Cost of research per patient on a phase III oncology trial was from $75,000 to $125,000 in 2013. Assuming two trials and 200 patients per trial, Spectrum need to spend from $30M to $50M.
Add Folotyn/CHOP. A new big study will compare Folotyn/CHOP and Bel/CHOP against CHOP alone in frontline PTCL. They should concentrate resources on this trial and SPI-2012. BTW, Spectrum needs to complete Phase 3 trial with Marqibo.
ARIA's drug is unique. It works for patients which have one specific mutation and saves their lives. No other drug can do it. Therefore, even causing severe side effects it stays on market. Big difference from Spectrum's drugs.
You might be right. I want to add that outcome of the trial cannot be not successful since both experimental chemotherapy regimens of the study include Marqibo. Whichever regimen wins, Marqibo will be a part of it.
Sometimes time frame for clinical study means time from the beginning of recruitment. Interesting is that last update was on Feb 26, 2014, two years after recruitment was completed and trial is open label. They knew what is going on and projected completion in September 2014.
1. To Evaluate the Safety, Activity and Pharmacokinetics of Marqibo in Children and Adolescents With Refractory Cancer. Phase2. Sponsor: Spectrum. Completion date: December 2014.
2. Combination Chemotherapy and Radiation Therapy in Treating Young Patients With Newly Diagnosed Hodgkin Lymphoma. Phase 3. Sponsor: Children's Oncology Group. Completion date: September 2014.
3. Bortezomib and Combination Chemotherapy in Treating Young Patients With Relapsed Acute Lymphoblastic Leukemia or Lymphoblastic Lymphoma. Phase 2, Sponsor: National Cancer Institute (NCI). Completion date: September 2014.
4. Safety and Efficacy of Marqibo in Metastatic Malignant Uveal Melanoma. Phase 2. Sponsor: Spectrum. Completion date: December 2013. Safety data are already published. Waiting for efficacy results.
Balugrastim was studied against Neulasta in two phase 3 trials with breast cancer patients. Lipegfilgrastim was studied against Neulasta in one phase 3 trial in breast cancer and against placebo in one phase 3 trial in NSCLC. Spectrum is going to study SPI-2012 in one phase 3 trial with breast cancer patients and one phase 3 trial with NHL patients. I guess, first trial will be conducted against Neulasta and the second one could be against placebo. Wonder why Spectrum/FDA chose this path?
I think, if one third of the dose of SPI-2012 and full dose of Neulasta have same effect on bone marrow, it should result in same number of AEs.
Their Phase 3 study enrolled patients from 1 to 17 years of age. If I remember correctly, this pediatric population has same incidence of SBS per person as the adult one.
Some interesting points:
1. Gattex launch is one of the top five best rare disease launches in US
2. ADH patient population is 2000-3000 worldwide. Results from phase 2 study of NPSP795 in ADH are expected in 1stQ 2015
3. Top line results from Phase 3 study in pediatric SBS are expected in 2014
4. Sensipar-backed debt will be paid off in 3dQ 2015.
I could not post full abstract because it is too long. Here are missing parts which help to explain the design of the study.
Background: Cumulative neurotoxicity is a major issue and dose limiting factor of vincristine in the treatment of diffuse large B‐cell‐lymphoma (DLBCL) and other malignancies, especially in elderly patients. In the RICOVER‐60 trial of the German High Grade Non‐Hodgkin Lymphoma Study Group (DSHNHL) [Pfreundschuh et al., Lancet Oncol. 2008] only 59% of patients received full‐dose vincristine at cycle 6, 22% of patients received no vincristine at cycle 6 and 36% at cycle 8 due to the neurotoxicity of the drug. Thus, vincristine‐induced polyneuropathy is a long‐term toxicity that heavily affects the quality of life of successfully treated elderly patients with lymphoma, because of slow and often incomplete recovery. Moreover, retrospective analyses suggest that dose reductions of vincristine lead to a considerable loss of efficacy [Fisher et al., Am J Med. 1977; De VV et al., Cancer Res. 1987]. Since preclinical and clinical studies suggest a high activity and excellent tolerability of liposomale vincristine, a randomized comparison of the two formulations is warranted.
Methods: In the OPTIMAL 60 study elderly patients with DLBCL (61‐ to 80 years‐old, ECOG 0‐4, IPI 1 [age 60] with bulk, or IPI 2‐5) are randomized to receive eight 2‐week applications or an optimized schedule of 12 application of rituximab in combination with 6 cycles of CHOP‐14. In a 2x2 factorial design, patients receive a second randomization into
conventional vincristine (1.4 mg/m , capped at 2 mg) or liposomal vincristine (2mg/m , uncapped). One secondary endpoint of the OPTIMAL 60 trial of the DSHNHL is the determination of the median dose of either conventional or liposomal vincristine, that can be given to patients applying identical dose reduction rules. Neurotoxicity was determined
as polyneuropathy according to the Common Terminology Criteria for Adverse Events
Most likely, $100M or $130M is a total Melphalan market in all indications. Melphalan for stem cell transplantation in multiple myeloma is only a fraction of it. Could be $10M or $20M. More expensive CE-Melphalan could generate much more revenue in SCT. Therefore, peak of CE-Melphalan sales could reach $100M while market for generic Melphalan will not change much.