On December 2, 2018 Aprea Therapeutics, a clinical-stage biotechnology company developing novel anticancer therapies targeting the p53 tumor suppressor protein, reported the closing of a EUR 50 million Series C financing round led by the Redmile Group, with participation by new investor Rock Springs Capital and existing investors 5AM Ventures, Versant Ventures, HealthCap, Sectoral Asset Management and Karolinska Development AB (Nasdaq Stockholm: KDEV) (Press release, Aprea, DEC 3, 2018, View Source [SID1234531806]). A representative from Redmile Group will also be appointed as Director to the Aprea Therapeutics Board of Directors.

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"We are thrilled to close this financing, initiate a Phase III clinical study in myelodysplastic syndromes (MDS) with our lead compound, APR-246, and drive the company toward its next value inflection point," said Christian S. Schade, President and Chief Executive Officer of Aprea. "The addition of Redmile and Rock Springs to our investor group broadens the US investor base and we believe positions the company for future strategic opportunities. We are committed to bringing this important new therapy to cancer patients with limited therapeutic options."

Proceeds from the financing will be used to advance the clinical development of APR-246, a first-in-class anticancer agent that reactivates mutated p53 tumor suppressor protein. Aprea is planning to begin a Phase 3 clinical study in myelodysplastic syndromes (MDS) and is nearing completion of a Phase Ib/II clinical trial in p53 mutated high-risk myelodysplastic syndromes (MDS) and oligoblastic acute myeloid leukemia (AML) with APR-246 and azacitidine. The Phase II part of the study is ongoing, with updated data to be presented at the 2018 American Society of Hematology (ASH) (Free ASH Whitepaper) annual meeting.

About Myelodysplastic Syndromes

Myelodysplastic syndromes (MDS) represents a spectrum of hematopoietic stem cell malignancies in which bone marrow fails to produce sufficient numbers of healthy blood cells. Approximately 30-40% of MDS patients progress to acute myeloid leukemia (AML) and mutation of the p53 tumor suppressor protein is thought to contribute to disease progression. Mutations in p53 are found in up to 20% of MDS and AML patients and are associated with poor overall prognosis.

About p53 and APR-246

The p53 tumor suppressor gene is the most frequently mutated gene in human cancer, occurring in approximately 50% of all human tumors. These mutations are often associated with resistance to anti-cancer drugs and poor overall survival, representing a major unmet medical need in the treatment of cancer.

APR-246 has been shown to reactivate mutant and inactivated p53 protein – by restoring wild-type p53 conformation and function – and thereby induce programmed cell death in human cancer cells. APR-246 has demonstrated pre-clinical anti-tumor activity in a wide variety of solid and hematological (blood) tumors, including MDS, AML, and ovarian cancer, among others. Additionally, strong synergy has been seen with both traditional anti-cancer agents, such as chemotherapy, as well as newer mechanism-based anti-cancer drugs and immuno-oncology checkpoint inhibitors. In addition to pre-clinical testing, a Phase I/II clinical program with APR-246 has been completed, demonstrating a favorable safety profile and both biological and confirmed clinical responses in hematological malignancies and solid tumors with mutations in the TP53 gene.

On December 3, 2018 Trillium Therapeutics Inc. (NASDAQ/TSX: TRIL), a clinical stage immuno-oncology company developing innovative therapies for the treatment of cancer, reported that new clinical data from the ongoing intratumoral trial of its CD47-blocking agent, TTI-621 (SIRPa-IgG1 Fc), were presented at the American Society of Hematology (ASH) (Free ASH Whitepaper) 60th Annual Meeting, December 1-4, in San Diego, California (Press release, Trillium Therapeutics, DEC 3, 2018, View Source [SID1234531823]).

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"These results build upon the clinical data reported at the EORTC Cutaneous Lymphoma Task Force meeting in September and provide further evidence that intratumoral TTI-621 is well tolerated and biologically active in patients with cutaneous T-cell lymphoma," said Dr. Niclas Stiernholm, president and CEO of Trillium Therapeutics. "The signs of anti-tumor activity in non-injected lesions are particularly encouraging, as are the translational data demonstrating recruitment of cells of both the innate and adaptive immune systems. We believe that this is the most compelling evidence of single-agent activity of any CD47-targeting agent in the clinic, and we are continuing to execute a focused development plan in T-cell lymphoma."

Poster Presentation 1653:

Intralesional Administration of the CD47 Antagonist TTI-621 (SIRPaFc) Induces Responses in Both Injected and Non-injected Lesions in Patients with Relapsed/Refractory Mycosis Fungoides and Sézary Syndrome: Interim Results of a Multicenter Phase I Trial
Presenter: Christiane Querfeld, M.D., Ph.D., City of Hope National Medical Center

This poster presentation provided a further update on the safety and anti-tumor activity observed in the ongoing open label Phase 1 study of local TTI-621 administration in highly pretreated patients with relapsed or refractory mycosis fungoides or Sézary syndrome. Intratumoral TTI-621 was well tolerated in 27 treated patients, with no grade 3 or higher toxicity observed. A rapid reduction in Composite Assessment of Index Lesion Severity (CAILS) scores, which measure local lesion responses, was observed in 91% (20/22) of patients with available scores across all disease stages, with 41% (9/22) exhibiting a 50% or greater decrease in CAILS scores. Similar CAILS-based changes were seen in adjacent non-injected lesions, suggesting local regional effects that were not confined to the site of injection. Continuation monotherapy beyond the initial two week induction period led to further reductions in CAILS scores in 3/4 evaluable patients and evidence of systemic effects were observed in one patient. In addition, emerging translational data demonstrate that local TTI-621 administration leads to a rapid influx of macrophages and CD8+ T cells.

On December 3, 2018 Gamida Cell Ltd. (Nasdaq: GMDA), a leading cellular and immune therapeutics company, reported translational data showing that recipients who received NiCord, an investigational cell therapy in Phase 3 clinical development for allogeneic hematopoietic stem cell (bone marrow) transplant had rapid and robust reconstitution of key immune cells (Press release, Gamida Cell, DEC 3, 2018, View Source [SID1234531840]). Successful immune reconstitution is an important factor in the recovery of patients undergoing bone marrow transplant. These translational data from the completed Phase 1/2 study of NiCord were presented in a poster session during the American Society of Hematology (ASH) (Free ASH Whitepaper) 2018 Annual Meeting being held December 1-4 in San Diego, CA.1

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"Immune reconstitution following transplantation is critical for disease and viral control, but historically cord blood transplantation has had limitations in timely immune reconstitution in patients," said Jaap-Jan Boelens, M.D., Ph.D., Chief, Pediatric Stem Cell Transplantation and Cellular Therapies Service, Memorial Sloan Kettering Cancer Center. "We were pleased to see that NiCord treatment resulted in rapid and robust immune reconstitution when compared to younger patients who typically achieve more rapid recovery than adults."

Despite the curative potential of bone marrow transplants, it is estimated that more than 40 percent of eligible patients in the U.S. do not receive one for various reasons, including finding a matched donor.2 While umbilical cord blood provides a source of stem cells for patients who do not have a matched related donor, it provides a smaller number of stem cells, which can delay engraftment and put patients at a greater risk for prolonged hospitalizations and life-threatening infections. NiCord is designed to address these limitations by offering a therapeutic dose of expanded cells while preserving the functional characteristics of stem cells.

Data Presented at ASH (Free ASH Whitepaper) 2018
The poster presentation, "Rapid and robust CD4+ and CD8+ T-, NK-, B- and monocyte cell reconstitution after nicotinamide-expanded cord blood transplantation" (Abstract 2123), described early, in-depth immune reconstitution data from the completed Phase 1/2, multicenter clinical study of NiCord as a stand-alone graft after myeloablative therapy in patients with high-risk hematologic malignancies.3 A random subgroup of 27 patients from this study had extensive immune monitoring evaluated throughout the first year after transplant. The primary endpoint was the probability of achieving CD4+ immune reconstitution (>50×106/L) within the first 100 days, and the secondary endpoints included the recovery of B cells, CD4+ T cells and natural killer (NK) cells during the first year after transplantation. These data were compared to cohorts of adolescent and young adults with hematologic malignancies receiving unmanipulated cord blood transplantation (n=27) or unrelated bone marrow transplantation (n=20). Analyses were performed at the University Medical Centre Utrecht, Laboratory of Translational Immunology.

Key findings from the analysis include the following:

91 percent of patients achieved successful immune reconstitution of CD4+ T cells at 100 days after transplantation with NiCord.
Immune reconstitution of T cells was similar in the NiCord group (median age: 41.5 years) compared to the younger cohorts receiving unmanipulated cord blood and unrelated bone marrow (median ages 15.4 and 14.3 years, respectively).
Immune reconstitution of B cells (p = 0.02) and NK cells (p < 0.001) was significantly faster after transplantation with NiCord compared to the other groups.
Immune reconstitution after NiCord transplantation was associated with recovery of a broad spectrum of T cell, B cell and NK cell subsets representing a range of effector functions similar to that observed with other graft sources.
"These data, combined with the clinical data from our Phase 1/2 study of NiCord in patients with high-risk blood cancers, suggest that NiCord has the potential to be an important treatment option for patients undergoing bone marrow transplant," stated Ronit Simantov, M.D., chief medical officer at Gamida Cell. "We are working to advance our NiCord clinical development program and expect to complete patient enrollment in our ongoing Phase 3 study in the second half of 2019."

About NiCord
NiCord, the company’s lead clinical program, is under development as a universal bone marrow transplant solution for patients with high-risk hematologic malignancies. NiCord has been granted breakthrough status by the U.S. Food and Drug Administration, making it the first bone marrow transplant alternative to receive this designation. It has also received U.S. and EU orphan drug designation. A Phase 3 study evaluating NiCord in patients with leukemia and lymphoma is ongoing in the United States, Europe and Asia.4 For more information on NiCord clinical trials, please visit www.clinicaltrials.gov.

On December 3, 2018 Under this agreement, Biotecnol reported that divested from it’s immuno-oncology pipeline and respective technologies (including Tb535H, and Trisoma (Press release, Biotecnol, DEC 3, 2018, View Source [SID1234570279]). Chiome will continue research and clinical development for Tb535H. In addition, Chiome will continue generating new immune-oncology products using Biotecnol’s Trisoma technology. Financial details were not disclosed.

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About Tb535H

Tb535H is a T-cell engager, trispecific antibody, directed against the 5T4/WAIF1 tumour antigen, a protein found on many different solid tumours and is thought to contribute to the spread of cancer cells. Tb535H recruits the patient’s T-cells –killer cells of the immune-system – and directs them to attack tumours. This highly targeted approach uses the patient’s own immune system to fight cancer. The WAIF1 antigen was discovered by scientists at the Cancer Research UK Manchester Institute. It could be a valuable target in many different cancer types, but the initial focus in this trial will be to treat cancers with high unmet-need. This includes thoracic cancers such as mesothelioma, small-cell lung carcinoma (SCLC) and non-small cell lung cancer (NSCLC), for which survival remains very low, and renal cell carcinoma.

About Trisoma technology

The Trisoma technology enables the generation of multi-specific antibody products. This unique technology overcomes the key shortcomings of conventional mono- as well as of currently developed bi-specific antibody formats. With the Trisoma technology, Biotecnol can engineer and assemble recombinant antibodies in a rational manner in order to design a chosen mechanism of action to address the killing of a specific tumour type.

On December 3, 2018 Shenzhen Salubris Pharmaceuticals Co. Ltd. (Salubris, SZSE: 002294) and Viracta Therapeutics, Inc. reported the initial closing of a financing, with Salubris committing $10 million as the lead investor (Press release, Viracta Therapeutics, DEC 3, 2018, View Source [SID1234534657]). New investor Virtus Inspire Ventures, as well as Viracta’s existing investors, NantKwest, Inc., Latterell Venture Partners and Forward Ventures, all participated in the financing. In addition to the financing, Salubris and Viracta have entered into an exclusive Collaboration and License Agreement to bring Viracta’s novel treatment approach for virus-associated malignancies to China.

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Viracta is advancing its innovative approach for viral-associated cancers in a Phase 1b/2 clinical trial for Epstein Barr Virus (EBV)-associated lymphomas in the US and Brazil. The approach is the first targeted, orally administered therapy for EBV-associated malignancies. It incorporates Viracta’s proprietary drug candidate, nanatinostat (VRx-3996) in combination with an antiviral as a targeted treatment to eradicate a range of viral-associated cancers. Viracta plans to initiate a clinical trial for the treatment of EBV-associated solid tumors in the coming year.

EBV-associated nasopharyngeal carcinoma is endemic in Southern China. EBV is also highly associated with the incidence and progression of other solid tumors, including gastric carcinoma, as well as NK/T cell lymphomas found throughout China. Salubris CEO, Kevin Ye highlighted, "EBV-driven cancers disproportionately impact patients in China. We look forward to partnering with Viracta to develop this new treatment option in China. The approach holds the potential to provide a valuable new treatment option for these patients, who currently still face considerable morbidity and mortality."

"We are committed to bringing our treatment approach to cancer patients around the world," commented Viracta CEO, Ivor Royston, MD. "We look forward to developing nanatinostat in collaboration with Salubris to address these major healthcare needs in China."

Under the terms of the license agreement, in addition to the equity investment made by Salubris, Viracta is eligible to receive up to $58 million in pre-commercial milestones as well as significant sales level-triggered commercial milestones and tiered royalties on sales. The Companies will collaborate for clinical development of the treatment approach for viral-associated cancers with Salubris taking on responsibility for development within the Peoples Republic of China (excluding Hong Kong, Macau and Taiwan). Viracta retains development responsibility as well as commercial rights outside of China.