prepare reproxalap for NDA submission and a potential commercial launch, if approved, while investing in the clinical development of ADX-629, ADX-2191, and other product candidates in retinal and systemic immunological diseases with unmet medical need."

Recent Highlights and Program Updates

Primary, Key Secondary, and All Secondary Endpoints Met in Phase 3 INVIGORATE Allergic Conjunctivitis Clinical Trial: In the first-ever Phase 3 clinical trial of a novel investigational product in an allergen chamber, 0.25% reproxalap ophthalmic solution (reproxalap) demonstrated statistically significant improvement over vehicle for the primary endpoint of ocular itching (p<0.0001), the key secondary endpoint of ocular redness (p<0.0001), and the secondary endpoints of ocular tearing and total ocular severity score (each p<0.0001). The results of INVIGORATE, the second positive Phase 3 trial for reproxalap in allergic conjunctivitis, indicate potential clinical utility before and during exposure to moderate to high levels of pollen.
Phase 3 TRANQUILITY and TRANQUILITY-2 Dry Eye Disease Trial Results Expected in Second Half of 2021: Patient enrollment has begun in the dry eye chamber Phase 3 TRANQUILITY Trial of reproxalap. The primary endpoint of the trial is ocular redness, which was statistically lower (p=0.03) for reproxalap relative to vehicle in the TRANQUILITY run-in cohort results announced in January 2021. Tear RASP (reactive aldehyde species) levels will also be assessed. Approximately 150 dry eye disease patients are expected to be enrolled per arm. Reproxalap will be administered four times the day prior to entry into the dry eye chamber, just before entry into the chamber, and 45 minutes after chamber entry. Enrollment in TRANQUILITY is ongoing, and enrollment in the confirmatory TRANQUILITY-2 Trial is expected to begin in the second quarter of 2021. Aldeyra plans to report top-line results from both trials in the second half of this year.
Phase 2 Clinical Trial Results from ADX-629, an Orally Available RASP Inhibitor, Expected Second Half of 2021: Initial Phase 2 clinical results from ADX-629, a novel orally available RASP inhibitor currently undergoing testing in asthma, psoriasis, and COVID-19, are expected in the second half of 2021. ADX-629 represents a first-in-class systems-based therapeutic approach for an orally administered RASP inhibitor, the potential applicability of which could extend to a myriad of immune-mediated diseases that today are treated with single-target drugs that can lead to serious toxicity.
Public Offering Completed: Aldeyra sold 10,000,000 shares of its common stock at a public offering price of $12.50 per share in an underwritten public offering. The offering generated gross proceeds of $125.0 million and net proceeds of $117.3 million after deducting underwriting discounts, commissions, and estimated offering expenses.
First-Quarter 2021 Financial Summary

Cash and cash equivalents as of March 31, 2021 were $138.4 million. Based on Aldeyra’s current operating plan, the company believes that existing cash and cash equivalents, as of March 31, 2021, together with the net proceeds from the sale of common stock in the underwritten public offering in May 2021, will be sufficient to fund currently projected operating expenses through the end of 2023, including potential NDA submission for reproxalap; initial commercialization of reproxalap, if approved; and continued early and late-stage development of the company’s product candidates in ocular and systemic immune-mediated diseases.

For the quarter ended March 31, 2021, Aldeyra reported a net loss of $11.3 million, compared with a net loss of $9.9 million for the quarter ended March 31, 2020. Net loss per share was $0.25 for the quarter ended March 31, 2021, compared with $0.34 for the same period in 2020. Losses have resulted from the costs of Aldeyra’s clinical trials and research and development programs, as well as from general and administrative expenses.

Research and development (R&D) expenses were $7.7 million for the quarter ended March 31, 2021, compared with $6.6 million for the same period in 2020. The increase of $1.1 million is primarily related to clinical development and manufacturing costs, partially offset by lower personnel related costs and a decrease in preclinical costs.

General and administrative expenses were $3.1 million for the quarter ended March 31, 2021, compared with $3.0 million for the quarter ended March 31, 2020.

For the quarter ended March 31, 2021, total operating expenses were $10.8 million, compared with total operating expenses of $9.6 million for the same period in 2020.

Conference Call & Webcast Information

Aldeyra will host a conference call at 8:00 a.m. ET today to discuss its first-quarter 2021 financial results and recent highlights. The dial-in numbers are (866) 211-4098 for domestic callers and (647) 689-6613 for international callers. The Conference ID number is 6779202. Due to the expected high demand on our conference call provider, please plan to dial in to the call at least 15 minutes prior to the start time.

A live webcast of the conference call will also be available on the Investor Relations page of the company’s website at View Source After the live webcast, the event will remain archived on the Aldeyra Therapeutics website for 90 days.