On May 15, 2020 UroGen Pharma Ltd. (Nasdaq:URGN) reported the presentation of positive data from the UGN-101 (Jelmyto(mitomycin) for pyelocalyceal solution) Phase 3 OLYMPUS trial in patients with low-grade upper tract urothelial cancer (LG-UTUC) (Press release, UroGen Pharma, MAY 15, 2020, View Source [SID1234558182]). The study was accepted for the 2020 American Urological Association (AUA) Annual Meeting, published as a supplement to the April 2020 issue of The Journal of Urology and presented as part of the AUA Virtual Experience. The presentation can be accessed via the AUA website here. More information can also be accessed via UroGen’s virtual AUA experience here.

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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 Jelmyto (mitomycin) for pyelocalyceal solution to evaluate the safety, tolerability and tumor ablative effect of Jelmyto in patients with low-grade upper tract urothelial cancer. The trial enrolled 74 patients at clinical sites across the U.S. and Israel. Study participants were treated with six weekly instillations of Jelmyto administered via a standard ureteral 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. 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 Jelmyto (mitomycin) for pyelocalyceal solution

Jelmyto (mitomycin) for pyelocalyceal solution is a drug formulation of mitomycin for the treatment of low-grade upper tract urothelial cancer (LG-UTUC) in adult patients. 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. Food and Drug Administration granted Orphan Drug, Fast Track, and Breakthrough Therapy Designations to Jelmyto for the treatment of LG-UTUC. Jelmyto is the first drug approved for the treatment of LG-UTUC in adult patients.

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 15, 2020 Mirati Therapeutics, Inc. (NASDAQ: MRTX), a clinical-stage targeted oncology company, reported it will present preclinical data on the Company’s pipeline of novel therapeutics in three presentations at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Virtual Annual Meeting II, taking place June 22-24, 2020 (Press release, Mirati, MAY 15, 2020, View Source [SID1234558181]). The data to be presented will include preclinical findings on MRTX849, a novel and optimized KRAS G12C inhibitor.

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"We continue to develop an increasing understanding of the role of KRAS in the pathogenesis of cancers as well as the consequences of direct therapeutic inhibition of KRAS. We continue to aspire to apply these learnings toward rational drug development and to make a real and timely impact on patient lives," said James G. Christensen, Ph.D. Executive Vice President and Chief Scientific Officer, Mirati Therapeutics.

Late-Breaking Poster Presentation Details:
Title: The anti-tumor activity of the KRAS G12C inhibitor MRTX849 is augmented by cetuximab in CRC tumor models
Poster Session Title: Late-Breaking Research: Experimental and Molecular Therapeutics 2
Presentation Date: June 22, 2020 (available via AACR (Free AACR Whitepaper)’s online platform)
Presenter: Jill Hallin, Principal Scientist, Biology, Mirati Therapeutics
Poster Number: LB-098

Minisymposium Session Details:
Title: Drug-anchored in vitro and in vivo CRISPR screens to identify targetable vulnerabilities and modifiers of response to MRTX849 in KRAS G12C-mutant models
Session Title: Emerging Mechanisms of Resistance to Targeted Therapies
Presentation Date: June 24, 2020 (available via AACR (Free AACR Whitepaper)’s online platform)
Presenter: Lars Engstrom, Principal Scientist, Biology, Mirati Therapeutics
Abstract Number: 5684

Poster Presentation Details:
Title: MRTX2219 is an imidazopyrimidine binder to EED that inhibits polycomb repressive complex 2 (PRC2) and demonstrates robust in vivo activity in EZH2 and SMARCB1-mutant, but not other SWI/SNF-mutant cancer models
Poster Session Title: Epigenetic Targets
Presentation Date: June 22, 2020 (available via AACR (Free AACR Whitepaper)’s online platform)
Presenter: David Briere, Principal Scientist, Biology, Mirati Therapeutics
Poster Number: 1769

About MRTX849

MRTX849 is an investigational, orally available small molecule that is designed to potently and selectively inhibit a form of KRAS which harbors a substitution mutation (G12C). KRAS G12C mutations are present in approximately 14% of non-small cell lung cancer adenocarcinoma patients, 4% of colorectal cancer patients, and subsets of other types of cancer. Tumors characterized by KRAS G12C mutations are commonly associated with poor prognosis and resistance to therapy, and patients with these mutations have few treatment options. MRTX849 is being evaluated in a Phase 1/2 trial treating patients with molecularly identified, KRAS G12C-positive advanced solid tumors.

On May 15, 2020 CRISPR Therapeutics (Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, reported that four abstracts have been accepted for poster presentation at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Virtual Annual Meeting II, which will take place from June 22 to 24, 2020 (Press release, CRISPR Therapeutics, MAY 15, 2020, View Source [SID1234558180]).

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Session information is available online via the Annual Meeting Itinerary Planner through the AACR (Free AACR Whitepaper) website at www.aacr.org.

Title: Functional and single-cell assessment of CRISPR-modified CAR-T cells from NSCLC patients and healthy donors
Session Title: Adoptive Cell Therapy 1
E-Poster Number: 879
Abstract Number: 3338

Title: Allogeneic CAR-T cell products containing 10 gene edits using CRISPR/Cas9 can retain full functionality in vivo and in vitro
Session Title: Adoptive Cell Therapy 1
E-Poster Number: 880
Abstract Number: 4647

Title: Allogeneic anti-PTK7 CAR-T cells for the treatment of solid tumors
Session Title: Adoptive Cell Therapy 3
E-Poster Number: 3243
Abstract Number: 6231

Title: Targeting T cell lymphomas with CRISPR/Cas9-generated anti-CD70 allogeneic CAR-T cells
Session Title: Adoptive Cell Therapy 5
E-Poster Number: 6595
Abstract Number: 3308

On May 15, 2020 Precigen, Inc., a biopharmaceutical company specializing in the development of innovative gene and cell therapies to improve the lives of patients, reported preclinical data for its innovative investigational PRGN-3005 UltraCAR-T in patients with advanced, recurrent platinum resistant ovarian, fallopian tube or primary peritoneal cancer will be presented as an e-poster at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Virtual Annual Meeting II, to be held from June 22-24, 2020 (Press release, Precigen, MAY 15, 2020, View Source [SID1234558178]). The e-poster presentation titled PRGN-3005 UltraCAR-T: Multigenic CAR-T Cells Generated Using Non-viral Gene Delivery and Rapid Manufacturing Process for the Treatment of Ovarian Cancer (Abstract 6593) is part of the Immunology/Adoptive Cell Therapy session and will be accessible on the AACR (Free AACR Whitepaper) e-poster website on June 22, 2020.

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PRGN-3005 utilizes Precigen’s transformative UltraCAR‑T therapeutic platform, which eliminates ex vivo expansion and reduces manufacturing time to allow for rapid next day administration of autologous UltraCAR-T cells following non-viral gene transfer at a medical center’s cGMP facility. PRGN-3005 UltraCAR-T cells simultaneously co-express a CAR specifically targeting MUC16, which is highly expressed on ovarian tumors with limited normal tissue expression; membrane bound IL-15 (mbIL15) for enhanced in vivo expansion and persistence; and a kill switch for improved therapeutic control. Ovarian cancer is the most lethal of gynecologic cancers with high unmet medical need.

"We are pleased to be able to share preclinical data for PRGN-3005 that led to the IND clearance and initiation of the Phase I study," said Helen Sabzevari, PhD, President and CEO of Precigen. "Our preclinical results demonstrate that PRGN-3005 UltraCAR-T administered one day after non-viral gene transfer has superior anti-tumor efficacy and persistence compared to traditional CAR-T cells and represents a promising opportunity for ovarian cancer treatment. We anticipate sharing the first clinical data for PRGN-3005 in the second half of 2020."

Based on these preclinical results, the FDA approved the IND application, and the first-in-human PRGN-3005 Phase I clinical trial for advanced ovarian cancer is currently under way (clinical trial identifier: NCT03907527). The PRGN‑3005 UltraCAR-T clinical study is an open-label, first-in-human Phase I dose escalation study to evaluate the safety and maximal tolerated dose of PRGN‐3005 UltraCAR-T delivered by intraperitoneal infusion (IP) or intravenous infusion (IV). The study population includes patients with advanced stage (III/IV) recurrent ovarian, fallopian tube, and primary peritoneal cancer who are platinum-resistant and have progressed after receiving standard-of-care therapies or are not eligible to receive available therapies with known clinical benefit.

About Ovarian Cancer
Worldwide, nearly 300,000 women are diagnosed with ovarian cancer every year1 with approximately 22,000 of them in the US2. Since early ovarian cancer is often without obvious symptoms, the disease is frequently diagnosed at an advanced stage where cancer has spread to distant parts of the body, such as the liver or lungs2,3. Five-year survival rates depend on stage and type of ovarian cancer with rates decreasing for advanced stage cancers that have spread to distant parts of the body3.

Precigen: Advancing Medicine with Precision
Precigen (Nasdaq: PGEN) is a dedicated discovery and clinical stage biopharmaceutical company advancing the next generation of gene and cell therapies using precision technology to target urgent and intractable diseases in our core therapeutic areas of immuno-oncology, autoimmune disorders, and infectious diseases. Our technologies enable us to find innovative solutions for affordable biotherapeutics in a controlled manner. Precigen operates as an innovation engine progressing a preclinical and clinical pipeline of well-differentiated unique therapies toward clinical proof-of-concept and commercialization. For more information about Precigen, visit www.precigen.com or follow us on Twitter @Precigen and LinkedIn.

Precigen’s UltraCAR-T Therapeutic Platform
Precigen’s UltraCAR-T platform has the potential to disrupt the CAR-T treatment landscape by increasing patient access through shortening manufacturing time, decreasing manufacturing-related costs, and improving outcomes using advanced approaches for precise tumor targeting and control of the immune system. The platform brings several key advancements: 1) Non-viral gene transfer using multigenic vectors for expression of multiple effector genes leads to better precision and control of tumor targeting and eliminates the need for virus; 2) Sustained persistence and desired phenotype of infused UltraCAR-T helps address T-cell exhaustion, a common issue with current CAR-T therapies; 3) T-cell control by incorporation of kill switch technology to potentially improve the safety profile; and 4) Rapid manufacturing of UltraCAR-T cells using our proprietary non-viral gene transfer process, which eliminates the need for ex vivo propagation, thus dramatically reducing wait times for patients from weeks to one day after gene transfer.

Trademarks
Precigen, UltraCAR-T, and Advancing Medicine with Precision are trademarks of Precigen and/or its affiliates. Other names may be trademarks of their respective owners.

On May 15, 2020 Autolus Therapeutics plc (Nasdaq: AUTL), a clinical-stage biopharmaceutical company developing next-generation programmed T cell therapies, reported presentations related to its AUTO1 and AUTO3 programs, the company’s CAR T cell therapies being investigated in Phase 1/2 studies of adult Acute Lymphocytic Leukemia (ALL) and relapsed/refractory Diffuse Large B Cell Lymphoma (DLBCL), respectively, at the European Hematology Association (EHA) (Free EHA Whitepaper) EHA (Free EHA Whitepaper)25 Virtual Congress on June 11 – 14, 2020 (Press release, Autolus, MAY 15, 2020, View Source [SID1234558177]). The AUTO1 data to be presented will expand on the positive data presented at the American Society of Hematology (ASH) (Free ASH Whitepaper) in December 2019 with additional patients as well as longer term data on previously treated patients.

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Oral Presentation Title: ALLCAR19: Updated data using AUTO1, a novel fast-off rate CD 19 CAR in relapsed/refractory B-Acute Lymphoblastic Leukaemia
Session Title: Cellular, antibody and targeted therapy
Abstract: S119
Date/Time: All oral abstract presentations will be made available on the on-demand Virtual Congress platform as of Friday, June 12 at 08:30 CEST and will be accessible until October 15, 2020.
Presenter: Dr. Claire Roddie MD, PhD, FRCPath, Consultant Haematologist and Honorary Senior Lecturer, Cancer Institute, University College London (UCL)

Oral Presentation Title: Phase 1 Alexander Study of AUTO3 the first Bicistronic Chimeric Antigen Receptor (CAR) targeting CD19 and CD22 with Pembrolizumab in patients with Relapsed/Refractory Diffuse Large B Cell Lymphoma
Session Title: Aggressive Lymphomas: Cellular and bispecific antibody therapies
Abstract: S240
Date/Time: All oral abstract presentations will be made available on the on-demand Virtual Congress platform as of Friday, June 12 at 08:30 CEST and will be accessible until October 15, 2020
Presenter: Dr Wendy Osborne, MBBS (Hons) MRCP FRCPath, Consultant Haematologist, Freeman Hospital, Newcastle upon Tyne Hospitals NHS Foundation Trust

Investor call on Monday June 15, 2020
Management will host a conference call and webcast at 8:30 am EDT/1:30 pm BST to discuss the EHA (Free EHA Whitepaper) data. To listen to the webcast and view the accompanying slide presentation, please go to: View Source

The call may also be accessed by dialing (866) 679-5407 for U.S. and Canada callers or (409) 217-8320 for international callers. Please reference conference ID 4838626. After the conference call, a replay will be available for one week. To access the replay, please dial (855) 859-2056 for U.S. and Canada callers or (404) 537-3406 for international callers. Please reference conference ID 4838626.