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Cytokinetics Is Planning New Phase 3 Trial For Tirasemtiv In ALS Using New Endpoint
- Cytokinetics announced that it is planning a new phase 3 trial for tirasemtiv in 2015 using sustained vital capacity as the primary endpoint.
- I think that the trial will only go forward if the FDA agrees to a Special Protocol Assessment using this measure as an endpoint.
- If the FDA does not agree to a Special Protocol Assessment, I think tirasemtiv will be dropped from clinical development.
- This renews hope that tirasemtiv could be approved for ALS in the future. There seems to be no expectation of this in a stock price in the mid $3.00s.
Discovery Laboratories: Collaboration With Battelle Is Validation Of Aerosurf's Potential By A Sophisticated Third Party
- This agreement partners Discovery with a world class medical device developer and meaningfully de-risks development of Aerosurf.
- It also gives meaningful validation from a sophisticated third party of the revolutionary potential for Aerosurf in treating respiratory distress syndrome.
- The terms of the collaboration reduces capital needs for Discovery and Battelle only wins if Aerosurf is successfully developed.
- Phase 2a Aerosurf results will be reported in 4Q, 2014. These relate primarily to safety and may not have any efficacy signal. They may not affect the stock price.
- Phase 2b results should be available in 4Q, 2015. Positive results would be the first proof of concept in a controlled trial and would be very positive for the stock.
Northwest Biotherapeutics: U.K. May Grant Early Patient Access For DCVax-L Prior To Formal Approval
- UK designates DCVax-L as first drug to be considered for early access approval under recently enacted EAMS program.
- Earlier this year, Germany granted approval to DCVax-L under its early access program.
- Early access means that DCVax-L can be prescribed prior to formal approval that might come in 2016. I expect revenues from German sales in 2014.
- These actions by regulatory agencies are unsurpassed validation for the potential of the DCVax-L technology, in my opinion.
- On another positive note, the phase 2 trial of DCVax Direct should begin in 4Q, 2014.
ImmunoCellular: Thoughts On Management's Plan To Move ICT-107 Into Phase 3
- Management hopes to begin a Phase 3 trial in a subgroup of patients who are HLA-A2 positive, possibly in 1H, 2015. It might conclude in late 2018 or 2019.
- I estimate that HLA-A2 sub-group is 34% to 40% of the 3,500 newly diagnosed GBM patients annually in the US.
- I estimate the price of ICT-107, if approved, would be $150,000 per treatment or more, which is in line with the price point of the newly introduced anti-PD-1 drugs.
- I agree that ICT-107 should be taken forward based on the data.
- However, I think that others are less optimistic and it may be very difficult for IMUC to raise the capital needed to fund the trial. Hence, I am neutral.
Antares: An Analysis Of FDA Briefing Documents For September 17th Advisory Committee Meeting On Testosterone Replacement Products
- The FDA does not appear to be concerned about cardiovascular risk with testosterone replacement products so this is a major positive.
- The FDA is asking the panel to consider if these products are used too much in unapproved indications.
- The testosterone replacement market has shrunk significantly in 2014 due to the cardiovascular safety issue. There could be continued shrinkage in 2015.
- This market is not going away. I see opportunity for Antares’ QST accounting for $50+ millions of product sales by the end of the decade.
- QST is a major product opportunity for Antares and I remain a buyer.
Cytokinetics: Will The FDA Agree To A New Phase 3 Trial For Tirasemtiv?
- Tirasemtiv failed to reach the primary endpoint of ALSFRS-r in the Phase 2b BENEFIT-ALS trial, causing investors to write off any potential for the drug.
- I believe that Cytokinetics will attempt to persuade the FDA and EMEA to accept sustained vital capacity as the primary endpoint in a new Phase 3 trial.
- If the agencies agree to SVC as a primary endpoint, I think there is a high probability for success. If not, I think Cytokinetics will abandon development of tirasemtiv.
- Because expectations are so low for tirasemtiv, there is asymmetric upside potential if a Phase 3 trial is undertaken and is successful.
Northwest Bio: The Data Monitoring Committee Addresses The Status Of The DCVax-L Trial
- Chairman of DMC says that there has not yet been an interim analysis performed for efficacy in the phase 3 trial of DCVax-L.
- He says that NWBO is totally blinded to the data and has no idea of how results are trending.
- This is a serious blow to the bear thesis that maintains that the interim analysis had been performed, the results were bad and that NWBO hid the results from investors.
Northwest Biotherapeutics: Responding To Richard Pearson's Article Alleging A Promotion Scheme
- I categorically deny all charges that Pearson has raised in his article that I engaged in the promotion of NWBO or any other company.
- I categorically deny that I received any compensation to write about NWBO, or any other company.
- Pearson presents only circumstantial arguments to support his case. I have asked Pearson for objective evidence that I have been paid to write on companies.
Discovery Laboratories: Surfaxin Launch Has Been Slow, But Clinical Trials Of Aerosurf Are Beginning At Last
- The Surfaxin launch has been slow, and analysts' expectations are for $2 million of sales in 2014.
- The potential for Aerosurf to become a blockbuster in neonatology is the key issue for the stock.
- Phase IIa results for Aerosurf are due in 3Q 2014, and Phase IIb proof-of-concept results could be available in 4Q 2015.
ImmunoCellular Plans To Move ICT-107 Forward Into Phase III Trials Based On New Sub-Group Analysis Presented At ASCO
- IMUC has identified two prospectively defined sub-groups of patients based on HLA-A2 and MGMT status that show encouraging results in GBM.
- The company plans to speak to regulatory agencies and move forward into Phase III trials.
- These two sub-groups comprise about 31% of GBM patients.
Northwest Biotherapeutics: Management Discusses Interim Data On Phase I/II Trial Of DCVax-Direct
- Interim results are for 19 patients who received two weeks of treatment; the protocol calls for 8 months of treatment.
- Early results indicate that 11 patients have shown tumor necrosis and an immunological effect on the tumor.
- Six patients have shown tumor shrinkage or stable disease, with one patient showing tumor shrinkage of 28%, just short of 30% which qualifies as a partial response.
- The key is whether these early responses translate into complete or partial responses as patients progress through the next 7 1/2 months of treatment.
Northwest Biotherapeutics Reports On Sarcoma Patient Treated With DCVAx-Direct
- NWBO provides information on sarcoma patient treated with DCVax-Direct.
- This is the first report on any patient treated with DCVax-Direct.
- The data is limited and early, but still quite encouraging.
Ed Quilty, CEO Of Derma Sciences, Outlines His Strategy To Build A Premier Wound Healing Company
- DSCI is a pure play investment opportunity for the treatment of diabetic foot ulcers; the incidence for which is about 1.2 million cases per year.
- DSCI's products are applicable to 300,000 to 500,000 cases which develop into serious conditions for which the treatment cost is $20,000 to $40,000.
- Its Advanced Wound Care Products are projected to have sales of $45 in 2014 and are growing at 30% per year justifying much of the current $280 million market capitalization.
- There is not much in the stock price for DSC-127, a potential blockbuster drug in phase 3 that will report data in mid-2015.
Refuting The Latest Negative Blog On Northwest Biotherapeutics
- There is no previously undisclosed new information in the risk factors section of NWBO's 2013 10-K.
- Progression free survival is a solid primary endpoint for DCVax-L phase 3.
- NWBO management states that it is confident that the design of the phase 3 trial of DCVax-L, if successful, is properly and robustly designed to achieve regulatory approval.
Chimerix: A Company Focused On Infectious Viral Diseases With Investment Characteristics Similar To Trius
- CMRX's brincidofovir is a novel anti-viral drug for preventing cytomegalovirus infections in hematopoeitc cell transplant patients.
- There is no drug approved for this indication.
- Cytomegalovirus infections can result in graft rejection, hospitilizations and death.
- I think that brincidofovir could gain approval in 2016 and my model projects potential sales of $800 million by 2021.
Analysis: Northwest Biotherapeutics DCVax-L Germany Approval Under Hospital Exemption Early Access Program
- DCVax-L gains approval in Germany through new (two years old) hospital exemption early access program.
- Approval is for all forms of glioma not just glioblastoma multiforme, both newly diagnosed and recurrent.
- This is first systemic drug approved through this new regulatory process. It is a great coup for management.
- Please read this report very carefully, this is a new and unprecedented investment situation.
- Agenus: Acquisition Of 4-Antibody Is A Transformational Move
- Cytokinetics: Institutional Investors Give BENEFIT-ALS Potential Results A Vote Of Confidence
- Derma Sciences: A Not Well Followed Growth Company With Solutions For Diabetic Foot Ulcers
- Cadence: What To Do With The Proceeds And A Post Mortem On A Successful Investment
- Cadence: Patent Settlement With Sandoz Increases Confidence That Ofirmev Will Have Exclusivity Through December 6, 2020
- Neuralstem: Sizing Up Potential Key Events Of 2014
- Agenus: Key Opinion Leader Support Builds For Prophage
- Highlights Of Recent PSivida Presentation
- Thoughts On The Price Surge Of Cytokinetics
- Senesco Offers A New Approach To Cancer Treatment And Dr. Phillip Frost Is Now With The Company
- Celldex: Some Thoughts On Rindopepimut
- PSivida: Game On
- Agenus: How Meaningful Is The Prophage Phase II Data?
- ImmunoCellular: Post Hoc Analysis Of Disappointing Phase II Trial Of ICT-107
- Northwest Biotherapeutics: An Analysis Of Its Transforming Balance Sheet Restructuring
- Repligen - My Projections For The Income Statement: 2013-2019