News

News2018-12-03T23:50:27+00:00

2017

Allan B. Haberman, Ph.D. was one of about 25 experts from pharmaceutical, biotechnology, and consulting firms who attended Aptuit’s one-day think-tank event, ”Improving Candidate Selection: Translating Molecules into Medicines”. This was the third and final such networking and discussion symposium, which was held in downtown Boston. The previous two events in this series had been held in San Francisco (18th & 19th Sept 2017) and in Hertfordshire, UK (22nd & 23rd Oct 2017).

The focus of the meeting was on improving drug candidate selection in order to improve development success. Only about 10% of drug candidates make their way from first-in-humans trials to regulatory approval. The greatest amount of attrition occurs in Phase 2. Approximately half of candidates fail at that stage, mainly due to lack of efficacy.

For more information about this event, see our blog article.

Cambridge Healthtech Institute (CHI) announced the publication of a new book-length report, Cancer Immunotherapy: Building on Initial Successes to Improve Clinical Outcomes, by Allan B. Haberman, Ph.D.

The new 2017 report includes an updated discussion of approved and clinical stage agents in immuno-oncology. It also addresses the means by which researchers and companies are attempting to build on prior achievements in immuno-oncology to achieve improved outcomes for more patients. This approach is often referred to as “immuno-oncology 2.0.” The American Society of Clinical Oncology (ASCO) named “immunotherapy 2.0” as its “Advance of the Year” for 2017.

As discussed in the report, researchers have found that checkpoint inhibitors such as pembrolizumab (Merck’s Keytruda) and nivolumab (Bristol-Myers Squibb’s Opdivo) produce tumor responses by reactivating TILs (tumor infiltrating lymphocytes). As a result, researchers have been developing biomarkers that distinguish inflamed (i.e. TIL-containing) tumors—which are susceptible to checkpoint inhibitor therapy—from “cold” tumors, which are not. They have also been working to develop means to render “cold” tumors inflamed, via treatment with various conventional therapies and/or development of novel agents. These studies are the major theme of immuno-oncology 2.0. Meanwhile, cellular immunotherapy has also been advancing, with two chimeric antigen receptor (CAR) T-cell therapies (from Novartis and Kite Pharma) in preregistration with the FDA as of March 2017.

These and other areas of current cancer immunotherapy R&D are discussed in the new report.

The report is designed to enable readers to understand current and future developments in immuno-oncology. It is also designed to inform the decisions of leaders in companies and in academic groups that are working in areas that relate to cancer R&D and treatment.

For more information on the report, or to order it, see the CHI Insight Pharma Reports website.

2016

Allan B. Haberman, Ph.D. was quoted in an article in the October 2016 issue of Nature Biotechnology, entitled “Early clinical data raise the bar for hemophilia gene therapies”, by Elie Dolgin.

The article is an update on progress in development of gene therapies for hemophilia A and B, based in part on presentations at the World Federation of Hemophilia 2016 World Congress (Orlando, FL, July 24-28).

Notably, the article included discussions of progress in development of gene therapies for hemophilia A, especially by BioMarin Pharmaceuticals (San Rafael, CA). Commercial development of gene therapies for hemophilia B is far ahead of that for hemophilia A. This is because the gene coding for factor IX (mutated in hemophilia B) is smaller and easier to insert into commonly used gene-therapy vectors than the factor VIII gene mutated in hemophilia A. Nevertheless, BioMarin’s reported promising results from the Phase 1/2 trial of its hemophilia A gene therapy at the July 2016 meeting.

Haberman Associates has produced two publications that address gene therapy for hemophilia:

Because progress in gene therapy for hemophilia B is far ahead of that for hemophilia A, these materials by necessity emphasize hemophilia B. However, Chapter 5 of our book-length report includes strategies by which researchers and companies are now developing gene therapies for hemophilia A.

The Nature Biotechnology article also includes a discussion of a novel experimental treatment for hemophilia A that is not a gene therapy: Roche/Chugai’s emicizumab, a bispecific factor VIII mimetic. This promising, but early-stage product (Phase 1/2) must be given to patients periodically—perhaps monthly. In contrast, a successful gene therapy may be active over the life of the patient, or at least over many years. Nevertheless, the half-life of emicizumab is much longer than that of current treatments.

As we say in the article, the data on these therapies is early-stage, and it is not known how long the therapeutic effects are going to last. However, the results are promising, and at least some commentators believe that hemophilia could prove to be the most competitive gene therapy race to date.

Read the full Nature Biotechnology article. (Subscription required).

2015

Cambridge Healthtech Institute (CHI) announced the publication of a new book-length report, Gene Therapy: Moving Toward Commercialization, by Allan B. Haberman, Ph.D.

This report looks at how researchers have been working to overcome critical barriers to development of safe and efficacious gene therapy, from 1990 to 2015. It then focuses on clinical-stage gene therapy programs that are aimed at commercialization, and the companies that are carrying out these programs. A major theme of the report is whether gene therapy can attain commercial success by the early-to-mid 2020s, which types of gene therapy programs have the greatest likelihood of success, and what hurdles might stand in the way of clinical and commercial success of gene therapy development.

Topics covered in the report:

  • Development of improved vectors (integrating and non-integrating vectors)
  • Gene therapy for ophthalmological diseases
  • Gene therapy for hemophilias and other rare diseases
  • Gene therapy for more common diseases (e.g., Parkinson’s disease, osteoarthritis, and heart failure)
  • Companies whose central technology platform involves ex vivo gene therapy
  • Gene editing technology
  • Outlook for gene therapy
  • Outlook for eight gene therapy products expected to reach the market before 2020

The report also includes:

  • An expert interview with Sam Wadsworth, Ph.D., the Chief Scientific Officer of Dimension Therapeutics and former Head of Gene Therapy R&D at Genzyme
  • Survey data from 88 researchers involved in gene therapy
  • Companies profiled: uniQure, Spark Therapeutics, GenSight, Dimension Therapeutics, Voyager Therapeutics, Oxford BioMedica, GeneQuine, bluebird, Juno Therapeutics, Kite Pharma, Editas, and others.

For more information on the report, or to order it, see the CHI Insight Pharma Reports website.

2014

Cambridge Healthtech Institute (CHI) announced the publication of a new book-length report, Cancer Immunotherapy: Immune Checkpoint Inhibitors, Cancer Vaccines, and Adoptive T-cell Therapies, by Allan B. Haberman, Ph.D.

This report focuses on the three principal types of therapeutics that have become the major focuses of research and development in immuno-oncology in recent years:

  • Checkpoint inhibitors
  • Therapeutic anticancer vaccines
  • Adoptive cellular immunotherapy

The discussions of these three types of therapeutics are coupled with an in-depth introduction and history as well as data for market outlook.

Also featured are three expert interviews and a survey of individuals working in immuno-oncology R&D, which focuses on market outlook, and portrays industry opinions and perspectives.

The report is designed to enable readers to understand current and future developments in immuno-oncology. It is also designed to inform the decisions of leaders in companies and in academic groups that are working in areas that relate to cancer R&D and treatment.

For more information on the report, or to order it, see the CHI Insight Pharma Reports website.

2013

Allan B. Haberman, Ph.D. was quoted in an article in The Pink Sheet entitled “Cystic Fibrosis Market Snapshot: Disease-Modifying Drugs Elusive 24 Years After Discovery Of Root Cause”. The article focused on the newly-approved disease modifying drug ivacaftor (Vertex’ Kalydeco), as well as drug discovery and development programs in cystic fibrosis at Vertex, PTC Therapeutics, Proteostasis Therapeutics, Pfizer, and Genzyme. It also discussed pipeline products aimed at treating or preventing life-threatening infections in cystic fibrosis patients at such companies as KaloBios, Insmed, and Savara.

Read full article.  (subscription required)

2012

Allan B. Haberman, Ph.D. was quoted in an article in Chemical & Engineering News (C&EN) by senior editor Lisa M Jarvis. The article is entitled “Agios Takes A Long View In Cell Metabolism.”

The article focuses on Agios Pharmaceuticals’ (Cambridge, MA) strategy for building a platform company that can endure as an independent firm over a long period of time, and that can also demonstrate sustained performance. This contrasts with the recent trend toward “virtual biotech companies”–lean companies, which are designed for early acquisition by a Big Pharma or large biotech company. Agios’ goal is to dominate the field of cancer metabolism, and to discover and develop innovative drugs for both cancer and rare metabolic diseases based on its technology platform. Read full article.

For a commentary on issues raised by this C&EN article, see our Biopharmconsortium Blog.

Informa’s Scrip Insights announced the publication of a new book-length report, Advances in the Discovery of Protein-Protein Interaction Modulators, by Allan B. Haberman, Ph.D.

Protein-protein interactions (PPIs) are of central importance in biochemical pathways, including pathways involved in disease processes. However, PPIs have been considered the prototypical “undruggable” or “challenging” targets. The discovery of small-molecule drugs that can serve as antagonists or agonists of PPIs, and which are capable of being successfully taken into human clinical trials, has been extremely difficult. Nevertheless, over the last twenty years, researchers have developed a set of technologies and strategies that have enabled them, in a several cases, to discover developable small-molecule PPI modulators. One direct PPI agonist, the thrombopoietin mimetic eltrombopag (Ligand/GlaxoSmithKline’s Promacta/Revolade), has reached the market. Several other small-molecule PPI modulators are in clinical trials. Despite this progress, the discovery and development of small-molecule PPI modulators has been one-at-a-time, slow and laborious.

This report discusses technologies and strategies that enable the discovery of drugs targeting PPIs, including both small-molecule and synthetic peptide modulators. It includes case studies on the discovery of compounds that address specific target classes, with emphasis on agents that have reached human clinical studies. This includes addressing the issue of the need to produce PPI modulatory agents that have pharmacological properties that will enable them to be good clinical candidates.

The report also includes discussions of second-generation technologies for the discovery of small-molecule and peptidic PPI modulators, which have been developed by such companies as Forma, Ensemble, and Aileron, and by academic laboratories. In part as the result of the development of these technologies, and of the increasing strategic importance of PPI modulator development, Big Pharma companies have been moving into the field. Examples include Bristol-Myers Squibb, Pfizer, Novartis, and Roche. A key issue is to what extent the new technologies for PPI modulator R&D will enable this area to be commercially successful, and to meet the strategic needs of the industry for expanding the universe of targets for which drugs can be developed.

Download the free report.

2011

Allan B. Haberman, Ph.D. led a pre-conference workshop, entitled “Developing Improved Animal Models in Oncology and CNS Diseases to Increase Drug Discovery and Development Capabilities” at Hanson Wade’s World Drug Targets Summit (July 20-21, 2011, Cambridge, MA).

The workshop was a discussion of four case studies involving development of novel animal models in oncology and CNS diseases (one zebrafish cancer model, two mouse cancer models, and one mouse autism model), aimed at more closely modeling human disease than current models. Drug discovery and development in these therapeutic areas has been severely hampered by animal models that are poorly predictive of efficacy. This is a major cause of clinical attrition.

We discussed the implications of these case studies for developing novel therapeutic strategies, target identification and validation, drug discovery, preclinical studies, and reducing clinical attrition.

Cambridge Healthtech Institute (CHI) announced the publication of a new book-length report, Multitargeted Therapies: Promiscuous Drugs and Combination Therapies, by Allan B. Haberman, Ph.D.

In the past 20 years, pharmaceutical and biotechnology industry R&D has been increasingly aimed at developing drugs to treat complex diseases. However, the one drug-one target-one disease paradigm that has become dominant in the post-genomic era has proven to be inadequate to address complex diseases, which have multiple “causes”. Moreover, researchers have found that most of the successful, FDA-approved small-molecule drugs that were developed prior to the year 2000 are promiscuous, i.e., they are single drugs that address multiple targets. The great majority of kinase inhibitors, one of the most successful drug classes of the early 21st century, are also promiscuous.

Meanwhile, the development of targeted drugs such as kinase inhibitors and monoclonal antibodies has resulted in the need to develop multitargeted combination therapies. This has been especially true in cancer, where disease causation may involve multiple signaling pathways. In particular, the development of resistance to targeted antitumor drugs has spawned the need to develop second-generation treatments, many of which are multitargeted combination therapies.

This new Insight Pharma Report covers both discovery and design of small-molecule promiscuous/multitargeted drugs, and of multitargeted combination therapies.

For more information on the report, or to order it, see the CHI Insight Pharma Reports website.

2010

Allan B. Haberman, Ph.D. was quoted in an article entitled “Bristol-Myers Squibb reaps biologics in ZymoGenetics windfall”, in the November 2010 issue of Nature Biotechnology. The article focused on the acquisition of ZymoGenetics (Seattle, WA) by Bristol-Myers Squibb (BMS), with a special focus on ZymoGenetics’ hepatitis C drug pegylated interferon-lambda.

Read full article here.  (subscription required)

Cambridge Healthtech Institute (CHI) announced the publication of a new book-length report, RNAi Therapeutics: Second Generation Candidates Build Momentum, by Allan B. Haberman, Ph.D. This new Insight Pharma Report examines the science behind therapeutic RNAi (RNA interference) and miRNA (microRNA), technologies for design of therapeutic oligonucleotides that work via an RNAi or miRNA-modulating mechanism, technologies for design of delivery vehicles, and leading specialty companies in the therapeutic RNAi/miRNA industry sector.

The report also includes a discussion of the outlook for the therapeutic RNAi/miRNA industry sector, including strategic issues such as technological prematurity and the development of enabling technologies, the role of Big Pharma investment, and the impact of patent litigation and cross-licensing in shaping the RNAi/miRNA sector. It also includes a scenario for the development of RNAi and miRNA-based drugs. Also included are transcripts of interviews with five leaders of biotech companies in the RNAi/miRNA industry sector.

For more information on the report, or to order it, see the CHI Insight Pharma Reports website.

Haberman Associates’ Biopharmconsortium Blog, formerly at http://biopharmconsortium.blogspot.com, has moved to our own site. You can now find it at https://biopharmconsortium.com/blog/.

We started the Biopharmconsortium Blog in July of 2009. The blog contains expert commentary on recent life science industry news and recent published scientific reports and business articles.

We hope that you will continue to visit and subscribe to our blog in its new location. We also welcome comments and discussions of our blog articles from people in the life science community, including companies, academic institutions, and disease organizations and patient advocates.

Reducing Attrition In Silico” by Allan B. Haberman, Ph.D. was published in the March-April 2010 issue of Bio-IT World. This article is based on an excerpt from Chapter 8 of the recently published book-length report Animal Models for Therapeutic Strategies.

Cambridge Healthtech Institute (CHI) announced the publication of a new book-length report, Animal Models for Therapeutic Strategies, by Allan B. Haberman, Ph.D. This new Insight Pharma Report discusses the use of animal models to develop new paradigms for drug discovery and development in important human diseases. The report also discusses strategies for developing more predictive models of drug efficacy. Poorly predictive animal models are a major reason for Phase II and Phase III pipeline drug attrition. Thus this new report complements our May 2009 Insight Pharma Report, Approaches to Reducing Phase II Attrition.

For more information on the report, or to order it, see the CHI Insight Pharma Reports website.