Piper Jaffray analyst Edward Tenthoff reiterated an Overweight rating on Celyad, but lowered his price target on shares to $41 from $51 as he pushes out approvals based on clinical progress. The analyst noted that Celyad intends to initiate a potentially registrational Phase II AML study in the second half of 2019, has begun a Phase I study of allogeneic CYAD-101 in mCRC with data in the second half of 2019, and will file INDs on CYAD-211 BCMA and CYAD-221 CD-19 CAR-Ts in 2020.
Monday, April 1, 2019
Catalyst Biosciences receives orphan drug desgination for MarzAA in EU
Catalyst Biosciences announced that the European Commission has awarded orphan designation of its Factor VIIa variant marzeptacog alfa, or MarzAA, for the treatment of haemophilia B. Catalyst has also completed dosing in the Phase 2 portion of the Phase 2/3 subcutaneous trial of MarzAA for the treatment of hemophilia A or B with inhibitors. Nine subjects successfully completed dosing and top-line results will be presented in the third quarter of 2019. “Orphan designation is another important acknowledgement of the significant benefits of subcutaneous MarzAA and will complement our orphan drug designation already granted in the U.S. by the Food and Drug Administration,” said Nassim Usman, Ph.D., CEO of Catalyst. “We have completed dosing in the Phase 2 portion of the Phase 2/3 trial for the treatment of hemophilia A or B with inhibitors and have clearly demonstrated efficacy as measured by greater than 90% reduction in annualized bleed rate, as well as bleeding density. We expect to present top-line results from the study in the third quarter of 2019. We also plan to initiate a Phase 3 MarzAA registrational study in 2020 and believe that subcutaneous MarzAA has significant commercial potential in the $2.2B hemophilia inhibitor market.”
Novartis to face lawsuit over doctor kickbacks
A Manhattan district court judge has ruled that Novartis (NVS -0.3%) must face a U.S. lawsuit over alleged kickbacks to physicians aimed at boosting prescriptions for its drugs.
The case began in 2011 when a former sales rep filed a whistleblower suit.
In 2010, the company agreed to pay $422M to settle various civil and criminal charges, including paying kickbacks to doctors. In 2015, it agreed to pay $390M to settle claims that it paid rebates to specialty pharmacies to promote two of its drugs.
Novartis spokesman Eric Althoff says, “We are disappointed in today’s decision and look forward to presenting our case at trial. We continue to believe that the government has insufficient evidence to support its claims.”
Celldex presents data from its bispecific and TAM receptor programs at AACR
Celldex Therapeutics presented promising data from the company’s growing bispecific and TAM receptor programs during poster sessions at the American Association for Cancer Research, AACR, Annual Meeting 2019. “Our research and discovery efforts have yielded multiple promising candidates that address important needs in cancer immunotherapy and complement our pipeline,” said Tibor Keler, Ph.D., Executive Vice President and Chief Scientific Officer of Celldex Therapeutics. “CDX-527 joins two powerful pathways in the immune system, PD-1 blockade and CD27 costimulation. The data we presented at AACR demonstrate that this bispecific antibody candidate has greater activity than the combination of the two individual antibodies and support advancing the program into IND-enabling studies. In addition, we also presented data on our lead candidate antibodies targeting the TAM receptors, which act as checkpoints on myeloid cells. We have uncovered a unique mechanism for antibody-mediated activation of dendritic cells and macrophages and demonstrated that a surrogate MerTK mAb promotes anti-tumor immunity alone and enhances the activity of PD-1 blockade. We look forward to continuing to progress both CDX-527 and our TAM program over the course of the year,” concluded Keler.
Dynavax: SD-101, domatinostat induce systemic anti-tumor response in models
Dynavax Technologies Corporation and 4SC AG announced the combination of 4SC’s orally available class I selective HDAC inhibitor domatinostat with Dynavax’s intratumoral TLR9 agonist SD-101 induced a systemic anti-tumoral immune response in tumor mouse models, resulting in the significant decrease in tumor size of both target tumors and distant site metastases. Combination of SD-101 with domatinostat showed better results than combinations of SD-101 with competing HDAC inhibitors. The triple combination of both compounds with PD-1 blockade demonstrated even higher efficacy. SD-101 is a TLR9 agonist, which was specifically developed for cancer based upon its ability to stimulate both IFN-alpha production and the maturation of plasmacytoid dendritic cells into tumor antigen presenting cells. This results in increased activation and proliferation of tumor-specific CD8+ T cells which attack distant site non-injected tumors. Domatinostat, acting via epigenetic regulation, renders tumor cells more visible to the immune system and promotes a general immune response against tumor tissue as well as infiltration of T cells into the tumor tissue. Preclinical data demonstrate that the combination of intra-tumoral SD-101 and systemic domatinostat strongly synergize to induce substantial regression of the primary tumor, as well as distant site non-injected tumors, including lung metastases. Checkpoint inhibitors, such as anti-PD-1 antibodies further boost the anti-tumor T-cell response, leading to rejection in mice with high metastatic burden. These data indicate that the combination of these three different treatment classes result in induction of a more potent tumor-specific immune response and better recognition and elimination of tumors by immune cells, especially in cancer patients refractory to anti-PD-1 treatment.
Aptose Biosciences presents preclinical data on CG-806, APTO-253 at AACR
Aptose announced that new preclinical data for CG-806, its highly potent oral small molecule pan-FLT3/pan-BTK inhibitor, and APTO-253, its MYC inhibitor, are being presented in two separate posters at the 2019 AACR Annual Meeting in Atlanta, GA. The poster, CG-806, a pan-FLT3 / pan-BTK inhibitor, demonstrates superior potency against cells from IDH-1 mutant and other non-favorable risk groups of AML patients, explores the activity of CG-806 on primary patient samples with acute myeloid leukemia, in studies that were conducted in collaboration with the Beat AML Initiative. CG-806 demonstrated significant potency across sub-groups of AML cells, including relapsed/refractory AML and those with genetic abnormalities related to poor prognoses in AML patients. CG-806 demonstrated superior potency when compared to other FLT3 inhibitors, including midostaurin, sorafenib, sunitinib, dovitinib, quizartinib, crenolanib and gilteritinib. While patient samples with FLT3-ITD mutations were expected to have greater sensitivity to CG-806, the sensitivity of patient cells with IDH1 R132 mutations was an unexpected finding. In 28-day GLP toxicity and toxicokinetic studies, CG-806 continued to demonstrate a favorable safety profile. The poster, Resistance to APTO-253 caused by internal deletion and alternate promoter usage of the MYC gene in Raji B cells, presents in vitro studies that further define the mechanism of action of APTO-253. A novel small molecule, APTO-253 inhibits expression of the MYC oncogene, leading to apoptosis in human-derived solid tumor and hematologic cancer cells without the myelosuppression seen with other chemotherapies. Researchers found that APTO-253 targets a G-quadruplex motif in the P1/P2 promoter region of the MYC gene and inhibits MYC gene expression to induce apoptosis, resulting in its ability to potently kill hematologic malignant cell lines and primary samples from AML and chronic lymphocytic leukemia patients.
Trovagene jumps after reporting results from Acute Myeloid Leukemia trial
Shares of TrovaGene are up 86c, or 23%, to $4.61 following the company’s report of new data from its ongoing Phase 1b/2 study evaluating onvansertib in combination with standard-of-care chemotherapy in Acute Myeloid Leukemia, or AML. Objective response rate has been observed at last 3 highest dose levels of onvansertib with 5 of 19 evaluable patients achieving a complete response, the company reported.
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