Innovative and improved drugs therapies developed through novel target discovery and technology platforms have originated from the backyards of small research focused companies (Rising Stars, RS, non-profitable biotech companies). The universe continues to grow despite the high risk and the wait to transform into a successful profitable Mature Biotech is long. 2012 saw new drugs approvals for unmet needs, first in class drugs for Anti-obesity, Myelofibrosis, cancers and better drug options for HCV, RA, MS, psoriasis, HCC, dyslipidemia etc. We expect approval of “better drugs for bad bugs”, targeted therapies for cancer, Alzheimer’s, antivirals and orphan diseases in the next five years.

Despite negative outcome of pivotal clinical trials or ruling by the FDA, no one (investors and large pharma/biotech companies) have given up on innovation which is reflected in the healthy cash position within the sector. Hope of resurrection will prevail in the next five years and successful commercialization of anti-obesity, dyslipidemia etc. could boost the RS sector. The expectation bar of investors for M&A is on the rise and with several RS companies accepting the challenge of marketing drugs on their own. Coming years will witness a mix of both- acquisitions as well as entering the Mature Biotech club. The race in innovations is killing newly launched novel drugs (ex. INCIVEK protease inhibitor for HCV) and patents do not offer protection from competition by better drugs in development. Diversified strategies will be essential both for the small and big players to mitigate these challenges as the life cycle of new drugs may be shorter than before in some therapy areas.

The New Avatar’s of Microbes is a growing threat world wide and we expect “GAINS” for companies focused in the anti-infective therapy class. “Pill vs. Prick”- is gaining popularity as new oral options are entering the market for RA, Psoriasis, MS etc. Pricing and experience of patients and physicians will drive compliance and define market opportunity of these drugs. Orphan disease focus will continue to remain attractive and success of Eteplirsen (PhII, Sarepta) for the treatment of Duchenne muscular dystrophy (DMD) and other antisense, RNAi drugs in the pipeline could see the final maturing of oligonucleotide based drug therapies.

Global Biopharmaceutical Outlook 2013 of the Rising Stars sector released by MP Advisors provides an in depth analyses of biotech companies and their drug pipeline amidst competition and dynamic regulatory environment. The report discusses the competitive landscape of the targeted therapy areas and the innovative strategy each company is deploying to surmount challenges and remain attractive for acquisitions, partnerships or march toward profitability. Sum of parts valuations of drugs in the pipeline are included after factoring clinical development risks and competition. Risk reward analyses of important upcoming milestones of the companies are included which should help fund managers as well as pharma company management to make critical decisions.