On May 12, 2022 Synlogic, Inc. (Nasdaq: SYBX), a clinical-stage biotechnology company developing medicines for metabolic and immunological diseases through its proprietary approach to synthetic biology, reported financial results for the first quarter ended March 31, 2022 and provided an update on pipeline programs (Press release, Synlogic, MAY 12, 2022, View Source [SID1234614379]).

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"We are pleased to share this progress as we seek to advance our novel phenylketonuria (PKU) program towards Phase 3 initiation," said Synlogic President and Chief Executive Officer Aoife Brennan, M.B. Ch.B. "We believe that we have a unique opportunity to advance a potential treatment for the 55,000 people around the globe with PKU, of whom more than 75% remain in need of additional treatment options – either monotherapy or adjunctive. We are pleased with the progress across our additional programs with expected milestones in 2022, including our homocystinuria (HCU) and enteric hyperoxaluria programs, as well as research progress including our ongoing collaborations with Ginkgo Bioworks and Roche."

Anticipated Upcoming Milestones

Report additional Phase 2 data and final candidate selection for PKU program in H2 2022

Initiate the Phase 3 trial for the PKU program in H1 2023 with Phase 3 readiness activities currently underway

Share data from the Phase 1 trial in healthy volunteers for SYNB1353 in H2 2022

Share proof of concept data for SYNB8802 for enteric hyperoxaluria in H2 2022
Recent Business Highlights

Michael Jensen joined Synlogic as CFO in March 2022

Participation and data presentations at scientific congresses described below:

PKU and HCU program data were presented at inborn errors of metabolism congresses including the Society for Inherited Metabolic Disorders (SIMD) Annual Meeting, the Genetic Metabolic Dietitians International (GMDI) 2022 Conference, and the Garrod Symposium 2022

From Synlogic’s Research team, five different programs were represented at the 2022 Synthetic Biology: Engineering, Evolution & Design (SEED) Conference covering preclinical and clinical work in PKU, HCU, enteric hyperoxaluria, inflammatory bowel disease (IBD), and gout

From Process Development & Manufacturing Sciences (PDMS) at Synlogic, a presentation at the 2022 18th Annual Protein Engineering & Cell Therapy Summit (PEGS) Boston Conference & Expo
First Quarter 2022 Financial Results

As of March 31, 2022, Synlogic had cash, cash equivalents and short-term investments of $120.5 million.

Revenue was $0.2 million for the three months ended March 31, 2022. Revenue in 2022 was associated with the ongoing research collaboration with Roche for the discovery of a novel Synthetic Biotic for the treatment of IBD. There was no revenue for the three months ended March 31, 2021.

For the three months ended March 31, 2022, Synlogic reported a consolidated net loss of $15.7 million, or $0.22 per share, compared to a consolidated net loss of $15.0 million, or $0.36 per share, for the corresponding period in 2021.

Research and development expenses were $11.7 million for the three months ended March 31, 2022 compared to $11.2 million for the corresponding period in 2021.

General and administrative expenses were $4.3 million for the three months ended March 31, 2022 compared to $3.9 million for the corresponding period in 2021.

Financial Outlook

Based upon its current operating plan and balance sheet as of March 31, 2022, Synlogic expects to have sufficient cash to be able to fund operations into 2024.

Investor & Industry Conference Participation

Synlogic will participate in the following upcoming investor and industry conferences:

RBC Capital Markets Global Healthcare Conference. Dr. Brennan will provide a Company presentation on Wednesday, May 18, 2022 at 8:30 am ET during the RBC Capital Markets Global Healthcare Conference to be held in New York City from May 17-18, 2022.

The H.C. Wainwright Global Investment Conference. Chief Financial Officer Michael Jensen and Chief Business Officer Molly Harper will provide a company presentation on Wednesday, May 25, 2022 at 10:30 am ET during the H.C. Wainwright Global Investment Conference taking place from May 23- 26, 2022 in Miami, Florida.

BIO International Convention. Synlogic will participate in the BIO International Convention taking place from June 13-16, 2022 in San Diego, California, including a company presentation by Ms. Harper.
Live webcasts of the presentations, if available, will be accessible under the "Event Calendar" in the Investors & Media section of the Synlogic website. Archived versions will be available afterwards at the same location.

Conference Call & Webcast Information

Synlogic will host a conference call and live webcast at 8:30 a.m. ET today, May 12, 2022. To access the live webcast, please visit the "Event Calendar" page within the Investors and Media section of the Synlogic website. Investors may listen to the call by dialing +1 (844) 815-2882 from locations in the United States or (213) 660-0926 from outside the United States. The conference ID number is 5149412. A replay will be available for 30 days on the Investors and Media section of the Synlogic website.

On May 12, 2022 Moleculin Biotech, Inc., (Nasdaq: MBRX) ("Moleculin" or the "Company"), a clinical stage pharmaceutical company with a broad portfolio of drug candidates targeting highly resistant tumors and viruses, reported its financial results for the quarter ended March 31, 2022 (Press release, Moleculin, MAY 12, 2022, View Source [SID1234614396]). The Company also provided an update on its portfolio of oncology drug candidates for the treatment of highly resistant tumors and viruses.

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"We have continued to make excellent progress across our entire pipeline since the start of 2022, paving the way for important data readouts throughout this year," commented Walter Klemp, Chairman and CEO of Moleculin. "Importantly, our clinical and regulatory strategies have expanded significantly outside of the U.S. and we recently bolstered the expertise of our leadership team to drive our EU clinical trials forward. Our development programs for Annamycin in STS lung mets and AML, as well as WP1122 for COVID-19 and GBM continue to advance. With the progress we’ve made and the milestones ahead, 2022 is poised to be an important year for not only providing more clarity for each clinical program’s path towards registration, but also an opportunity to unlock value for all stakeholders, and importantly, addressing unmet needs for people with highly resistant tumors and viruses."

Recent Highlights
Received required authorizations for an amendment to commence a Phase 1a clinical trial of WP1122 in the United Kingdom (UK) for the treatment of COVID-19.
Received allowance from the Polish Department of Registration of Medicinal Products (URPL), as well as the requisite Ethics Committee approval, to proceed with its Phase 1/2 clinical trial in Poland of Annamycin (L-ANN) in combination with Cytarabine (Ara-C) in the treatment of subjects with acute myeloid leukemia (AML) who are refractory to or relapsed after induction therapy.
Engaged Wolfram C. M. Dempke, MD, PhD, MBA as its European Chief Medical Officer and part-time contractor for its European clinical trials.
Received IND clearance to conduct Phase 1 study of WP1066 for the treatment of recurrent malignant glioma.
Presented preclinical data at the AACR (Free AACR Whitepaper) 2022 Annual Meeting demonstrating Annamycin exhibited robust antitumor activity in experimental colorectal cancer liver and lung metastasis models.
Launched new corporate branding and website.
Programs Update
Next Generation Anthracycline – Annamycin
Annamycin is the Company’s next-generation anthracycline that has been shown in animal models to accumulate in the lungs at up to 30-fold the level of doxorubicin, as well as demonstrating the ability to avoid the multidrug resistance mechanisms that typically limit the efficacy of doxorubicin and other currently prescribed anthracyclines. Importantly, Annamycin has also demonstrated a lack of cardiotoxicity in multiple human clinical trials, including ongoing trials for the treatment of relapsed or refractory acute myeloid leukemia (AML) and soft tissue sarcoma (STS) lung metastases, and the Company believes that the use of Annamycin may not face the same usage limitations imposed on doxorubicin, one of the most common currently prescribed anthracyclines. Annamycin is currently in development for the treatment of AML and STS lung metastases and the Company believes it may have the potential to treat a number of additional indications.

Annamycin currently has Fast Track Status and Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for the treatment of STS lung metastases, in addition to Orphan Drug Designation for the treatment of relapsed or refractory acute myeloid leukemia.

For more information about the Phase 1b/2 study evaluating Annamycin for the treatment of STS lung metastases, please visit clinicaltrials.gov and reference identifier NCT04887298.

Upcoming Milestones Expectations
Q2 2022: Commence Phase 1/2 study in Europe for the treatment of AML evaluating combination therapy of Annamycin + Ara-C.
Q2 2022: Commencement of an investigator-funded, second Phase 1b/2 clinical trial of Annamycin in sarcoma lung metastases in Europe.
Q2 2022: Report topline data from Phase 1b portion of ongoing Phase 1b/2 study of Annamycin for the treatment of sarcoma lung metastases in the US.
Metabolism/Glycosylation Inhibitor – WP1122
WP1122, the Company’s lead metabolism/glycosylation inhibitor, is a prodrug of a well-known glucose decoy called 2-deoxy-D-glucose (2-DG), currently being developed by an unrelated third party in India for inhibition of viral replication and disease manifestations in humans infected with SARS-CoV-2, the virus responsible for COVID-19. The Company is also evaluating WP1122 for the treatment of Glioblastoma Multiforme (GBM). The mechanism of action of 2-DG includes both the inhibition of glycolysis and the disruption of glycosylation, two processes that are important to both viral activity and tumor development. WP1122 was developed as a 2-DG prodrug to provide a more favorable pharmacological profile, and was found to have greater potency than 2-DG alone in preclinical models where tumor cells require higher glycolytic activity than normal cells. WP1122 has also been shown to have a more potent antiviral effect than 2-DG against SARS-CoV-2 in MRC-5 cells (one of the most common human-derived cell lines used for antiviral research) in culture.

COVID-19

The Company has received authorization from the Medicines and Healthcare Products Regulatory Agency (MHRA) to commence a Phase 1a clinical trial of WP1122 in the United Kingdom (UK). The Company also received a favorable opinion from the London – Riverside Research Ethics Committee in the UK to begin the study, which is expected to be conducted at the Medicines Evaluation Unit in Manchester, United Kingdom. In May 2022, the Company received approval from both the Riverside Ethics Committee and the MHRA for its amended study protocol to update the dilution of the oral solution to achieve full dissolution of WP1122. No risk/benefit to the study was affected because of this change. The Phase 1a study in healthy human volunteers will investigate the effects of a single ascending dose (SAD) and multiple days of ascending dosing (MAD) of WP1122 administered as an oral solution. Dose escalation will take place in sequential SAD cohorts, and MAD will start as soon as SAD has completed at least 3 dosing cohorts in which WP1122 is found to be safe and well-tolerated. This study in healthy volunteers will explore safety and pharmacokinetics (PK), and subsequent clinical development will be in patients infected with SARS-CoV-2 to further evaluate safety and establish a favorable risk/benefit profile. The Company expects to enroll approximately 80 healthy volunteers in the United Kingdom.

Glioblastoma Multiforme

Additionally, Moleculin recently received IND clearance from the U.S. Food and Drug Administration (FDA) to initiate a Phase 1 open label, single arm, dose escalation study of the safety, pharmacokinetics and efficacy of oral WP1122 in adult patients with GBM, which the Company expects to commence in 2022.

Upcoming Milestones Expectations
Q2 2022: Commence recruitment in Phase 1a study of WP1122 for the treatment of COVID-19 in the UK.
H2 2022: Potential to launch Phase 2 study of WP1122 for the treatment of COVID-19 outside of the US.
2022: Identify investigators interested in initiating a Phase 1 open label, single arm, dose escalation study of the safety, pharmacokinetics and efficacy of oral WP1122 in adult patients with GBM.
Ongoing preclinical development work in additional anti-viral indications such as HIV, Zika, and Dengue. Collaborations targeted for 2022.
Summary of Financial Results for the First Quarter 2022
Research and development (R&D) expense was $4.6 million and $4.1 million for the three months ended March 31, 2022 and 2021, respectively. The increase of $0.5 million is mainly related to increased clinical trial activity as described above, a license termination fee, and costs related to manufacturing of additional drug product.

General and administrative expense was $2.4 million and $1.9 million for the three months ended March 31, 2022 and 2021, respectively. The increase of $0.5 million is mainly related to an increase in regulatory legal services, consulting and advisory fees.

For the three months ended March 31, 2022 and 2021, the Company reported a net loss of $6.9 million and $4.4 million, respectively, and had net cash flows used in operating activities of $4.8 million and $3.6 million, respectively.

The Company ended the quarter with $66.1 million of cash. The Company believes that this cash is sufficient to meet its projected operating requirements, which include a forecasted increase over its current R&D rate of expenditures, into 2024.

On May 12, 2022 Biodesix, Inc. (Nasdaq: BDSX), a leading data-driven diagnostic solutions company with a focus in lung disease, reported new clinical utility data highlighting the impact of the Nodify XL2 Risk Assessment Test on clinical management decisions in patients with lung nodules will be presented at the American Thoracic Society (ATS) 2022 International Conference (Press release, Biodesix, MAY 12, 2022, View Source [SID1234614416]). The meeting will take place May 13-18, 2022, in San Francisco, California.

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Data from the abstract titled, The Impact of Plasma Proteomics Biomarker on Clinical Management Decision in Lung Nodules, presented by Dr. J. P. Uribe of Deaconess Medical Center, Boston, Massachusetts, United States, will highlight data that show the impact of the Nodify XL2 integrated proteomic classifier on management decisions in a "real world" clinical setting. Conclusions show that when used in patients with lung nodules with a nodule probability of malignancy (pCA) of ≤ 50%, the blood-based lung nodule risk assessment test was able to support a decrease in chest imaging, outpatient clinic visits and additional invasive procedures without misclassifying benign lung nodules.

An additional presentation titled, ALTITUDE trial design: A Multicenter, Randomized Controlled Trial, Prospectively Evaluating the Clinical Utility of the Nodify XL2 Proteomic Classifier in Incidentally Discovered Low to Moderate Risk Lung Nodules, will review the ALTITUDE study, a first-in-class biomarker study which aligned with the recommendations from the official 2018 American Thoracic Society (ATS) policy statement on the early detection of lung cancer. The abstract, authored by Michael N. Kammer, Ph.D. of Vanderbilt University Medical Center, Nashville, Tennessee, Steve Springmeyer, MD, Biodesix, Boulder, Colorado and Gerard Silvestri, MD of Medical University of South Carolina, Charleston, South Carolina will be presented at ATS by Dr. Michael Kammer.

Both poster presentations will occur during ATS discussion Session B30 – THE QUEST FOR THE HOLY GRAIL: MODELING AND BIOMARKERS FOR NODULES AND LUNG CANCER. The session, moderated by Edwin Ostrin, MD, PhD. from MD Anderson Cancer Center, Houston, Texas and Adam Fox, MD, from Medical University of South Carolina, Charleston, South Carolina, will occur at the ATS meeting on May 16th, 2022, from 9:30-11:00AM PT, Room 203-204 (South Building, Level 2), Moscone Center.

On May 12, 2022 Jazz Pharmaceuticals plc (Nasdaq: JAZZ) reported that the Company and its partners will present seven abstracts at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting from June 3 – June 7, 2022, and eight abstracts at the 27th Annual Congress of the European Hematology Association (EHA) (Free EHA Whitepaper) from June 9 –12, 2022. Research findings to be presented include data on Rylaze/JZP458, Zepzelca (lurbinectedin), Defitelio (defibrotide sodium) and Vyxeos/Vyxeos Liposomal (daunorubicin and cytarabine), also known as JZP351 (Press release, Jazz Pharmaceuticals, MAY 12, 2022, View Source [SID1234614434]).

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"As part of Jazz’s commitment to explore potential new applications of our oncology medicines to address critical needs, we continue to advance programs that could impact difficult-to-treat therapeutic areas," said Rob Iannone, M.D., M.S.C.E., executive vice president, global head of research and development of Jazz Pharmaceuticals. "Our growing, early-stage pipeline, combined with ongoing Jazz-sponsored and partner research across our portfolio, is making significant progress when it comes to addressing unmet patient needs in cancers that have historically lacked scientific advancements."

Highlights from Jazz and its investigational sponsors at the congresses feature data for our medicines across a range of solid tumors and hematological malignancies, including:

An oral presentation at ASCO (Free ASCO Whitepaper) featuring results from Cohort 1 of a Phase 2/3 trial conducted with the Children’s Oncology Group, evaluating the efficacy and safety of Rylaze administered intramuscularly (IM) on a Monday/Wednesday/Friday dosing schedule, for patients living with acute lymphoblastic leukemia (ALL) or lymphoblastic lymphoma (LBL).
Two poster presentations for JZP351 including preliminary results from Arm B (JZP351 in combination with midostaurin) from the Phase 1b V-FAST trial in adults with previously untreated FLT3-mutated acute myeloid leukemia (AML) and data from the Phase 1b trial evaluating lower-intensity JZP351 + venetoclax in adults with newly diagnosed AML who are unfit for intensive chemotherapy. These data will be presented at both ASCO (Free ASCO Whitepaper) and EHA (Free EHA Whitepaper).
Four poster presentations at ASCO (Free ASCO Whitepaper) evaluating Zepzelca in a range of small cell lung cancer (SCLC) settings, both as a monotherapy and in combination with other therapies, and in BRCA1/2-associated metastatic breast cancer.
The Jazz-supported presentations at the 2022 ASCO (Free ASCO Whitepaper) Annual Meeting are:

Rylaze Presentations

Presentation Title

Author

Presentation Details

Efficacy and safety of intramuscular (IM) recombinant Erwinia asparaginase in acute lymphoblastic leukemia (ALL) or lymphoblastic lymphoma (LBL): The Children’s Oncology Group (COG) AALL1931 study

Maese L, et al.

Type: Oral presentation

Session: Oral Abstract Session/ Hematologic Malignancies—Leukemia, Myelodysplastic Syndromes, and Allotransplant

Date: June 7 at 10:45 a.m. EDT

Abstract number: 7001

Abstract Link

Zepzelca Presentations

Presentation Title

Author

Presentation Details

Analysis of patients with relapsed small cell lung cancer (SCLC) receiving single-agent lurbinectedin in the phase 3 ATLANTIS trial

Navarro A, et al.

Type: Poster

Session: Lung Cancer – Non-Small Cell Local-Regional/Small Cell/Other Thoracic Cancers

Date: June 6 at 9:00 a.m. EDT

Abstract number: 8524

Abstract link

Efficacy and safety of lurbinectedin as second-line therapy in Chinese patients with small cell lung cancer: Preliminary results of a phase 1 study

Cheng Y, et al.

Type: Poster

Session: Lung Cancer – Non-Small Cell Local-Regional/Small Cell/Other Thoracic Cancers

Date: June 6 at 9:00 a.m. EDT

Abstract number: 8580

Abstract link

A phase 1/2 trial of lurbinectedin (L) in combination with pembrolizumab (P) in relapsed small cell lung cancer (SCLC): The LUPER study

Calles Blanco A, et al.

Type: Poster

Session: Lung Cancer – Non-Small Cell Local-Regional/Small Cell/Other Thoracic Cancers

Date: June 6 at 9:00 a.m. EDT

Abstract number: 8581

Abstract link

Lurbinectedin in patients with pretreated BRCA1/2-associated metastatic breast cancer: Results from a phase II basket study

Boni V, et al.

Type: Poster

Session: Breast Cancer – Metastatic

Date: June 6 at 9:00 a.m. EDT

Abstract number: 1092

Abstract link

Vyxeos Presentations

Presentation Title

Author

Presentation Details

V-FAST master trial: Preliminary results of treatment with CPX-351 plus midostaurin in adults with newly diagnosed FLT3-mutated acute myeloid leukemia

McCloskey J, et al.

Type: Poster

Session: Hematologic Malignancies – Leukemia, Myelodysplastic Syndromes, and Allotransplant

Date: June 4 at 9:00 a.m. EDT

Abstract number: 7043

Abstract link

Lower-intensity CPX-351 + venetoclax for patients with newly diagnosed AML who are unfit for intensive chemotherapy

Uy G.L. et al.

Type: Poster

Session: Hematologic Malignancies – Leukemia, Myelodysplastic Syndromes, and Allotransplant

Date: June 4 at 9:00 a.m. EDT

Abstract number: 7031

Abstract link

The Jazz-supported presentations at the EHA (Free EHA Whitepaper) 27th Congress are:

Vyxeos Liposomal Presentations

Presentation Title

Author

Presentation Details

Lower-intensity CPX-351 + venetoclax for patients with newly diagnosed acute myeloid leukemia who are unfit for intensive chemotherapy

Uy L. G. et al.

Type: Poster

Date: June 10 at 16:30 CEST

Abstract number: P515

Abstract link

CPX-351 treatment for acute myeloid leukemia in England: Real-world outcomes in adults aged <60 years versus >60 years

Legg A. et al.

Type: Poster

Date: June 10 at 16:30 CEST

Abstract number: P513

Abstract link

V-FAST master trial: Preliminary results of treatment with CPX-351 plus midostaurin in adults with newly diagnosed FLT3-mutated acute myeloid leukemia

McCloskey J, et al

Type: Poster

Date: June 10 at 16:30 CEST

Abstract number: P514

Abstract link

Real life experience using front-line CPX-351 for therapy-related and AML-MRC: results from the Spanish PETHEMA registry (IST)

Bernal T, et al

Type: Poster

Date: June 10 at 16:30 CEST

Abstract number: P508

Abstract link

A randomised comparison of CPX-351 and FLAG-Ida in high risk acute myeloid leukaemia. Results from the NCRI AML19 trial

Russel N, et al

Type: Oral presentation

Date: June 11

Abstract number: S128

Abstract link

Defitelio Presentations

Presentation Title

Author

Presentation Details

Veno-occlusive disease/sinusoidal obstruction syndrome (VOD/SOS) after autologogous hematopoietic cell transplantion (HCT): Outcomes of defibrotide-treated adult patients from the DefiFrance study

Mohty M, et al

Type: Poster

Date: June 10 at 16:30 CEST

Abstract number: P1355

Abstract link

A systematic literature review (SLR) of the manifestations of veno-occlusive disease/sinusoidal obstruction syndrome (VOD/SOS) after hematopoietic cell transplant (HCT) in adults versus children

Angus J, et al.

Type: Poster

Date: June 10 at 16:30 CEST

Abstract number: P1354

Abstract link

Asparaginase Presentations

Presentation Title

Author

Presentation Details

Phase 1 trial of pegcrisantaspase in combination with venetoclax in adults with relapsed or refractory acute myeloid leukemia (R/R AML) – Safety, efficacy and PK/PD in the first two cohorts

Liu Y, et al

Type: Poster

Date: June 10 at 16:30 CEST

Abstract number: P527

Abstract link

All ASCO (Free ASCO Whitepaper) virtual poster presentations and poster discussion presentations will be available on-demand to registered participants for 180 days beginning June 3, 2022. EHA (Free EHA Whitepaper) presentations will be available on-demand beginning June 20, 2022.

About Zepzelca (lurbinectedin)
Zepzelca is an alkylating drug that binds guanine residues within DNA. This triggers a cascade of events that can affect the activity of DNA binding proteins, including some transcription factors, and DNA repair pathways, resulting in disruption of the cell cycle and eventual cell death.2

Zepzelca for injection 4 mg is a prescription medicine used to treat adults with a kind of lung cancer called small cell lung cancer that has spread to other parts of the body (metastatic) and who have received treatment with chemotherapy that contains platinum, and it did not work or is no longer working. Zepzelca is approved based on response rate and how long the response lasted. Additional studies will further evaluate the benefit of Zepzelca for this use.

Important Safety Information for ZEPZELCA

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

have liver or kidney problems.
are pregnant or plan to become pregnant. ZEPZELCA can harm your unborn baby.
Females who are able to become pregnant:
Your healthcare provider should do a pregnancy test before you start treatment with ZEPZELCA.
You should use effective birth control (contraception) during treatment with and for 6 months after your final dose of ZEPZELCA.
Tell your healthcare provider right away if you become pregnant or think that you are pregnant during treatment with ZEPZELCA.
Males with female partners who are able to become pregnant should use effective birth control during treatment with and for 4 months after your final dose of ZEPZELCA.

Are breastfeeding or plan to breastfeed. It is not known if ZEPZELCA passes into your breastmilk. Do not breastfeed during treatment with ZEPZELCA and for 2 weeks after your final dose of ZEPZELCA. Talk to your healthcare provider about the best way to feed your baby during treatment with ZEPZELCA.
Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Certain other medicines may affect how ZEPZELCA works.

What should I avoid while using ZEPZELCA?

Avoid eating or drinking grapefruit, or products that contain grapefruit juice during treatment with ZEPZELCA.

ZEPZELCA can cause serious side effects, including:

Low blood cell counts. Low blood counts including low neutrophil counts (neutropenia) and low platelet counts (thrombocytopenia) are common with ZEPZELCA, and can also be severe. Some people with low white blood cell counts may get fever, or an infection throughout the body (sepsis), that can cause death. Your healthcare provider should do blood tests before you receive each treatment with ZEPZELCA to check your blood cell counts.
Tell your healthcare provider right away if you develop:

fever or any other signs of infection
unusual bruising or bleeding
tiredness
pale colored skin
Liver problems. Increased liver function tests are common with ZEPZELCA, and can also be severe. Your healthcare provider should do blood tests to check your liver function before you start and during treatment with ZEPZELCA.
Tell your healthcare provider right away if you develop symptoms of liver problems including:

loss of appetite 
nausea or vomiting 
pain on the right side of your stomach area (abdomen)
Your healthcare provider may temporarily stop treatment, lower your dose, or permanently stop ZEPZELCA if you develop low blood cell counts or liver problems during treatment with ZEPZELCA.

The most common side effects of ZEPZELCA include:

tiredness
low white and red blood cell counts
increased kidney function blood test (creatinine)
increased liver function blood tests
increased blood sugar (glucose)
nausea
decreased appetite
muscle and joint (musculoskeletal) pain
low level of albumin in the blood
constipation
trouble breathing
low levels of sodium and magnesium in the blood
vomiting
cough
diarrhea
These are not all of the possible side effects of ZEPZELCA.

Call your doctor for medical advice about side effects. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088. You may also report side effects to Jazz Pharmaceuticals at 1-800-520-5568.

More information about Zepzelca, including Full Prescribing Information and Patient Information, is available here.

ZEPZELCA is a trademark of PharmaMar, S.A. used by Jazz Pharmaceuticals under license.

About RYLAZE (asparaginase erwinia chrysanthemi (recombinant)-rywn)
RYLAZE, also known as JZP458, is approved in the U.S. for use as a component of a multi-agent chemotherapeutic regimen for the treatment of acute lymphoblastic leukemia (ALL) and lymphoblastic lymphoma (LBL) in adult and pediatric patients one month or older who have developed hypersensitivity to E. coli-derived asparaginase. RYLAZE has orphan drug designation for the treatment of ALL/LBL in the United States. RYLAZE is a recombinant erwinia asparaginase that uses a novel Pseudomonas fluorescens expression platform. JZP458 was granted Fast Track designation by the U.S. Food and Drug Administration (FDA) in October 2019 for the treatment of this patient population. RYLAZE was approved as part of the Real-Time Oncology Review program, an initiative of the FDA’s Oncology Center of Excellence designed for efficient delivery of safe and effective cancer treatments to patients.

The full U.S. Prescribing Information for RYLAZE is available here.

Important Safety Information for Rylaze

RYLAZE should not be given to people who have had:

Serious allergic reactions to RYLAZE
Serious swelling of the pancreas (stomach pain), serious blood clots, or serious bleeding during previous asparaginase treatment
RYLAZE may cause serious side effects, including:

Allergic reactions (a feeling of tightness in your throat, unusual swelling/redness in your throat and/or tongue, or trouble breathing), some of which may be life-threatening
Swelling of the pancreas (stomach pain)
Blood clots (may have a headache or pain in leg, arm, or chest)
Bleeding
Liver problems
Contact your doctor immediately if any of these side effects occur.

Some of the most common side effects with RYLAZE include: liver problems, nausea, bone and muscle pain, tiredness, infection, headache, fever, allergic reactions, fever with low white blood cell count, decreased appetite, mouth swelling (sometimes with sores), bleeding, and too much sugar in the blood.

RYLAZE can harm your unborn baby. Inform your doctor if you are pregnant, planning to become pregnant, or nursing. Females of reproductive potential should use effective contraception (other than oral contraceptives) during treatment and for 3 months following the final dose. Do not breastfeed while receiving RYLAZE and for 1 week after the final dose.

Tell your healthcare provider if there are any side effects that are bothersome or that do not go away.

These are not all the possible side effects of RYLAZE. For more information, ask your healthcare provider.

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088 (1-800-332-1088).

About Vyxeos/Vyxeos Liposomal (daunorubicin and cytarabine), also known as JZP351
Vyxeos is a liposomal combination of daunorubicin, an anthracycline topoisomerase inhibitor, and cytarabine, a nucleoside metabolic inhibitor.

In the U.S., Vyxeos is indicated for the treatment of newly-diagnosed therapy-related acute myeloid leukemia (t-AML) or AML with myelodysplasia-related changes (AML-MRC) in adults and pediatric patients 1 year and older.1

More information about Vyxeos in the United States, including Full Prescribing Information, BOXED Warning and Medication Guide, is available here.

In Europe, Vyxeos Liposomal (daunorubicin/cytarabine) is indicated for the treatment of adults with newly diagnosed, therapy-related acute myeloid leukemia (t-AML) or AML with myelodysplasia-related changes (AML-MRC). Backed by a robust clinical development program including Phase 3 data, Vyxeos is currently approved in more than 30 countries, and Jazz continues to work with regulatory authorities worldwide to bring this innovative therapy to appropriate patients.

The full Summary of Product Characteristics of Vyxeos Liposomal in Europe is available here.

Important Safety Information for VYXEOS/VYXEOS LIPOSOMAL
WARNING: VYXEOS has different dosage recommendations from other medications that contain daunorubicin and/or cytarabine. Do not substitute VYXEOS for other daunorubicin and/or cytarabine-containing products.

VYXEOS should not be given to patients who have a history of serious allergic reaction to daunorubicin, cytarabine, or any of its ingredients.

VYXEOS can cause a severe decrease in blood cells (red and white blood cells and cells that prevent bleeding, called platelets) which can result in serious infection or bleeding and possibly lead to death. Your doctor will monitor your blood counts during treatment with VYXEOS. Patients should tell the doctor about new onset fever or symptoms of infection or if they notice signs of bruising or bleeding.

VYXEOS can cause heart-related side effects. Tell your doctor about any history of heart disease, radiation to the chest, or previous chemotherapy. Inform your doctor if you develop symptoms of heart failure such as:

shortness of breath or trouble breathing
swelling or fluid retention, especially in the feet, ankles, or legs
unusual tiredness
VYXEOS may cause allergic reactions including anaphylaxis. Seek immediate medical attention if you develop signs and symptoms of anaphylaxis such as:

trouble breathing
severe itching
skin rash or hives
swelling of the face, lips, mouth, or tongue
VYXEOS contains copper and may cause copper overload in patients with Wilson’s disease or other copper-processing disorders.

VYXEOS can damage the skin if it leaks out of the vein. Tell your doctor right away if you experience symptoms of burning, stinging, or blisters and skin sores at the injection site.

VYXEOS can harm your unborn baby. Inform your doctor if you are pregnant, planning to become pregnant, or nursing. Do not breastfeed while receiving VYXEOS. Females and males of reproductive potential should use effective contraception during treatment and for 6 months following the last dose of VYXEOS.

The most common side effects were bleeding events, fever, rash, swelling, nausea, sores in the mouth or throat, diarrhea, constipation, muscle pain, tiredness, stomach pain, difficulty breathing, headache, cough, decreased appetite, irregular heartbeat, pneumonia, blood infection, chills, sleep disorders, and vomiting.

Call your doctor for medical advice about side effects. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088. You may also report side effects to Jazz Pharmaceuticals at 1-800-520-5568.

About Defitelio (defibrotide sodium)
In the U.S., Defitelio (defibrotide sodium) injection 80mg/mL received U.S. Food and Drug Administration (FDA) marketing approval on March 30, 2016, and it is indicated for the treatment of adult and pediatric patients with hepatic veno-occlusive disease (VOD), also known as sinusoidal obstruction syndrome (SOS), with renal or pulmonary dysfunction following hematopoietic stem-cell transplantation (HSCT) and is the first and only FDA-approved therapy for patients with this rare, potentially fatal complication. Defitelio is not approved for the prevention of VOD.

Please see full Prescribing Information for Defitelio in the United States.

In Europe, defibrotide is marketed under the name Defitelio ▼ (defibrotide). In October 2013, the European Commission granted marketing authorization to Defitelio under exceptional circumstances for the treatment of severe VOD in patients after HSCT therapy. In Europe, Defitelio is indicated in patients over one month of age. It is not indicated in patients with hypersensitivity to defibrotide or any of its excipients or with concomitant use of thrombolytic therapy.

▼This medicinal product is subject to additional monitoring. This will allow quick identification of new safety information. Healthcare professionals are asked to report any suspected adverse reactions via the national reporting system found under section 4.8 of the SmPC.
(View Source)

The full Summary of Product Characteristics of Defitelio in Europe is available here.

Important Safety Information for Defitelio
Defitelio should not be given to patients who are:

Currently taking anticoagulants or fibrinolytics
Allergic to Defitelio or any of its ingredients
Defitelio may increase the risk of bleeding in patients with VOD and should not be given to patients with active bleeding. During treatment with Defitelio, patients should be monitored for signs of bleeding. In the event that bleeding occurs during treatment with Defitelio, treatment should be temporarily or permanently stopped. Patients should tell the doctor right away about any signs or symptoms of hemorrhage such as unusual bleeding, easy bruising, blood in urine or stool, headache, confusion, slurred speech, or altered vision.

Defitelio may cause allergic reactions including anaphylaxis. Patients who develop signs and symptoms of anaphylaxis such as trouble breathing, severe itching, skin rash or hives, or swelling of the face, lips, mouth or tongue should seek medical attention immediately.

The most common side effects of Defitelio are decreased blood pressure, diarrhea, vomiting, nausea and nose bleeds.

On May 12, 2022 Molecular Templates, Inc. (Nasdaq: MTEM, "Molecular Templates," or "MTEM"), a clinical-stage biopharmaceutical company focused on the discovery and development of proprietary targeted biologic therapeutics, engineered toxin bodies (ETBs), reported financial results for the first quarter of 2022 (Press release, Molecular Templates, MAY 12, 2022, View Source [SID1234614480]).

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"We continue to make meaningful progress in advancing our pipeline of ETBs," said Eric Poma, Ph.D., Chief Executive and Chief Scientific Officer of Molecular Templates. "We recently commenced dosing in Cohort 3 of our Phase 1 study of MT-6402 in PD-L1+ patients. We intend to report additional data from this study in the second half of the year. Dose finding in the MT-5111 and MT-0169 programs is ongoing with clinical data expected this year. We look forward to continued momentum across our pipeline in 2022, including filing an IND for MT-8421, our ETB targeting CTLA-4, and advancing our earlier stage pipeline of ETBs targeting TIGIT, TROP-2, and BCMA."

Company Highlights and Upcoming Milestones

Corporate

MTEM expects to provide periodic updates on MT-6402, MT-5111, and MT-0169 throughout 2022.
MTEM hosted a webinar on MT-6402 (PD-L1 ETB with Antigen Seeding Technology) with David Spigel, M.D. of the Sarah Cannon Research Institute on April 13. A replay of the event (including slides) can be accessed here.
Abstracts on MT-6402 and MT-5111 (HER2 ETB) have been accepted for presentation at the 2022 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) annual meeting, to take place June 3-7, 2022, in Chicago, IL.
Gabriela Gruia, M.D. appointed to Board of Directors.
Megan Filoon promoted to General Counsel.
Immuno-oncology ETBs:

MT-6402 (PD-L1 ETB with Antigen Seeding Technology)

Patient enrollment continues in the Phase 1 study of MT-6402 which began in July 2021. MT-6402 is a 3rd generation ETB designed to induce potent anti-tumor effects via PD-L1 targeting through multiple mechanisms that may overcome the limitations of approved checkpoint inhibitors.
The Phase 1 study is a multi-center, open-label, dose escalation and dose expansion trial in the United States. Patients with confirmed PD-L1 expressing tumors or confirmed PD-L1 expression in the tumor microenvironment (TME) are eligible for enrollment.
As of March 2022, twelve patients with relapsed/refractory tumors that express PD-L1 have been treated to date across two dose cohorts: 16 mcg/kg (n=6) and 24 mcg/kg (n=6). Dosing continues with three patients currently enrolled in the 32 mcg/kg cohort (cohort 3).
One patient in cohort 1 (16 mcg/kg) with non-small cell lung cancer (NSCLC) that had progressed after prior checkpoint therapy (PD-1 and CTLA-4) had evaluable-only multiple sites of bone disease that appeared to have resolved on bone scan with only one remaining site which showed decreased uptake. This patient remained on MT-6402 up to cycle 8 when increased uptake was noted on bone scan and treatment was discontinued.
The 16 mcg/kg cohort was completed with no dose-limiting toxicities (DLTs) observed. One DLT was observed in a single patient in cohort 2 (24 mcg/kg). The patient experienced dermatitis that resolved rapidly with systemic steroids. The patient was rechallenged without incident at 24 mcg/kg. No other DLTs have been reported.
Following determination of the maximum tolerated dose (MTD), MTEM plans expansion cohorts to evaluate MT-6402 as a monotherapy in tumor-specific and PD-L1 positive basket tumor cohorts.
MTEM continues to observe pharmacodynamic (PD) effects including monocyte depletion and T cell activation in the 24 mcg/kg cohort. The extent and timing of these PD effects appear dose-related with patients in the 24 mcg/kg generally showing a more rapid and profound PD effect, including monocyte depletion and T cell activation, potentially in a dose-dependent manner.
These PD effects associated with immune activation were seen across the majority of patients irrespective of HLA type or level of tumor PD-L1 staining. The patient that demonstrated tumor regression was one of two patients treated with high tumor PD-L1 expression and may represent engagement of direct tumor cell-kill and antigen seeding.
MT-8421 (CTLA-4 ETB)

Preclinical data from MTEM’s CTLA-4 program were featured in a poster at the AACR (Free AACR Whitepaper) annual meeting held April 8-13, 2022. In a transgenic mouse model expressing human CTLA-4 and bearing syngeneic subcutaneous tumors, MT-8421 treatment depleted immune suppressive regulatory T cells (Tregs) in the TME.
MT-8421 was well tolerated in a non-human primate toxicology study and achieved serum levels well-above projected IC50 concentrations for Tregs in the TME.
IND filing for MT-8421 is expected in 2H22, with clinical studies expected to commence in 2023.
MT-6402 and MT-8421 represent MTEM’s unique approach to immuno-oncology based on dismantling the TME through direct cell-kill of tumor and immune cells and not just the blocking of ligand-ligand interactions seen with current antibody therapeutics.
Research

MTEM continues to expand its unique approach to immuno-oncology targets with lead optimization on a TIGIT-targeting ETB on-going and additional exploration around new immuno-oncology targets.
Targeted Solid Tumor ETBs:

MT-5111 (HER2 ETB)

The Phase 1 study of MT-5111 in HER2-positive cancers is ongoing with multiple sites open for enrollment.
The HER2-positive breast cancer expansion cohort initiated in November 2021 at a dose of 10 mcg/kg.
As of January 2022, 30 patients had been treated with MT-5111 across eight dose escalation cohorts ranging from 0.5 mcg/kg to 13 mcg/kg without any DLTs. Enrollment in the 17 mcg/kg cohort has been initiated.
Dose escalation will continue to determine the MTD, while the breast cancer expansion cohort collects efficacy and safety data.
No signs of capillary leak syndrome (CLS) or significant cardiotoxicity have been observed to date with MT-5111.
Research

Lead optimization on a 3rd generation ETB targeting TROP-2 continues.
Hematologic Malignancy Targeted ETBs:

MT-0169 (CD38 ETB)

The revised protocol for the ongoing Phase 1 study in patients with relapsed/refractory multiple myeloma or non-Hodgkin’s lymphoma is now open. The revised protocol will explore a lower dose of MT-0169 to reduce the risk of adverse events observed at the initial dose and to enable patients to continue MT-0169 therapy for a longer duration that may drive tumor benefit. Importantly, the robust and rapid NK cell depletion that was observed at the starting dose is expected to be observed at lower doses.
MTEM is opening new sites for the Phase 1 study and anticipates enrollment beginning in the second quarter of 2022.
Research

Lead optimization on BCMA, SLAMF-7, and CD45 continues.
Financial Results

The net loss attributable to common shareholders for the first quarter of 2022 was $21.6 million, or $0.38 per basic and diluted share. This compares with a net loss attributable to common shareholders of $26.8 million, or $0.51 per basic and diluted share, for the same period in 2021.

Revenues for the first quarter of 2022 were $8.5 million, compared to $3.2 million for the same period in 2021. Revenues for the first quarter of 2022 were comprised of revenues from collaborative research and development agreements with Takeda and Bristol Myers Squibb.

Total research and development expenses for the first quarter of 2022 were $21.5 million, compared with $21.4 million for the same period in 2021. Total general and administrative expenses for the first quarter of 2022 were $7.6 million, compared with $8.2 million for the same period in 2021.

As of March 31, 2022, MTEM’s cash and investments totaled $124.5 million. MTEM’s current cash and investments are expected to fund operations to the end of 2023.

For more details on MTEM’s financial results for the first quarter 2022, refer to Form 10Q filed with the SEC.