S&S On Biotech

To continue listening to podcast on biotech, pharma, and medtech visit our new channel: biobizbuzz.com .  When the first chimeric antigen receptor T-cell (CAR-T) therapy was approved in 2017, there was a general expectation that T-cell receptor T-cell (TCR-T) therapies would follow shortly afterwards and would greatly expand the range of addressable antigens. Despite considerable efforts, CAR-T th...

To continue listening to podcast on biotech, pharma, and medtech visit our new channel: biobizbuzz.com . In 1934, Mary Walker, a pioneering Scottish physician, successfully, albeit transiently, treated a myasthenia gravis patient with physostigmine, a traditional remedy for treating poisoning with curare. She had noticed that the signs and symptoms of myasthenia gravis resembled those caused by cu...

To continue listening to podcast on biotech, pharma, and medtech visit our new channel: biobizbuzz.com . Melanoma has been at the very centre of the cancer immunotherapy revolution over the past decade and a half. The CTLA-4 inhibitor Yervoy (ipilimumab), which gained approval in 2011, was the first agent to demonstrate a survival improvement in a phase 3 trial for metastatic melanoma. It was also...

To continue listening to podcast on biotech, pharma, and medtech visit our new channel: biobizbuzz.com . This is a sobering episode that traces the history of the treatment of brain cancer – principally malignant glioma, since it is the most common diagnosis in a thankfully rare oncology indication – and how, unlike many of the indications we have discussed, the treatment options have been limited...

To continue listening to podcast on biotech, pharma, and medtech visit our new channel: biobizbuzz.com . Ophthalmology has long been a fruitful area for biotechnology innovation. In highly prevalent conditions associated with ageing, such as age-related macular degeneration, or with chronic disease, such as diabetic macular oedema, vascular endothelial growth factor inhibitors have made important...

To continue listening to podcast on biotech, pharma, and medtech visit our new channel: biobizbuzz.com . For all the extraordinary progress we have seen in basic biological research and in the development of advanced therapies, allergy is an area that seems to be stuck and badly in need of innovation. For some, an allergy may be little worse than an irritant, but for those with severe allergies to...

A couple of years ago, PROTACs were ubiquitous. Start-ups were devising increasingly clever ways of making them, while large pharma companies were queuing up to tap into their expertise and their development programs. Proteolysis-targeting chimeras, to give them their full title, emerged as a clever and versatile way of degrading, with exquisite selectivity, disease-associated proteins that were h...

To continue listening to podcast on biotech, pharma, and medtech visit our new channel: biobizbuzz.com . Over 10% of the world’s population – or 850 million people – are estimated to have kidney disease, and the problem is growing rapidly. The vast majority lives in low-income or low-and-middle-income countries, and, without access to primary healthcare, may be unaware of the problem until it is t...

To continue listening to podcast on biotech, pharma, and medtech visit our new channel: biobizbuzz.com . Human longevity is a precious thing. Some people receive a tragically short amount of it. Others who have it in abundance want even more of it – and a reasonable chunk of the biopharmaceutical industry has organised itself around the goal of delivering it to them.  Age-associated diseases encom...

Therapeutic cancer vaccines have had a long and chequered history. The BCG (Bacillus Calmette-Guérin) vaccine, originally developed to protect against tuberculosis in the 1920s, was adopted back in the 1970s as treatment for bladder cancer. When delivered locally to the organ, it elicits an anti-tumor immune response. Progress in recent decades has been painfully slow, however. Only a clutch of th...

The flow of money is a constant preoccupation of the biotechnology sector, because without it nothing gets done. So, whether the markets are up or down, and whether investors are buying or selling is important, even if it is secondary to the actual business of biotech, which is the discovery and development of therapies that offer improvements over what is currently available. Investor sentiment i...

The failure of Relyvrio in a post-approval phase 3 study in amyotrophic lateral sclerosis (ALS) means its days as a commercial drug are probably numbered. It initially gained approval in Canada in June 2022 (where it is, confusingly, known as Albrioza) and secured FDA approval the following September. Both approvals rested on the 24-week phase 2 ‘Centaur’ trial, in which the drug showed modest eff...

A 40% share price drop after an investigational drug hits the primary endpoint of a phase 3 trial is not typical. But that’s what happened to Ironwood Pharmaceuticals when it reported data from a trial of apraglutide in patients with intestinal failure due to short bowel syndrome (SBS-IF).  The condition arises from the loss of gut function following surgical removal of a large portion of the smal...

By late June or early July we’ll know whether Alnylam’s decision to alter the design of its phase 3 ‘Healios B’ study of Amvuttra (vutrisiran) in transthyretin amyloidosis with cardiomyopathy (ATTR-CM) was a good one. The short-interfering RNA (siRNA) drug is already approved in ATTR with polyneuropathy (ATTR-PN), a debilitating, fatal condition in which misfolded transthyretin proteins clump toge...

Novartis’s recent €2.7 billion ($2.9 billion) offer for Morphosys is the very stuff of biotechnology. The medium-sized deal adds two clinical-stage cancer drug candidates to its pipeline.  Both operate through epigenetic mechanisms – they aim to alter dysfunctional gene expression programs and thereby interfere with the signals that maintain cancer growth. Pelabresib, a potential first-in-class in...

Hereditary angioedema (HAE), arguably, represents an ideal case of the Orphan Drug rules in action. Because of the incentives on offer, a mini-cluster of firms has, over the past fifteen years, developed a well-stocked cabinet of therapies for this rare disease.  The condition is linked to deficiencies in C1 esterase inhibitor activity and results in dysregulation of the kinin-kallikrein pathway,...

Non-alcoholic steatohepatitis (NASH) and its precursor non-alcoholic fatty liver disease (NAFLD) have recently been redesignated as MASH and MASLD – metabolic dysfunction-associated steatohepatitis and metabolic dysfunction-associated steatotic liver disease, respectively. What hasn’t changed is the sheer difficulty of establishing clinical efficacy in these indications. They have long been known...

Sanofi’s partial acquisition of Inhibrx recalls earlier transactions, such as Pfizer’s acquisition of BioHaven or, to go back even further, Johnson & Johnson’s acquisition of Actelion, in which assets deemed surplus to the acquirer’s needs were spun out into new entities. In the case of Inhibrx, Sanofi wanted just one programme, INBRX-101, its investigational drug for alpha1-anti-trypsin defic...

The use of radiation in medicine began with the work of the great pioneers Marie Curie, her husband Pierre, and Henri Becquerel. In the 1940s, Samuel Hertz, Samuel Seidlin, and William Beierwaltes established radioactive iodine (I-131) as a treatment for overactive thyroid and thyroid cancer.  The effects of radiation are not cell-specific – it is inherently damaging to all cells, so the focus of...

The annual JP Morgan Healthcare Conference, held in San Francisco every January, marks the start of the biotech industry’s new year. It’s the time when biotechnology companies of all shapes and sizes set out their stall for the year ahead, hoping to attract the attention – and the dollars – of big pharma.  It’s the ideal time to take the temperature of the industry. And right now, it appears to be...

In its own words, the U.S. Federal Trade Commission’s opposition to a licensing deal between Sanofi and the biotechnology firm Maze Therapeutics, “is a case about a monopolist seeking to eliminate a nascent threat to its monopoly”.   Cue howls of complaint by VC’s and public market investors that government scrutiny is preventing innovation. True enough, acquisitions, mergers and licensing transac...

Have antibody-drug conjugates peaked—or is there more innovation to come? Antibody-drug conjugates (ADCs) are complex products, and it has taken the field some time to iron out some of the difficulties that hampered their initial development.  The basic idea is simple—by attaching a cytotoxic payload to an antibody that targets a protein expressed (ideally uniquely) on the surface of cancer cells,...

The UK approval of the CRISPR-Cas9 gene editing therapy Casgevy (exagamglogene autotemcel or ‘exa-cel’) for treating the inherited blood disorders sickle cell disease (SCD) or transfusion-dependent b-thalassemia is an historic scientific and clinical milestone. Coming barely more than a decade after the technology was invented, it represents a major achievement for its developers Crispr Therapeuti...

When they first emerged, chimeric antigen receptor T-cell (CAR-T) therapies seemed like the stuff of science fiction. The basic idea of reprogramming T-cells is decades old. It involves isolating a patient’s T-cells, genetically modifying them to express a new receptor that recognizes a target protein, such as an antigen expressed by a cancer cell, and putting them back into the body, where they c...

When I was a global product manager in big pharma, my US counterpart mentioned to me one day that “we do generics properly in the US, not like you do in Europe.” He meant that when a small molecule went generic in the US, and the 180-day first-to-file exclusivity expired, the former branded price was massacred. It was true, in the first year SmithKline’s Tagamet went generic, they lost 80% of sale...