Stocks in News: CBIO, CTIC
Discussion: Catalyst Biosciences (CBIO) announced interim data from a Phase 2/3 clinical trial of evaluating subcutaneously administered prophylactic Factor VIIa variant marzeptacog alfa (activated) in patients with hemophilia A or B with inhibitors. One administered patient showed significant improvement in prevention of bleeding in the first 96 days. Two other patients are yet to complete the first 50 days of dosing to determine any significant improvement. Marzeptacog alfa’s subcutaneous half life is 9.5 or almost three times the intravenous half life of 3.5.
Till date, no anti-drug antibody was detected in any of the patients. The results were presented at the 64th Annual Scientific and Standardization Committee Meeting of the International Society on Thrombosis and Haemostasis in Dublin. Shares moved 25% up on this positive news. Haemophilia is caused by mutations in the F8 gene (Xq28) encoding coagulation factor VIII, or in the F9 gene (Xq27) encoding coagulation factor IX. Accordingly, the carriers show either types A and B of the disease.
It is estimated that hemophilia A occurs in 1 in 5,000 live male births. And this genetic disorder is about four times as common as hemophilia B. Prevalence of the disease in the U.S. is estimated to be about 20,000 individuals. Globally 0.4M people are thought to be affected by the disorder and ~75% of them are expected to receive inadequate treatment.
The candidate has the potential advantage of becoming the first FDA-approved drug for Hemophilia B patients as a prophylactic subcutaneous injection. Further, if the candidate succeeds, the candidate has a clear advantage over existing therapies like Bioverativ’s ECLOCTATE and APROLIX treatments. Both these are given as intravenous injections and hence require expert assistance. On the contrary, marzeptacog will be given as a subcutaneous administration and could be taken at home.
Some think that progress made by CBIO takes them closer to the acquisition by a big pharma player. For example, here an analyst explains why he thinks that Sanofi’s (NYSE:SNY) recent moves by acquiring Bioverativ and Ablynx could be seen as part of its attempt to establish itself as the dominant player in the Hemophilia and rare blood diseases.
The financials were improved in December 2017 with a cash raise of $10.5 million, meaning another raise and share price dilution because of such an event is not highly likely until sometime in 2019. After the latest dip in early June, the share price is now hovering around the lower range of its 52-wk range of $3.11-$37.
The share price has seen wide fluctuations with a high beta of 2.62 in price as the market responded to various catalysts. However, the most recent data should be taken as a positive sign for the company and the share price to move in a northward direction. Over the last couple of days, the positive news-fuelled spike in share price has corrected ~40% back to its earlier level, the share price seems to be good value.
Discussion: CTI BioPharma (CTIC) announced its plans to launch a Phase 3 clinical trial of its lead candidate pacritinib in patients with myelofibrosis (‘MF). The trial will begin in 2019. The dosing will be specified on the basis of the outcome of the Phase 2 study. The planned Phase 3 trial is expected to begin in early 2019 with top line data coming up in 2Q 2019. The trial is estimated to be completed by the end of the next year. The company’s decision to move to the next trial phase is encouraged by the positive assessment made by the FDA of the earlier trial data.
Previously, the candidate went through two different Phase 3 trials on patients with myelofibrosis (“MF”) patients with acute or life-threatening thrombocytopenia. Both the trials showed good results with the second trial met one of two co-primary endpoints. It also showed better results than the best available therapy (BAT). However, the trials were put on hold by the FDA due to a few deaths from treatment related adverse events (TEAEs).
For the time being, the company had withdrawn its NDA, but then the hold was lifted. One of the positive consequences of this development is the announcement made by the company regarding the launch of its upcoming dose-limiting pivotal trial. MAA has already been submitted on the basis of the earlier results and a fresh NDA can mature on the basis of the direction this upcoming trial takes.
Current sales data of the widely prescribed MF drug Jakafi, which pacritinib plans to challenge, is a little more than $1B. While MF is only ~1/3 of the total sales figure, there is a significant unmet condition of Jakafi prescription. Considering both these situations, the potential sales figures may be moderately estimated to be $300M/year.
In other News
Pfizer (PFE) and Eli Lilly (NYSE:LLY), its development partner, announced positive results from a Phase 3 clinical trial of tanezumab for the treatment of osteoarthritis (‘OA) pain. The candidate uses a subcutaneous administration route. The trial conclusively met all three endpoints.
Janssen Pharmaceutical, a unit of Johnson & Johnson receives FDA approval for its once-daily Symtuza [darunavir/cobicistat/emtricitabine/tenofovir alafenamide (D/C/F/TAF)] for treatment-naive and certain virologically suppressed patients with HIV-1 infection. This drug will be the first darunavir-based single-table regimen approved in the U.S.
Novartis’s (NYSE:NVS) Kisqali (ribociclib) is now FDA-approved for treatment of pre/perimenopausal or postmenopausal women with HR+/HER2- advanced/metastatic breast cancer. The drug will be treated as an initial endocrine-based therapy. This is the first approval under the new FDA pilot program to a more efficient development and review of cancer drugs.
Rolling submission for marketing application for selinexor of Karyopharm Therapeutics (NASDAQ:KPTI) has been started for the treatment of patients with penta-refractory multiple myeloma (MM). The candidate already has Fast Track and Orphan Drug designations. The U.S. filing is estimated to be completed by H2 and a European filing is expected in early 2019.
Genentech, a unit of Roche (OTCQX:RHHBY) receives Breakthrough Therapy designation for its Tecentriq (atezolizumab) in combination with Avastin (bevacizumab) for early phase treatment of patients with advanced or metastatic phases of most common form of liver cancer, hepatocellular carcinoma.
Apprehensive of sudden consequences of Brexit, AstraZeneca (NYSE:AZN) is reportedly increasing stockpiles of certain medicines up to 20%.
Enzo Biochem (NYSE:ENZ) announced validation of three cost-efficient biomarker detection tests for monitoring the progression of certain cancers. These developments complement the company’s development of Polyview in situ hybridization platform.
Adaptimmune Therapeutics plc (NASDAQ:ADAP) announced a positive review of the safety parameter of its transduced SPEAR T-cells targeting MAGE-A10 in its non-small cell lung cancer (NSCLC) study. No evidence of toxicity was reported in three patients who received the therapy. This allowed a further escalation of the dose.
Proprietary intraductal microcatheter administration technology to focus on the site of tumor initiation developed by Atossa genetics (NASDAQ:ATOS) receives further development support from the developers. Atos shares moved marginally ahead.
Takeda Pharma (OTCPK:TKPYY) and Ovid Therapeutics (NASDAQ:OVID) plans to expand their clinical development program to include three more clinical trials. The three trials are Phase 2 trial of paediatric patients with either CDD or Dup15q syndrome, Phase 2 trial of paediatric patients with either CDD or Dup15q syndrome and finally, a long-term extension study in patients with DEE who participated in an earlier TAK-935/OV935 clinical trial.
Moleculin Biotech (NASDAQ:MBRX) announced the beginning of preclinical toxicology testing of its WP1732. A new subsidiary in Australia is conducting these tests.
Immunomedics’ (NASDAQ:IMMU) Biologics License Application (BLA) seeking approval to use antibody-drug conjugate sacituzumab govitecan to treat patients with metastatic triple-negative breast cancer (mTNBC) is accepted by the FDA for review. PDUFA is January 18, 2019.
Cellectar Biosciences (NASDAQ:CLRB) announced positive preliminary data from its Phase 2 clinical trial of lead candidate CLR 131 in patients with diffuse large B-cell lymphoma (DLBCL).
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