On September 3, 2019 Achilles Therapeutics ("Achilles"), a biopharmaceutical company developing personalised cancer immunotherapies, reported that it has closed a £100 million Series B financing led by incoming U.S. investor RA Capital Management, corner-stoned by founding investor Syncona and joined by important new investors including Forbion, Invus, Perceptive Advisors and Redmile Group (Press release, Achilles Therapeutics, SEP 3, 2019, View Source [SID1234539180]).

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Proceeds from this financing will deliver two human proof-of-concept studies using a unique personalised T cell therapy approach targeting clonal neoantigens in non-small cell lung cancer and melanoma. These programmes are expected to enter the clinic this year. In addition, the financing will enable the Company to continue building out its manufacturing capabilities as well as broadening its growing solid tumour pre-clinical product pipeline.

"Achilles is leading the next wave of immuno-oncology drug development. We have moved from concept to clinic-ready in less than three years. We are extremely pleased to welcome this excellent group of new investors to Achilles and I would like to personally thank our existing founding investors for their continued support," said Dr Iraj Ali, CEO of Achilles Therapeutics. "With this fundraising we have made a clear statement about the scale and nature of our ambitions to bring novel cancer therapies rapidly to patients with a high unmet medical need."

"In 2016, we saw an opportunity to work with world-leading experts to found a company harnessing unique insight into the understanding of cancer evolution, bioinformatics and the development of cell-based immunotherapies to target the treatment of solid tumours. We are delighted with the outcome of this funding round, which is a testament to the Company’s globally differentiated proposition and best-in-class team. We look forward to continuing to support Achilles as it works toward its goal to bring treatments to patients," added Martin Murphy, CEO of Syncona Investment Management Limited.

"The Achilles approach integrates years of multi-disciplinary scientific and clinical knowledge from immuno-oncology, cell therapy, and genomics with the goal of creating a TIL-based therapeutic enriched with T cells reactive against clonal neoantigens. We believe this approach may represent the optimal way to expand the utility of polyclonal TIL therapy to multiple solid tumour types and has the potential to provide profound clinical benefit for patients living with cancer. RA Capital is impressed by the scientific co-founders, management team, board of directors, and vision of the Company, and we are excited to support Achilles as they progress into the clinic this year," added Dr Derek DiRocco, Principal of RA Capital Management.

In association with the financing, Achilles has added two leading life science executives to the Board: Dr Derek DiRocco, Principal of RA Capital Management and Dr Rogier Rooswinkel, Partner at Forbion.

Achilles is developing personalised T cell therapies for solid tumours targeting clonal neoantigens: protein markers unique to each patient that are present on the surface of all cancer cells. Using its PELEUS bioinformatics platform, Achilles can identify clonal neoantigens from each patient’s unique tumour profile which are present on every cancer cell. Achilles uses its proprietary process to manufacture T cells which seek to exquisitely target a specific set of clonal neoantigens in each patient. Targeting multiple clonal neoantigens that are present on all cancer cells, but not on healthy cells, reduces the risk that new mutations can induce immune evasion and therapeutic resistance, and allows individualised treatments to potentially target and destroy tumours without harming healthy tissue.

– Ends –

Further information:

Achilles Therapeutics
Dr Iraj Ali – Chief Executive Officer
+44 (0)1438 906 906
[email protected]

Julia Wilson – Head of Communications
+44 (0)7818 430877
[email protected]

Consilium Strategic Communications
Mary-Jane Elliott, Sukaina Virji, Melissa Gardiner
Tel: +44 (0) 203 709 5000
Email: [email protected]

On September 3, 2019 Kura Oncology, Inc. (Nasdaq: KURA), a clinical-stage biopharmaceutical company focused on the development of precision medicines for oncology, reported positive topline results from an investigator-sponsored Phase 2 trial of its lead drug candidate, tipifarnib, in patients with relapsed or refractory urothelial carcinomas that carry HRAS mutations (Press release, Kura Oncology, SEP 3, 2019, View Source [SID1234539198]).

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The ongoing, single-agent, single-arm trial is designed to enroll at least 18 patients, with a primary endpoint of progression-free survival (PFS) rate at 6 months. Secondary endpoints include objective response rate, duration of response and safety. The trial is being conducted at the Samsung Medical Center in Korea.

To date, more than 200 patients with relapsed or refractory urothelial carcinoma have been screened for the presence of tumor HRAS mutations. A total of 15 patients were identified to carry tumors with HRAS mutations. Two patients withdrew from the trial prior to their first response assessment. Of the 13 evaluable patients, five experienced confirmed objective responses, according to RECIST 1.1 criteria, for an overall response rate of 38%. Notably, four patients have experienced PFS of greater than 6 months. According to the trial protocol, the primary endpoint is met when at least four patients achieve PFS at 6 months.

"Although the treatment paradigm for advanced urothelial carcinoma has evolved with the introduction of checkpoint inhibitors, there remains a need for more precise and effective treatment options for these patients," said Se Hoon Park, M.D., Ph.D., Samsung Medical Center, principal investigator for the trial. "These biomarker-driven data in patients with relapsed or refractory urothelial carcinoma are promising and further underscore the potential for tipifarnib in HRAS mutant solid tumors."

All patients joined the trial upon progression from at least one prior systemic chemotherapy cycle, with a median of one prior therapy. Tipifarnib has been generally well-tolerated in the trial; adverse events observed are consistent with the known safety profile of tipifarnib. Further analyses of the trial are ongoing, and detailed data are expected to be presented at a future medical meeting.

"We are very encouraged to see that this investigator-sponsored trial in HRAS mutant urothelial carcinoma met its primary endpoint prior to the completion of enrollment," said Antonio Gualberto, M.D., Ph.D., Head of Development and Chief Medical Officer of Kura Oncology. "This study represents the fourth clinical proof-of-concept for tipifarnib and the second proof-of-concept in a HRAS mutant solid tumor indication. We believe that more than five percent of urothelial carcinoma patients carry the HRAS mutation, which represents a meaningful additional market opportunity for tipifarnib. Based on these results, we are currently evaluating next steps for tipifarnib in this indication and look forward to the presentation of the full data set at an upcoming medical meeting."

About Urothelial Carcinoma

Urothelial carcinoma, also known as transitional cell carcinoma, develops from urothelial cells that line the inside of the bladder. Urothelial carcinoma accounts for 90 percent of all bladder cancers, and can also arise in the renal pelvis and ureters. The American Cancer Society estimates approximately 80,470 new cases of bladder cancer in the United States for 2019. Despite the approval of checkpoint inhibitors in recent years, the treatment of patients with advanced urothelial carcinoma in the second-line setting remains a significant unmet need.

About Tipifarnib

Kura Oncology’s lead drug candidate, tipifarnib, is a potent, selective and orally bioavailable inhibitor of farnesyl transferase in-licensed from Janssen in December 2014. Previously, tipifarnib was studied in more than 5,000 cancer patients and showed compelling and durable anti-cancer activity in certain patient subsets; however, no molecular mechanism of action had been determined that could explain its clinical activity across a range of solid tumor and hematologic indications. Leveraging advances in next-generation sequencing as well as emerging information about cancer genetics and tumor biology, Kura is seeking to identify those patients most likely to benefit from tipifarnib. In November 2018, following an end of Phase 2 meeting with the U.S. Food and Drug Administration, Kura initiated its first registration-directed trial of tipifarnib in patients with recurrent or metastatic HRAS mutant head and neck squamous cell carcinoma (HNSCC). In addition to HRAS mutant HNSCC and HRAS mutant urothelial carcinoma, the Company has achieved clinical proof-of-concept with tipifarnib in angioimmunoblastic T-cell lymphoma and CXCL12-expressing peripheral T-cell lymphoma.

On September 3, 2019 Trovagene, Inc. (Nasdaq: TROV), a clinical-stage, Precision Cancer Medicine oncology therapeutics company developing drugs that target cell division (mitosis) for the treatment of various cancers including prostate, colorectal and leukemia, reported it will be presenting at the H.C. Wainwright 21st Annual Global Investment Conference at 10:50 a.m. Eastern time on Tuesday, September 10, 2019 (Press release, Trovagene, SEP 3, 2019, https://www.prnewswire.com/news-releases/trovagene-to-present-at-the-hc-wainwright-21st-annual-global-investment-conference-300909790.html [SID1234539215]). The conference is being held September 8-10, 2019 at the Lotte New York Palace Hotel.

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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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Dr. Thomas Adams, Chief Executive Officer and Chairman, and Dr. Mark Erlander, Chief Scientific Officer of Trovagene, will provide an overview of the Company’s business during the live presentation and will be available for one-on-one meetings with investors.

Institutional investors who would like to attend the company’s presentation should click here to register for the conference. Once registered, you can log into the conference website to request a one-on-one meeting.

The presentation will be webcast live. To access the webcast, please click here. The webcast replay will remain available for 90 days following the live presentation.

On September 3, 2019 Exicure, Inc. (Nasdaq: XCUR), a pioneer in gene regulatory and immunotherapeutic drugs utilizing spherical nucleic acid (SNA) constructs, reported presentations at the following investor conferences during the months of September and early October (Press release, Exicure, SEP 3, 2019, View Source [SID1234539232]):

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H.C. Wainwright 21st Annual Global Investment Conference
Corporate update
Presented by: CEO David Giljohann
Monday, September 9, 2019 at 10:25am ET
Location: New York, NY
Ladenburg Thalmann 2019 Healthcare Conference
Corporate update
Presented by: CEO David Giljohann
Tuesday, September 24, 2019 at 1:30pm ET
Location: New York, NY
Chardan’s 3rd Annual Genetic Medicines Conference
Corporate update
Presented by: Dr. David Giljohann
Monday, October 7, 2019 at 2:00pm ET
Location: New York, NY

The Next Big Thing in Genetic Medicines
Panelist: Dr. David Giljohann
Tuesday, October 8, 2019 at 9:30am ET
Location: New York, NY
An audio webcast will be available on the Investors section of Exicure’s website: www.exicuretx.com. The webcast will be archived for approximately 30 days following the event.

On September 3, 2019 Helix BioPharma Corp. (TSX: HBP), ("Helix" or the "Company"), an immuno-oncology company developing innovative drug candidates for the prevention and treatment of cancer, is reported that they, together with Moffitt Cancer Center ("Moffitt"), will be presenting a poster entitled "Pharmacodynamics of targeted urease and checkpoint blockade using Chemical Exchange Saturation Transfer ("CEST") and 31P-magnetic resonance spectroscopy ("31P-MRS") (Press release, Helix BioPharma, SEP 3, 2019, View Source [SID1234539248])." The question asked, "Is there a role for hyperpolarized 13C and 15N?"

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The poster presentation will take place at the World Molecular Imaging Congress ("WMIC"), which is taking place at the Palais des congrès de Montréal in Montréal, Quebec, Canada (View Source) on September 4th through the 7th, 2019.

The presentation describes the use of functional imaging techniques to measure the effect of L-DOS47 on changing tumor pH in a pancreatic mouse model. The pH effect can be measured by CEST MRI using a CT contrast agent iopamidol and by 31P-MRS with 3-aminopropyl phosphate ("3-APP") as reporting agent. In both cases, L-DOS47 injection is shown to induce a localized pH increase at the target tumor. L-DOS47 pH effect may also have a direct impact on therapy as combined treatment of L-DOS7 with an anti-PD-1 antibody is more efficacious than either agent alone in a preliminary round of animal study.

In a separate experiment, high resolution magnetic resonance spectroscopy of hyperpolarized 13C and 15N urea was able to show how urease (L-DOS47 enzymatic active component) breaks down urea directly and induces the pH effect. Both CEST and hyperpolarization are functional imaging techniques being studied in clinics to monitor therapeutic purposes. Helix is exploring the potential application of these techniques to its L-DOS47 clinical development program.

"I would like to thank and congratulate Dr. Robert (Bob) Gillies and the Moffitt team," said Dr. Heman Chao, Helix’s Chief Executive Officer. "This work complements data that was presented at the recent American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2019, in Atlanta, where the Company presented a poster presentation on "Improving survival in pancreatic cancer using Doxorubicin in combination with L-DOS47". We look forward to continuing collaboration with Bob and the team."

On August 7, 2019 the Company announced that it had received approval from the U.S. Food and Drug Administration to initiate a Phase Ib/II study of L-DOS47 and doxorubicin in advanced metastatic pancreatic cancer.
About Helix BioPharma Corp.

Helix BioPharma Corp. is an immuno-oncology company specializing in the field of cancer therapy. The company is actively developing innovative products for the prevention and treatment of cancer based on its proprietary technologies. Helix’s product development initiatives include its novel L-DOS47 new drug candidate and Chimeric Antigen Receptor ("CAR") based cell therapies. Helix is currently listed on the TSX under the symbol "HBP".

About L-DOS47

L-DOS47 is Helix’s first immunoconjugate based drug candidate in development based on the Company’s novel DOS47 platform technology, which is designed to use an innovative approach to modify the microenvironmental conditions of cancer cells in a manner that leads to their destruction.

Investor Relations

Helix BioPharma Corp.
9120 Leslie Street, Suite 205
Richmond Hill, Ontario, L4B 3J9
Tel: 905-841-2300
Email: [email protected]