An Industry Update: December 2019, What is New in the Field of Therapeutic Delivery This month?

Keywords:

• competitor intelligence
• intellectual property
• nasal
• parenteral delivery
• partnering

Company launch

Sumitomo Dainippon completes strategic alliance with Roivant & launches Sumitovant Biopharma

The year 2019 came to a close with the announcement on 30 December 2019 that Sumitomo Dainippon Pharma Co., Ltd (Tokyo/Osaka, Japan) had completed its strategic alliance with Roivant Sciences GmbH (Basel, Switzerland), resulting in the formation of a subsidiary; Sumitovant Biopharma Ltd (NY, USA) that is fully owned by Sumitomo Dainippon [1]. The deal involved Sumitomo Dainippon acquiring Roivant’s stake in five of its subsidiaries, the rights to two business-related technologies and taking an 11% stake in the Swiss-based company in exchange for $3 billion (USD). The newly formed Sumitovant Biopharma will act as the parent company to the five former Roivant firms namely, Myovant Sciences (Basel, Switzerland), Urovant Sciences (CA, USA), Enzyvant Therapeutics (Basel, Switzerland), Altavant Sciences (NC, USA) and Spirovant Sciences Ltd (PA, USA) [2]. Roivant has a ‘hub and spoke’ decentralized structure [3] and therefore each of these subsidiaries is focused on a particular therapeutic area. Myovant develops treatments for prostate cancer and women’s health, Urovant is focused on the therapy of an over-active bladder in men and pain in irritable bowel syndrome, Enzyvant Therapeutics has a T-cell generation platform to treat severe immunodeficiency and is investigating an enzyme replacement therapy for dysregulation of ceramide metabolism. The company has a Biologics Licence Application under review by the US FDA for its novel regenerative medicine; RVT-802, for the treatment of pediatric congenital athymia. Altavant Sciences’ pipeline is centered on tryptophan hydroxylase inhibitors for the treatment of pulmonary conditions, such as pulmonary arterial hypertension, and Spirovant’s development efforts are on the gene therapy of cystic fibrosis and other lung conditions [2,3].

The two technologies, which formed part of the deal, are DrugOme technology, which through computational analytics and other techniques identifies high-value assets and facilitates clinical development and commercial strategy and Digital Innovation, which is used to improve business decision-making. Both these technologies are now under the control of Sumitovant Biopharma, but both Sumitomo Dainippon and Roivant have access to them as part of the agreement [1].

The agreement also provides Sumitomo Dainippon with options to purchase Roivant’s ownership stake in six additional biopharma companies within Roivant’s portfolio.

Acquisitions

Sanofi strengths its immuno-oncology pipeline with Synthorx purchase

On 9 December came the news that Sanofi (Paris, France) would acquire Synthorx Inc. (CA, USA) for approximately $2.5 billion (USD) [4]. The California company has a novel Expanded Genetic Alphabet drug discovery platform based on new DNA base pairs, such as d5SICSTP (X) and dNaMTP (Y), which can be replicated, transcribed and then translated in vivo [5]. The technology employs a semi-synthetic organism, based on an established Escherichia coli (E. coli) strain, to incorporate nonnatural amino acids into proteins, in order to modify their biological activity and create new entities with therapeutic promise. It can even be used to incorporate molecules, such as polyethylene glycol, to extend protein half-life.

These proprietary proteins are known as Synthorins™. The modified E. coli strain expresses a nucleotide transporter that maintains the integrity of the genetic code containing the novel base pairs for the production of the engineered mRNA and tRNA. The X and Y nucleotides fed to these bacterial cultures accumulate within the cells. On induction of transcription, the cell expresses the Synthorin mRNA containing noncanonical codons and the tRNAs to decode them. Modified tRNA synthetase enzymes recognize and present the Synthorin tRNAs with the correct novel amino acids which have been added to the medium. Translation then occurs as normal with the nonnatural amino acids being incorporated into the new protein in a site-specific manner. The process is scalable and has been used by the company to develop a number of product candidates.

Synthorx’s lead compound, THOR-707 (‘not-alpha’ IL-2), is being evaluated for the treatment of a number of solid tumors, both alone and in combination with immune checkpoint inhibitors and other immuno-oncology drugs.

The acquisition fits well and expands Sanofi’s existing portfolio in the field of immuno-oncology. It is hoped that the addition of the Synthorx platform to Sanofi will be synergistic with its existing internal technologies, including the Nanobody^® technology for the production of single chain antibody fragments, which it acquired through its purchase of Ablynx in 2018 [6]. Also, it is hoped that this will enable the company to develop optimal treatments for cancer and other indications. As a result of the agreement, Synthorx becomes a fully owned subsidiary of Sanofi.

Astellas acquires Xyphos Biosciences

On 26 December 2019 Astellas Pharma Inc. (Tokyo, Japan) and Xyphos Biopharma Inc. (CA, USA) made public that Astellas had purchased Xyphos in a transaction worth up to $665 million (USD) including $120 million at closing, with the remainder dependent on the achievement of development milestones [7].

Xyphos has a proprietary technology platform called Advanced Cellular Control through Engineered Ligands for the development of novel cancer immunotherapies [8], which fits with Astellas’ internal expertise in this area. This technology seeks to address some of the disadvantages of the current chimeric antigen receptor T-cell (CAR-T) therapy, such as the targeting to only one single antigen, its association with life-threatening events for example cytokine storm and neurotoxicity and the development of antidrug antibodies, which ultimately limit treatment effectiveness.

The Xyphos convertible CAR technology is based on the fact that NKG2D receptors can interact with eight proteins of the MHC-class I-like complex (MIC). NKG2D receptors are found on the surface of natural killer (NK) cells, T cells and some macrophages but are seldom present on healthy cells. However, this situation changes under certain stress conditions when one or more of these MIC ligands for NKG2D can be expressed on the cell surfaces, forming the binding site for the NKG2D receptor through their α1-α2 domain. The resultant binding between the NKG2D receptors on NK cells and these ligands set off a chain of events that results in the death of the stressed cells.

The Xyphos technology exploits the NKG2D system by creating mutant ligands that selectively bind to proprietary, genetically engineered, CAR-T cells. The latter express a mutant of the native NKG2D receptor (iNKG2D), which cannot bind with natural ligands because of the aforementioned receptor. Human antibodies are fused to the modified ligands to target them to specific cells such as cancer cells. This resulting fusion product (a MicAbody), is therefore bispecific and can bind both to the iNKG2D expressed on the engineered T cell and the targeted cancer cell.

Activation of cancer cell destruction only occurs when both cells bearing the iNKG2D receptor and the MicAbodys are present. Control of the therapeutic effects and minimization of potential side effects, such as a cytokine storm are managed through dose titration of the MicAbodys. The system also allows the targeting of multiple targets through selection of the antibody portion of the fusion product, without having to submit patients to further CAR-T therapy. This is because the iNK2D-T-cell component binds to all MicAbodys. The components of the convertible CAR technology are also human-derived, which minimizes the risk of development of antibodies against the system. The company is also working on bispecific molecules known as MicAdaptor molecules to further modulate the activity of the convertible CAR-T cells, for example through the delivery of interleukins.

Xyphos’s proprietary bispecific molecules can be delivered to both natural immune cells or the CAR-T cells to enable immuno-oncology therapy. The lead product candidate, targeting the CD20 surface antigen for the treatment of relapsed or refractory non-Hodgkin lymphoma, is at the preclinical development stage with the first study in humans planned for 2021.

Licensing agreements

Neurocrine obtains rights to Xenon’s investigational treatment for epilepsy

At the start of the month Neurocrine Biosciences Inc. (CA, USA) and Xenon Pharmaceuticals Inc. (BC, Canada) announced that they had entered into a licensing and collaboration agreement for Xenon’s first-in-class, selective Nav1.6 sodium channel inhibitor; XEN901, under development for the treatment of epilepsy [9]. As a result, Neurocrine obtained exclusive rights to XEN901, which has demonstrated promise in preclinical models as a therapeutic agent for the treatment of SCN8A developmental and epileptic encephalopathy (SCN8A-DEE) in children and potentially other indications, such as focal epilepsy in adults [10]. SCN8A-DEE is caused by mutations in the SCN8A gene that result in a gain-of-function in the Nav1.6 sodium channel. Seizures typically start between birth and 18 months of age, with many sufferers having multiple attacks per day that are difficult to control. This rare condition is also associated with many other severe symptoms, including learning difficulties, developmental delay, muscle spasm and physical disability. In addition, patients may be at risk of sudden unexpected death in epilepsy.

Xenon has already conducted a Phase I clinical trial of XEN901 in healthy adult volunteers in Canada, with a powder-in-capsule formulation that demonstrated good safety and tolerability [11]. They have also developed a granule formulation and initiated toxicology studies to support trials studying children. It is expected that an Investigational New Drug application will be filed with the US FDA in mid-2020.

The license between Neurocrine and Xenon also extends to other preclinical compounds within the Canadian company’s pipeline, including selective Nav1.6 inhibitors and dual Nav1.2/1.6 inhibitors. The deal also foresees a 3-year research collaboration focused on the discovery and development of further Nav1.6 and Nav1.2/1.6 inhibitors.

Financial details of the agreement include a $50 million (USD) payment to Xenon, split between $30 million (USD) upfront in cash and a $20 million (USD) equity investment based on a Xenon share price of $14.196, a further $25 million (USD) on FDA acceptance of the Investigational New Drug for XEN901 and potentially up to $1.7 billion (USD) in milestone payments across all licensed products, in addition to royalties on commercialized products. Under the terms of the agreement Neurocrine will meet development and clinical costs with respect to the licensed compounds and fund the research collaboration, with Xenon being responsible for the preclinical work and some near-term manufacturing costs. However, Xenon can also elect to co-fund 50% of the cost of developing XEN901 or another candidate for the US market in return for an increase in the percentage of royalty payments, if the product is commercialized.

Denovo & Sunesis reach an agreement on vosaroxin

On 6th December 2019 Denovo Biopharma LLC (CA, USA) made public that it has licensed world-wide rights for the development, production and commercialization of vosaroxin; a first-in-class quinolone derivative, nonanthracycline topoisomerase II inhibitor, which Sunesis Pharmaceuticals (CA, USA) has been evaluating in Phase III studies for the treatment of acute myeloid leukemia (AML) and solid tumors [12]. So far, the drug has been evaluated in 16 clinical studies and demonstrated an acceptable safety profile but variable efficacy. However, its administration in the Phase III VALOR trial, involving 711 patients, resulted in significant improvements in overall survival and relapse rates in certain sub-groups with very poor prognosis. These include cancer sufferers over 60 years old with relapsed and refractory AML. Patients in this age group with newly diagnosed AML or those with relapsed or refractory disease have a survival prognosis of less than 1 year, therefore more effective treatments are urgently needed.

Denovo plan to employ their biomarker technology platform to uncover the genomic biomarkers in archived patient clinical samples, which correlate with positive clinical outcomes of vosaroxin. This is in order to guide its further development for AML and possibly other oncology indications.

Vosaroxin is the fifth compound to be licensed by Denovo to date. Its clinical profile fits with the company’s strategy of gaining the rights to late stage assets that have failed in larger Phase III trials. The company intend to use their technology platform to identify the biomarkers associated with success and employ this information to design smaller biomarker-informed studies in the patient population most likely to benefit from treatment [13].

Financial details of the agreement were not disclosed.

Jazz pharmaceuticals licenses lurbinectedin from PharmaMar

PharmaMar S.A. (Madrid, Spain) and Jazz Pharmaceuticals plc. (Dublin, Ireland) announced on 19 December 2019 that Jazz Pharmaceuticals Ireland Ltd had obtained an exclusive license to PharmaMar’s lurbinectedin in the US market [14]. PharmaMar retains manufacturing rights and will supply Jazz with the product.

A new drug application for lurbinectedin for the treatment of relapsed small-cell lung cancer, was submitted to the FDA in the same month. Lurbinectedin is a selective inhibitor of RNA polymerase II; an essential enzyme for the transcription process that is up-regulated in certain tumors. In addition, lurbinectedin inhibits oncogenic transcription in tumor-associated macrophages and reduces cytokine levels on which tumor growth is dependent [15]. Submission is based on the positive outcome of a Phase II trial involving 105 patients with relapsed small-cell lung cancer on monotherapy, which demonstrated that lurbinectedin had an overall response rate of 35.2% based on investigator assessment compared with 16.9% based on historical data with topotecan. Product approval and launch of this drug, which has orphan drug designation in the US, is anticipated in 2020.

The deal is potentially worth over $1 billion (USD) to the Spanish firm, split between an upfront payment of $200 million (USD), an additional $250 million (USD) if the FDA accelerated and/or full regulatory approval of lurbinectedin is achieved within certain timelines and a further $550 million (USD) contingent on potential commercial milestones. PharmaMar may also receive up to 30% royalties on future net sales plus additional payments if lurbinectedin is approved for other indications [14].

Product approvals

FDA approves Padcev™ for advanced urothelial cancer

Astellas Pharma Inc. (Tokyo, Japan) and Seattle Genetics Inc. (CA, USA) received an early Christmas present on 18 December 2019 in the form of FDA-accelerated approval of Padcev™ (enfortumab vedotin-ejfv), their jointly developed treatment for urothelial cancers [16,17]. Approval was received approximately 3 months ahead of the target date.

Padcev is a first-in-class antibody directed microtubule disrupting conjugate targeting nectin-4, a cell adhesion molecule, which is found in high numbers on urothelial cancer cells. Urothelial cells form the lining of the bladder, nearby organs and 90% of all bladder cancers can be traced back to cancerous changes in these cells.

The conjugate is composed of a human anti-nectin-4 IgG1 kappa monoclonal antibody (AGS-22C3) conjugated to the anticancer drug, monomethyl auristatin E, through a maleimidocaproyl valine-citrulline linker (SGD-1006). The linker is capable of being cleaved by proteases in the body [18]. The drug-to-antibody ratio is approximately 3.8:1. It is formulated as a lyophilised powder for reconstitution and is available in single use vials containing 20 mg or 30 mg enfortumab vedotin-ejfv. Padcev is administered at 1.25 mg/kg (up to a maximum dose of 125 mg) following reconstitution in sterile water and further dilution in specified infusion fluids by intravenous infusion over 30 min on days 1, 8 and 15 of a 28-day cycle. Dosing at this level continues to the point of disease progression or unacceptable toxicity.

Padcev is indicated for the second line treatment of adults with a locally advanced or metastatic disease, who have been previously treated with a programmed death receptor-1 (PD-1) or programmed death ligand 1 (PD-L1) inhibitor and a platinum-containing chemotherapy.

The FDA approved Padcev based on data from a single-arm Phase II study involving 125 patients with locally advanced or metastatic urothelial cancer who had been previously treated with a PD-1 or PD-L1 inhibitor and platinum-based chemotherapy. Treatment with Padcev resulted in an overall response rate (in terms of tumour shrinkage per blinded independent central review) of 44%, with 12% having a complete response and 32% having a partial response. The response to Padcev had a median duration of 7.6 months. As Padcev was approved under the FDA’s accelerated approval system, continuing approval requires a further clinical trial to verify and further evaluate the clinical response.

Janssen bags EU approval for esketamine nasal spray

On the same day the Janssen Pharmaceutical Companies (Beerse, Belgium) of Johnson and Johnson made public that the European Commission had approved Spravato^® nasal spray, containing esketamine for treatment-resistant major depressive disorder (TRD) in adults, when prescribed in combination with a selective serotonin reuptake inhibitor or serotonin and norepinephrine reuptake inhibitor [19]. TRD sufferers are defined in the approval as those currently with moderate-to-severe depression who have not demonstrated improvement after at least two different antidepressant treatments. There are estimated to be up to 40 million sufferers of major depressive disorder in Europe and around 33% do not respond to existing therapies, resulting in a potentially large market for improved antidepressant therapy.

Esketamine is the S-enantiomer of ketamine and a N-methyl-D-aspartate glutamate receptor antagonist. It therefore has a different mechanism of action to existing treatments. Its activity in depression is believed to be associated with restoration of synaptic connections between brain cells in TRD patients, thus, improving communication between specific parts of the brain. It has a quicker onset of action than existing therapies with administration of Spravato nasal spray, in addition to an oral antidepressant starting to ameliorate depressive symptoms as early as day 2 from initiation of treatment. The medicine was approved based on data from three Phase III trials: three short-term, one randomized withdrawal and maintenance of effect trial and one long-term safety study [19–21]. The data demonstrated that around 70% of esketamine-treated patients had a ≥50 percent symptom reduction, while approximately 50% were in remission at 4 weeks. Continuing therapy with esketamine in combination with an oral antidepressant significantly reduced the risk of relapse, compared with oral antidepressant medication alone. This reduction in relapse was 70% in the case of patients with a stable response and 51% in those in stable remission. The recommended dose of Spravato for adults under 65 years is 56–84 mg twice weekly for the first 4 weeks, once weekly for the next 4 weeks and subsequently once every 1–2 weeks. The starting dose on day 1 is 56 mg. It is presented as a single use spray containing 28 mg esketamine, to be delivered in two sprays (one per nostril).

Spravato was also approved by the FDA earlier in 2019 [22]. However, in both the USA and Europe the response to its approval has been tinged with caution [23]. On the one hand there is an urgent need to provide better treatment for sufferers of TRD, while on the other hand there has been a discussion in the medical literature about the robustness of the study results and concern that side effects associated with the drug, particularly sedation and dissociation, although transient, require it to be administered under the direct supervision of a healthcare professional and the patient to be monitored for at least 2 h after administration. It is also contra-indicated in those with blood vessel wall weaknesses, for example patients who have previously suffered brain bleeds and recent heart attack victims, due to its propensity to raise blood pressure. In addition, it is likely to have addictive potential [20,22]. As a result, it is the subject of risk management strategies in both the USA and in Europe.

Clinical trials

Positive Phase III outcome for tenapanor in chronic kidney disease patients

It was a good start to the month for Ardelyx, Inc. (CA, USA) who announced positive efficacy and safety results from its Phase III PHREEDOM study, which evaluated the phosphate absorption inhibitor; tenapanor, for the treatment of hyperphosphatemia in patients with chronic kidney disease (CKD) on dialysis [24]. Hyperphosphatemia is a common feature of CKD and is currently treated with phosphate binders. However, approximately 70% of CKD patients on dialysis still have high phosphate levels at any one-time despite treatment. Unlike phosphate binders, tenapanor decreases phosphate absorption by inhibiting the sodium hydrogen exchanger 3 (NHE3) in the gut wall, thus, causing a reduction in the paracellular absorption of phosphate across tight junctions. It is also dosed twice daily, which compares favourably with the significantly higher pill burden with phosphate binders that must be taken before every meal or snack [25].

The study started with a 26-week, open-label treatment period in which 564 CKD patients on dialysis were randomized to receive either tenapanor or the phosphate binder; sevelamer in a 3:1 ratio. The patients had a serum phosphorus level ranging from 6.0–10.0 mg/dl and an increase in serum phosphorous of ≥1.5 mg/dl up to 3 weeks after the cessation of phosphate binder therapy. At the end of the open-label stage, the patients treated with tenapanor were re-randomized (1:1) prior to their inclusion in a 12-week, double-blind, placebo-controlled withdrawal period. Finally, the withdrawal period was followed by a 3-month safety extension. Those assigned to the sevelamer arm received this medication for the entire 12 months.

The trial met its primary end point with tenapanor-treated patients achieving a statistically significant difference in serum phosphorus change (-1.4 mg/dl; p < 0.0001), compared with the placebo from completion of the 26-week phase to the end of the withdrawal period. In addition, 77% of patients in the tenapanor arm experienced a mean reduction in serum phosphorous from a baseline of 2.0 mg/dl during the 26-week treatment part of the trial.

The study demonstrated the safety profile of tenapanor to be generally good with loose stools/diarrhea being the most common adverse effect with the majority of cases occurring within the first 5 days of treatment and being of a transient, mild-to-moderate nature. Overall, in the 26-week treatment phase, 17.2% patients in the tenapanor treatment arm experienced a severe adverse effect compared with 22.6% in the sevelamer-treated cohort.

The company has previously reported positive results from two previous Phase III trials, one with tenapanor as monotherapy and one where it was administered in combination with phosphate binders [24]. The drug was also approved by the FDA under the tradename Ibsrela^® earlier in 2019 for the treatment of irritable bowel syndrome with constipation.

Biohaven’s vazegepant meets primary end points in Phase II/III trial in migraine sufferers

On 17 December 2019 Biohaven Pharmaceutical Holding Company Ltd (CT, USA) made public the positive headline results from a Phase II/III trial of their third generation, small molecule calcitonin gene-related peptide (CGRP) receptor antagonist [26]. Vazegepant is part of Biohaven’s NOJECTION™ Migraine Platform, which is focused on developing therapies for migraine through targeting the CGRP receptor. CGRP release from the trigeminal nerve is considered to be the trigger for migraine attacks [27,28]. Vazegepant is structurally different from Biohaven’s other migraine drug, rimegepant, which was recently approved by the FDA [29] and is formulated for intra-nasal delivery using the Aptar Pharma Unidose System [30].

In the trial 5, 10 and 20 mg vazegepant or placebo were administered intra-nasally to 1673 patients suffering an acute migraine attack. Both the 10 and 20 mg doses met the co-primary regulatory end points of freedom from pain and freedom from the patient’s most bothersome symptom (photophobia, phonophobia or nausea) at the 2-h time-point. In addition, vazegepant demonstrated rapid onset of pain relief and had beneficial effects that were maintained over 48 h, despite patients only receiving a single dose. The study also indicated that vazegepant had a good safety profile with >80% of the side effects being of mild-to-moderate intensity. There was no evidence of liver toxicity.

Positive top-line results for investigational diabetes Type 1 treatment

Diamyd Medical AB (Stockholm, Sweden) had good news to report on their DIAGNODE-1 clinical trial in newly diagnosed Type 1 diabetics on 20 December 2019 [31]. This aim of this open label study was to test their diabetes vaccine; Diamyd^®, in 12 patients for its safety, efficacy and immunological response, in particular its ability to maintain endogenous insulin production, after administration at a low dose (4 μg) three-times at monthly intervals, directly into the lymph node. The patients also received oral vitamin D for 4 months and their progress was reported at 6, 15 and 30 months. As an extension of the trial, three patients received a fourth injection after approximately 2.5 years and were followed for a further 11 months.

The top-line results demonstrated that all 12 patients had a near normal long-term blood sugar (measured as HbA1c) with low requirements for supplementation with insulin injection. Overall, their endogenous insulin production (measured as stimulated C-peptide, AUC) decreased on average by 19 and 42% over 15 and 30 months, respectively. This is in contrast to the loss of 35–50% anticipated in untreated patients of a similar age over 15 months, based on previous studies.

Those receiving the additional injection maintained their own insulin production between the 30 and 43-month checks and had lower long-term blood sugar. They had a reduced need for insulin even compared with the 30-month time-point. Overall, the safety profile of the vaccine was good and no serious adverse effects were reported.

The vaccine is based on GAD65, a neuroendocrine protein to which autoantibodies are directed in Type 1 diabetes [32,33]. The company utilizes the intralymphatic route to deliver its vaccine, in order to boost immune response to the antigen and facilitate the induction of immune tolerance to protect against and preserve the patient’s ability to produce their own insulin. Based on the success of DIAGNODE-1, the company has initiated a double-blind, placebo-controlled Phase IIb trial using this route of administration that is currently underway.

Patents

USPTO grants patent to Aphios for oral bryostatin nanoparticles for neurodegenerative diseases

On 5 December 2019 the Aphios Corporation (MA, USA) made public that it had been granted a US patent for its proprietary bryostatin nanoparticles, as a method of treatment by oral administration for neurodegenerative diseases [34], such as Huntington’s disease, Parkinson’s disease, multiple sclerosis and Alzheimer’s disease. The US Patent No. 10485766 entitled ‘Drug delivery system and method for the treatment of neuro-degenerative disease’ [35] covers bryostatin-1 nanoparticles and those containing other bryostatin analogs.

Aphois is developing a bryostatin-1 analog, APH-1104, which is more potent than bryostatin-1, for the treatment of Alzheimer’s [36]. This molecule has been shown to activate (α)-secretase by protein kinase C dependent mechanisms. The enzyme cleaves the myloid precursor protein ‘APP’ into ‘sAPP-α’, which facilitates new synapse formation, while being more soluble than other breakdown products of APP, such as those formed by the action of β- and γ-secretase. It is therefore more easily cleared from the brain, thus, reducing the risk of Aβ plaque formation.

The nanoparticles are prepared from poly(D,L-lactide-co-glycolic acid) and may be manufactured using the company’s super-critical fluid technology, SuperFluids™ [36]. The Massachusetts’ firm has demonstrated that bryostatin-1 loaded nanoparticles restore cognitive abilities in a transgenic mouse model of Alzheimer’s Disease and improve inter-trial latency, with respect to inter-trial water-maze performance in a transgenic murine model of Down’s syndrome. This latter model displays many of the same characteristics as Alzheimer’s disease.

Summary

Company and product news from December 2019 feature in this month’s Industry Update. This was the month that Sumitomo Dainippon launched a new subsidiary, Sumitovant Biopharma Ltd, following completion of its strategic alliance with Roivant; Sanofi acquired Synthorx; while Astellas purchased Xyphos Biosciences. It was also the month that positive top-line clinical trial results were released for Diamyd’s Type 1 diabetes vaccine, Biohaven’s migraine drug, vazegepant and Ardelyx’s tenapanor, for the treatment of hyperphosphatemia in patients with chronic kidney disease on dialysis. In addition, therapy of urothelial cancer received a boost when the FDA approved the antibody–drug conjugate, Padcev (enfortumab vedotin-ejfv) from Astellas. The information is sourced predominately from company press releases and websites.

Financial & competing interests disclosure

The author has no relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties.

No writing assistance was utilized in the production of this manuscript.