On November 5, 2020 CytomX Therapeutics, Inc. (Nasdaq: CTMX), a clinical-stage oncology-focused biopharmaceutical company pioneering a novel class of investigational antibody therapeutics based on its Probody technology platform, reported the treatment of the first patient in the Phase 2 expansion study of CX-2029, an anti-CD71 Probody drug conjugate (Press release, CytomX Therapeutics, NOV 5, 2020, View Source [SID1234570004]). The study, being conducted under a partnership with AbbVie, is evaluating CX-2029 as monotherapy in four cohorts; squamous non-small cell lung cancer (sqNSCLC), squamous head and neck cancer (sqHNSCC), esophageal cancer, and diffuse large B-cell lymphoma (DLBCL).

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"Our Phase 2 advancement of CX-2029 against the previously undruggable target CD71 marks a major milestone in our broadening clinical pipeline and highlights the progress we continue to make in applying our Probody platform to unique therapeutic opportunities in cancer," said Amy Peterson, MD, chief development officer of CytomX Therapeutics. "These expansion cohorts build on our Phase 1 clinical experience with CX-2029 in which we achieved meaningful therapeutic activity for this first-in-class drug candidate, setting the stage for Phase 2 exploration of its potential."

This open-label, multi-center Phase 2 cohort expansion study (NCT003543813) will enroll approximately 25 evaluable patients in each of the cohorts and assess the efficacy and tolerability of 3 mg/kg of CX-2029 administered every three weeks. The primary objective is overall response rate (ORR) with secondary objectives evaluating safety and tolerability. CytomX anticipates initial data from this study in late 2021.

About the CytomX and AbbVie Collaboration

In April 2016, AbbVie and CytomX entered into a Co-Development and Licensing Agreement under which the two companies are co-developing CX-2029, a Probody drug conjugate against CD71 conjugated to the cytotoxic payload MMAE. CD71, also known as the transferrin receptor 1 ("TfR1"), is a cell surface protein essential for iron uptake in dividing cells. CD71 is highly expressed in a number of solid and hematologic cancers and has attractive molecular properties for efficient delivery of cytotoxic payloads to tumor cells. CD71 has high potential as an anti-cancer target but is widely considered undruggable due to its presence on most dividing healthy cells. CX-2029 is designed to potentially create a therapeutic window for this novel target.

Under the agreement, CytomX is responsible for clinical development up to initial clinical proof of concept. AbbVie will lead late-stage clinical development and global commercial activities with CytomX eligible to receive up to $390 million in development, regulatory and commercial milestone payments, pending the achievement of pre-determined outcomes. In addition, CytomX is eligible to receive a profit share in the U.S. and tiered double-digit royalties on net product sales outside of the U.S. with CytomX retaining an option to co-promote in the U.S. In the first quarter of 2020, CytomX earned a $40 million milestone payment from AbbVie following the achievement of pre-specified criteria for the dose escalation phase of the ongoing Phase 1/2 clinical trial (NCT003543813).

On November 5, 2020 Magenta Therapeutics (Nasdaq: MGTA), a clinical-stage biotechnology company developing novel medicines to bring the curative power of immune reset to more patients, reported recent business highlights and financial results for the third quarter ended September 30, 2020 (Press release, Magenta Therapeutics, NOV 5, 2020, View Source [SID1234570003]).

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"Magenta showed steady progress throughout the third quarter, bringing aboard new scientific leadership in Lisa Olson to broaden our technical expertise across research and discovery. We continued to progress both clinically and preclinically, showcasing findings at the European Society for Blood and Marrow Transplantation annual meeting," said Jason Gardner, D.Phil., President and Chief Executive Officer, Magenta Therapeutics. "We are driven by what’s ahead, including presentations at the upcoming American Society of Hematology (ASH) (Free ASH Whitepaper) annual meeting, and into 2021, and the anticipation of clinical data in our conditioning and mobilization programs."

Recent Program Updates:

Mobilization –

By the end of 2020, Magenta plans to initiate multiple Phase 2 clinical trials of MGTA-145, the Company’s first-line stem cell mobilization agent. These trials, including both allogeneic and autologous transplant settings across multiple diseases, are intended to evaluate mobilization and collection of functional hematopoietic stem cells ("HSCs") and engraftment of these cells in patients after transplant. The MGTA-145 Phase 1 trial in healthy volunteers was completed earlier this year and met all primary and secondary endpoints.
Magenta presented data from its Phase 1 trial of MGTA-145 at the European Society for Blood and Marrow Transplantation ("EBMT") annual meeting, held August 29 to September 1, 2020. These data provide further confirmation that MGTA-145, in combination with plerixafor, enables the same-day mobilization and collection of high numbers of functional HSCs for transplant.
Magenta will present data from the MGTA-145 program at the upcoming American College of Rheumatology Convergence 2020, to be held November 5th through 9th, 2020 and the American Society of Hematology (ASH) (Free ASH Whitepaper) ("ASH") annual meeting, to be held December 5th through 8th, 2020.
Conditioning –

MGTA-117, Magenta’s clinical candidate for antibody drug conjugate ("ADC")-based conditioning for stem cell transplant and gene therapy and Magenta’s most advanced conditioning program, is on track with IND-enabling studies ongoing and progressing in GMP manufacturing. Magenta expects to generate initial clinical data in 2021.
Magenta has identified a lead antibody for its CD45-ADC program for blood and immune system reset and preclinical work continues to advance.
Magenta presented two sets of preclinical data on its CD45-ADC program at the EBMT annual meeting. The first abstract showed that a single dose of CD45-ADC removed disease-causing T-cells, was well tolerated and enabled successful immune reset to halt disease progression. In the second study, the data demonstrate that a single dose of its CD45-ADC is fully myeloablative and enables complete chimerism in a full mismatch allogeneic hematopoietic stem cell transplant, potently and safely enabling immune reset.
Magenta will also present data on its MGTA-117 and CD45-ADC conditioning programs at the ASH (Free ASH Whitepaper) annual meeting in December 2020.
Recent Business Highlights:

In September 2020, Magenta announced it was named as co-recipient along with the University of Southern California, University of Washington and Fred Hutchinson Cancer Research Center, Harvard University and Massachusetts General Hospital, and the Ragon Institute of a multi-project, interdisciplinary U19 grant from the National Institutes of Health (NIH) to explore the use of novel targeted conditioning agents with gene editing approaches to advance research in a cure for HIV.

Financial Results:

Cash Position: Cash, cash equivalents and marketable securities as of September 30, 2020, were $161.7 million, compared to $145.7 million as of December 31, 2019. Magenta anticipates that its cash, cash equivalents and marketable securities will be sufficient to fund operations and capital expenditures into the second half of 2022.

Research and Development Expenses: Research and development expenses were $11.8 million in the third quarter of 2020, compared to $16.5 million in the third quarter of 2019. The decrease was driven primarily by lower clinical trial costs for the MGTA-145 Phase 1 clinical trials which were completed in the first quarter of 2020, decreased preclinical costs for manufacturing related to the conditioning programs and lower manufacturing and clinical trial costs due to the discontinuance of enrollment in the Phase 2 trial of MGTA-456 in inherited metabolic diseases in June 2020.

General and Administrative Expenses: General and administrative expenses were $6.6 million for the third quarter of 2020, compared to $6.1 million for the third quarter of 2019. The increase was primarily due to an increase in personnel costs associated with the growth of the Company.

Net Loss: Net loss was $17.7 million for the third quarter of 2020, compared to net loss of $21.0 million for the third quarter of 2019.

On November 5, 2020 Fate Therapeutics, Inc. (NASDAQ: FATE), a clinical-stage biopharmaceutical company dedicated to the development of programmed cellular immunotherapies for cancer and immune disorders, reported that four oral and eight poster presentations for the Company’s induced pluripotent stem cell (iPSC) product platform were accepted for presentation at the 62nd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition being held virtually from December 5-8, 2020 (Press release, Fate Therapeutics, NOV 5, 2020, View Source [SID1234570002]).

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Accepted abstracts include a clinical case study of a patient treated with FT596 at the first dose level (30 million cells) as a monotherapy in the Company’s Phase 1 clinical trial for the treatment of relapsed / refractory B-cell lymphoma (NCT04245722). FT596 is the Company’s universal, off-the-shelf, chimeric antigen receptor (CAR) natural killer (NK) cell product candidate derived from a clonal master induced pluripotent stem cell (iPSC) line engineered with three anti-tumor functional modalities: a proprietary CAR optimized for NK cell biology that targets CD19 (CAR19); a novel high-affinity, non-cleavable CD16 (hnCD16) Fc receptor that enhances antibody-dependent cellular cytotoxicity (ADCC); and an IL-15 receptor fusion (IL-15RF) that augments NK cell activity. The accepted abstracts are available online through the ASH (Free ASH Whitepaper) conference website (www.hematology.org/Annual-Meeting/Abstracts/).

In addition, the Company plans to host a virtual investor event entitled "The Power of hnCD16" to highlight the unique therapeutic features and functionality of its novel hnCD16 Fc receptor, a core component incorporated in its iPSC-derived NK cell product candidates. The Company’s hnCD16 Fc receptor is designed to maximize ADCC, a potent anti-tumor mechanism by which NK cells recognize, bind and kill antibody-coated cancer cells, through enhanced binding to tumor-targeting antibodies and prevention of down-regulation commonly observed in cancer patients. Scientists from the Company have shown in a peer-reviewed publication (Blood. 2020;135(6):399-410) that hnCD16 iPSC-derived NK cells, compared to peripheral blood NK cells, elicit a more durable anti-tumor response and extend survival in combination with anti-CD20 monoclonal antibodies in an in vivo xenograft mouse model of human lymphoma.

Oral Presentations

CAR19 iPSC-Derived NK Cells Utilize the Innate Functional Potential Mediated through NKG2A-Driven Education and Override the HLA-E Check Point to Effectively Target B Cell Lymphoma
93; Session Name: 203. Lymphocytes, Lymphocyte Activation, and Immunodeficiency, including HIV and Other Infections: Pathogenesis and Immunotherapy; December 5, 2020
Development of a Novel MICA/B-Specific CAR as a Pan-Tumor Targeting Strategy for Off-the-Shelf, Cell-Based Cancer Immunotherapy
613; Session Name: 703. Adoptive Immunotherapy: Mechanisms and New Approaches: Adoptive Cell Therapy beyond CAR T cells; December 7, 2020
Generation of Multiplexed Engineered, Off-the-Shelf CAR T Cells Uniformly Carrying Multiple Anti-Tumor Modalities to Prevent Tumor Relapse
566; Session Name: 802. Chemical Biology and Experimental Therapeutics: Innovations in Therapy and Drug Screening; December 7, 2020
Engineered iPSC-Derived NK Cells Expressing Recombinant CD64 for Enhanced ADCC
67; Session Name: 704. Immunotherapies: Beyond T to NK; December 5, 2020
Poster Presentations

Initial Clinical Activity of FT596, a First-in-Class, Multi-Antigen Targeted, Off-the-Shelf, iPSCDerived CD19 CAR NK Cell Therapy in Relapsed / Refractory B-Cell Lymphoma
2356; Session Name: 704. Immunotherapies: Poster II; December 6, 2020
A Phase I Study of FT819, a First-of-Kind, Off-the-Shelf, iPSCDerived TCRLess CD19 CAR T Cell Therapy for the Treatment of Relapsed / Refractory B-Cell Malignancies
3267; Session Name: 704. Immunotherapies: Poster III; December 7, 2020
A Phase I Study of FT538, a First-of-Kind, Off-the-Shelf, Multiplexed Engineered, iPSC-Derived NK Cell Therapy As Monotherapy in Relapsed / Refractory Acute Myelogenous Leukemia and in Combination with Daratumumab or Elotuzumab in Relapsed / Refractory Multiple Myeloma
1449; Session Name: 704. Immunotherapies: Poster I; December 5, 2020
Triple Gene-Modified iPSC-Derived NK Cells Combined with Daratumumab for Targeted Immunotherapy Against AML
1947; Session Name: 615. Acute Myeloid Leukemia: Commercially Available Therapy, excluding Transplantation: Poster II; December 6, 2020
cGMP Mass Production of FT538, a First-of-Kind, Off-the-Shelf, Multiplexed Engineered Natural Killer Cell Cancer Immunotherapy Derived from a Clonal Master Induced Pluripotent Stem Cell Line
3279; Session Name: 711. Cell Collection and Processing: Poster III; December 7, 2020
FT576: Multi-Specific Off-the-Shelf CAR-NK Cell Therapy Engineered for Enhanced Persistence, Avoidance of Self-Fratricide and Optimized Mab Combination Therapy to Prevent Antigenic Escape and Elicit a Deep and Durable Response in Multiple Myeloma
1402; Session Name: 653. Myeloma/Amyloidosis: Therapy, excluding Transplantation: Poster I; December 5, 2020
Multiplexed Engineered, Off-the-Shelf T Cells Carrying Three Tumor-Associated Antigen-Targeting Modalities: CAR + Pan-Tumor Targeting TCR + CD16 Fc Receptor
1434; Session Name: 703. Adoptive Immunotherapy: Poster I; December 5, 2020
Novel Method for Clonal Selection of Multiplexed Engineered CAR-T Cells Which Uniquely Demonstrate Anti-Tumor Functionality in the Tumor Microenvironment
3263; Session Name: 703. Adoptive Immunotherapy: Mechanisms and New Approaches: Poster III; December 7, 2020
About Fate Therapeutics’ iPSC Product Platform
The Company’s proprietary induced pluripotent stem cell (iPSC) product platform enables mass production of off-the-shelf, engineered, homogeneous cell products that can be administered with multiple doses to deliver more effective pharmacologic activity, including in combination with other cancer treatments. Human iPSCs possess the unique dual properties of unlimited self-renewal and differentiation potential into all cell types of the body. The Company’s first-of-kind approach involves engineering human iPSCs in a one-time genetic modification event and selecting a single engineered iPSC for maintenance as a clonal master iPSC line. Analogous to master cell lines used to manufacture biopharmaceutical drug products such as monoclonal antibodies, clonal master iPSC lines are a renewable source for manufacturing cell therapy products which are well-defined and uniform in composition, can be mass produced at significant scale in a cost-effective manner, and can be delivered off-the-shelf for patient treatment. As a result, the Company’s platform is uniquely capable of overcoming numerous limitations associated with the production of cell therapies using patient- or donor-sourced cells, which is logistically complex and expensive and is subject to batch-to-batch and cell-to-cell variability that can affect clinical safety and efficacy. Fate Therapeutics’ iPSC product platform is supported by an intellectual property portfolio of over 300 issued patents and 150 pending patent applications.

On November 5, 2020 Seagen Inc. (Nasdaq:SGEN) reported multiple ADCETRIS (brentuximab vedotin) data presentations at the upcoming 62nd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition, taking place virtually December 5-8, 2020 (Press release, Seagen, NOV 5, 2020, View Source [SID1234570001]). Data presentations will include five-year updates from the phase 3 ECHELON-1 and ECHELON-2 clinical trials evaluating ADCETRIS plus a chemotherapy combination regimen in frontline advanced stage Hodgkin lymphoma (HL) or frontline peripheral T-cell lymphoma (PTCL). ADCETRIS is an antibody-drug conjugate (ADC) directed to CD30, a defining marker of classical HL and expressed on the surface of several types of PTCL. ADCETRIS is being evaluated globally in more than 70 corporate- and investigator-sponsored clinical trials across multiple settings in lymphoma and other indications.

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"At this year’s ASH (Free ASH Whitepaper) meeting, ADCETRIS will be featured in 18 data presentations, including five-year analyses from the ECHELON-1 and ECHELON-2 phase 3 frontline trials," said Roger Dansey, M.D., Chief Medical Officer at Seagen. "Reaching five-years without disease recurrence represents a significant clinical milestone for patients, as they may be considered disease-free at this time point. The five-year data presentations from our phase 3 ECHELON-1 and ECHELON-2 trials demonstrate ADCETRIS plus chemotherapy resulted in superior clinical activity and durable benefit when compared to outcomes with a standard chemotherapy regimen. We will also present additional analyses from trials evaluating ADCETRIS in combination with other therapies, with the goal of identifying potential new treatment strategies that improve outcomes for patients."

Details of Seagen Presentations at ASH (Free ASH Whitepaper):

Abstract Title

Abstract #

Presentation

Type / Date

Presenter

Company-Sponsored Trials

Frontline Brentuximab Vedotin as Monotherapy or in Combination for Older Hodgkin Lymphoma Patients

#471

Oral presentation / Sunday, Dec. 6 at 2:15 p.m. PT

C. Yasenchak

Brentuximab Vedotin with Chemotherapy for Patients with Previously Untreated, Stage III/IV Classical Hodgkin lymphoma: 5-year Update of the ECHELON-1 Study

#2973

Poster presentation / Monday, Dec. 7, 7:00 a.m. – 3:30 p.m. PT

D. Straus

The ECHELON-2 Trial: 5-Year Results of a Randomized, Double-Blind, Phase 3 Study of Brentuximab Vedotin and CHP (A+CHP) Versus CHOP in Frontline Treatment of Patients with CD30-positive Peripheral T-cell Lymphoma

#1150

Poster presentation / Saturday, Dec. 5, 7:00 a.m. – 3:30 p.m. PT

S. Horwitz

Economic Assessment of Diagnostic Revision in Peripheral T-cell Lymphoma

#1606

Poster presentation / Saturday, Dec. 5, 7:00 a.m. – 3:30 p.m. PT

N. Liu

Nivolumab Combined with Brentuximab Vedotin for Relapsed/Refractory Mediastinal Gray Zone Lymphoma: Primary Efficacy and Safety Analysis of the Phase 2 CheckMate 436 Study

#2045

Poster presentation / Sunday, Dec. 6, 7:00 a.m. – 3:30 p.m. PT

A. Santoro

Real-World Characteristics of Patients with Classical Hodgkin Lymphoma Receiving Frontline Brentuximab Vedotin with Chemotherapy: A Retrospective Analysis with Propensity Score Matching

#2499

Poster presentation / Sunday, Dec. 6, 7:00 a.m. – 3:30 p.m. PT

T. Phillips

Real-World Adherence to National Comprehensive Cancer Network (NCCN) Guidelines Regarding the Usage of PET/CT and Reported Deauville Scores in Advanced Stage Classical Hodgkin Lymphoma: A Community Oncology Practice Perspective

#2033

Poster presentation / Sunday, Dec. 6, 7:00 a.m. – 3:30 p.m. PT

C. Yasenchak

SGN-CD30C, an Investigational CD30-Directed Camptothecin Antibody-Drug Conjugate (ADC), Shows Strong Anti Tumor Activity and Superior Tolerability in Preclinical Studies

#2089

Poster presentation / Sunday, Dec. 6, 7:00 a.m. – 3:30 p.m. PT

M. Ryan

Real-World Characteristics of Patients with Peripheral T-Cell Lymphoma Receiving Frontline Brentuximab Vedotin with Chemotherapy: A Retrospective Analysis with Propensity Score Matching

#3418

Poster presentation / Monday, Dec. 7, 7:00 a.m. – 3:30 p.m. PT

J. Burke

Nodal Peripheral T-Cell Lymphoma with T Follicular-Helper Phenotype: A Different Entity? Results of the Spanish Retrospective Real-T Study

#2972

Poster presentation / Monday, Dec. 7, 7:00 a.m. – 3:30 p.m. PT

AM. Garcia-Sancho

Results from the International, Multi-Center, Retrospective B-Holistic Study: Describing Treatment Pathways and Outcomes for Classical Hodgkin Lymphoma

#2979

Poster presentation / Monday, Dec. 7, 7:00 a.m. – 3:30 p.m. PT

B. Ferhanoglu

Investigator-Sponsored Trials

Increased Tumor Specific Cytotoxic T Cell Responses and Reversion to a Favorable Cytokine Profile after Treatment in Patients with Newly Diagnosed High Risk Hodgkin Lymphoma Treated on Children’s Oncology Group Trial- AHOD1331

#595

Oral presentation / Monday, Dec. 7 at 9:15 a.m. PT

H. Dave

Addition of Brentuximab Vedotin to Gemcitabine in Relapsed or Refractory T-Cell Lymphoma: Results of a LYSA Multicenter, Phase II Study. "The TOTAL Trial"

#1161

Poster presentation / Saturday, Dec. 5, 7:00 a.m. – 3:30 p.m. PT

O. Tournilhac

A Pilot Study of Brentuximab Vedotin, Rituximab and Dose Attenuated CHP in Patients 75 Years and Older with Diffuse Large B-Cell Lymphoma

#2102

Poster presentation / Sunday, Dec. 6, 7:00 a.m. – 3:30 p.m. PT

P. Reagan

Dose-Dense Brentuximab Vedotin Plus Ifosfamide, Carboplatin, and Etoposide (ICE) Is Highly Active for Second Line Treatment in Relapsed/Refractory Classical Hodgkin Lymphoma: Final Results of a Phase I/II Study

#2964

Poster presentation / Monday, Dec. 7, 7:00 a.m. – 3:30 p.m. PT

R. Lynch

A Phase I Trial Assessing the Feasibility of Romidepsin Combined with Brentuximab Vedotin for Patients Requiring Systemic Therapy for Cutaneous T-Cell Lymphoma

#2970

Poster presentation / Monday, Dec. 7, 7:00 a.m. – 3:30 p.m. PT

S. Barta

Trials-in-Progress

Trial-in-Progress: Brentuximab Vedotin in Combination with Lenalidomide and Rituximab in Subjects with Relapsed or Refractory Diffuse Large B-Cell Lymphoma (DLBCL)

#2112

Poster presentation / Sunday, Dec. 6, 7:00 a.m. – 3:30 p.m. PT

N. Bartlett

A Phase 1 Study of Sea-CD70 in Myeloid Malignancies

#2874

Poster presentation / Monday, Dec. 7, 7:00 a.m. – 3:30 p.m. PT

A. Aribi

Trial-in-Progress: Frontline Brentuximab Vedotin and CHP (A+CHP) in Patients with Peripheral T-Cell Lymphoma with Less Than 10% CD30 Expression

#2976

Poster presentation / Monday, Dec. 7, 7:00 a.m. – 3:30 p.m. PT

D. Jagadeesh

On November 5, 2020 Replimune Group, Inc. (Nasdaq: REPL), a biotechnology company developing oncolytic immuno-gene therapies derived from its Immulytic platform, reported financial results for the fiscal second quarter ended September 30, 2020 and provided a business update (Press release, Replimune, NOV 5, 2020, View Source [SID1234570000]).

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"In October, we released clinical data with our second product candidate, RP2, demonstrating durable single agent responses in heavily pre-treated patients with immune insensitive tumor types that provides validation of our platform," said Philip Astley-Sparke, CEO of Replimune. "We also presented updated data for RP1 in combination with Opdivo in melanoma and non-melanoma skin cancers that continues to improve upon our recent June update in terms of depth and durability of response, and supports our two registration-directed clinical programs in both cutaneous squamous cell carcinoma and anti-PD1 failed melanoma. Following a successful follow-on offering in October, we are well-financed to advance and expand our pipeline of product candidates and to fund the initial build of our commercial infrastructure with many exciting milestones ahead. In particular, we look forward to initiating clinical development with RP3 as we seek to establish our products more broadly beyond immune-responsive tumor types, as a cornerstone of immune-based cancer treatments."

Recent Events and Corporate Updates

Presented updated clinical data with RP1 combined with Opdivo in melanoma and non-melanoma skin cancer (NMSC). The updated data from the Phase 1 expansion of RP1 in combination with Opdivo and the Phase 2 cohorts in melanoma and NMSC showed eight of eleven patients with CSCC have achieved response (5 CR) with one patient currently assessed as stable disease at their first scan who remains on treatment. Five of 16 patients (87.5% with advanced visceral disease) with anti-PD-1 failed melanoma have achieved response, four of whom had also previously failed ipilimumab. A further patient is a surgical CR remaining tumor free currently more than 5 months from surgery and a further stable disease patient remains on treatment. Further responses have been observed in angiosarcoma and in anti-PD1 failed mucosal melanoma. All responses have been durable with only one patient with CSCC and one patient with any type of melanoma having progressed after response. Treatment has continued to be well tolerated and remains ongoing in many of the patients. RP1 is an enhanced potency oncolytic immunotherapy that expresses a GALV-GP R- fusogenic protein and GM-CSF.

The Company has been selected to participate in the SITC (Free SITC Whitepaper) 2020 Virtual Press Conference being held on Monday, November 9, 2020 at 7:45 AM ET where the principal investigator will discuss the Company’s poster titled "An Open-label, multicenter, Phase 1/2 clinical trial of RP1, an enhanced potency oncolytic HSV, combined with nivolumab: Updated results from the skin cancer cohorts".
Presented single agent data from Phase 1 trial evaluating RP2 in heavily pre-treated patients. The data presented demonstrated compelling monotherapy clinical activity in patients with immune insensitive tumor types. Nine patients were treated with single agent RP2. One patient (with mucoepidermoid carcinoma) has an ongoing complete response and two other patients (with uveal melanoma and esophageal cancer) have ongoing partial responses. All three of these responses are durable; ongoing at between eight and 11 months from the first dose. RP2 was observed to be well-tolerated with side effects consistent with RP1. Following from this single agent data, enrollment of patients being treated with RP2 combined with Opdivo is currently underway. RP2 is an enhanced potency oncolytic immunotherapy which expresses an anti-CTLA-4 molecule, intended to improve on the safety and efficacy profile of systemic antibody approaches to targeting CTLA-4.

Clinical trials authorization (CTA) accepted by the MHRA for RP3. The CTA for RP3, Replimune’s third product candidate, which in addition to the GALV-GP R- fusogenic protein and anti-CTLA-4 also expresses CD40L and 4-1BBL, has been accepted by the Medicines and Healthcare Products Regulatory Agency (MHRA) in the United Kingdom allowing initiation of the Phase 1 clinical trial with RP3 alone and combined with anti-PD1 therapy, which is expected to initiate by the end of 2020. RP3, in addition to maximizing so-called Signal 1 (antigen presentation), is intended to also maximize Signal 2 (immune co-stimulation) and Signal 3 (the production of inflammatory cytokines, stimulated by CD40L and 4-1BBL).
Extended cash runway into the second half of 2024. In October 2020, the Company raised gross proceeds of approximately $287 million through a public offering of common stock and pre-funded warrants. The Company believes that the existing cash, cash equivalents and short-term investments at September 30, 2020, together with the proceeds raised in October, will enable the Company to conduct additional clinical trials including potentially registration-directed clinical development of RP2 and/or RP3, build initial commercial infrastructure, and to fund overall operations into the second half of 2024.
Announced new appointment to the Board of Directors. In October, the Company announced the appointment of Tanya Lewis to the Board of Directors. The appointment adds deep strategic expertise in developing and executing regulatory strategies.
Completed manufacturing facility to support late-stage development and commercialization. The 63,000-square-foot state-of-the-art facility in Framingham, MA provides multi-product manufacturing capabilities for the Company’s product candidates with sufficient capacity to support full commercialization. The facility is now fully operational and technology transfer activities for RP1 complete. GMP batch production is underway with product expected to be released for use in 2021.
COVID-19 potential impact on milestones: Enrollment into our clinical trials, such as the Company’s clinical trial of RP1 in solid organ transplant patients with CSCC, representing a highly immunocompromised patient population, has been slower than expected, which the Company attributes to the global pandemic. As the clinical sites continue to evaluate their capacity to treat patients, the Company could see additional impact on the pace of enrollment in the final quarter of 2020 and the first half of 2021 across its clinical trial programs.

Program Highlights

RP1 in combination with Libtayo in cutaneous squamous cell carcinoma (CSCC): The Company is actively enrolling patients into its 240-patient, registration-directed Phase 2, randomized, controlled, global clinical trial.

RP1 in combination with Opdivo in melanoma, non-melanoma skin cancers, and MSI-H/dMMR tumors: Enrollment and accrual of the initial melanoma cohort (including anti-PD1 naïve and failed patients) was completed in the first half of 2020. The Company continues to enroll into a cohort of patients with non-melanoma skin cancers which has been expanded from 30 patients to 45 to include anti-PD1 failed patients. The Company is accumulating data from the MSI-H/dMMR (anti-PD1 naïve) cohort to inform a decision as to whether to pursue MSI-H/dMMR tumors into registration-directed development in 2021.

RP1 in combination with Opdivo in anti-PD-1 failed melanoma: The Company initiated recruitment into a new registration-directed 125-patient cohort in the Phase 2 clinical trial of RP1 in combination with Opdivo in the first half of 2020 and continues to enroll patients.

RP1 in anti-PD1 failed patients with non-small cell lung cancer (NSCLC): The Company plans to initiate the 30 patient cohort of anti-PD1 refractory patients with NSCLC to the RP1 combined with Opdivo clinical trial later this year with the first patient expected to be dosed by the end of 2020 or in early 2021.

RP1 as monotherapy in solid organ transplant recipients with CSCC: A 30 patient Phase 1b clinical trial assessing the safety and efficacy of RP1 in liver and kidney transplant recipients with recurrent CSCC is open for enrollment.

RP2 alone and in combination with Opdivo: RP2 is being evaluated in a Phase 1 clinical trial evaluating the safety and efficacy of RP2 alone and combined with Opdivo. Following the monotherapy phase, enrollment is currently underway in a 30-patient expansion cohort in combination with Opdivo.

RP3 alone and in combination with anti-PD-1 therapy: The Phase 1 clinical trial remains on track to be initiated in 2020.

Financial Highlights

Cash Position: As of September 30, 2020, cash, cash equivalents and short-term investments were $244.6 million, as compared to $168.6 million as of March 31, 2020. This increase was primarily related to $109.5 million in net proceeds from financing activities offset by cash utilized in operating activities largely associated with advancing our expanded clinical development plan.

In October the company closed on an offering of common stock and pre-funded warrants raising approximately $287 million in gross proceeds and received aggregate net proceeds of approximately $270 million after deducting underwriting discounts, commissions, and other offering expenses. This includes the exercise in full by the underwriters of their option to purchase additional shares of common stock.
R&D Expenses: Research and development expenses were $14.0 million for the second quarter ended September 30, 2020, as compared to $8.2 million for the second quarter ended September 30, 2019. This increase was primarily due to increased clinical and manufacturing expenses driven by the Company’s lead programs and increased personnel expenses. Research and development expenses included $1.3 million in stock-based compensation expenses for the second quarter ended September 30, 2020.

G&A Expenses: General and administrative expenses were $5.6 million for the second quarter ended September 30, 2020, as compared to $4.1 million for the second quarter ended September 30, 2019. The increase was primarily driven by personnel-related costs, professional fees, and facility expansion. General and administrative expenses included $1.5 million in stock-based compensation expenses for the second quarter ended September 30, 2020.

Net Loss: Net loss was $20.1 million for the second quarter ended September 30, 2020, as compared to a net loss of $11.1 million for the second quarter ended September 30, 2019.

About RP1

RP1 is Replimune’s lead Immulytic product candidate and is based on a proprietary new strain of herpes simplex virus engineered to maximize tumor killing potency, the immunogenicity of tumor cell death and the activation of a systemic anti-tumor immune response through the expression of a GALV-GP R- fusogenic protein and GM-CSF.

About RP2 & RP3

RP2 and RP3 are derivatives of RP1 that express additional proteins. RP2 expresses an anti-CTLA-4 antibody-like molecule and RP3 additionally expresses the immune co-stimulatory pathway activating proteins CD40L and 4-1BBL. RP2 and RP3 are intended to provide targeted and potent delivery to the sites of immune response initiation in the tumor and draining lymph nodes, with the goal of focusing systemic immune-based efficacy on tumors and limiting off-target toxicity.