Below is a summary of updates to the BioMedReports.com database of 239 entries included in the FDA and Clinical Trial Calendars.
The FDA Calendar includes companies with pending new drug, biological agent, or medical device new product decisions at the FDA sorted by their PDUFA deadline dates. The entries are updated on a daily basis as new information becomes available with a total of 112 entries through 4/6/09. The FDA Calendar includes the following information: company name, ticker, decision date, and description.
The Clinical Trial Calendar includes 127 entries through 4/6/09 and encompasses pending clinical trial results (with a focus on late-stage, Phase 3 trials), pending new submissions to the FDA (e.g. NDA, BLA, 510k, PMA, sNDA, sBLA filings), and pending re-submissions to the FDA for complete response rulings by the agency which require more information before an approval can be granted.
1.) Theravance (THRX). The FDA accepted Theravance's NDA today for Telavancin as a once-daily injectable antibiotic in the treatment of hospital-acquired pneumonia (HAP), including MRSA strains of antibiotic-resistant and other Gram-positive bacteria. The FDA has established a goal of a standard 10-month review of the telavancin NDA, resulting in PDUFA decision date deadline of 11/26/09 and triggering a milestone payment of $10M from THRX partner, Astellas Pharma (ALPMF.PK).
This is the second telavancin NDA filed with the FDA, which is currently reviewing telavancin for the treatment of complicated skin and skin structure infections (cSSSI), for which a Complete Response Letter (CRL) was received on 2/26/09. The CRL requires a Risk Evaluation and Mitigation Strategy (REMS), data on patients with certain renal risk factors from the cSSSI and HAP studies, revisions to the draft label, and a safety update.
2.) Gilead Sciences (GILD) and Teva Pharma (TEVA): GILD recieved notice today of a Paragraph IV challenge by Teva as part of an Abbreviated New Drug Application (ANDA) filing to the FDA requesting permission to manufacture and market a generic version of HIV drug Atripla (a three-drug combination in a single tablet).
Gilead is currently reviewing the notice letter and has 45 days from the date of receipt to commence a patent infringement lawsuit against Teva. Such a lawsuit would restrict the FDA from approving Teva's ANDA for up to 30 months or until a district court decision that is adverse to Gilead, whichever occurs first. Atripla is currently protected by 15 patents, which are listed in the FDA's Approved Drugs Products List and all 15 patents would need to be invalidated or expired before a generic version of Atripla could be marketed.
3.) Alnylam Pharma (ALNY), a leading RNA interference (RNAi) therapeutics company, today announced that it has initiated a Phase 1 human clinical trial of ALN-VSP to evaluate its safety and pharmacological parameters in patients with advanced liver cancers, including hepatocellular carcinoma and other solid tumors with liver involvement.
ALN-VSP is an RNAi therapeutic comprised of two small interfering RNAs (or siRNAs, the molecules that mediate RNAi) formulated in a lipid nanoparticle and designed to target two genes which are crucial to the growth and development of cancer cells. Pre-clinical data in mice tumor models have demonstrated robust efficacy of ALN-VSP, including suppression of targeted genes, demonstration of an RNAi mechanism of action, tumor reduction, and extension of survival.
4.) ReNeuron Group PLC (UK: RENE.L) (US: RNUGF.PK) announced positive preclinical data today for its CTX stem cell line in the treatment of peripheral artery disease (PAD), which is a chronic and progressive condition that restricts the flow of blood in the limbs.
The Company's CTX stem cell line was injected IM into mice with restricted blood supply in their limbs and the cells resulted in a significant recovery of blood flow and tissue perfusion. The results of this study will be presented later this week at the UK National Stem Cell Network Conference.
ReNeuron plans to continue gathering preclinical data in an effort to begin clinical studies within the next two years. The Company will also begin a clinical trial for disabled stroke patients using the same CTX stem cell line as the basis for ReN001 therapy for this indication since receiving UK regulatory approval to conduct the trial earlier this year in January.
ReNeuron is one of the 40 companies included in the ETF Innovators Global Stem Cell and Regenerative Medicine index, featured along with other healthcare stock indexes at BioMedReports.com at http://biomedreports.com/health-indexes.html.
Cell Therapeutics (CTIC) is a micro-cap cancer biotech which is down substantially in the past year in terms of stock price and market value, but not out after recently establishing a joint venture with Spectrum Pharma (SPPI) to market Zevalin, the potential for two FDA approvals this year (one new product, one label expansion), the potential for EMEA approval of Opaxio in the E.U., and a goal to become cash flow break-even by year-end.
A World Health Organization report released late last year Cell Therapeutics (CTIC) is a micro-cap cancer biotech which is down substantially in the past year in terms of stock price and market value, but not out after recently establishing a joint venture with Spectrum Pharma (SPPI) to market Zevalin, the potential for two FDA approvals this year (one new product, one label expansion), the potential for EMEA approval of Opaxio in the E.U., and a goal to become cash flow break-even by year-end.
A World Health Organization report released late last year estimates that cancer will overtake heart disease as the top cause of death in the world by 2010, which is part of an overall trend that predicts global cancer cases and deaths will more than double by 2030. The report predicts 27 million new cases of cancer will occur in 2030, which is a sharp increase from the 12 million cases of cancer in 2007.
Near-term events for CTIC include a special meeting for shareholders scheduled for 2/6/09 to obtain approval to increase the number of authorized shares of common stock (from 400M to 800M), conduct a reverse split (between 1:2-1:20), and increase the number of shares authorized for equity incentive + employee stock purchase plans - with the first two proposals aimed at regaining compliance for continued listing on the Nasdaq Capital Market by a deadline of 2/12/09. CTIC will also be presenting at the 11th Annual BIO CEO & Investor Conference on Tuesday, February 10th at 9:30 AM in the Jade Room of the Waldorf-Astoria Hotel in New York City - click here for a link to the webcast for this event.
CTIC and SPPI have an upcoming PDUFA decision date (one of 14 extreme FDA trades, including companies with market caps below $100M with pending regulatory decisions) of 4/2/09 with a priority review designation for their sBLA to expand the label of Zevalin (Ibritumomab Tiuxetan) as consolidation therapy after remission induction in previously untreated patients with follicular non-Hodgkin's lymphoma (NHL). Zevalin is currently approved for the treatment of relapsed or refractory low-grade or follicular B-cell NHL, which is now being marketed by the 50/50 joint venture between CTIC and SPPI called RIT Oncology.
CTIC received $15M in cash along with a 50% interest as part of the agreement with SPPI to form the joint venture to further develop and market Zevalin. CTIC is also eligible to receive an additional $15M in milestone payments based on achieving sales targets for the drug while doubling the size of the sales and marketing team behind the drug. There is also an option for CTIC to sell its 50% interest in RIT Oncology to SPPI for $18M at any time from 1/15/09 to 7/15/09, which is an important feature of the deal since CTIC has indicated that it will require additional financing during 2009 and the low stock price precludes equity-based financings.
In late January, CTIC announced that its plans to begin submission of a rolling NDA for pixantrone (BBR-2778) during 1Q09 for the treatment of relapsed, aggressive NHL - with FDA approval possible before year-end if a priority review is granted by the agency. There is a high likelihood of a priority review for this indication since it represents an unmet medical need with no currently approved drugs on the market. Last November, CTIC reported Phase 3 clinical trial results for pixantrone, which achieved its primary and secondary efficacy endpoints under a Special Protocol Assessment (SPA) approval by the FDA for the study.
Patients randomized to receive pixantrone in the study achieved an 11% (8 of 70) rate of confirmed complete remissions, as compared to no patients (out of 70) who were randomized to receive standard chemotherapy. Pixantrone also achieved a higher overall response rate of 37.1% (26 of 70) compared to patients received standard chemo at 14.3% (10 of 70). Patients receiving pixantrone also demonstrated a statistically significant improvement in progression-free survival compared to standard chemo (4.7 months versus 2.6 months). The FDA approval and launch of pixantrone could result in upfront + milestone payments from Novartis (NVS) if NVS chooses to exercise its exclusive, worldwide license to commercialize pixantrone.
Pixantrone is less damaging to the heart compared to the widely used and effective class of chemo drugs called anthracyclines and has demonstrated synergistic activity when combined with Rituxan and other treatments. Pixantrone also offers advantages in the way it is delivered (via regular IV infusion), compared to other agents which require pre-medication and central lines to administer.
Opaxio (paclitaxel poliglumex, CT-2103, formerly Xyotax) has a pending marketing authorization application (MAA) to the European Medicines Agency (EMEA) which was filed in March 2008 for the first-line treatment of patients with non-small cell lung cancer (NSCLC) (with performance status 2) as part of an exclusive licensing deal with NVS. Opaxio is designed to reduce the side effects of paclitaxel by linking it to a biodegradable polymer to create a new chemical entity which results in targeted delivery to solid tumors such as breast, ovarian, and lung cancers.
Clinical studies suggest that the metabolism of Opaxio by lung cancer cells may be influenced by estrogen, resulting in higher levels of the drug in women with lung cancer and the potential for increased efficacy. The MAA was filed based on non-inferiority or equivalent effectiveness + an improved safety profile for the treatment of NSCLC while the U.S. regulatory strategy will seek approval for maintenance treatment of ovarian cancer following complete remission after first-line treatment.
Brostallicin is currently in Phase 2 clinical development as a new type of synthetic cancer drug (a DNA minor groove binding agent) which has the potential to be a highly selective cancer treatment which is synergistic with standard chemo drugs and newer targeted therapies. CTIC owns the exclusive, worldwide rights to brostallicin and has completed six clinical trials for the drug, with results for three of the trials still pending.
While shares of CTIC have declined sharply in the past year, 2009 holds the promise for better days with the potential for three regulatory approvals (Zevalin sBLA, Pixantrone NDA, and Opaxio MAA), the potential for increased Zevalin sales from the RTI Oncology joint venture, the option to sell its stake in the joint venture to SPPI for $18M through mid-July, and the goal of becoming cash flow break-even by year-end.
EXACT Sciences (EXAS) provides a useful comparison of a micro-cap cancer diagnostics company which traded as low as 22 cents late last year and now trades around $1.50 thanks to a hostile takeover bid by Sequenom (SQNM) and a subsequent licensing deal with Genzyme (GENZ) which included a $24.5M cash infusion and an extensive collaboration for product development and marketing.
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