On may 6, 2019 Adaptimmune Therapeutics plc (Nasdaq:ADAP), a leader in T-cell therapy to treat cancer, reported financial results for the first quarter ended March 31, 2019 (Press release, Adaptimmune, MAY 6, 2019, View Source [SID1234535735]). Adaptimmune shared a clinical update in a separate release (https://bit.ly/2IJpltR).

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Financial Results for the three-month period ended March 31, 2019

·Cash / liquidity position: As of March 31, 2019, Adaptimmune had cash and cash equivalents of $49.9 million and Total Liquidity(1) of $168.2 million.

·Revenue: Revenue for the three-month period ended March 31, 2019 was nil, compared to $8.2 million for the same period in 2018. No revenue has been recognised for the three months ended March 31, 2019 as the NY-ESO SPEAR T-cell transition program and the PRAME pre-clinical development program were completed in 2018, and work has not commenced on the third target nominated by GSK under the Collaboration and License Agreement.

·Research and development ("R&D") expenses: R&D expenses for the three-month period ended March 31, 2019 were $22.0 million, compared to $25.7 million for the same period of 2018; this decrease being primarily due to a reduction in expenditure associated with NY-ESO, which was transferred to GSK on July 23, 2018.

· General and administrative ("G&A") expenses: G&A expenses for the three-month period ended March 31, 2019 were $11.8 million, compared to $11.2 million for the same period of 2018.

· Other income, net: Other income for the three-month period ended March 31, 2019 was $5.4 million, compared to $7.1 million for the same period of 2018. Other income primarily comprises unrealized foreign exchange gains, which fluctuate depending on exchange rate movements and the amount of foreign currency assets and liabilities.

· Net loss: Net loss attributable to holders of the Company’s ordinary shares for the three-month period ended March 31, 2019 was a loss of $27.4 million, ($(0.04) per ordinary share) compared to a loss of $21.1 million ($(0.04) per ordinary share) in the same period of 2018.

Financial guidance

The Company believes that its existing cash, cash equivalents and marketable securities will fund the Company’s current operations into the third quarter of 2020.

(1) Total liquidity is a non-GAAP financial measure, which is explained and reconciled to the most directly comparable financial measures prepared in accordance with GAAP below.

On May 6, 2019 Unum Therapeutics Inc. (Nasdaq: UMRX), a clinical-stage biopharmaceutical company focused on the development of cellular immunotherapies to treat cancer using its novel T cell technology platforms, reported that the company will host a conference call and live audio webcast on Monday, May 13, 2019 at 8:00 a.m. ET to discuss financial results for the first quarter of 2019 (Press release, Unum Therapeutics, MAY 6, 2019, View Sourcenews-releases/news-release-details/unum-therapeutics-host-first-quarter-2019-financial-results" target="_blank" title="View Sourcenews-releases/news-release-details/unum-therapeutics-host-first-quarter-2019-financial-results" rel="nofollow">View Source [SID1234535734]). Unum management will also provide an update on the Company’s recent progress and upcoming milestones.

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Participants may access the conference call by dialing 866-300-3411 (domestic) or 636-812-6658 (international) and refer to conference ID number 1443149. To join the live webcast, please visit the investor relations section of the Unum Therapeutics website at View Source at least 10 minutes before the event begins.

A webcast replay will be available at the same location on the Unum Therapeutics website beginning approximately two hours after the event and will be archived for 90 days.

On May 6, 2019 Adaptimmune Therapeutics plc (Nasdaq:ADAP), a leader in T-cell therapy to treat cancer, reported significant clinical progress with partial responses in 4 out of 5 synovial sarcoma patients treated with ~10 billion cells in the ADP-A2M4 pilot study, and tumor shrinkage seen in nearly all assessed synovial sarcoma patients (Press release, Adaptimmune, MAY 6, 2019, View Source;p=RssLanding&cat=news&id=2397170 [SID1234535732]). Based on these data, the Company will initiate the SPEARHEAD-1 trial in patients with synovial sarcoma and myxoid/round cell liposarcoma (MRCLS) later this year.

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Beyond sarcoma, there is evidence of antitumor activity with ADP-A2M4 and ADP-A2M10 in other solid tumors. Based on these data and translational findings, the Company is expanding its clinical trial program by initiating a radiation sub-study, as well as opening a next-generation ADP-A2M4CD8 study (SURPASS trial), for which the IND has been filed. Finally, the first patient with HCC was treated in Cohort 2 (1 billion SPEAR T-cells) of the ADP‑A2AFP study and showed good tolerability to treatment and a transient decrease in serum AFP as well as tumor shrinkage at first scan.

"Today is a watershed moment for the Company. We now have confirmed responses in an unmet medical indication, synovial sarcoma, with our wholly owned ADP-A2M4 SPEAR T-cells. As we prepare to start the SPEARHEAD-1 study, we are one step closer to realizing our ambition to be the first T-cell company with an approved therapy in solid tumors in 2022," said James Noble, Adaptimmune’s Chief Executive Officer. "There is also early evidence of activity in other solid tumors with all three products and I am delighted to announce that we have filed an IND for a more potent, next-generation program with MAGE-A4 as the target. Working with world-class clinical trial centers and having a robust manufacturing and supply capability, we look forward to making further progress across the entire portfolio in the coming months."

ADP-A2M4 responses and data in synovial sarcoma patients

10 patients treated
8 patients assessed, with 6 showing tumor shrinkage
3 confirmed partial responses, 1 unconfirmed partial response
3 stable diseases (SD) and 1 progressive disease (PD)
ADP-A2M4 SPEAR T-cells appear to show a favorable benefit:risk profile in patients with synovial sarcoma
Good tolerability overall. Most adverse events are consistent with those typically experienced by cancer patients undergoing cytotoxic chemotherapy or other cancer immunotherapies
SPEARHEAD‑1 protocol summary
Single-arm, Phase 2 study in more than 20 centers (North America & Europe) to include 60 patients with:
Advanced (metastatic or inoperable) synovial sarcoma or MRCLS patients who have received prior chemotherapy
HLA-A*02 & MAGE-A4 antigen positive
MAGE-A4 expression 30% (2+, 3+)
Primary endpoint will be overall response rate by RECIST v1.1 by independent review
Interim futility: 3 or more responses in the first 15 patients for study continuation
Safety endpoints with Independent Data Safety Monitoring Board
Exploratory endpoints with translational data and patient-reported outcomes
Treatment
Lymphodepletion: Flu: (30 mg/m2/ day) x 4 days; Cy (1800 mg/ m2/ day) x 2 days
Dose: up to 10 billion transduced SPEAR T-cells
Antitumor activity in other indications with ADP-A2M4 and ADP-A2M10

Tumor shrinkage seen in lung patients (ADP-A2M10), and melanoma and ovarian patients (ADP-A2M4)
7 patients treated with ADP-A2M4 in indications other than sarcoma in Cohorts 3 and Expansion phase
3 SDs, 3 PDs, and 1 patient expired due to disease progression before the first scan
7 patients treated with ADP-A2M10 in Cohort 3 and Expansion phase in the NSCLC and triple tumor studies
4 SDs, with one patient receiving a second infusion at Week 16, and 3 PDs to date
Adaptimmune continues to examine patient data to gain a clearer understanding of the best path forward to enhance RECIST responses
Adaptimmune is planning to start two new studies to transform currently observed activity in epithelial tumors into durable responses
Radiation sub-study at MD Anderson Cancer Center (MDACC) to initiate 2H 2019
Sub-study of the ADP-A2M4 clinical trial with up to 10 patients to be treated
Primary endpoint is safety and secondary endpoint is RECIST v1.1 responses
Radiation is 7Gy (low dose) per lesion or isocenter to be administered before lymphodepletion
SURPASS trial (ADP-A2M4CD8) – the first next-generation approach in the clinic in multiple solid tumors to initiate later this year
IND filed April 2019
Preclinical proof-of-concept data for this next-generation SPEAR T-cell therapy were presented at AACR (Free AACR Whitepaper) 2019 (https://bit.ly/2FGlRHN)
These data indicate that addition of the CD8α homodimer to the ADP-A2M4 cells effectively enables CD4+ "helper" T-cells to adopt a CD8+ "killer" like phenotype in vitro
This is intended to speed up initial antitumor activity and broaden the antitumor response in patients
Protocol summary:
Up to 30 subjects (HLA-A*02 with MAGE-A4+)
Primary endpoint: safety and tolerability
Secondary endpoint: antitumor activity
Lymphodepletion: Flu (30 mg/m2/day) × 4 days; Cy (1800 mg/m2/day) × 2 days
Shorter stagger between patients – anticipate faster dose escalation
Starting doses of ~1 billion cells (Cohort 1) and escalation through:
Cohort 2 (1.2 to 3.0 billion cells)
Cohort 3 (3.0 to 6.0 billion cells)
Expansion phase with up to 10 billion cells
Data expected in 2020

ADP-A2M10 studies are continuing and research plans will be reassessed as more data is accumulated across the two studies
ADP-A2AFP

Data from first HCC patient treated in Cohort 2
Continuing favorable safety data as was seen in Cohort 1 presented at AACR (Free AACR Whitepaper) 2019 (https://bit.ly/2FR8Lse)
Shows transient serum AFP decrease and tumor shrinkage at first scan
Data update will be provided in 1H 2020
Off-the-shelf program (allogeneic platform)

Substantial progress with update provided in an oral presentation at the American Society of Gene & Cell Therapy Annual Meeting 2019 by Dr. Jo Brewer, VP Allogeneic Research (https://bit.ly/2UWmqma)
Conference Call and Webcast Link for Clinical and Business Update Slide Presentation today (May 6th)
The Company will host a live teleconference and slide presentation at 8:00 a.m. EDT (1:00 p.m. BST) today (Monday May 6, 2019). The live webcast of the conference call and slides will be available at View Source An archive will be available after the call at the same address. To participate in the live webinar, if preferred, please dial (833) 652-5917 (U.S. and Canada) or
+1 (430) 775-1624 (International).

On May 5, 2019 Harbour BioMed and Chia Tai Tianqing Pharmaceutical Group Ltd. (CTTQ) reported they are entering into a strategic alliance to discover, develop and commercialize next-generation biologics for multiple therapeutic targets in oncology and immunology (Press release, Harbour BioMed, MAY 5, 2019, View Source [SID1234535722]).

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The collaboration will combine Harbour BioMed’s discovery capabilities, including its transgenic mouse technologies for generating fully human antibodies, with CTTQ’s preclinical development resources and expertise. The companies will be responsible for clinical development and commercialization of therapeutic candidates emerging from the collaboration in their respective regions. Harbour BioMed will be responsible for the United States, Japan, and the rest of the world outside, while CTTQ will have responsibility for Greater China and Europe. The companies will pay royalties to each other based on sales in their respective territories. Additional financial terms were not disclosed.

"The agreement with CTTQ, is part of our strategy to build a robust therapeutic pipeline for global markets through co-discovery, development and commercialization partnerships that capitalize on our patented platforms for generating fully human antibodies," said Dr. Jingsong Wang, Founder, Chairman and CEO of Harbour BioMed. "CTTQ, a well-known pharmaceutical company that is making strategic investments in therapeutic innovation in oncology and immunology, is a strong partner in that effort and directly complements our internal discovery programs," added Dr. Wang. It is expected that both sides will jointly own the IP and development and commercialization rights for the new drugs coming out of this collaboration.

"CTTQ will be committed to bring valuable treatment agents for diseases with unmet need. The collaboration with Harbour BioMed is well aligned with CTTQ’s strategy for innovative biological medicine development, it also marks significant progress to strengthen our capability of R&D of innovative therapeutic antibodies," said Shanchun Wang, President of CTTQ. CTTQ currently has several monoclonal antibody drugs, including a PD-L1 inhibitor, which have progressed into Phase 2 and Phase 3 clinical development.

On May 5, 2019 UroGen Pharma Ltd. (Nasdaq: URGN), a clinical-stage biopharmaceutical company developing treatments to address unmet needs in the field of urology, reported findings from a secondary analysis from the pivotal Phase 3 OLYMPUS trial which showed that UGN-101 (mitomycin gel) for instillation, an investigational mitomycin formulation, demonstrated a 59 percent complete response rate in a subset of patients with endoscopically unresectable low-grade upper tract urothelial cancer (UTUC) (Press release, UroGen Pharma, MAY 5, 2019, View Source [SID1234535721]). Findings were presented by Seth Paul Lerner, M.D., FACS, Professor of Urology at Baylor College of Medicine, in an oral presentation during the plenary session at the 114th American Urological Association (AUA) Annual Meeting in Chicago.

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The analysis showed that in the OLYMPUS intent-to-treat population, 71 patients had undergone PDE at the time of the analysis and 42 of the 71 patients (59 percent) achieved a CR. Forty-one patients entered follow-up. Of the evaluated complete responses to date, 27 patients have undergone a six-month evaluation, and 24 out of 27 patients (89 percent) have remained disease free at six months. Overall, 5 of 41 patients who achieved a CR have relapsed at any time during the study.

Of these 71 patients, 34 were initially characterized by the treating physician as having endoscopically unresectable tumor at baseline, and 20 of 34 of these patients (59 percent) achieved a CR at the PDE.

*Forty-one patients entered follow-up. At the time of the analysis, 66 percent (27/41) of patients have completed a six-month evaluation.

The most common adverse events observed were urinary tract infection, ureteral narrowing and stricture formation. The majority of ureteral events were reported as mild to moderate and have resolved.

"The results from the OLYMPUS trial continue to be compelling for new and recurrent LG UTUC, as well as for those who have unresectable tumors and would be immediate candidates for kidney removal. For this typically elderly patient population, kidney preservation is paramount, and these findings provide evidence-based support for the concept of chemoablation with UGN-101 as an initial kidney-sparing treatment option for low-grade UTUC," said Mark P. Schoenberg, MD, Chief Medical Officer at UroGen. "The analysis also advances our understanding of durability of response, which we are pleased to see has remained consistent as the number of patients who reach the six-month follow-up timepoint increases."

The Company initiated its rolling submission of the UGN-101 New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) in December 2018. The FDA previously granted Orphan Drug, Fast Track, and Breakthrough Therapy Designations to UGN-101 for the treatment of UTUC. If approved, UGN-101 would be the first drug approved for the non-surgical treatment of LG UTUC.

About The Phase 3 OLYMPUS Trial

OLYMPUS (Optimized DeLivery of Mitomycin for Primary UTUC Study) is a pivotal, open-label, single-arm Phase 3 clinical trial of UGN-101 (mitomycin gel) for instillation to evaluate the safety, tolerability and tumor ablative effect of UGN-101 in patients with low-grade UTUC. The trial enrolled 71 patients at clinical sites across the United States and Israel. Study participants were treated with six weekly instillations of UGN-101 administered via a standard catheter. Four to six weeks following the last instillation, patients underwent a primary disease evaluation (PDE) to determine response, 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 complete response at the PDE timepoint were then followed for up to 12 months to determine the durability of disease control with UGN-101.

About UGN-101

UGN-101 (mitomycin gel) for instillation is an investigational drug formulation of mitomycin in Phase 3 development for the treatment of low-grade upper tract urothelial cancer (LG UTUC). Utilizing the RTGel technology platform, UroGen’s proprietary sustained release, hydrogel-based formulation, UGN-101 is designed to enable longer exposure of urinary tract tissue to mitomycin, thereby enabling the treatment of tumors by non-surgical means. UGN-101 is delivered to patients using standard ureteral catheters. The Company initiated its rolling submission of the UGN-101 New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) in December 2018. The FDA previously granted Orphan Drug, Fast Track, and Breakthrough Therapy Designations to UGN-101 for the treatment of UTUC. If approved, UGN-101 would be the first drug approved for the non-surgical treatment of LG UTUC.