Posted by Mike Havrilla on Fri, Jan 29, 2010 @ 08:57 AM
On 1/29/10, KERX announced that an article entitled "Clinical and Translational Studies of a Phase II Trial of the Novel Oral Akt Inhibitor Perifosine in Relapsed or Relapsed/Refractory Waldenstrom's Macroglobulinemia," reporting Phase 2 data demonstrating the single agent activity of KRX-0401 (Perifosine) for the treatment of advanced Waldenstrom's Macroglobulinemia will appear in the 2/1/10 issue of Clinical Cancer Research.
Perifosine, the Company's oral PI3K/Akt pathway inhibitor is currently being investigated in a Phase 3 trial, under Special Protocol Assessment (SPA), for the treatment of Advanced Multiple Myeloma. Similar to Multiple Myeloma and Non-Hodgkin's Lymphoma, Waldenstrom's is a hematologic disease in which the cancer cells target the bone marrow. There are currently no drugs FDA approved for the treatment of Waldenstrom's.
The progression-free survival (PFS) of 12.6 months is considered long compared to other targeted agents used in a similar population such as bortezomib (Velcade), where the median time to progression was reported at 7.9 months. KERX provided guidance for further clinical studies to evaluate perifosine for this disease, either as a single agent or in combination with agents such as Rituxan or Velcade.
Disclosure: Long KERX
Posted by Mike Havrilla on Wed, Jan 27, 2010 @ 08:38 AM
The
ProActive News Room website for Keryx Biopharma (NASDAQ: KERX) has been updated to include the Company's most recent corporate presentation and the GI-ASCO 2010 poster presentation from last weekend. In the randomized Phase 2 trial, perifosine + capecitabine (P-CAP) demonstrated a statistically significant improvement in TTP (time to progression) and OS (overall survival) compared to placebo + capecitabine (CAP) in patients with second or third-line mCRC (metastatic colorectal cancer). ORR (overall response rate) was also improved for P-CAP over CAP.
Perifosine is in-licensed by KERX from Aeterna Zentaris (NASDAQ: AEZS). During 2010, KERX expects to report updated data from its Phase 2 colon cancer study, conduct / update additional earlier stage Phase I/II studies, finalize late-stage trial protocol for colon cancer study, and begin a late-stage study for colon cancer patients in addition to the ongoing pivotal Phase 3 study for multiple myeloma under a Special Protocol Assessment (SPA) with the FDA. KERX is preparing to begin a pivotal Phase 3 trial of perifosine in patients with mCRC and an additional SPA with the FDA for this indication is expected within the next three months.
Below are some highlights from the GI-ASCO poster presentation:
1.) The improvements in TTP and OS with P-CAP compared to CAP are also seen in patients with 5-FU refractory disease. One patient with 5-FU refractory disease had a PR on P-CAP.
2.) P-CAP was well tolerated compared to CAP alone.
3.) A Phase I study to evaluate the combination of perifosine + capecitabine using a higher dose of capecitabine at 1000 mg/m2PO BID days 1 -14 is underway. PK of perifosine and capecitabine are being examined.
4.) Given the improved clinical efficacy of P-CAP compared to CAP in 5-FU refractory patients, a randomized Phase III study comparing P-CAP to CAP in patients with refractory mCRC is planned.
Below is a summary of additional expected catalysts and milestones for KERX:
On 12/16/09, KERX announced the initiation of a Phase 3 pivotal study of perifosine (KRX-0401), the Company's PI3K/Akt pathway inhibitor, in multiple myeloma patients. It is structured as a double-blind, placebo-controlled trial comparing the efficacy and safety of perifosine vs. placebo when combined with bortezomib (Velcade) and dexamethasone and will enroll 400 patients with relapsed or relapsed / refractory multiple myeloma. The primary endpoint is progression-free survival and secondary endpoints include overall response rate, overall survival and safety. Approximately 265 events (defined as disease progression or death) will trigger the un-blinding of the data. KERX expects a patient recruitment period of approximately 16-18 months and expects to report data from this study during 2H11.
On 1/5/10, KERX announced that it has reached agreement with the FDA regarding a SPA on the design of a Phase 3 clinical program for Zerenex (ferric citrate), its iron-based phosphate binder for the treatment of elevated serum phosphorous levels, or hyperphosphatemia, in patients with end-stage renal disease (ESRD). In accordance with the Company's SPA agreement with the FDA, the Phase 3 clinical program for Zerenex will consist of two clinical studies, including (1) a short-term efficacy study that is expected to commence by the end of 1Q10 with date expected during 2H10; and (2) a long-term safety and efficacy study that is expected to begin mid-2010 with data expected and a NDA filing expected during 1H12.
KERX also has a partnership in Japan with JT Torii that includes over $100 million in potential milestone payments. The ongoing Phase 2 study in Japan is near completion and KERX expects the Phase 3 trial to begin during 2H10, which will trigger a development milestone payment that is in the mid single digit million dollar range. An additional $15 million milestone is possible upon regulatory approval in Japan, with approximately $55 million in remaining milestone payments based on the achievement of sales targets upon commercialization.
Disclosure: Long KERX
Posted by Mike Havrilla on Sun, Jan 24, 2010 @ 02:48 PM
Perifosine is in-licensed by KERX from Aeterna Zentaris (AEZS). During 2010, KERX expects to report updated data from its Phase 2 colon cancer study, conduct / update additional earlier stage Phase I/II studies, finalize late-stage trial protocol for colon cancer study, and begin a late-stage study for colon cancer patients in addition to the ongoing pivotal Phase 3 study of for multiple myeloma.
KERX is preparing to begin a pivotal Phase 3 trial of perifosine in colon cancer patients with a Special Protocol Assessment (SPA) with the FDA possible for this indication within the next 2-3 months.
On 1/24/10, KERX presented updated results at GI-ASCO for a randomized Phase 2 study of perifosine in combination with capecitabine (P-CAP) versus capecitabine plus placebo (CAP) in patients with second- or third-line metastatic colon cancer.
For refractory 5-FU patients (P-CAP vs. CAP), median TTP (time to progression) is 18 vs. 10.3 weeks (p = 0.0188) while OS (overall survival) is 24.3 vs. 11.7 mo (p = 0.0346). Median OS for P-CAP vs. CAP is 24.3 vs. 16.3 mo (p = 0.1348) or 49% benefit on P-CAP. Most frequent G1/2 AE's (grade 1 / 2 adverse events) (P-CAP vs. CAP): diarrhea (65% vs. 29%), nausea (45% vs. 29%), fatigue (45% vs. 35%), hand/foot syndrome (25% vs. 24%). Most frequent G3/4 AE's: hand/foot syndrome (25% vs. 0%), anemia (15% vs. 0%), fatigue (0% vs. 12%).
Disclosure: Long KERX
Posted by Mike Havrilla on Thu, Jan 21, 2010 @ 09:21 AM
Perifosine is in-licensed by Keryx from Aeterna Zentaris (AEZS). Approximately 265 events (defined as disease progression or death) will trigger the un-blinding of the data. During 2010, KERX expects to report updated data from its Phase 2 colon cancer study, conduct / update additional earlier stage Phase I/II studies, finalize late-stage trial protocol for colon cancer study, and begin additional late-stage studies for colon cancer in addition to the pending pivotal Phase 3 study of perifosine under a SPA agreement with FDA for multiple myeloma.
KERX is preparing to begin a pivotal Phase 3 trial of perifosine in colon cancer patients with a Special Protocol Assessment (SPA) with the FDA possible for this indication within the next 2-3 months (i.e. mid-April 2010). On 1/24/10, KERX is scheduled to present updated results at GI-ASCO from a randomized Phase 2 study of perifosine in combination with capecitabine (P-CAP) versus capecitabine plus placebo (CAP) in patients with second- or third-line metastatic colon cancer.
Disclosure: Long KERX
Posted by Mike Havrilla on Sat, Jan 16, 2010 @ 01:16 PM
Below are some updates and developments from the past week, which I spent in San Francisco for the OneMed, JP Morgan, and other healthcare conferences.
I had the opportunity to meet with the CEO (Ron Bentsur) and CFO (James Oliviero) of Keryx Biopharma (NASDAQ: KERX) on Wednesday and was impressed with the Company's focused strategy and upcoming milestones for 2010 and beyond. As outlined in my overview article last week, I have purchased shares of KERX over the past week on weakness at a slightly higher price range ($2.70-2.75) than I originally projected.
Given the Company's history of in-licensing compounds for development and robust cash position, I inquired if anything was on the horizon in this regard. However, for the near to intermediate term, Keryx plans to remain focused on utilizing its resources (cash / personnel) to get the Phase 3 trials well established before considering any new acquisitions, which is a good strategy given that Keryx is well-funded but still a small-cap drug developer that needs to remain focused.
Approximately $10-11 million is the estimated cost to fund both the short and long-term Phase 3 studies of Zerenex while perifosine has estimated funding of $12 million each to fund the Phase 3 studies for multiple myeloma (ongoing) and colorectal cancer (pending), with an estimate of $25,000-$30,000 per patient to fund the Phase 3 cancer trials. Below is a summary of additional details for upcoming catalysts and milestones for Keryx over this year and beyond that were not included in my overview article last week.
During 2010, Keryx expects to report updated data from its Phase 2 colon cancer study, conduct / update additional earlier stage Phase I/II studies, finalize late-stage trial protocol for colon cancer study, and begin additional late-stage studies for colon cancer in addition to the pending pivotal Phase 3 study of perifosine under a SPA agreement with FDA for multiple myeloma. KERX expects to provide an update on Phase 2 colon cancer survival and other data for perifosine next weekend at GI-ASCO 2010 (Jan. 22-24) as it prepares to begin a pivotal Phase 3 trial of perifosine in colon cancer patients with a Special Protocol Assessment (SPA) with the FDA possible for this indication within the next 2-3 months (i.e. mid-April 2010).
The ongoing Zerenex Phase 2 study in Japan by JT Torii is near completion and Keryx expects the Phase 3 trial to begin during 2H10, which will trigger a development milestone payment that is in the mid single-digit million dollar range. An additional $15 million milestone is possible upon regulatory approval in Japan, with approximately $55 million in remaining milestone payments based on the achievement of sales targets upon commercialization.
Zerenex represents a niche market opportunity within well understood / established class of drugs (phosphate binders) that has a key differentiation of being iron-based compared to existing treatments such as PhosLo, Fosrenol, and Renagel/Renvela. Zerenex is expected to offer an improved safety profile / patient acceptance with reduced GI / bloating side effects and approximately 50% less dosing burden (i.e. taking less pills less often) compared to existing phosphate binders.
In addition, a major secondary endpoint being evaluated is the potential ability of Zerenex to decrease the need for IV-iron and blood cell stimulators (EPO agents such as Epogen and Aranesp) since it is iron based and expected to result in long-term accumulation of the mineral. This may result in a first-line phosphate binder indication (with a goal of capturing 20-25% market share) for Zerenex and advantages that include safety (there is a pending FDA Advisory Panel to discuss safety issues of EPO drugs) and economic (dialysis bundling payment issues - Zerenx has potential to save dialysis providers money by reducing need for IV-iron or EPO agents).
On 1/13/10, Cell Therapeutics (NASDAQ: CTIC) announced an agreement to raise $30 million through the sale of preferred stock and warrants. The deal included approximately 24.7 million shares of common stock and warrants to purchase 8.6 million shares of common stock at an exercise price of $1.18 per share. Interesting to note that shares of CTIC were traded up nearly 15% during the day on heavy volume prior to news of the financing, which also comes ahead of an upcoming FDA Advisory Panel meeting to review the Company's NDA for pixantrone.
On 6/24/09, CTIC announced that it completed the submission of the New Drug Application (NDA) to the FDA for pixantrone to treat relapsed or refractory, aggressive non-Hodgkin's lymphoma (NHL). On 5/5/09, CTIC announced that pixantrone is available on a named-patient basis for use in Europe to treat patients with aggressive NHL that has either relapsed or is refractory to standard treatment options. On 9/5/09, CTIC announced that the FDA established a PDUFA action date of 4/23/10 (representing a standard, 10-month review designation) for pixantrone as a potential treatment for relapsed or refractory aggressive non-Hodgkin's lymphoma (NHL).
Auxilium Pharma (NASDAQ: AUXL) provided guidance over the past week that it expects an FDA decision soon for Xiaflex (collagenase clostridium histolyticum), which is a novel, first-in-class, orphan-designated, biological agent for the treatment of Dupuytren's contracture (this is a progressive condition that affects the connective tissue that lies beneath the skin in the palm, resulting in collagen deposits which impair normal hand function).
AUXL filed its Biologics License Application (BLA) for Xiaflex for the treatment of Dupuytren's contracture on 2/27/09 and the FDA accepted the BLA filing with a priority review (six-month) designation on 4/28/09. On 12/16/09 AUXL announced Phase 2b results for Xiaflex for Peyronie's disease (a new indication that is not part of the pending BLA) and stated that a FDA decision is still pending for Xiaflex. On 9/16/09, the FDA Panel voted 12-0 in favor of Xiaflex to treat Dupuytren's disease.
On 7/17/09, Cytori Therapeutics (NASDAQ: CYTX) announced that the FDA will regulate the Company's Celution 700 System as a medical device, reviewed by the Agency's Center for Biologics and any required clinical studies would be conducted in accordance with the Investigational Device Exemption (IDE) regulations rather than the Investigational New Drug (IND) regulations applicable to new drugs / biologics.
On 1/7/10, the FDA granted marketing clearance for the PureGraft System, which will be launched as the first and only device in the US cleared for aesthetic body contouring and allows a patient's own fat tissue (autologous) to rapidly be prepared in about 15 minutes for re-injection and aesthetic contouring. Cytori expects to launch PureGraft in the US during 1Q10 with a formal launch at the American Society of Aesthetic Plastic Surgeons in May 2010. Marketing approval (CE Mark) is pending in Europe and expected during 1H10.
Cytori expects to receive ongoing FDA feedback in 90-day cycles to expand upon the initial marketing clearance announced last week to include specific therapeutic claims (e.g. wound healing) with the next feedback from the Agency anticipated to occur by early April. The other major looming catalysts for Cytori include pending clinical trial data from Europe for two heart trials, which are outlined in detail in my article from last year that includes promising interim results from the two studies in addition to an embedded CEO video interview and product demo for the Celution System.
Disclosure: Long CYTX, KERX