Archive for the 'Biotech' Category

Ebola Outbreak Raises Ethical Issues

The development of new treatments and preventives to combat the lethal Ebola virus has been slow, marked by caution at public health agencies to approve testing of high-risk compounds, and reluctance of biopharmaceutical companies to invest in a field with limited market potential. All that has changed now, as thousands of people have been sickened by the virus, and the death rate has escalated. The risk of harm from an untested treatment now is much less alarming than the prospect of infection, and the potential benefit of any effective treatment outweighs the need for regulatory caution.

Thus international and national authorities are working hard to facilitate access to experimental drugs and vaccines, despite a long history of distrust related to studying experimental treatments in Africa and other developing regions. Sponsors often face charges of using poor patients to test products that later will be unaffordable and unavailable. Authorities also question whether severely ill patients can make informed decisions about treatment options amid community fears of medical treatment. Vaccines raise added concerns, as they require widespread testing over long periods, often in healthy individuals and children. While the possibility of severe side effects from test drugs can generate a backlash against potential treatment, withholding therapy raises even more serious questions.

An important step was for the World Health Organization (WHO) to declare it ethical to use unproven therapies to try to contain the growing Ebola outbreak in West Africa. FDA similarly permitted a small biopharma company to distribute an experimental product that had been put on clinical hold due to concerns about a possible serious side effect.

These actions, though, raise similarly difficult questions about how to distribute the very limited supply of an experimental drug. Some experts cite the importance of treating children first. Others believe that patients with access to effective care are more likely to recover, making that a factor in using scarce medical resources.

Healthcare workers often get top priority, on the basis that doctors and nurses are needed to care for the sick, and that the prospect of treatment may encourage critical professionals to volunteer for service. But the treatment of two Westerners with the potentially effective drug Zmapp raised protests that the therapy was not available to sick Africans.

Data critical
One rationale for treating health professionals is that they are familiar with protocols and can understand the risks and the informed consent process. Treatment in a hospital setting also is more likely to support scientific assessment of an experimental regimen. One area of agreement is the importance of collecting and evaluating data on the safety and efficacy of experimental drugs used during the outbreak, and to share that data with all affected parties.

Researchers are continuing to assess factors related to the recovery of the two health workers who received Zmapp, acknowledging that they don’t know if that good result is are due to the drug, to effective care, or to other health factors. Treatment with this monoclonal antibody therapy produced by tiny Mapp Biopharmaceutical of San Diego may facilitate FDA approval of initial clinical trials, once the company can rebuild its exhausted supply.

Tekmira Pharmaceuticals of Canada may gain important safety information on its test product, TKM-Ebola, now that FDA converted a “full clinical hold” to a “partial hold” on its phase 1 trial, which permits the company to provide the drug to infected patients – but not to healthy volunteers — due to concerns about a potentially serious side effect.

While US and international authorities grapple with these ethical and logistical issues, manufacturers are struggling to ramp up production. Three centers in the US have capacity to quickly scale up production of a vaccine or therapy in response to an epidemic or biological threat and may be tapped to produce candidate vaccines.

A low-priority Ebola vaccine development project at GlaxoSmithKline’s Okairos unit now expects to move from preclinical development to clinical trials this year. Several small biotech firms are looking to gain needed funding to revive abandoned Ebola vaccine development programs. But optimistic statements from biopharma companies raise the risk of appearing “opportunistic” and  “self-promoting,” noted biotech commentator Adam Feuerstein in his “The Street” blog. A lethal outbreak of contagious disease provides an opportunity for biopharma companies to demonstrate their capacity to respond with innovative therapies, based on ethical and scientific factors, as opposed to the potential for financial gain.

by Jill Wechsler

Sandoz Wins Biosimilar Filing Race

After months of speculation about prospects for biosimilar development in the United States, Novartis announced on July 24 that FDA has accepted Sandoz’ biologics license application (BLA) for a similar version of Amgen’s Neupogen (filgrastim). Assuming FDA approval within a year, this action sets the stage for testing whether and how biosimilars will gain acceptance in the US healthcare market, and the impact these products will have on new drug development and product pricing and marketing.

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Clusters set to benefit from improved funding climate but IP rights are even more critical

Guest blog written by John Dean, partner and patent attorney at Withers & Rogers LLP.

Life sciences and biotech research clusters across the UK, including those linked to university research departments are benefiting from an improved funding climate and ongoing consolidation in the pharmaceutical sector. However, as big pharma companies increasingly vie for the best spin-out investment opportunities, those with strategic intellectual property plans are likely to find favour. Read more »

Myriad Ruling Will Undermine Global Biotech investment But All Is Not Lost

Guest blog written by Adrian Tombling, partner and patent attorney at Withers & Rogers LLP.

The US Supreme Court ruling in the so-called ‘Myriad case’ regarding the patentability of human genes will undermine global biotech investment but all is not lost. Judge Thomas, in Association for Molecular Pathology v. Myriad Genetics, effectively tightened the rules on the patentability of human genes, finding that all naturally occurring gene sequences, even when isolated in the laboratory, are not patentable. In future, only non-naturally occurring gene sequences (e.g., sequences that have been modified or entirely created in the laboratory), will be patentable, and therefore, attract investment from biotech companies. Read more »

Praise and Perils for Biotechnology Patent Policy

The Supreme Court decision blocking patents on naturally-occurring genes has generated predictions of doom for biotech innovation, along with expectations of more healthy competition in discovering new treatments and diagnostics. Although some commentators regarded the decision, Association for Molecular Pathology v. Myriad Genetics, as a “major reversal” in longstanding patent policy, many leaders of the biopharmaceutical research community described the ruling as likely to spur innovation and the development of therapies and companion diagnostics necessary for advances in personalized medicine.

All sides had legitimate reasons to claim victory:  patient and research groups anticipated easier access to more effective and efficient tests and therapies; biotech companies were relieved that the Justices did not invalidate ancillary patents surrounding genetic discoveries. A number of testing firms announced plans to develop newer, better, less costly tests using the BRCA genetic mutations. Myriad Genetics said its test for breast cancer would remain a leading option for patients and that it would continue its R&D program based on hundreds of other patents.

The ultimate outcome remains to be seen.  The U.S. Patent & Trademark Office (PTO) is expected to revise practices for granting patents for isolated DNA, although the court specifically permits protection for companies that modify genetic material or create new products based on human genes. But in upholding patents on complementary, or cDNA, the Court left the door open to further debate and legal battles over just what genetic manipulations qualify for patent protection.

Yet, there is reason to hope that the unanimous, non-partisan Court ruling will help clarify patent policy related to biotechnology, a subject that remains tangled up in multiple legal cases and legislative proposals.  And the non-political nature of this fairly technical ruling should provide grounds for optimism that the system can address some issues on the legal merits of the case.

Global Biosimilars Market to Reach $2.445 Billion in 2013

VLUU L110  / Samsung L110The global market for biosimilar drugs has been forecasted to be worth $2.445 billion this year, according to a new report by British market research specialist Visiongain. The growth represents a 20% increase from last year and accounts for approximately 2% of the overall biologics market. Moreover, the global biosimilars market is expected to experience a steady growth over the next 10 years, driven by worldwide launches of such products, particularly in the EU and US. Read more »

Adapting to Change

Evidence of health outcomes is what payers want to see. As a result, biotech companies are now focussing more on demonstrating economic value, for example, by showing survival benefit of a cancer drug or superiority to branded or generic competitors. Read more »

Overcoming the Challenges in Biopharmaceutical Stability Testing

Patricia Van Arnum PharmTech editorAs pharmaceutical and biopharmaceutical companies intensify product development in biologics, they are tasked with meeting the challenges of biologic-based drug development and manufacturing. Unlike traditional small-molecule drugs, stability studies for biopharmaceuticals can be one of the most critical and challenging aspects of large-molecule drug development. The size and complexity of most proteins provide fertile ground for intramolecular changes and multiple routes of degradation, and to assess their effects, each must be correlated to the bioactivity of the drug. Pharmaceutical Technology will examine biopharmaceutical stability studies in more depth by gaining input from leading industry experts, in a live webcast, “New Strategies for Biopharmaceutical Stability Testing,” on Thursday May 9th from 2:00 to 3:00 PM EST. Read more »

Biotech Needs to Focus on Demonstrating Product Value

VLUU L110  / Samsung L110Ernst & Young recently released its annual biotechnology industry report, Beyond Borders: Matters of Evidence, stating that while the major players are performing well, it is essential that small- to mid-size biotech companies focus on demonstrating the value of products in their pipelines instead of just creating a drug that works. If not, they will lose out in a challenging environment, especially now that there is a global shift towards evidence-based healthcare. Read more »

Big Pharma Leverages Early-Stage Risk with Innovative Options

In recent years, large pharmaceutical companies have launched a variety of initiatives to restock ailing pipelines and boost business performance including mergers and acquisitions, diversifying business portfolios to non-pharmaceutical products, downsizing, spinoffs, and entering the biopharmaceutical arena.

Whatever the approach, pharmaceutical companies want balanced portfolios with programs at various stages and risk profiles, says Melinda Richter, founder and CEO of San Francisco-based Prescience International in a BioPharm International podcast.

To date, most Big Pharma companies have partnered or acquired assets of biopharmaceutical companies with products in late-stage development, says Richter. However, as the availability of late-stage development opportunities shrink and the landscape becomes more competitive, Big Pharma is turning to more early-stage partnerships with academia and early-stage companies.

It is attractive to for the pharmas to go after early-stage companies because “by nature, they are smaller, they are nimbler, and they are willing to take the risks that the large pharmas just can’t. These small companies have to swing for the fences and they have to win. Pharmas have a lot to protect. They have to be more conservative,“ says Richter.

More scalable innovation opportunities are another part of the story, says Richter.

For example, last year, Merck announced a $90 million, seven-year commitment for the California Institute for Biomedical Research (Calibr), an independent, not-for-profit organization established to accelerate the translation of basic biomedical research to innovative new medicines.

However, for hands-on research, startup companies need laboratory and office space, as well as specialized equipment. Janssen Labs, located on the West Coast Research Center of Janssen Research & Development in La Jolla, Calif., offers short-term leases on wet laboratory and office space.  Tenants also have access to core research facilities and instruments.

The facility, operated by Prescience International, has a “no strings attached” policy. Janssen R&D does not take an equity stake or first right of refusal in the work of tenants, protecting the entrepreneurial rights of startup companies that choose independence.

Janssen Labs and Calibr are two options offered by Big Pharma that will be explored in the session “And Now for Something Completely Different: How Will Pharma Access External Early-Stage Innovation?” at the 2013 BIO International convention on April 23, 2013.


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