On May 7, 2020 AngioDynamics, Inc. (NASDAQ: ANGO), a leading provider of innovative, minimally invasive medical devices for vascular access, peripheral vascular disease, and oncology, reported that Jim Clemmer, President and Chief Executive Officer, and Stephen Trowbridge, Executive Vice President and Chief Financial Officer, will participate in a virtual fireside chat at the UBS Virtual Global Healthcare Conference at 9:10 a.m. ET on Monday, May 18, 2020 (Press release, AngioDynamics, MAY 7, 2020, View Source [SID1234557358]).

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A live webcast of the fireside chat will be accessible through the "Investors" section of the Company’s website at www.angiodynamics.com and will be available for replay following the event.

On May 7, 2020 UroGen Pharma Ltd. (Nasdaq: URGN), a biopharmaceutical company dedicated to building and commercializing novel solutions that treat specialty cancers and urologic diseases, reported financial results for the first quarter ended March 31, 2020 and provided an overview of the Company’s recent developments (Press release, X4 Pharmaceuticals, MAY 7, 2020, View Source [SID1234557357]).

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"The first quarter of 2020 was marked by flawless execution by our team as we prepared for the landmark FDA approval of Jelmyto, the first and only non-surgical treatment for patients with low-grade upper tract urothelial cancer. It has been our mission to pioneer novel treatments for patients in areas of unmet need, and our team has developed innovative solutions to engage key stakeholders so we can bring this effective, kidney-sparing therapy to patients who have been waiting for new treatment options," said Liz Barrett, President and Chief Executive Officer of UroGen. "Our momentum continues as we advance our portfolio of product candidates, highlighted by our recent UGN-102 data readout for the treatment of patients with low-grade intermediate-risk non-muscle invasive bladder cancer, a disease associated with high rates of recurrence and the need for repetitive surgical intervention. The recently published positive interim UGN-102 data, combined with pivotal data for Jelmyto, further supports our confidence in the broad potential of our pipeline. We remain on track to initiate a pivotal Phase 3 study later this year."

Recent Highlights and Upcoming Milestones

Jelmyto (mitomycin) for pyelocalyceal solution, formerly known as UGN-101, for adult patients with low-grade upper tract urothelial cancer (LG-UTUC)

UroGen announced the U.S. FDA granted approval of Jelmyto for the treatment of patients with LG-UTUC on April 15, 2020. Jelmyto is the first and only FDA approved non-surgical treatment option for patients with LG-UTUC.
The most commonly reported adverse events (≥ 20%) were ureteric obstruction, flank pain, urinary tract infection, hematuria, renal dysfunction, fatigue, nausea, abdominal pain, dysuria, and vomiting. No treatment-related deaths occurred.
The Lancet Oncology published pivotal results from the Phase 3 OLYMPUS trial, reporting that 59% of LG-UTUC patients treated with Jelmyto achieved a Complete Response (CR). Durability at 12-months (based on interim data) was estimated to be 84% by Kaplan-Meier analysis.
At the 12-month time point for assessment of durability, 19 patients remained in CR, seven had experienced recurrence of disease, nine patients continued to be followed for the 12-month duration of response, and median duration of response was not reached as of the FDA-approval date.
Jelmyto Commercial Readiness

The Company expects to commence the official commercial launch of Jelmyto on June 1, 2020.
To maximize engagement with health care professionals and key stakeholders in this current environment, the Company has developed innovative solutions, including a virtual platform, to allow for an effective launch.
The team has completed the application for submission of the C-code, and submission of the J-code application to CMS is in process. The company continues to expect a C-code will be secured by October and a J-code by the end of the year.
UGN-102 (mitomycin) for intravesical solution for patients with low-grade intermediate risk non-muscle invasive bladder cancer (LG-IR-NMIBC)

UroGen announced a positive interim data analysis of UGN-102 in patients with LG-IR-NMIBC, featured in a late-breaking abstract published in the April Supplement to The Journal of Urology.
The Phase 2b OPTIMA II trial demonstrated a CR rate at three months following onset of treatment of 65% (41/63 patients). In this subset of patients, 31/32 patients (97%) and 17/20 patients (85%) remained free of disease at six- and nine-months follow-up, respectively.
The most commonly reported adverse events seen to date (≥ 10%) were reported as mild to moderate and include dysuria, hematuria, urinary frequency, fatigue, urgency and urinary tract infection.
The detailed results presentation from the Phase 2b OPTIMA II trial will be available online via the American Urological Association (AUA) in mid-May 2020.
The Company remains on track to initiate a pivotal Phase 3 trial in 2H 2020.
There are no drugs currently approved by the FDA as first-line treatment for LG-IR-NMIBC. UGN-102 has the potential to provide a non-surgical treatment alternative for approximately 80,000 patients diagnosed with LG-IR-NMIBC in the United States alone.
First Quarter 2020 Financial Results; 2020 Guidance

As of March 31, 2020, cash, cash equivalents and marketable securities totaled $159.2 million, excluding restricted cash.
Research and development expenses for the three months ended March 31, 2020 were $16.6 million, including non-cash share-based compensation expense of $1.9 million, and a one-time payment of $6.6 million to unwind our obligation to the IIA.
Selling, general and administrative expenses for the three months ended March 31, 2020 were $22.0 million, including non-cash share-based compensation expense of $5.7 million.
UroGen reported a net loss of $37.8 million, or basic and diluted net loss per ordinary share of $1.79, for the three months ended March 31, 2020.
The 2020 financial guidance set forth during the Company’s full year and fourth quarter 2019 quarterly call on March 2, 2020 remains the same based on current business goals and anticipated activities.
Conference Call & Webcast Information

Members of UroGen’s management team will host a live conference call and webcast today at 8:30 AM Eastern Time to review the Company’s financial results and provide a general business update.

The live webcast can be accessed by visiting the Investors section of the Company’s website at View Source Please connect at least 15 minutes prior to the live webcast to ensure adequate time for any software download that may be needed to access the webcast. Alternatively, please call (855) 765-5685 (U.S.) or (615) 247-5916 (International) to listen to the live conference call. The conference ID number for the live call will be 4575088. An archive of the webcast will be available for two weeks on the Company’s website.

About the Phase 3 OLYMPUS Trial

OLYMPUS (Optimized DeLivery of Mitomycin for Primary UTUC Study) is an open-label, single-arm Phase 3 clinical trial of UGN-101, Jelmyto (mitomycin) for pyelocalyceal solution, to evaluate the safety, tolerability and tumor ablative effect of Jelmyto in patients with low-grade UTUC. Seventy-one patients were treated at clinical sites across the United States and Israel. Study participants were treated with six weekly instillations of Jelmyto administered via a standard catheter. Four to six weeks following the last instillation, patients underwent a Primary Disease Evaluation (PDE) to determine Complete Response (CR), the primary endpoint of the study. PDE involved a ureteroscopy and wash cytology, a standard microscopic test of cells obtained from the urine to detect cancer and for cause biopsy. Patients who achieved a CR at the PDE timepoint were then followed for up to 12 months to determine the durability of response with Jelmyto.

About JelmytoTM

Jelmyto (mitomycin) for pyelocalyceal solution, is a drug formulation of mitomycin indicated for the treatment of adult patients with low-grade upper tract urothelial cancer (LG-UTUC). Utilizing the RTGel technology platform, UroGen’s proprietary sustained release, hydrogel-based formulation, Jelmyto is designed to enable longer exposure of urinary tract tissue to mitomycin, thereby enabling the treatment of tumors by non-surgical means. Jelmyto is delivered to patients using standard ureteral catheters or nephrostomy tube. The U.S. FDA previously granted Orphan Drug, Fast Track, and Breakthrough Therapy Designations to Jelmyto for the treatment of LG-UTUC. On April 15, 2020, the FDA approved Jelmyto, making it the first drug approved for the treatment of LG-UTUC in adult patients.

About Upper Tract Urothelial Cancer (UTUC)

Urothelial cancer is the ninth most common cancer globally and the eighth most lethal neoplasm in men in the U.S. Between five percent and ten percent of primary urothelial cancers originate in the ureter or renal pelvis and are collectively referred to as upper tract urothelial cancers (UTUC). In the U.S., there are approximately 6,000 – 7,000 new or recurrent low-grade UTUC patients annually. Most cases are diagnosed in patients over 70 years old, and these older patients often face comorbidities. There are limited treatment options for UTUC, with the most common being endoscopic surgery or nephroureterectomy (removal of the entire kidney and ureter). These treatments can lead to a high rate of recurrence and relapse.

IMPORTANT SAFETY INFORMATION

You should not receive JELMYTO if you have a hole or tear (perforation) of your bladder or upper urinary tract.

Before receiving JELMYTO, tell your healthcare provider about all your medical conditions, including if you:

are pregnant or plan to become pregnant. JELMYTO can harm your unborn baby. You should not become pregnant during treatment with JELMYTO. Tell your healthcare provider right away if you become pregnant or think you may be pregnant during treatment with JELMYTO.
Females who are able to become pregnant: You should use effective birth control (contraception) during treatment with JELMYTO and for 6 months after the last dose.

Males being treated with JELMYTO: If you have a female partner who is able to become pregnant, you should use effective birth control (contraception) during treatment with JELMYTO and for 3 months after the last dose.

are breastfeeding or plan to breastfeed. It is not known if JELMYTO passes into your breast milk. Do not breastfeed during treatment with JELMYTO and for 1 week after the last dose.
Tell your healthcare provider if you take water pills (diuretic).
How will I receive JELMYTO?

Your healthcare provider will tell you to take a medicine called sodium bicarbonate before each JELMYTO treatment.
You will receive your JELMYTO dose from your healthcare provider 1 time a week for 6 weeks. It is important that you receive all 6 doses of JELMYTO according to your healthcare provider’s instructions. If you miss any appointments, call your healthcare provider as soon as possible to reschedule your appointment. Your healthcare provider may recommend up to an additional 11 monthly doses.
JELMYTO is given to your kidney through a tube called a catheter.
During treatment with JELMYTO, your healthcare provider may tell you to take additional medicines or change how you take your current medicines.
After receiving JELMYTO:

JELMYTO may cause your urine color to change to a violet to blue color. Avoid contact between your skin and urine for at least 6 hours.
To urinate, males and females should sit on a toilet and flush the toilet several times after you use it. After going to the bathroom, wash your hands, your inner thighs, and genital area well with soap and water.
Clothing that comes in contact with urine should be washed right away and washed separately from other clothing.
JELMYTO may cause serious side effects, including:

Swelling and narrowing of the tube that carries urine from the kidney to the bladder (ureteric obstruction). If you develop swelling and narrowing, and to protect your kidney from damage, your healthcare provider may recommend the placement of a small plastic tube (stent) in the ureter to help the kidney drain. Tell your healthcare provider right away if you develop side pain or fever during treatment with JELMYTO.
Bone marrow problems. JELMYTO can affect your bone marrow and can cause a decrease in your white blood cell, red blood cell, and platelet counts. Your healthcare provider will do blood tests prior to each treatment to check your blood cell counts during treatment with JELMYTO. Your healthcare provider may need to temporarily or permanently stop JELMYTO if you develop bone marrow problems during treatment with JELMYTO.
The most common side effects of JELMYTO include: side pain, urinary tract infection, blood in your urine, kidney problems, tiredness, nausea, stomach pain, trouble with urination, vomiting, low red blood cell count, frequent urination, itching, chills, and fever.

On May 7, 2020 Syros Pharmaceuticals (NASDAQ:SYRS), a leader in the development of medicines that control the expression of genes, reported financial results for the quarter ended March 31, 2020 and provided an update on recent accomplishments and upcoming events (Press release, Syros Pharmaceuticals, MAY 7, 2020, View Source [SID1234557356]).

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"While COVID-19 has created tremendous uncertainty across the globe, it has also reminded us of the critical importance of medical innovation," said Nancy Simonian, M.D., Chief Executive Officer of Syros. "Over the last few months, we have focused on ensuring the safety and well-being of our employees and our community, while continuing to advance our ongoing clinical trials and critical earlier-stage research. There is no playbook for what we are going through, and I am extremely proud of the dedication, creativity and flexibility our team has shown during this time. Thanks to their efforts, we recently completed enrollment in our Phase 2 trial cohort evaluating SY-1425 in combination with azacitidine in RARA-positive relapsed or refractory AML patients. We remain on track to report data from this cohort and mature data on SY-1425 from our cohort in newly diagnosed AML patients, as well as initial dose-escalation data from the Phase 1 trial of SY-5609, all in the fourth quarter. While these are difficult times, we have a strong foundation and a strong team, and I am confident we will adapt to the challenges ahead as we pursue our mission of delivering medicines that provide a profound benefit for patients."

Anticipated Milestones:

SY-1425

Report potential proof-of-concept data in the fourth quarter of 2020 from the ongoing, fully enrolled Phase 2 trial cohort evaluating SY-1425 in combination with azacitidine in RARA-positive relapsed or refractory acute myeloid leukemia (AML) patients.
Report mature data in the fourth quarter of 2020 from the ongoing, fully enrolled Phase 2 trial cohort evaluating SY-1425 in combination with azacitidine in newly diagnosed AML patients who are not suitable candidates for standard chemotherapy.
SY-5609

Present new preclinical data on the anti-tumor activity of SY-5609 in models of colorectal cancer, as well as details on the design of its ongoing Phase 1 trial, at the Virtual 2020 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting.
Report initial safety, tolerability, pharmacokinetic and pharmacodynamic data in the fourth quarter of 2020 from the dose-escalation portion of the ongoing Phase 1 trial evaluating SY-5609 in patients with breast, colorectal, lung and ovarian cancers, as well as in patients with solid tumors of any histology that harbor Rb pathway alterations.
Report additional dose-escalation data, including clinical activity data, in mid-2021.
Preclinical Pipeline

Nominate next development candidate by the end of 2021.
First Quarter 2020 Financial Results:

Cash, cash equivalents and marketable securities as of March 31, 2020 were $121.9 million, compared with $91.4 million on December 31, 2019. This increase reflects the $20 million upfront payment received in connection with Syros’ entry into a collaboration with Global Blood Therapeutics, Inc. (GBT) in December 2019, the $20 million that Syros drew down from its senior secured loan facility with Oxford Finance, LLC in February 2020, and $12.3 million from the sale of common stock under Syros’ at-the-market sales facility in the first quarter.

For the first quarter of 2020, Syros reported a net loss of $17.2 million, or $0.39 per share, compared to a net loss of $16.5 million, or $0.49 per share, for the same period in 2019.

Revenues were $2.4 million for the first quarter of 2020, compared to $0.5 million for the same period in 2019. In the first quarter of 2020 $2.2 million in revenue was recognized under Syros’ collaboration with GBT and $0.2 million was recognized under its collaboration with Incyte Corporation (Incyte). All revenues recognized in the first quarter of 2019 were under Syros’ collaboration with Incyte.
Research and development (R&D) expenses were $14.6 million for the first quarter of 2020, as compared to $12.6 million for the same period in 2019. This increase was primarily attributable to continued advancement of Syros’ existing clinical trials and preclinical programs, including its sickle cell disease program.
General and administrative (G&A) expenses $5.1 million for the first quarter of 2020, as compared to $4.9 million for the same period in 2019. This increase was primarily attributable to an increase in employee-related costs, including salary, benefits and stock-based compensation due to our increased headcount.
Financial Guidance:

Based on its current plans, Syros believes that its existing cash, cash equivalents and marketable securities will be sufficient to fund its planned operating expenses and capital expenditures requirements into 2022, beyond key milestones expected for both SY-1425 and SY-5609.

Conference Call and Webcast:

Syros will host a conference call today at 8:30 a.m. ET to discuss these first quarter 2020 financial results and provide a corporate update.

To access the live conference call, please dial (866) 595-4538 (domestic) or (636) 812-6496 (international) and refer to conference ID 1585949. A webcast of the call will also be available on the Investors & Media section of the Syros website at www.syros.com. An archived replay of the webcast will be available for approximately 30 days following the call.

On May 7, 2020 Moderna, Inc. (Nasdaq: MRNA), a clinical stage biotechnology company pioneering messenger RNA (mRNA) therapeutics and vaccines to create a new generation of transformative medicines for patients, reported financial results and provided business updates for the first quarter of 2020 and highlighted pipeline progress (Press release, Moderna Therapeutics, MAY 7, 2020, View Source [SID1234557355]).

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"The imminent Phase 2 study start is a crucial step forward as we continue to advance the clinical development of mRNA-1273, our vaccine candidate against SARS-CoV-2. With the goal of starting the mRNA-1273 pivotal Phase 3 study early this summer, Moderna is now preparing to potentially have its first BLA approved as soon as 2021. We are accelerating manufacturing scale-up and our partnership with Lonza puts us in a position to make and distribute as many vaccine doses of mRNA-1273 as possible, should it prove to be safe and effective," said Stéphane Bancel, Moderna’s Chief Executive Officer. "We also are continuing to progress our development pipeline and invest in our future. We are very pleased with Vertex’s decision, based on our preclinical progress, to extend our strategic collaboration working to develop the technology to allow for delivery of mRNA in the lung."

New updates and recent progress include:

Infectious Diseases

The U.S. Food and Drug Administration (FDA) completed its review of the Company’s Investigational New Drug (IND) application for its novel coronavirus (SARS-CoV-2 or COVID-19) vaccine candidate (mRNA-1273) allowing it to proceed to Phase 2 study, expected to begin shortly; finalizing protocol for Phase 3 study of mRNA-1273, expected to begin in early summer of 2020
Positive interim results announced from Phase 1 Zika vaccine candidate (mRNA-1893) study
Oncology

Positive data from the monotherapy arm of the Phase 1/2 study of OX40L (mRNA-2416) presented at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Virtual Annual Meeting
Rare Diseases

Due to COVID-19, enrollment and new site initiation paused for methylmalonic acidemia (MMA; mRNA-3704) and propionic acidemia (PA; mRNA-3927) clinical trials. During the pause, the Company is implementing changes that the Company believes will ultimately help to accelerate clinical development
Given current strategic priorities, the Fabry disease program (mRNA-3630) is being discontinued
Research Update

In March 2020, based on promising preclinical data generated to date, Vertex Pharmaceuticals, Inc. extended the conduct of the initial cystic fibrosis (CF) research plan for an additional 18 months by making an additional payment to Moderna
Moderna currently has 23 mRNA development candidates in its portfolio with 13 in clinical studies. Across Moderna’s pipeline, more than 1,900 participants have been enrolled in clinical studies. The Company’s updated pipeline can be found at www.modernatx.com/pipeline. Moderna and collaborators have published more than 45 peer-reviewed papers.

Summary of Program Highlights by Modality

Core Modalities

Prophylactic Vaccines: Moderna is developing vaccines against viral diseases where there is unmet medical need – including complex vaccines with multiple antigens for common diseases, as well as vaccines against threats to global public health. The Company’s global public health portfolio is focused on epidemic and pandemic diseases for which funding has been sought from governments and non-profit organizations.

Infections transmitted from mother to baby

Cytomegalovirus (CMV) vaccine (mRNA-1647): In March, Moderna announced completion of enrollment for the Phase 2 dose-confirmation study of mRNA-1647. The first Phase 2 interim analysis is expected in the third quarter of 2020. Manufacturing and planning are underway for the pivotal Phase 3 study, which is designed to evaluate the efficacy of mRNA-1647 against primary CMV infection in women of childbearing age and is expected to start in 2021. Moderna owns worldwide commercial rights for mRNA-1647.
Zika virus vaccine (mRNA-1893): The 10 µg, 30 µg and 100 µg cohorts in the Phase 1 study of mRNA-1893 have completed enrollment. In April 2020, Moderna announced positive interim Phase 1 data showing the 10 µg and 30 µg dose levels of mRNA-1893 induced a neutralizing antibody response in both flavivirus infection-naïve (seronegative) participants and in participants with pre-existing flavivirus antibodies (seropositive) following a two-dose vaccination schedule given 28 days apart. The 10 µg and 30 µg dose levels were both generally well-tolerated, and there were no vaccine-related serious adverse events (SAEs) or adverse events of special interest (AESI). The most frequent solicited adverse reaction was local pain at the injection site. The safety profile did not appear affected by the second vaccination nor a flavivirus-positive baseline serostatus. mRNA-1893 is being developed in collaboration with the U.S. Biomedical Advanced Research and Development Authority (BARDA) within the Office of the Assistant Secretary for Preparedness and Response at the U.S. Department of Health and Human Services. Moderna owns worldwide commercial rights to mRNA-1893.
Vaccines against respiratory infections

Novel coronavirus (SARS-CoV-2) vaccine (mRNA-1273): The U.S. Food and Drug Administration (FDA) completed its review of the Company’s Investigational New Drug (IND) application for its novel coronavirus (SARS-CoV-2 or COVID-19) vaccine candidate (mRNA-1273) allowing it to proceed to the Phase 2 study. A 600 participant Phase 2 study is expected to begin shortly. The Company is finalizing the protocol for the Phase 3 study, which is expected to begin in the early summer of 2020.
Human metapneumovirus (hMPV) and parainfluenza type 3 (PIV3) vaccine (mRNA-1653): Due to the pandemic, the Company previously decided to pause new enrollment of participants in the ongoing hMPV/PIV3 study (mRNA-1653), which had been actively enrolling seropositive pediatric participants (12-36 months of age). The Company intends to work with appropriate medical and site personnel to determine when to resume new enrollment. Moderna owns worldwide commercial rights to mRNA-1653.
Pediatric respiratory syncytial virus (RSV) vaccine (mRNA-1345): mRNA-1345 is a vaccine against RSV in young children encoding for a prefusion F glycoprotein, which elicits a superior neutralizing antibody response compared to the postfusion state. The Company intends to combine mRNA-1345 with mRNA-1653, its vaccine against hMPV and PIV3, to create a combination vaccine against RSV, hMPV and PIV3. There is no approved vaccine for RSV. Moderna owns worldwide commercial rights to the combined mRNA-1345/mRNA-1653 vaccine.
RSV vaccine (mRNA-1172 or V172): The Phase 1 study of mRNA-1172 led by Merck is ongoing. Moderna has licensed worldwide commercial rights to mRNA-1172 to Merck.
Influenza H7N9 vaccine (mRNA-1851): Discussions regarding funding the Company’s influenza H7N9 vaccine program through approval are ongoing.
Vaccines against highly prevalent viral infections

Epstein-Barr virus (EBV) vaccine (mRNA-1189): mRNA-1189 is a vaccine against EBV containing five mRNAs that encode viral proteins (gp350, gB, gp42, gH and gL) in EBV. Similar to Moderna’s CMV vaccine (mRNA-1647), the viral proteins in mRNA-1189 are expressed in their native membrane-bound form for recognition by the immune system. There is no approved vaccine for EBV. Moderna owns worldwide commercial rights to mRNA-1189.
Systemic Secreted & Cell Surface Therapeutics: In this modality, mRNA is delivered systemically to create proteins that are either secreted or expressed on the cell surface.

Antibody against the chikungunya virus (mRNA-1944): Moderna has been notified that the enrollment of further subjects in the Phase 1 study of mRNA-1944 has been paused by the site due to the impact of COVID-19. Moderna owns worldwide commercial rights to mRNA-1944.
IL-2 (mRNA-6231): mRNA-6231 is an mRNA encoding for a long-acting tolerizing IL-2. This new autoimmune development candidate is designed to preferentially activate and expand the regulatory T cell population. The Company plans to conduct a Phase 1 study of mRNA-6231 in healthy adult volunteers. mRNA-6231 uses the same LNP formulation as mRNA-1944. The Phase 1 study of mRNA-6231 will be the first clinical demonstration of subcutaneous administration of this delivery technology. Moderna owns worldwide commercial rights to mRNA-6231.
PD-L1 (mRNA-6981): mRNA-6981 is an mRNA encoding for PD-L1. This new autoimmune development candidate is designed to augment cell surface expression of PD-L1 on myeloid cells to provide co-inhibitory signals to self-reactive lymphocytes. As an initial step to addressing a range of autoimmune indications, the Company intends to pursue proof-of-concept in a Phase 1 study of mRNA-6981 in type 1 autoimmune hepatitis (AIH), a condition that involves liver inflammation and can lead to cirrhosis and liver failure. mRNA-6981 uses the same LNP formulation as mRNA-1944. Moderna owns worldwide commercial rights to mRNA-6981.
Relaxin (AZD7970): Partnered with AstraZeneca, AZD7970 is in preclinical development for the treatment of heart failure. Under the terms of the collaboration, AstraZeneca would sponsor the Phase 1 trial to assess safety, tolerability and duration of systemic exposure to the Relaxin protein. Moderna shares worldwide commercial rights to AZD7970 with AstraZeneca.
Fabry disease (mRNA-3630): Given current strategic priorities, the Fabry disease program (mRNA-3630) is being discontinued.
Publication of note: In April, in collaboration with Seattle Children’s Research Institute, Moderna published preclinical data in Molecular Therapy Nucleic Acids showing administration of mRNA encoding the factor VIII (FVIII) protein led to rapid and prolonged FVIII expression in mouse models of Hemophilia A. This work is part of Moderna’s research to explore the potential of mRNA therapy across various rare diseases.
Exploratory Modalities

Cancer Vaccines: These programs focus on stimulating a patient’s immune system with antigens derived from tumor-specific mutations to enable the immune system to elicit a more effective anti-tumor response.

Personalized cancer vaccine (PCV) (mRNA-4157): The randomized Phase 2 study investigating a 1 mg dose of mRNA-4157 in combination with Merck’s pembrolizumab (KEYTRUDA), compared to pembrolizumab alone, for the adjuvant treatment of high-risk resected melanoma is ongoing. The Phase 1 study is ongoing. The Company is evaluating the impact of COVID-19 related challenges that are leading to delays in enrollment. Moderna shares worldwide commercial rights to mRNA-4157 with Merck.
KRAS vaccine (mRNA-5671 or V941): The Phase 1 open-label, multi-center study to evaluate the safety and tolerability of mRNA-5671 both as a monotherapy and in combination with pembrolizumab, led by Merck, is ongoing. Moderna shares worldwide commercial rights to mRNA-5671 with Merck.
Intratumoral Immuno-Oncology: These programs aim to drive anti-cancer T cell responses by injecting mRNA therapies directly into tumors.

OX40L (mRNA-2416): The Phase 1/2 study of mRNA-2416 alone and in combination with durvalumab (IMFINZI) is ongoing. The Company is evaluating the impact of COVID-19-related challenges that are leading to delays in enrollment. Moderna owns worldwide commercial rights to mRNA-2416.
Presentation of Note: Data from the monotherapy arm of the Phase 1/2 study of OX40L (mRNA-2416) were presented at the AACR (Free AACR Whitepaper) Virtual Annual Meeting showing that mRNA-2416 was well-tolerated at all dose levels studied with the majority of adverse events reported as grade 1 and 2; no grade 3 adverse events were reported. Best overall response observed was stable disease in 14/39 patients with 6 of these patients at stable disease for >14 weeks. Patients treated with monotherapy mRNA-2416 showed increased OX40L protein expression, upregulation of PD-L1 levels and evidence of increased pro-inflammatory activity demonstrating proof of mechanism and supporting the evaluation of intratumoral mRNA-2416 with the anti-PD-L1 inhibitor durvalumab in solid tumors, which is ongoing in Part B of this study with a focus on advanced ovarian carcinoma.
OX40L/IL-23/IL-36γ (Triplet) (mRNA-2752): The Phase 1 trial evaluating mRNA-2752 as a single agent and in combination with durvalumab in patients with advanced solid tumor malignancies and lymphoma is ongoing. mRNA-2752 is an investigational mRNA immuno-oncology therapy that encodes a novel combination of three immunomodulators. The Company is evaluating the impact of COVID-19-related challenges that are leading to delays in enrollment. Moderna owns worldwide commercial rights to mRNA-2752.
IL-12 (MEDI1191): The Phase 1 open-label, multi-center study of intratumoral injections of MEDI1191 alone and in combination with durvalumab in patients with advanced solid tumors, led by AstraZeneca, is ongoing. MEDI1191 is an mRNA encoding for IL-12, a potent immunomodulatory cytokine. Moderna shares worldwide commercial rights to MEDI1191 with AstraZeneca.
Localized Regenerative Therapeutics: Localized production of proteins has the potential to be used as a regenerative medicine for damaged tissues.

VEGF-A (AZD8601): The Phase 2a study of AZD8601 for VEGF-A for ischemic heart disease in patients undergoing coronary artery bypass grafting (CABG) surgery with moderately impaired systolic function, led by AstraZeneca, is ongoing at one site. Moderna has licensed worldwide commercial rights to AZD8601 to AstraZeneca.
Systemic Intracellular Therapeutics: These programs aim to deliver mRNA into cells within target organs as a therapeutic approach for diseases caused by a missing or defective protein.

Methylmalonic acidemia (MMA) (mRNA-3704): Due to the COVID-19 pandemic, Moderna previously decided to pause new enrollment and new site initiation for its Phase 1/2 study of mRNA-3704 to ensure the safety of these pediatric patients and their caregivers. No patients have been dosed to date. During the pause, the Company is implementing changes that the Company believes will ultimately help to accelerate clinical development. mRNA-3704 uses the same LNP formulation as mRNA-1944. Moderna owns worldwide commercial rights to mRNA-3704.
Propionic acidemia (PA) (mRNA-3927): Due to the COVID-19 pandemic, Moderna previously decided to pause new enrollment and new site initiation for its Phase 1/2 study of mRNA-3927 to ensure the safety of these pediatric patients and their caregivers. No patients have been dosed to date. During the pause, the Company is implementing changes that the Company believes will ultimately help to accelerate clinical development. mRNA-3927 uses the same LNP formulation as mRNA-1944. Moderna owns worldwide commercial rights to mRNA-3927.
MMA and PA Natural History Study (MaP): This is a global, multi-center, non-interventional study for patients with confirmed diagnosis of MMA due to MUT deficiency or PA and is designed to identify and correlate clinical and biomarker endpoints for these disorders. Enrollment in the study has been completed.
Phenylketonuria (PKU) (mRNA-3283): Individuals with PKU have a deficiency in phenylalanine hydroxylase (PAH) resulting in a reduced or complete inability to metabolize the essential amino acid phenylalanine into tyrosine. mRNA-3283 encodes human PAH to restore the deficient or defective intracellular enzyme activity in patients with PKU. mRNA-3283 is in preclinical development. Moderna owns worldwide commercial rights to mRNA-3283.
Glycogen storage disease type 1a (GSD1a) (mRNA-3745): Individuals with GSD1a have a deficiency in glucose-6-phosphatase resulting in pathological blood glucose imbalance. mRNA-3745 is an IV-administered mRNA encoding human G6Pase enzyme, designed to restore the deficient or defective intracellular enzyme activity in patients with GSD1a. mRNA-3745 is in preclinical development. Moderna owns worldwide commercial rights to mRNA-3745.
Information about each development candidate in Moderna’s pipeline, including those discussed in this press release, can be found on the investor relations page of its website: investors.modernatx.com.

Research Update

Vertex cystic fibrosis research collaboration: In July 2016, Moderna and Vertex announced an exclusive research collaboration and licensing agreement aimed at the discovery and development of mRNA therapeutics for the treatment of CF. In July 2019, Vertex elected to extend the initial research period by six months. Based on promising preclinical work to date, in March 2020 Vertex extended this collaboration through August 2021 with options to extend further based on future progress. Pulmonary mRNA delivery represents a potential new route of administration for Moderna.
Management Updates

Moderna’s Chief Financial Officer, Lorence Kim, M.D., will be leaving the Company in August after six years of service. The Company has retained Russell Reynolds to recruit for a new CFO with public company, global and commercial experience as the Company scales up to file several biologics license applications (BLAs) over the next few years with the Company’s first potential launch, for its SARS-CoV-2 vaccine, as soon as 2021.

"I would like to thank Lorence for his tremendous impact on Moderna’s success over the last six years. Lorence brought unique skills with a medical degree and financial market experience, which has helped us build our financial foundation. His leadership and contributions were vital in helping Moderna get to where we are today. I have enjoyed having him as my partner and wish him all the best as he embarks on the next leg of his successful career," said Stéphane Bancel.
Charbel Haber, M.P.H., Ph.D. joined Moderna on April 21 as SVP, Regulatory Affairs joining from Biogen where he served as Vice President, Global Safety and Regulatory Sciences since 2017. Prior to Biogen, Dr. Haber was Head, Global Regulatory Affairs-Immunology and Neurology at EMD Serono, Inc. Dr. Haber brings significant global regulatory experience and led numerous successful regulatory development and registration of drugs in different modalities and disease areas.
Jacqueline Miller, M.D., FAAP will join Moderna on May 11 as SVP, Infectious Disease Development. Dr. Miller joins the Company from GlaxoSmithKline where she held a variety of leadership roles since 2005. Most recently, Dr. Miller was the Vice President and Head, Clinical R&D and Epidemiology where she built and led the clinical and epidemiology research teams at the first GSK vaccines research and development center in the U.S.
Patrick Bergstedt will join Moderna on June 1 as SVP, Commercial Vaccines. Prior to Moderna, Mr. Bergstedt held various progressive leadership positions with the Infectious Diseases and Global Human Health groups at Merck & Co, Inc. since 2001. Mr. Bergstedt most recently served as Head of Global Marketing & Commercial Operations: Vaccines at Merck where he led global initiatives with a focus on revenue growth and access expansion, and played a pivotal role in the transformation of the vaccines business.

"We are excited to welcome these three new senior leaders, who bring extensive clinical development, regulatory and commercial experience, as we begin to pivot towards late-stage development and commercialization," said Stéphane Bancel.
Corporate Updates

The Company hosted its first Vaccines Day on April 14, 2020.
Financial Guidance

The Company has up to $2.4 billion to invest, including cash and investments of $1.7 billion and up to $0.7 billion in potentially available grants and awards.
In 2020, the Company expects net cash used in operating activities and for purchases of property and equipment to be approximately $500 million.
While the Company expects to incur significant expenses this year in relation to the BARDA award for the development of mRNA-1273 to FDA licensure and manufacturing process scale-up, the Company expects in general a close matching of expenses and reimbursements for those expenses covered by the BARDA award.
Key 2020 Investor and Analyst Event Dates

Science Day – June 2 (virtual)
R&D Day – September 17
First Quarter 2020 Financial Results (Unaudited)

Cash Position: Cash, cash equivalents and investments as of March 31, 2020 and December 31, 2019 were $1.7 billion and $1.3 billion, respectively.
Net Cash Used in Operating Activities: Net cash used in operating activities was $106.2 million for the three months ended March 31, 2020 compared to $144.3 million for the three months ended March 31, 2019. Net cash used in operating activities includes $22.0 million for the three months ended March 31, 2019, of in-licensing payments to Cellscript, LLC and its affiliate, mRNA RiboTherapeutics, Inc., to sublicense certain patent rights. After the first quarter of 2019, the Company has no further in-licensing payment obligations to Cellscript and its affiliate.
Cash Used for Purchases of Property and Equipment: Cash used for purchases of property and equipment was $6.2 million for the three months ended March 31, 2020 compared to $7.6 million for the three months ended March 31, 2019.
Revenue: Total revenue was $8.4 million for the three months ended March 31, 2020 compared to $16.0 million for the three months ended March 31, 2019. The total revenue decrease in 2020 was mainly attributable to cumulative catch-up adjustments in revenue due to changes in estimated costs for our future performance obligations under the collaboration agreements with Merck and AstraZeneca, and a decrease in revenue from Merck, primarily driven by the timing of amortization of deferred revenue due to the satisfaction of the Company’s performance obligation.
Research and Development Expenses: Research and development expenses were $115.1 million for the three months ended March 31, 2020 compared to $130.4 million for the three months ended March 31, 2019. The decrease was primarily attributable to decreases in lab supplies and materials, clinical trial and manufacturing costs, consulting and outside services, partially offset by increases in personnel related costs, depreciation and amortization and stock-based compensation.
General and Administrative Expenses: General and administrative expenses were $24.1 million for the three months ended March 31, 2020 compared to $27.3 million for the three months ended March 31, 2019. The decrease was mainly due to a decrease in legal related costs and consulting and outside services.
Net Loss: Net loss was $124.2 million for the three months ended March 31, 2020 compared to $132.6 million for the three months ended March 31, 2019.
Investor Call and Webcast Information

Moderna will host a live conference call and webcast at 8:00 a.m. ET on Thursday, May 7, 2020. To access the live conference call, please dial 866-922-5184 (domestic) or 409-937-8950 (international) and refer to conference ID 6698719. A webcast of the call will also be available under "Events and Presentations" in the Investors section of the Moderna website at investors.modernatx.com. A replay of the webcast will be archived on Moderna’s website for one year following the presentation.

On May 7, 2020 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 first quarter ended March 31, 2020, announced new clinical trials in systemic inflammatory diseases, and provided an update on ocular disease programs (Press release, Aldeyra Therapeutics, MAY 7, 2020, View Source [SID1234557354]).

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"Based on the success of the novel RASP inhibitor ADX-629 in Phase 1 clinical testing, we are pleased to announce a new comprehensive clinical initiative in systemic inflammatory diseases, complementing our late-stage pipeline in ocular disease," said Todd C. Brady, M.D., Ph.D., President and CEO of Aldeyra. "Although the COVID-19 pandemic has affected clinical trial enrollment timelines, our cash position remains strong, and we are extending our projected cash runway guidance into 2022."

New Clinical Programs in Systemic Inflammatory Diseases

Aldeyra plans to assess the activity of ADX-629 in three types of severe inflammation: cytokine release syndrome, autoimmune disease, and allergy. In preclinical models, ADX-629 treatment reduced levels of TH1, TH2, and TH17-related cytokines, suggesting potential activity across a broad array of inflammatory diseases. The timing of clinical trial initiation depends, in part, on restrictions related to COVID-19, the availability of clinical research facilities and staffing, and the ability to recruit patients.

COVID-19 Respiratory Compromise: A Phase 2 clinical trial of ADX-629 is expected in subjects with COVID-19-associated respiratory compromise, defined as hypoxia and pulmonary radiographic involvement, immediately following admission to the hospital. Severe inflammation, characterized in part by cytokine release syndrome, leads to acute respiratory distress syndrome and other conditions that require mechanical ventilation. The clinical trial, contingent on FDA review of information submitted via the Coronavirus Treatment Acceleration Program (CTAP), is expected to begin in the third quarter of 2020.
Autoimmune Disease: A Phase 2a clinical trial of ADX-629 in patients with psoriasis, an autoimmune condition associated with TH1 cytokines, is expected to begin in the second half of 2020.
Allergy: A Phase 2a allergen-challenge clinical trial of ADX-629 in patients with atopic asthma, an allergic inflammatory disease associated with TH2 cytokines, is expected to begin in the second half of 2020.
Late-Stage Ocular Disease Programs

Reproxalap, a first-in-class RASP inhibitor for topical ocular administration, continues to advance towards a new drug application (NDA) filing in allergic conjunctivitis and dry eye disease. The Phase 3 GUARD Trial of ADX-2191, a novel formulation of methotrexate for intravitreal administration, in patients with proliferative vitreoretinopathy currently remains active, although enrollment has been significantly delayed due to the COVID-19 pandemic.

Dry Eye Disease: A Type C meeting with the U.S. Food and Drug Administration (FDA) is scheduled for mid-2020 to discuss remaining NDA requirements for reproxalap in dry eye disease. Reproxalap has demonstrated clinically relevant improvement from baseline in two well-controlled clinical trials: Part 1 of the Phase 3 RENEW Trial announced late last year and a Phase 2 formulation trial announced earlier this year. Dry eye disease remains poorly served by available therapies, and represents one of the largest markets in ophthalmology, affecting an estimated 34 million patients in the United States. Aldeyra plans to provide an update on dry eye disease clinical development plans following receipt and review of FDA feedback.
Allergic Conjunctivitis: Based on delays primarily associated with an extended allergy season, results from the Phase 3 INVIGORATE Trial of reproxalap are currently expected in the first half of 2021. Based on the successful Phase 3 ALLEVIATE Trial announced in 2019, and assuming continued clinical success and positive regulatory review, reproxalap has the potential to be the first new mechanistic approach in decades for the treatment of allergic conjunctivitis. The current therapeutic landscape of allergic conjunctivitis is generally limited to antihistamines, which do not lead to satisfactory activity in up to one-third of patients, and corticosteroids, which cannot be used chronically due to potentially serious adverse events. Allergic conjunctivitis is one of most common ocular surface diseases, affecting an estimated 66 million patients in the United States, and is often associated with dry eye disease.
Proliferative Vitreoretinopathy (PVR): Patient enrollment in Part 1 of the adaptive the Phase 3 GUARD Trial of ADX-2191 for the prevention of PVR has been significantly delayed due to lack of clinical site availability and staffing resulting from the COVID-19 pandemic. PVR is a rare but vision-threatening retinal disease associated with recurrent retinal detachments. There is no approved therapy for PVR. Aldeyra expects to update the enrollment and completion timeline by year-end, and is also exploring additional indications for ADX-2191, including primary intraocular lymphoma, a rare but serious ocular cancer that can affect the retina, uvea, optic nerve, and other ocular structures.
Financial Review for the Quarter Ended March 31, 2020

For the quarter ended March 31, 2020, Aldeyra reported a net loss of $9.9 million, compared with a net loss of $15.6 million for the quarter ended March 31, 2019. Net loss per share was $0.34 for the quarter ended March 31, 2020, compared with $0.58 for the same period in 2019. 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 expenses were $6.6 million for the quarter ended March 31, 2020, compared with $7.8 million for the same period in 2019. The decrease of $1.2 million is primarily related to the decreases in clinical and preclinical development and manufacturing costs. Expenses for the 2019 period also included $6.6 million of in-process research and development expenses incurred in connection with the acquisition of Helio Vision.

General and administrative expenses were $3.0 million for each of the quarters ended March 31, 2020 and 2019. Increases in personnel related costs, including stock-based compensation, were offset by a decrease in legal and other miscellaneous administrative costs.

For the quarter ended March 31, 2020, total operating expenses were $9.6 million, compared with total operating expenses of $17.4 million for the same period in 2019.

Cash, cash equivalents, and marketable securities were $61.4 million as of March 31, 2020. Based on current operating plans, Aldeyra believes that its cash, cash equivalents, and marketable securities as of March 31, 2020 will be sufficient to fund currently anticipated operating expenses into 2022, including the completion of the Phase 3 INVIGORATE Trial for reproxalap, as well as the Phase 2 clinical trials of ADX-629 in COVID-19-associated respiratory compromise, atopic asthma, and psoriasis; the commencement of one or more additional clinical trials in dry eye disease, subject to the outcome of the FDA meeting scheduled for mid-year 2020; and the continuation of Part 1 of the adaptive Phase 3 clinical trial in PVR contingent on patient enrollment.

Conference Call & Webcast Information

Aldeyra will host a conference call today at 8:00 a.m. ET to announce new clinical trials in systemic inflammatory diseases, provide an update on ocular disease programs, and report first-quarter 2020 financial results. The dial-in numbers are (866) 211-4098 for domestic callers and (647) 689-6613 for international callers. The Conference ID number is 6982314. Due to the expected high demand on our conference provider, please plan to dial in to the call at least 30 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 corporate website at View Source After the live webcast, the event will remain archived on the Aldeyra Therapeutics website for 90 days.