Tim Wright, Editor, Contract Pharma07.15.20
Headquarters: Basel, Switzerland
twitter.com/Roche
www.roche.com
Headcount: 97,735
Pharma Revenues: $49,960 (+11%)
Net Income: $13,583 (+23%)
R&D: $10,293 (-16%)
TOP SELLING DRUGS
New products were the major growth driver for Roche in 2019. Demand for recently launched medicines for multiple sclerosis (Ocrevus) and haemophilia (Hemlibra) were particularly high and the same goes for the cancer immunotherapy Tecentriq. These three products were the major contributors to Roche’s pharma sales growth of 11% in 2019 to $49.9 billion.
Another highlight from the year was the approval of Rozlytrek, a targeted cancer treatment that marked a new chapter for Roche in the development of personalized healthcare. Biomarker testing for neurotrophic tyrosine receptor kinase (NTRK) gene fusions is the only way to identify people who may be eligible for treatment with Rozlytrek. Roche is leveraging its expertise in advanced diagnostics to help identify people with NTRK gene fusions using a companion diagnostic that is undergoing review.
During the year Roche made progress in other therapeutic areas besides oncology as well. It submitted documentation for two new medicines in the second half of the year: risdiplam in the hereditary disorder spinal muscular atrophy, and satralizumab for a specific disease of the central nervous system. For risdiplam, the FDA granted priority review and for satralizumab breakthrough therapy designation.
Gene therapy expansion
Gene therapies represent the future of life sciences. They are particularly promising for a number of rare diseases because they directly address the genetic cause of such conditions. Roche bolstered its presence substantially in this area, expanding its portfolio through the acquisition of the U.S. gene therapy pioneer Spark Therapeutics for $4.3 billion.
Based in Philadelphia, PA, Spark discovers, develops and delivers gene therapies for genetic diseases, including blindness, hemophilia, lysosomal storage disorders and neurodegenerative diseases. Its lead clinical asset is SPK-8011, a novel gene therapy for the treatment of hemophilia A, which entered Phase III in 2019. Spark also has SPK-8016 in a phase 1/2 trial aimed at addressing the hemophilia A inhibitor population.
Spark was the first company to receive FDA approval for a gene therapy for a genetic disease in 2017. That was for Luxturna, a one-time gene therapy indicated for the treatment of biallelic RPE65 mutation-associated retinal dystrophy.
Spark’s additional clinical assets include: SPK-9001, an investigational gene therapy for the potential treatment of hemophilia B in Phase III and SPK-7001 for choroideremia in Phase 1/2. The company is also developing SPK-3006 for Pompe disease and SPK-1001 for CLN2 disease (a form of Batten disease) which were on target for clinical development in 2019, as well as additional preclinical programs for Huntington's disease and Stargardt disease.
In another significant acquisition, Roche bought Promedior for $1.39 billion, giving it the biotech’s entire portfolio of molecules for serious fibrotic diseases, notably PRM-151. Based in Lexington, MA, Promedior successfully advanced PRM-151 in human clinical trials and received breakthrough designation from the FDA for idiopathic pulmonary fibrosis (IPF).
PRM-151, a recombinant form of human pentraxin-2 (PTX-2) protein, has demonstrated both prevention and reversal of fibrosis and opens up new opportunities to treat a wide range of systemic fibrotic diseases. Phase II trial results demonstrated that PRM-151 is the first molecule to show significant lung function improvements on top of current therapies in IPF. PRM-151 has also shown promising early clinical trial data in myelofibrosis (MF) and its anti-fibrotic mechanism has therapeutic potential in other fibrotic diseases.
R&D alliances
During 2019, Roche, through its biotech subsidiary Genentech, entered several notable research collaborations. With Xencor, Inc. it entered into a research and license agreement to develop and commercialize novel IL-15 cytokine therapeutics, including XmAb24306. XmAb24306 is an IL-15/IL-15Rα cytokine complex engineered with Xencor's bispecific Fc domain and Xtend Fc technology and is Xencor's most advanced preclinical cytokine program.
The companies will co-develop XmAb24306 and other potential IL-15 programs, in which the companies will share development costs and profits. Additionally, the companies will engage in a two-year research program to discover new IL-15 drug candidates, including ones targeted to specific immune cell populations. Genentech paid Xencor $120 million upfront, and Xencor is eligible to receive up to $160 million in development milestones for the XmAb24306 program and up to $180 million in development milestones for each new IL-15 drug candidate.
In another deal, Skyhawk Therapeutics entered into a multi-target exclusive option and license agreement with Genentech to develop and commercialize small molecules that modulate RNA splicing. Skyhawk is using its SkySTAR technology platform to discover and develop innovative small molecule treatments directed to certain oncology and neurological disease targets.
The agreement grants Genentech an exclusive worldwide license to develop and commercialize potential therapeutics directed to multiple targets while Skyhawk received an upfront payment and is eligible to receive future payments and royalties. As part of the agreement, Genentech is responsible for clinical development and commercialization.
Convelo Therapeutics and Genentech formed a partnership for the discovery and development of novel remyelinating medicines for neurological disorders such as multiple sclerosis (MS).
Convelo receives an upfront payment and research support from Genentech, which has the option to acquire Convelo and downstream milestones.
Lastly, Genentech teamed up with Enable Injections in a drug delivery partnership. The agreement includes the potential for multiple molecule development programs including enFuse, under development by Enable to allow patient-administered subcutaneous delivery of high-volume therapeutics.
Enable's enFuse is an on-body drug delivery platform with a drug transfer system compatible with standard syringes or vial container formats. The wearable enFuse platform is being developed for subcutaneous administration of large-volumes potentially ranging up to 50 mL. Designed for ease of use, enFuse has the potential to provide patients and their caregivers an alternative delivery method for subcutaneous administration of parenteral therapies outside of a clinical setting.
twitter.com/Roche
www.roche.com
Headcount: 97,735
Pharma Revenues: $49,960 (+11%)
Net Income: $13,583 (+23%)
R&D: $10,293 (-16%)
TOP SELLING DRUGS
| Drug | Indication | 2019 Sales | (+/-%) |
| Avastin | breast cancer | $7,118 | 2% |
| MabThera/Rituxan | rheumatoid arthritis | $6,518 | -6% |
| Herceptin | cancer | $6,078 | -15% |
| Ocrevus | Relapsing-Remitting MS (RRMS) | $3,732 | 55% |
| Perjeta | breast cancer | $3,544 | 25% |
| Xolair | asthma | $1,982 | 1% |
| Actemra/RoActemra | rheumatoid arthritis | $1,925 | 4% |
| Tecentriq | Bladder, Lung and Breast cancer | $1,887 | 139% |
| Lucentis | macular degeneraton | $1,838 | 8% |
| Kadcyla | Breast cancer | $1,402 | 40% |
New products were the major growth driver for Roche in 2019. Demand for recently launched medicines for multiple sclerosis (Ocrevus) and haemophilia (Hemlibra) were particularly high and the same goes for the cancer immunotherapy Tecentriq. These three products were the major contributors to Roche’s pharma sales growth of 11% in 2019 to $49.9 billion.
Another highlight from the year was the approval of Rozlytrek, a targeted cancer treatment that marked a new chapter for Roche in the development of personalized healthcare. Biomarker testing for neurotrophic tyrosine receptor kinase (NTRK) gene fusions is the only way to identify people who may be eligible for treatment with Rozlytrek. Roche is leveraging its expertise in advanced diagnostics to help identify people with NTRK gene fusions using a companion diagnostic that is undergoing review.
During the year Roche made progress in other therapeutic areas besides oncology as well. It submitted documentation for two new medicines in the second half of the year: risdiplam in the hereditary disorder spinal muscular atrophy, and satralizumab for a specific disease of the central nervous system. For risdiplam, the FDA granted priority review and for satralizumab breakthrough therapy designation.
Gene therapy expansion
Gene therapies represent the future of life sciences. They are particularly promising for a number of rare diseases because they directly address the genetic cause of such conditions. Roche bolstered its presence substantially in this area, expanding its portfolio through the acquisition of the U.S. gene therapy pioneer Spark Therapeutics for $4.3 billion.
Based in Philadelphia, PA, Spark discovers, develops and delivers gene therapies for genetic diseases, including blindness, hemophilia, lysosomal storage disorders and neurodegenerative diseases. Its lead clinical asset is SPK-8011, a novel gene therapy for the treatment of hemophilia A, which entered Phase III in 2019. Spark also has SPK-8016 in a phase 1/2 trial aimed at addressing the hemophilia A inhibitor population.
Spark was the first company to receive FDA approval for a gene therapy for a genetic disease in 2017. That was for Luxturna, a one-time gene therapy indicated for the treatment of biallelic RPE65 mutation-associated retinal dystrophy.
Spark’s additional clinical assets include: SPK-9001, an investigational gene therapy for the potential treatment of hemophilia B in Phase III and SPK-7001 for choroideremia in Phase 1/2. The company is also developing SPK-3006 for Pompe disease and SPK-1001 for CLN2 disease (a form of Batten disease) which were on target for clinical development in 2019, as well as additional preclinical programs for Huntington's disease and Stargardt disease.
In another significant acquisition, Roche bought Promedior for $1.39 billion, giving it the biotech’s entire portfolio of molecules for serious fibrotic diseases, notably PRM-151. Based in Lexington, MA, Promedior successfully advanced PRM-151 in human clinical trials and received breakthrough designation from the FDA for idiopathic pulmonary fibrosis (IPF).
PRM-151, a recombinant form of human pentraxin-2 (PTX-2) protein, has demonstrated both prevention and reversal of fibrosis and opens up new opportunities to treat a wide range of systemic fibrotic diseases. Phase II trial results demonstrated that PRM-151 is the first molecule to show significant lung function improvements on top of current therapies in IPF. PRM-151 has also shown promising early clinical trial data in myelofibrosis (MF) and its anti-fibrotic mechanism has therapeutic potential in other fibrotic diseases.
R&D alliances
During 2019, Roche, through its biotech subsidiary Genentech, entered several notable research collaborations. With Xencor, Inc. it entered into a research and license agreement to develop and commercialize novel IL-15 cytokine therapeutics, including XmAb24306. XmAb24306 is an IL-15/IL-15Rα cytokine complex engineered with Xencor's bispecific Fc domain and Xtend Fc technology and is Xencor's most advanced preclinical cytokine program.
The companies will co-develop XmAb24306 and other potential IL-15 programs, in which the companies will share development costs and profits. Additionally, the companies will engage in a two-year research program to discover new IL-15 drug candidates, including ones targeted to specific immune cell populations. Genentech paid Xencor $120 million upfront, and Xencor is eligible to receive up to $160 million in development milestones for the XmAb24306 program and up to $180 million in development milestones for each new IL-15 drug candidate.
In another deal, Skyhawk Therapeutics entered into a multi-target exclusive option and license agreement with Genentech to develop and commercialize small molecules that modulate RNA splicing. Skyhawk is using its SkySTAR technology platform to discover and develop innovative small molecule treatments directed to certain oncology and neurological disease targets.
The agreement grants Genentech an exclusive worldwide license to develop and commercialize potential therapeutics directed to multiple targets while Skyhawk received an upfront payment and is eligible to receive future payments and royalties. As part of the agreement, Genentech is responsible for clinical development and commercialization.
Convelo Therapeutics and Genentech formed a partnership for the discovery and development of novel remyelinating medicines for neurological disorders such as multiple sclerosis (MS).
Convelo receives an upfront payment and research support from Genentech, which has the option to acquire Convelo and downstream milestones.
Lastly, Genentech teamed up with Enable Injections in a drug delivery partnership. The agreement includes the potential for multiple molecule development programs including enFuse, under development by Enable to allow patient-administered subcutaneous delivery of high-volume therapeutics.
Enable's enFuse is an on-body drug delivery platform with a drug transfer system compatible with standard syringes or vial container formats. The wearable enFuse platform is being developed for subcutaneous administration of large-volumes potentially ranging up to 50 mL. Designed for ease of use, enFuse has the potential to provide patients and their caregivers an alternative delivery method for subcutaneous administration of parenteral therapies outside of a clinical setting.
