Here’s a look at the drugs that may shape 2017, compliments of FirstWord Pharma. It’s a list of some of the products I agree are likely to dominate industry news over the next 12 months.Drugs that may shape 2017 and the trends molding new-drug discovery Click To Tweet
But equally, if not more, important are the trends in new-drug discovery that are shaping the next generation of treatments with genomics and other rarified technologies moving ever closer to the heart (no pun intended) of the academic/scientific and commercial push forward.
Dupixent (dupilumab) is an investigational human monoclonal antibody in development for the treatment of adult patients with inadequately controlled moderate-to-severe atopic dermatitis. In addition to being explored as a monotherapy for AD (atopic dermatitis), Regeneron and Sanofi are also researching Dupixent (dupilumab) for other indications. Dupixent is expected to enable Regeneron reduce its over-reliance on its Eylea’s revenues. Analysts suggest Dupixent will enable Regeneron to pose strong competition to other biotechnology players such as Biogen and United Therapeutics.
Last month, AstraZeneca and its global biologics research and development unit, MedImmune, announced that the Food and Drug Administration has accepted the first Biologics License Application (BLA) for durvalumab, a PD-L1 human monoclonal antibody (mAb), and granted priority review status with a Prescription Drug User Fee Act (PDUFA) set for the second quarter of 2017. The BLA submission, for the treatment of patients with locally advanced or metastatic urothelial carcinoma (UC) whose disease has progressed during or after one standard platinum‑based regimen, is based on the results of the UC cohort of Study 1108 and follows the FDA’s February 2016 “Breakthrough Therapy” designation for durvalumab.
Performance of the PD-(L)1 inhibitors will continue to attract investor attention in 2017, especially since Merck & Co.’s Keytruda is used increasingly in first-line non-small-cell lung cancer (NSCLC) patients.
GW Pharmaceuticals announced new Epidiolex positive Phase 3 data in Dravet Syndrome and Lennox-Gastaut syndrome (LGS). The British-based company found a significant difference between Epidiolex and the placebo in seizure frequency reduction during the 14-week period. Caregivers reported an improvement in overall condition and the drug was well tolerated.
GW pharma said, “In the LGS study, the drop seizure responder analysis showed a statistically significant separation between Epidiolex and placebo at the 50% seizure reduction threshold.”
Expectations for the drug remain high, and 2017 will see full presentation of previously top-lined data. GW pharma uses cannabidiol (derived from cannabis plants) in its drugs that are used to fight multiple sclerosis and a variety of rare epileptic diseases.
Roche’s new immuno-oncology drug Tecentriq is ready for its debut in lung cancer. The PD-L1 checkpoint inhibitor snagged FDA approval for patients who’ve failed on a previous treatment, putting it in head-to-head competition with Merck & Co.’s Keytruda and Bristol-Myers Squibb’s Opdivo. Tecentriq was already approved to treat bladder cancer, but the lung cancer arena is a much larger market. Though Keytruda and Opdivo have already made inroads, analysts see a genuine opportunity for Tecentriq, particularly after strong survival data recently presented at the European Society for Medical Oncology meeting in Copenhagen.
There is a rush of R&D activity these days that is uncovering alternative ways of manipulating the immune system; one approach is chimeric antigen receptor (CAR) T-cells. CAR T-cell therapy is a rapidly growing field that involves extracting a patient’s own T cells, expanding and modifying them ex vivo to recognize tumor associated antigens, before transferring back to the patient. Novartis, Juno Therapeutics and Kite Pharma are the key CAR-T players. But the flood of data updates is indicative of progress in this hot field. Kite Pharma and Novartis will file KTE-C19 (for aggressive B-cell non-Hodgkin lymphoma) and CTL019 (for pediatric and young adult patients with acute lymphoblastic leukemia), respectively, with the FDA in early 2017.
Acadia Pharmaceuticals’s Nuplazid drug, which treats hallucinations associated with Parkinson’s disease, generated third-quarter sales that more than doubled analysts’ estimates.
“In our view, both the greater-than-expected sales and positive sentiment from management support that the launch is progressing well,” JMP Securities analysts said in a note.
Wall Street has also cheered the results, with shares up sharply recently. The company’s Nuplazid treatment brought in $5.3 million in revenue during the period, easily topping consensus estimates of $2.6 million. All eyes are now focused on other trials, including Nuplazid’s potential benefit for Alzheimer’s agitation.
Manufacturing issues were to blame again in December for another delayed 2016 approval of Ocrevus as Roche announced its speedy FDA review was canceled as it must now wait another 3 months to late March for a green light. In a brief update the Swiss drug giant said the PDUFA (Prescription Drug User Fee Act) date for Ocrevus (ocrelizumab), which had been December 28, will now not be met after the agency demanded “additional data by Roche regarding the commercial manufacturing process of Ocrevus.”
Roche stressed the FDA had no concerns with safety or efficacy. The biologic has been submitted for the treatment of relapsing remitting MS, but is also set for approval as the first treatment of primary progressive MS, following the publication of positive Phase 3 data.
The UK’s National Institute for Health and Care Excellence (NICE) rejected Roche’s breast-cancer therapy Perjeta last year, saying its long-term benefits are “unclear.” The draft guidance suggests NICE wants more data on the benefits of adding Perjeta (pertuzumab) to treatment with chemotherapy or Roche’s Herceptin (trastuzumab)–particularly its effect on overall survival–in people with HER2-positive breast cancer. Perjeta is used in combination with Herceptin or docetaxel to reduce the volume of breast tumors before surgery, making it easier to carry out the operation.
A federal judge last week banned the sale of Regeneron Pharmaceuticals’ cholesterol-lowering drug Praluent, handing a surprise and significant triumph to Amgen and its competing drug Repatha in a long-running patent lawsuit. Amgen had already won the trial in which Regeneron was shown to have infringed on two patents covering a class of cholesterol-lowering drugs known as PCSK9 inhibitors. Regeneron markets Praluent with the French drugmaker Sanofi. Regeneron and Sanofi must stop selling Praluent in the US, although the ban has been delayed 30 days to allow the companies to appeal. Regeneron and Sanofi say they intend to appeal both the original patent infringement decision and the judge’s injunction against Praluent.
The Food and Drug Administration recently granted accelerated approval to Clovis Oncology’s ovarian cancer drug in patients with a specific gene mutation whose disease had advanced despite multiple rounds of chemotherapy. The drug, rubraca, which gained approval some two months earlier than scheduled, targets patients whose tumors have a mutation called BRCA–identifiable using an FDA-approved companion diagnostic test. The FDA also just approved a test made by Foundation Medicine Inc. to help detect the presence of BRCA mutations. If one or more mutations are detected, the patient qualifies for rubraca.
Rubraca is expected to compete with Tesaro’s niraparib and AstraZeneca Plc’s lynparza. Tesaro applied for US marketing last month after data released in October showed niraparib improved outcomes for all women with recurrent ovarian cancer. All three drugs belong to a closely watched class of new medicines called PARP inhibitors, which block enzymes involved in repairing damaged DNA, thereby staving off cancer cells.
The Food and Drug Administration in December announced its approval of Spinraza (nusinersen), making the Biogen drug the first for treating spinal muscular atrophy in adults and children. SMA is typified by a motor neuron loss, leading to progressive weakness and degeneration in the muscles. Those with the most severe type of the disease can eventually become paralyzed and have trouble performing basic life functions like swallowing and breathing.
“There has been a long-standing need for a treatment for spinal muscular atrophy…We could not be more pleased to have the first approved treatment for this debilitating disease,” said Dr. Billy Dunn, Division of Neurology Products director at the FDA’s Center for Drug Evaluation and Research.
Alzheimer’s remains the world’s most common form of dementia and is the sixth leading cause of death in the US. But a viable and safe drug remains hard to pin down. According to the Alzheimer’s Association, about 5.4 million Americans suffer from the disease. There may now be a new drug on the way to treat Alzheimer’s. A drug from Merck has just shown hopeful results from a small trial and has shown no side effects in participants. The drug, verubecestat, targets the protein plaques in the brains of Alzheimer’s patients that eventually lead to neurodegeneration.
Verubecestat works because it’s a BACE1 inhibitor–an enzyme essential to the production of deadly amyloid beta proteins, clumps of which form the plaques. By blocking the enzyme, the drug keeps proteins from forming or sufficiently reduces them to prevent the clumping.
Yes, new, exciting innovations in pharma are expensive, but they are saving lives and giving people hope based upon rational and reasonable scientific data. Like the Hep c breakthrough medicines; expensive? Yes. But the cost of previous treatment for years and years far exceeded the relatively few pills needed to eradicate the affliction in a matter of months.
Yet one reason so many people detest pharmaceutical companies is because while they get wealthier selling their products, mostly at the highest price possible, many people aren’t getting better says Luke Timmerman for Forbes.
Smoking is a good example. A doctor can prescribe a smoking-cessation product like Pfizer’s varenicline tartrate (Chantix) or GlaxoSmithKline’s bupropion hydrochloride (Zyban), then send the patient home. But clinical trials reveal that taking the medicine only goes so far for most smokers. While the big companies bring single-minded focus to selling their products, researchers and health insurers know that an integrated combination of drug therapy and behavioral therapy is far more effective at helping people kick the habit.
Timmerman cites one San Francisco Bay Area startup, Chrono Therapeutics, which employs an ingenious combination of drugs, digital sensors and human coaching into an integrated approach. If they can put the puzzle pieces together, Timmerman says, Chrono should do a better job of helping a person quit than just writing a prescription, sending them home and hoping for the best.
Next, the science of the human microbiome has taken biology by storm the past few years. (In a nutshell, the human microbiome–all of our microbes’ genes–can be considered a counterpart to our human genome–all of our genes. The genes in our microbiome outnumber genes in our human genome by 100:1.)
This captivation of the pharmaceutical sector is due in large part to DNA sequencing tools that are yielding an increasingly clear understanding of how we as humans coexist with the trillions of bacteria in our guts, and on our skin, Timmerman points out.
When we get along well with the bugs, we’re usually what people would consider healthy. But when something goes awry with a certain kind of bug or bugs, our immune systems can function erratically, attacking healthy tissue, for example, and causing autoimmune disease.
Some research suggests gut bacterial composition affects our mood, opening up interesting new ideas for treatment of depression and other forms of central nervous system disorders and mental illnesses. Exactly how biotech and pharma companies can gain the upper hand in this science and design wonder drugs based on it, is anyone’s guess.
Cambridge, MA-based Seres Therapeutics, a pioneer in the field, went bust last year with a microbiome-based treatment for C. difficile infections. But money continued to flow toward a variety of microbiome-based drug discovery efforts, such as Vendanta Biosciences, Synlogic, and Second Genome.
Timmerman points out that it’s we taxpayers who invest in basic biomedical research, largely through the National Institutes of Health. When a discovery occurs, universities sometimes get a patent, and Pharma businesses occasionally license these innovations for further development.
Traditionally, these raw discoveries need a mammoth amount of investment and labor before they can be forged into a functional product, like a bottle of sinus spray. Knowing this, big Pharma has often acquired intellectual property from universities for a modest cost to them (but not to the grateful school) then carried the ball through the clinical development and regulatory gauntlet all the way to the commercial marketplace.
But as universities have become more skilled at some of the early steps like basic manufacturing of biologic drugs and use of tools commonly employed by molecular biologists to deliver genetic material into cells for gene therapy, they’ve been able to push their innovative ideas further along that same commercial development path. As a result the schools have been getting much higher prices from Pharma for their nascent assets.
Harvard, for example, wrung a $20 million upfront payment from Merck last March for the right to develop a set of small molecule drugs for acute myeloid leukemia. Juno Therapeutics and Spark Therapeutics, prominent cancer immunotherapy and gene-therapy players, respectively, also had to pay dearly for their building-block intellectual property.
Of course the potential for conflicts of interest, and abuse of taxpayer resources, is always suspect in such deals. It’s also become common for the vast majority of these academic/Pharma deals to wind up in the mass media.
Still, academia will continue to hone its bargaining skills and cut more lucrative deals in 2017 and cause more hand wringing in Pharma and increasingly judicious media coverage.Steve's Take: Pharma is an insatiable furnace and we need more fuel (product) Click To Tweet
Bottom line is that Pharma is an insatiable furnace; we keep needing more fuel, namely, more product. We’re just scratching the surface of the mirror beneath which lie the myriad of cures mankind will discover, not some day in a galaxy far, far away, but already in progress right here, right now.
Which companies will lead the way and possibly augment your portfolio? Some of the likely ones are mentioned above.