Recently, Bristol-Myers Squibb Co. (BMY) received approval from the U.S. Food and Drug Administration (FDA) to market Abilify (aripiprazole) for the treatment of irritability in pediatric patients having autistic disorder. Abilify can be used to treat children aged 6 to 17 years for symptoms of aggression toward others, deliberate self injury, temper tantrums and quick [...]

(BMY) Bristol-Myers Squibb Earns FDA Approval

Recently, Bristol-Myers Squibb Co. (BMY) received approval from the U.S. Food and Drug Administration (FDA) to market Abilify (aripiprazole) for the treatment of irritability in pediatric patients having autistic disorder. Abilify can be used to treat children aged 6 to 17 years for symptoms of aggression toward others, deliberate self injury, temper tantrums and quick mood swings. 

Bristol-Myers and Otsuka Pharmaceutical Co. jointly develop and distribute the drug in the U.S. and Europe. The FDA approval of Abilify for treating irritation in pediatric patients with autistic disorder is based on data from two eight-week late-stage studies in which Abilify significantly improved scores on the irritability subscale of the caregiver-rated Aberrant Behavior Checklist (ABC-I), compared to placebo. 

The most common adverse affects were sedation, fatigue, vomiting, somnolence, tremor and pyrexia. However, the efficacy of Abilify for the maintenance treatment of irritability associated with autistic disorder was not evaluated. Abilify, which is approved to treat depression, bipolar disorder, and schizophrenia, generated sales of $2,153 million in 2008, up 30% from 2007. Furthermore, sales in the third quarter of 2009 grew 16% to $653 million. 

Abilify has been growing at about five times the overall antipsychotic market. The company’s beefed-up sales force and new Direct to Consumer (DTC) campaigns are aiding sales growth. Abilify faces competition from other drugs in the market such as Merck’s (MRK) Saphris, Pfizer Inc.'s (PFE) Geodon, Johnson & Johnson's (JNJ) Risperdal, AstraZeneca PLC's (AZN) Seroquel and Eli Lilly & Co.'s (LLY) Zyprexa. 

Plavix, the antiplatelet blood thinner indicated to reduce the risk of heart attack in patients with atherosclerosis, is the top growth driver for Bristol. However, patent expirations loom large on the company starting 2011 when the Plavix patent expires. The company has lost patent protection on products worth about $4 billion in sales over the past four years. 

Drugs such as Cefzil, Paraplatin, Glucophage, Monopril and Taxol are also experiencing declining sales due to generic competition. However, the $1 billion addition in cost cuts in 2012-2013, the extension of the Abilify agreement with Otsuka, and the acquisition of Medarex indicate that management is taking meaningful steps to prepare for the loss of exclusivity of Plavix. Currently, we are Neutral on Bristol-Myers.
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The American Diabetes Association estimates that there are currently 23.6 million diabetics in the U.S. (7.8% of the total population, including 17.9 million diagnosed cases, 5.7 million undiagnosed, and 57 million pre-diabetics. Diabetes results in an estimated $174 billion in costs from diagnosed cases of diabetes in the U.S. (2007).[More...]

PPD, Inc. will acquire BioDuro, a preclinical CRO, as a follow-up to its purchase last month of Excel PharmaStudies, both of which are major transactions; we reflected on the huge difference between how China and the US go about creating “life science clusters”; China enrolled a record number of students in American universities during 2008-9; China Cord Blood listed on the NYSE and also completed a secondary offering; Advanced BioMedical signed an agreement in China to develop its bio-absorbable material in new applications;[More...]

On a global scale, the International Diabetes Federation (IDF) estimates 285 million diabetics worldwide in 2010. I have outlined some investing opportunities in this article, with a focus on emerging small / micro-cap companies from across the world which are developing innovative therapeutics, diagnostics, and monitoring products to improve the treatment outcomes for the growing, global diabetes epidemic.

Epinex Diagnostics, Inc. (privately held) is dedicated to the development of innovative point-of-care technologies using rapid diagnostic tests. Epinex has developed a proprietary platform for rapid tests that apply existing immunoassay strips and biosensors to create unique quantitative diagnostic tests, focused on a significant initial market opportunity for a new type of diabetes monitoring test that measures glycated albumin (G1a), with an expected FDA 510(k) filing in early 2010 seeking marketing clearance as a Class 2 medical device for an expected review period of 90 days. My 13-page profile report and the most recent corporate profile for Epinex are available to view or download at the BioMedReports.com research downloads section or at the ProActive News Room landing page for Epinex.

Novo Nordisk (NYSE: NVO) is based in Denmark and represents the largest (market cap of US$39.5 billion), globally diversified play on diabetes care, deriving over 73% of total revenue (US$5.6 million / US$7.6 million) during the first nine months of 2009 from this business segment. During the first three quarters of 2009, NVO posed total and diabetes care segment sales growth of 15% (Danish kroner) and 11% (local currencies).

NVO expects to receive formal feedback from the FDA during 4Q09 for its pending Victoza (liraglutide is a once-daily human GLP-1 analogue) New Drug Application (NDA) seeking U.S. marketing approval for the treatment of type 2 diabetes in adults. In early July, the European Commission granted marketing authorization for liraglutide. Victoza is used once-daily via subcutaneous injection, and the drug is a synthetic glucagon-like peptide-1 (GLP-1) that works by stimulating insulin release when glucose levels become high.

Another company awaiting a FDA decision for a new diabetes care product is MannKind Corp. (NASDAQ: MNKD), which submitted its NDA in mid-March that is seeking FDA approval of Afresa (insulin monomer human rDNA origin) Inhalation Powder and the AFRESA Inhaler for the treatment of adults with type 1 or type 2 diabetes. The FDA issued a standard, 10-month review for the NDA, with a decision expected in mid-January 2010. Afresa is an ultra rapid-acting form of insulin (achieving peak levels within 12-14 minutes to mimic the normal physiological release of insulin in healthy individuals) that is delivered through an inhaler rather than the typical route of injection by a syringe.

In early October, MNKD announced that it would not be able to sign a partnership deal for Afresa by year-end as it awaits the pending FDA decision, stating that the Company and potential partners will be better able to address appropriate deal terms and structure once the label for Afresa is clarified. In November 2007, Pfizer (NYSE: PFE) agreed to a settlement with Nektar Therapeutics (NASDAQ: NKTR) that included a one-time payment of $135 million to the latter after Exubera (the first FDA-approved inhaled insulin product) failed to generate meaningful sales despite a significant investment by Pfizer.

In early May, Amylin Pharma (NASDAQ: AMLN), Eli Lilly (NYSE: LLY), and Alkermes (NASDAQ: ALKS) announced that a NDA was filed seeking FDA approval of exenatide (Byetta LAR) as a once-weekly formulation for type 2 diabetes which is administered by subcutaneous injection, with a FDA decision expected in early 2010. Byetta is currently approved for use as a twice-daily injection for patients with Type 2 diabetes and is classified as an incretin that exhibits many of the same actions as GLP-1 (glucagon-like peptide-1) which act by stimulating insulin release when glucose levels become high.

Diabetes care companies in the Asia /Pacific region include Terumo Corp. (TYO: 4543) (OTC: TRUMY.PK) and Takeda Pharma (TYO: 4502) (OTC: TKPHY.PK). Terumo manufacturers medical devices / equipment such as insulin syringes while Takeda markets diabetes drug Actos with continued development of alogliptin as an experimental drug for Type 2 diabetes as both a single agent and in combination with Actos. In late June, Takeda received a Complete Response Letter (CRL) from the FDA for alogliptin, which is a selective dipeptidyl peptidase IV (DPP-4) inhibitor under investigation for the treatment of type 2 diabetes as an adjunct to diet and exercise that will require a cardiovascular safety study prior to possible FDA approval.

In September, Vivus Inc. (NASDAQ: VVUS) announced positive results from two final, Phase 3 pivotal 56-week studies, EQUIP (OB-302) and CONQUER (OB-303), evaluating the safety and efficacy of Qnexa (phentermine + topiramate) in more than 3,750 patients across 93 sites. The EQUIP and CONQUER studies met all primary endpoints by demonstrating statistically significant weight loss with all three doses of Qnexa, as compared to placebo. Patients taking Qnexa also achieved significant improvements in cardiovascular and metabolic risk factors including blood pressure, lipid levels, and Type 2 diabetes. Vivus expects to file a NDA before year-end and has an ongoing Phase 2 study evaluating Qnexa in Type 2 diabetics.

Access Pharma (OTC: ACCP.OB) has developed a nano-polymer drug delivery system for the oral administration of large molecules such as insulin, human growth hormone (hGH), and erythropoietin (EPO). This drug delivery technology involves coating a nano-particle with a B12 analog (cobalamin) that binds to intrinsic factor in the gut and triggers binding to cellular receptors which absorb the entire package, resulting in 1,000 to 1,000,000-fold increases in absorption through the gut of large molecule drugs typically administered by injection.

Licensing discussions are expected to formalize for the Company's basal (long-acting) oral insulin product following the mid-June announcement that two bio-pharmaceutical companies will conduct preclinical, animal studies before proceeding to more formal negotiations. The results of these preclinical studies will be made public, including one North American biotech company and one European biotech company. Access is providing the oral insulin while the two interested companies will conduct one animal study each (including a rat and dog model) with data expected before year-end.

Other investment opportunities related to diabetes care include (1) Unilife Medical Solutions (OTC: UNIFF.PK) (ASX: UNI.AX) (Unitract safety syringes, contract medical device manufacturing); (2) West Pharmaceutical Services (NYSE: WST) (syringe component manufacturer); (3) Retractable Technologies (AMEX: RVP) (VanishPoint safety syringes); (4) CPEX Pharma (NASDAQ: CPEX) (Nasulin is an experimental intranasal insulin product candidate); (5) Emisphere (OTC: EMIS.OB) (plans to change development focus to an oral formulation of a GLP-1 analog rather than continuing to pursue development of oral insulin).

The market for all diabetes monitoring products is the largest medical diagnostics market in the world at $12.4 billion in 2000 and estimated to grow to a level of $27 billion by 2010, representing a 12% CAGR (compound annual growth rate). The diabetes testing market includes diversified, global healthcare giants and smaller niche players, including those outlined below.

Johnson & Johnson (NYSE: JNJ) (LifeScan - One Touch), Abbott Labs (NYSE: ABT) (FreeStyle), Becton Dickinson (NYSE: BDX) (BD Diagnostics), Roche (OTC: RHHBY.PK) (Accu-Chek), Bayer (OTC: BAYRY.PK) (Ascenscia), Bio-Rad Labs (NYSE: BIO), Home Diagnostics (NASDAQ: HDIX), Insulet Corp. (NASDAQ: PODD), Echo Therapeutics (OTC: ECTE.OB), Trinity Biotech (NASDAQ: TRIB), and DexCom (NASDAQ: DXCM).

As I wrote last week, Echo Therapeutics announced positive results in a pilot clinical study for its needle-free Symphony Transdermal Continuous Glucose Monitoring (tCGM) System as a non-invasive, wireless, transdermal solution for monitoring blood sugar levels when used in conjunction with the needle-free Prelude SkinPrep System. The Prelude SkinPrep has been licensed to Ferndale Pharma in North America + UK for the enhanced delivery of its topical lidocaine skin-numbing agent (LMX4), with a FDA 510(k) submission expected during 1Q10 for potential marketing clearance while the Symphony tCGM will be evaluated in a pivotal trial that is expected to begin by late February 2010 with a FDA PMA submission expected to follow.

Coming soon is a premium service offered in collaboration with Investars YOU for full access to a global database of over 1,000 stocks which are organized and managed within my 23 ProActive HavRx stock indexes. This service will allow investors to create custom portfolios, personalized exchange-traded funds (ETFs), and generate investment ideas based on their personal preferences, beliefs, and opinions through specialized stock indexes that are organized and targeted toward a wide variety of themes.

The BioMedReports.com FDA Calendar service includes a database with over 400 entries of (1) pending new drug, biological agent, or medical device new product decisions at the FDA (e.g. NDA, BLA, 510k, PMA, sNDA, and sBLA filings); (2) pending new submissions to the FDA; (3) pending complete response letter (CRL) re-submissions to the FDA; and (4) pending clinical trial results.

Disclosure: Long ACCP.OB, Epinex Diagnostics (privately held)