On May 5, 2022 Codiak BioSciences, Inc. (NASDAQ: CDAK), a clinical-stage biopharmaceutical company focused on pioneering the development of exosome-based therapeutics as a new class of medicines, reported first quarter 2022 financial results and operational progress (Press release, Codiak Biosciences, MAY 5, 2022, View Source [SID1234613842]).

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"We are making exciting progress with our clinical and preclinical programs, which continue to generate further confirmatory evidence that our engEx Platform can harness exosomes for potent and selective delivery of therapeutics, not only in immuno-oncology, but in vaccines and gene therapy as well," said Douglas E. Williams, Ph.D., President and Chief Executive Officer of Codiak. "We remain on track to deliver on several key milestones by the end of this quarter, including results from all five dose cohorts in our exoSTING trial, initial CTCL patient data from the exoIL-12 program, and commencement of dosing in the Phase 1 study of our third candidate to enter the clinic, exoASO-STAT6."

First Quarter 2022 and Recent Highlights

Completed enrollment and dose escalation in cohorts 4 and 5 in the Phase 1/2 clinical trial of exoSTING (CDK-002) for the treatment of advanced/metastatic, recurrent and injectable solid tumors; patient follow-up continues in all dose cohorts, as well as enrollment of enrichment cohorts
Advanced cutaneous T cell lymphoma (CTCL) portion of Phase 1 trial of exoIL-12 (CDK-003); implemented plans for protocol amendment to include a broader range of CTCL patients, and developed plans to enroll patients with additional cutaneous malignancies responsive to rIL-12
Activated sites for Phase 1 trial of exoASO-STAT6 (CDK-004) for the intravenous treatment of hepatocellular carcinoma in preparation for patient dosing
Presented new preclinical data from the exoVACC pan-beta coronavirus vaccine program, as part of Codiak’s collaboration with the Ragon Institute, at the World Vaccine Congress 2022
Presented preclinical data for exoASO-C/EBPb, a proprietary engineered exosome loaded with antisense oligonucleotides targeting C/EBPβ, at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting
Published a manuscript describing the full exoASO-STAT6 preclinical program in the American Association for the Advancement of Science’s journal, Science Advances
Named industry veteran oncology drug developer David Mauro, M.D., Ph.D. as Chief Medical Officer
Anticipated Milestones and Events

First patients dosed in exoASO-STAT6 Phase 1 clinical trial in hepatocellular carcinomas anticipated during 1H 2022
Safety, PK, PD, objective response rate (ORR), and efficacy data in injected and non-injected tumors from dose escalation cohorts 1-5 in the Phase 1/2 trial of exoSTING and recommended Phase 2 dose expected in late 1H 2022
Initial safety, PK/PD and efficacy data from at least the first cohort of CTCL patients in the Phase 1 clinical trial of exoIL-12 anticipated in late 1H 2022
Presentation of new data from engEx-AAV discovery program describing the generation of potent and high-yield exosome-associated AAV constructs as a strategy for improving gene therapy delivery at the American Society of Gene and Cell Therapy Annual Meeting to be held May 16-19, 2022
Dr. Williams added, "As we have noted in prior quarters, enrollment in the exoIL-12 study at trial sites in the UK has been challenging, and we have worked to pursue options for expediting enrollment. We’re looking forward to the positive impact of protocol enhancements for this trial to include a broader CTCL population (stage IIIa) and the potential to enroll patients with cutaneous malignancies responsive to rIL-12 in past studies – including Kaposi’s sarcoma, Merkel cell carcinoma, and Squamous cell carcinoma – each orphan cutaneous diseases treated by the same physicians, where local treatment is common."

First Quarter 2022 Financial Results

Total revenues for the quarter ended March 31, 2022, were $12.7 million, compared to $13.2 million for the same period in 2021. These results reflect deferred revenue recognized under the Company’s collaboration with Jazz Pharmaceuticals.

Net loss for the quarter ended March 31, 2022, was $8.0 million, compared to a net loss of $10.3 million for the same period in 2021. The decrease in net loss for the quarter was driven primarily by a reduction in research and development expenses, some of which were in connection with the Company’s agreement with Lonza.

Research and development expenses were $14.2 million for the quarter ended March 31, 2022, compared to $16.6 million for the same period in 2021. The decrease in research and development expenses was driven primarily by decreases in lab expenses and personnel-related costs in connection with our agreement with Lonza.

General and administrative expenses were $6.7 million for the quarter ended March 31, 2022, compared to $6.6 million for the same period in 2021. The increase was driven primarily by an increase in personnel expenses.

As of March 31, 2022, Codiak had cash, cash equivalents, and marketable securities of approximately $56.5 million.

On May 5, 2022 The Upstate Cancer Center reported that has received a $10,000 grant from the American Cancer Society (ACS) aimed at addressing the transportation needs of cancer patients in Central New York (Press release, SUNY Upstate, MAY 5, 2022, View Source [SID1234613645]).

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Frequent treatments for cancer patients require much support for families, not only in time commitments, but also the cost of transportation.

To help patients get the critical care they need, ACS awards community transportation grants to health systems, treatment centers and community organizations. These grants are available in select communities through an application process and focus on addressing unmet transportation needs of cancer patients, particularly vulnerable populations experiencing an unequal burden of cancer.

Richard Kilburg, associate administrator of the Upstate Cancer Center, applauded the support from the ACS. "Any assistance offered that can remove transportation as an obstacle to getting timely and important treatments, is vital for our patients," he said. "This grant from the American Cancer Society is most important, and we are grateful for the support."

This year’s grant marks the second year Upstate Cancer Center has received a transportation grant.

Caption: From left, Richard Kilburg, associate administrator of the Upstate Cancer Center; Joni Richter, manager of American Cancer Society Control Strategic Partnerships; and Linda Naples, a financial counselor with Upstate Cancer Center, announcing the $10,000 grant to aid patients with transportation needs.

On May 5, 2022 Humanigen, Inc. (Nasdaq: HGEN) ("Humanigen"), a clinical stage biopharmaceutical company focused on preventing and treating an immune hyper-response called "cytokine storm" with its lead drug candidate, lenzilumab (LENZ), reported financial results for the first quarter ended March 31, 2022 (Press release, Humanigen, MAY 5, 2022, View Source [SID1234613660]).

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"A key highlight of the first quarter was the completion of enrollment in the ACTIV-5/BET-B study. We also held a productive Type B pre-EUA meeting with FDA where we gained alignment on the data and statistical analysis plan to be included as part of the amendment to our EUA for LENZ in COVID-19 patients. In concert with the NIH, we anticipate top-line data in the primary analysis population to be reported in the second quarter, with an amendment to our EUA submission planned to follow," stated Cameron Durrant, Chairman and Chief Executive Officer, Humanigen. "We anticipate hospitalizations from COVID-19 will continue for years to come. Published data on LENZ, confirmed by key opinion leaders and national guideline committees, including NIH, supports treatment guidance based on CRP levels and first-line utilization in hypoxic patients."

"Hospitalizations from COVID-19 in the US continue to remain steady with a 7-day average of 2,072 new daily hospitalizations. While there have been more than 900,000 people already hospitalized in the U.S. this year to date,1 synergizing results from multiple forecasting models prepared by leading experts in epidemiology in four different scenarios forecast additional COVID-19 hospitalizations in the United States, to range from approximately 500,000 to 1,200,000 for the remainder of 2022.2 Variant agnostic treatments for hospitalized patients are still desperately needed," commented Edward Jordan, Chief Commercial Officer, Humanigen.

"As well as its clinical benefit in reducing invasive mechanical ventilation and death, LENZ could deliver significant economic savings to health care systems. LENZ can be used in combination with remdesivir, which is currently used in 50% of hospitalized COVID-19 patients in the U.S.3 Sales of the top two hospital treatments for COVID-19 exceeded $7 billion in global revenue in 2021.4,5 We believe LENZ is well positioned to participate in this sizable and sustainable market," he added.

Lenzilumab is an investigational product and is not currently authorized or approved in any country.

First Quarter and Recent Highlights:

Lenzilumab in COVID-19 patients

Completed enrollment of the Phase 2/3 ACTIV-5/BET-B study, sponsored by the National Institute of Allergy and Infectious Diseases, part of the National Institutes of Health, and enrolled over 400 patients in the primary analysis population (patients with a C-reactive protein level at baseline of less than 150mg/L).
Gained alignment with the FDA during a Type B pre-EUA meeting on the data and statistical analysis plan to be included in the amendment to the EUA.
Announced a peer-reviewed publication in ClinicoEconomics and Outcomes Research outlining the potential clinical and health economic benefits of lenzilumab, if authorized or approved for use in the United Kingdom.
First subject dosed in the PK study in Korea.
Lenzilumab in Development in Other Therapeutic Areas

Gained alignment with the FDA on the protocol for the planned registrational Phase 3 SHIELD study of lenzilumab for the prevention of CAR-T therapy related toxicities including Immune Effector Cell-Associated Neurotoxicity ("ICANS"), in which Humanigen intends to enroll the first patient in the second quarter.
Announced a peer-reviewed publication in Leukemia, a leading oncology and hematology journal, entitled "GM-CSF disruption in CART cells modulates T cell activation and enhances CART cell anti-tumor activity."
Notified by the University of Birmingham, UK, that the amended Investigational Medicinal Product Dossier has been accepted by Medicines & Healthcare products Regulatory Agency for the "RATinG" study. Humanigen believes the first patient will be enrolled in this Phase 2/3 potentially registrational trial for lenzilumab to treat patients who have undergone allogeneic hematopoietic stem cell therapy, who are at high and intermediate risk for acute Graft versus Host Disease ("aGvHD") in the second quarter.
Continued enrollment in the PREACH-M study of lenzilumab in chronic myelomonocytic leukemia. Study sponsor planning for expansion of clinical sites.
First Quarter Ended March 31, 2022 Financial Results

Net loss for the quarter ended March 31, 2022 was $21.3 million, or $0.32 per share, as compared to $65.6 million, or $1.25 per share, for the quarter ended March 31, 2021. The decrease in net loss was due to a decrease in total expenses, mainly Research and Development ("R&D") expense. R&D expense decreased $42.7 million from $59.9 million for the three months ended March 31, 2021, to $17.2 million for the three months ended March 31, 2022. The decrease is primarily due to a decrease of $35.7 million in lenzilumab manufacturing costs.

Cash and Cash Equivalents

Net cash used in operating activities, net of balance sheet changes, was $19.4 million for the quarter ended March 31, 2022. During the first quarter of 2022, the company sold shares of its common stock under its At-the-Market or "ATM" facility, raising net proceeds of approximately $18 million. As of March 31, 2022, the company had cash and cash equivalents of approximately $69 million.

A summary of key financial highlights as of and for the three months ended March 31, 2022 and 2021 is as follows ($ in thousands):

About Lenzilumab

Lenzilumab is a proprietary Humaneered first-in-class monoclonal antibody that has been proven to neutralize GM-CSF, a cytokine of critical importance in the hyperinflammatory cascade, sometimes referred to as cytokine release syndrome, or cytokine storm, associated with COVID-19 and other indications. Lenzilumab binds to and neutralizes GM-CSF, potentially improving outcomes for patients hospitalized with COVID-19. Humanigen believes that GM-CSF neutralization with lenzilumab also has the potential to reduce the hyper-inflammatory cascade known as cytokine release syndrome common to chimeric antigen receptor T-cell (CAR-T) therapy and acute Graft versus Host Disease (aGvHD).

In CAR-T, lenzilumab successfully achieved the pre-specified primary endpoint at the recommended dose in a Phase 1b study with Yescarta in which the overall response rate was 100% and no patient experienced severe cytokine release syndrome or severe neurotoxicity. Based on these results, Humanigen plans to test lenzilumab in a randomized, multicenter, potentially registrational, Phase 3 SHIELD study to evaluate its efficacy and safety when combined with Yescarta and Tecartus CAR-T therapies in non-Hodgkin lymphoma. Lenzilumab will also be tested to assess its ability to prevent and/or treat aGvHD in patients undergoing allogeneic hematopoietic stem cell transplantation.

A study of lenzilumab is also underway for patients with chronic myelomonocytic leukemia exhibiting RAS pathway mutations. This study builds on evidence from a Phase 1 study, conducted by Humanigen, that showed RAS mutations are associated with hyper-proliferative features, which may be sensitive to GM-CSF neutralization.

On May 5, 2022 eyra Therapeutics, Inc. (Nasdaq: ALDX) (Aldeyra), a biotechnology company developing innovative therapies for the treatment of immune-mediated diseases, reported recent corporate highlights and financial results for the quarter ended March 31, 2022 (Press release, Aldeyra Therapeutics, MAY 5, 2022, View Source [SID1234613676]).

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"Consistent with our planned completion this quarter of clinical development for reproxalap in dry eye disease and the recently announced demonstration of clinical activity of ADX-629 in three inflammatory diseases, we are delivering on our strategy to expand our RASP platform from the front of the eye to systemic disease, including clinical trials in ethanol toxicity, chronic cough, minimal change disease, and Sjögren-Larsson Syndrome," stated Todd C. Brady, M.D., Ph.D., President and CEO of Aldeyra. "Additionally, we continue to advance our intravitreal drug platform for the treatment of rare retinal diseases, highlighted by Phase 3 clinical trial results in proliferative vitreoretinopathy and Phase 2 clinical trial results in retinitis pigmentosa expected in the second half of this year."

Recent Corporate Highlights

Completed Enrollment in the Phase 3 TRANQUILITY-2 Trial in Dry Eye Disease. Aldeyra completed enrollment in the Phase 3 TRANQUILITY-2 Trial of 0.25% reproxalap ophthalmic solution in patients with dry eye disease. The primary endpoints of the TRANQUILITY-2 Trial are Schirmer test on the first day of dosing and ocular redness on the second day of dosing during exposure to a dry eye chamber. In a Phase 2 clinical trial and in the Phase 3 TRANQUILITY Trial announced last year, reproxalap demonstrated statistically significant superiority over vehicle in ocular redness and Schirmer test, respectively.
Reported Positive Top-Line Data and Announced New Indications for ADX-629. At its Research & Development Day in March, Aldeyra reported positive top-line data from Phase 2 proof-of-concept trials of ADX-629, a first-in-class orally administered RASP modulator, suggesting broad-based activity across a number of biomarker and clinical endpoints. Accordingly, Aldeyra announced the advancement of ADX-629 to Phase 2 clinical trials in four new indications: ethanol toxicity, chronic cough, minimal change disease, and Sjögren-Larsson Syndrome.
Initiated Phase 2 Clinical Trials of ADX-629 in Ethanol Toxicity and Chronic Cough. Patient enrollment has begun in the Phase 2 clinical trials of ADX-629 in ethanol toxicity and chronic cough. Up to 10% of adults in the U.S. abuse ethanol, which when done chronically can lead to the development of liver disease. Chronic cough, defined as a cough that lasts eight weeks or longer in adults, affects an estimated 13 million adults in the U.S., and up to approximately 10% of people worldwide.
Dry Eye Disease Clinical Data Presented at 2022 ASCRS Annual Meeting. Edward J. Holland, M.D., Professor of Ophthalmology at the University of Cincinnati, presented results from the run-in cohort of the Phase 3 TRANQUILITY Trial of reproxalap in dry eye disease at the 2022 American Society of Cataract and Refractive Surgery (ASCRS) Annual Meeting. The TRANQUILITY run-in cohort demonstrated statistical superiority of reproxalap over vehicle in ocular redness during exposure to a dry eye chamber and in symptom scores after a single day of dosing.
Upcoming Planned Clinical and Regulatory Milestones

Dry Eye Disease: Results from the Phase 3 TRANQUILITY-2 Trial of reproxalap in dry eye disease are expected in the second quarter of 2022, followed by a planned New Drug Application (NDA) submission to the U.S. Food and Drug Administration, pending the outcome of TRANQUILITY-2 and enrollment in the 12-month safety trial of reproxalap in dry eye disease patients. Aldeyra is continuing to review data from the completed TRANQUILITY Trial to finalize analytical plans for the TRANQUILITY-2 results.
Allergic Conjunctivitis: Results from the Phase 3 INVIGORATE-2 allergen chamber trial of reproxalap in allergic conjunctivitis are expected in 2023. INVIGORATE-2 is a randomized, double-masked, crossover trial substantially similar in design to INVIGORATE, which demonstrated statistically significant superiority of reproxalap over vehicle for the primary endpoint of ocular itching and the key secondary endpoint of ocular redness.
Retinal Disease: Results from Part 1 of the Phase 3 GUARD Trial of ADX-2191 in proliferative vitreoretinopathy, and from the Phase 2 clinical trial of ADX-2191 in retinitis pigmentosa, are expected in the second half of 2022.
Systemic Disease: Results from the Phase 2 clinical trial of ADX-629 in ethanol toxicity are expected in the second half of 2022, and results from the Phase 2 clinical trials of ADX-629 in chronic cough, minimal change disease, and Sjögren-Larsson Syndrome are expected in 2023.
First-Quarter 2022 Financial Results

Cash, cash equivalents, and marketable securities as of March 31, 2022 were $216.9 million. Based on its current operating plan, Aldeyra believes that existing cash, cash equivalents, and marketable securities will be sufficient to fund currently projected operating expenses through the end of 2023, including potential NDA submissions; initial commercialization of reproxalap, if approved; and continued development of Aldeyra’s product candidates in ocular and systemic immune-mediated diseases.

Net loss for the three months ended March 31, 2022 was $16.8 million, or $0.29 per share, compared with a net loss of $11.3 million, or $0.25 per share, for the comparable period of 2021. Losses have resulted from the costs of clinical trials and research and development programs, as well as from general and administrative expenses.

Research and development expenses for the three months ended March 31, 2022 were $12.2 million, compared with $7.7 million for the same period in 2021. The increase of $4.5 million is primarily related to increases in clinical research and development expenditures.

General and administrative expenses for the three months ended March 31, 2022 were $4.2 million, compared with $3.1 million for the same period in 2021. The increase of $1.1 million is primarily due to increases in consulting expenditures.

Total operating expenses for the three months ended March 31, 2022 were $16.5 million, compared with total operating expenses of $10.8 million for the same period in 2021.

Conference Call & Webcast Information

Aldeyra will host a conference call at 8:00 a.m. ET today to discuss recent corporate highlights and financial results for the quarter ended March 31, 2022. The dial-in numbers are (844) 200-6205 for domestic callers and (929) 526-1599 for international callers. The access code is 742862. Please dial in at least 10 minutes prior to the start time.

A live webcast of the conference call can be accessed via the Investors & Media page of Aldeyra’s website at View Source After the live webcast, the event will remain archived on the website for 90 days

On May 5, 2022 MaaT Pharma (EURONEXT: MAAT – the "Company"), a French clinical-stage biotech and a pioneer in the development of microbiome-based ecosystem therapies dedicated to improving survival outcomes for patients with cancer reported its cash position as of March 31, 2022, and its revenues for the first quarter of 2022 (Press release, MaaT Pharma, MAY 5, 2022, View Source [SID1234613708]).

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Over the course of the first quarter 2022, the Company has continued its clinical development as previously announced in the context of its IPO in November 2021 such as:

– In January 2022, announcement of positive interim and preliminary data of its Phase 1b trial for MaaT033 in preventing complications of allogeneic hematopoietic stem cell transplantation
– In March 2022, inclusion of the first patient in a Phase 3 trial "ARES" for drug-candidate MaaT013 in the treatment of Graft-versus-Host Disease
– In April 2022, initiation of a Phase 2a trial, sponsored by AP-HP, evaluating MaaT013 in combination with immune checkpoint inhibitors for patients with melanoma

Moreover, the Company entered a partnership agreement in February 2022 with Skyepharma to build, to date, France’s largest cGMP manufacturing facility exclusively dedicated to the production of Microbiome Ecosystem Therapy (MET), thus increasing MaaT Pharma’s manufacturing capabilities ten-fold in line with supply needs required by 2030.

Cash position1

As of March 31, 2022, total cash and cash equivalents were EUR 41.1 million, as compared to EUR 43.3 million as of December 31, 2021. The Company believes it has sufficient cash to cover needs of the development programs presented during the IPO up until the end of the third quarter of 2023.

Revenues in Q1 20221

MaaT Pharma reported revenues of EUR 0.3 million for the quarter ended March 31, 2022, compared with 0.1 million for the same period of 2021. Revenues correspond to compensation invoiced in relation to the compassionate access program, as approved by the French National Drug Safety Agency (Agence Nationale de Sécurité du Médicament or ANSM). In 2022, the program was fully active whereas in 2021 the program began over the course of the first quarter, thus explaining the year-on-year increase.

Upcoming financial communication and investor conference participation

May 31, 2022 – Annual General Meeting
June 7, 2022 – Investor R&D Day
June 30, 2022 – 9th Portzamparc Annual Conference, Paris
July 28, 2022 – Revenues and Cash Position Quarter 2*
September 15 – 16, 2022 – KBCS Life Sciences Conference
September 29, 2022 – Half-year Results 2022*
*Indicative calendar that may be subject to change.