Looking ahead to 2018, the most promising products in pharma’s pipeline will compete in a landscape that increasingly rewards big risks and places an emphasis on novel mechanisms.

That comes with a catch. Consumers are more sensitive than ever to high prices, with more than 30 states introducing legislation on drug pricing. Given the current political climate, the market is likely to place a premium on novel therapies that truly advance the standard of care. Indeed, next year’s most promising late-stage candidates run the gamut, from drugs with never-before-seen mechanisms to old standbys with new potential.

Not surprisingly, attention remains laser-focused on gene therapy technologies that promise a one-and-done treatment approach. Along those lines, Spark Therapeutics is slated to land the first major gene therapy approval in early 2018. Meanwhile, BioMarin is moving a candidate through Phase III trials in hemophilia A, and BlueBird is forging ahead with Phase III trials on a treatment for childhood cerebral adrenoleukodystrophy, a rare disease.

In oncology, AstraZeneca was granted breakthrough therapy designation for its BTK inhibitor acalabrutinib, which was approved in Q4 2017. Juno is taking another stab at CAR-T therapy after its first attempt, JCAR015, was associated with five deaths in a trial. BlueBird and Celgene are moving forward with their own CAR-T candidate.

In 2017, the FDA issued its share of complete response letters for immunology candidates. The category should remain lively, with several, among them AbbVie/Boehringer Ingelheim’s anti-IL-23 risankizumab, Celgene’s selective S1PR1 and S1PR5 inhibitor ozanimod, and Biogen’s anti-BDCA2 monoclonal antibody BIIB059.

It’s been years since the market has seen a major step forward in neurodegenerative disorders, yet 2018 could bring one. Mitsubishi acquired NeuroDerm largely for a new approach that relies on subcutaneous levodopa/carbidopa delivery for Parkinson’s. Generating similar buzz is vTv Therapeutics’ azeliragon, which could become the first major Alzheimer’s drug approval since 2002.

Finally, with the nation reeling from the opioid epidemic, several companies are pursuing new pain medications that promise to be less addictive. Eli Lilly/Pfizer’s chronic pain med tanezumab landed fast track designation in June 2017, with Teva/Regeneron’s fasinumab not far behind. Medivir is moving forward with MIV-711 for osteoarthritis pain.

Agents profiled in this report are based on consultation with inThought Research, Adis R&D Insight, GfK Health, BioPharm Insight, and others. Analyses of featured products include the latest clinical data, revenue forecasts, expected launch dates, and likelihood of success as of press time.

Gene Therapy
Most-anticipated products

Luxturna, Voretigene neparvovec

Spark, Preregistration

Indication: Leber congenital amaurosis (blindness)

What the clinical trials found: Voretigene neparvovec, up for an FDA decision on Jan. 12, is poised to be the first true gene therapy approved in the United States — and, in October, received a unanimous thumbs up from an FDA advisory committee. A Phase III trial met its primary endpoint, with 93% of participants demonstrating some gain in functional vision and experiencing ongoing improvement one year later. There was a statistically significant and clinically meaningful difference between the intervention group and control group at one year.

BioPharm Insight probability and inThought comment: BioPharm senior journalist Alaric DeArment rates voretigene neparvovec’s approval chances as “neutral,” with sales expected in 2018. “People have been waiting for an ability to treat this condition for a long time. It meets that intersection of novel gene therapy and a massive unmet need, and there are a lot of patient support groups behind it.” — Dr. Leon Henderson-MacLennan, medical adviser, inThought Research

BioPharm Insight revenue forecast: $491 million in global annual sales by 2025

What physicians are saying: “This form of local delivery into an immune privileged space has been demonstrated to be safe and efficacious, but requires specialized surgical techniques and/or devices to deliver the vector. Thus it offers the promise of a novel one-off treatment regimen leading to potentially lifelong benefits, but is likely to pose major affordability challenges if paid for using traditional methods.” — Mark Slomiany, consultant, market access, GfK Health

BMN 270

BioMarin, Phase III

Indication: Hemophilia A

What the clinical trials found: Results from a Phase II study showed the drug was well-tolerated and met its primary endpoint, with patients continuing to improve in a subsequent efficacy analysis. The study demonstrated a 91% drop in the average annual bleeding rate and a 98% drop in prophylactic infusions.

Credit Suisse success probability and inThought comment: 35%, with expected launch in 2020. “Between BioMarin’s BMN 270 and Spark’s 8011 for hemophilia, BioMarin may have the best shot to make it to the market first.” — Henderson-MacLennan

Credit Suisse revenue forecast: $58 million in global annual sales in 2020

What physicians are saying: “Hemophilia treaters are excited about BMN 270, noting the data continue to show promising evidence of efficacy and safety, and the candidate has the potential to change the treatment paradigm for hemophilia A. Gene therapy has the promise of delivering, in as little as a single administration, the missing gene needed to produce factor VIII, which may eliminate the need for ongoing treatments.” — Carol Chang, VP, consulting, GfK


BlueBird, Phase III

Indication: Childhood cerebral adrenoleukodystrophy (CCALD)

What the clinical trials found: Lenti-D exceeded its primary endpoint in an interim analysis of the Phase II/III Starbeam Study (ALD-102). In the trial, which studied boys younger than 18 years old with CALD, also known as Lorenzo’s Oil disease, 15 out of the initial cohort of 17 patients who had completed two years of follow-upwere free of major functional disabilities. Meanwhile, the safety profile was on-par with myeloablative conditioning.

inThought comment: “People have been paying attention to BlueBird’s gene therapy construct, and Lenti-D has a shot at approval in 2019.” — Henderson-MacLennan 

BioPharm Insight revenue forecast with GfK Health comment: $172 million in global annual sales by 2023. “While peak sales could equate to a mere $200 million, the real value may come from receiving approval for its first treatment.” — Slomiany

What physicians are saying: “CALD is challenging, as it is the result of any of more than 600 different loss-of-function mutations to the peroxisomal transport protein ABCD1. Thus replacement with ABCD1-competent phagocytes via hematopoietic stem cell-directed gene therapy is considered a bar set high.” — Slomiany

Other key products in the pipeline:

AVXS 101
spinal muscular atrophy type 1, Phase III

Lentiglobin BB305
beta thalassemia, Phase III

GS 010
GenSight Biologics
Leber’s hereditary optic neuropathy, Phase III

adenosine deaminase deficiency, Phase II/III

Orchard Therapeutics
adenosine deaminase severe combined immunodeficiency (ADA-SCID), Phase II/III

hemophilia, Phase I/II

Vocimagene amiretrorepvec-flucytosine 
recurrent glioblastoma and anaplastic astrocytoma, Phase II/III

VBL Therapeutics
recurrent glioblastoma and anaplastic astrocytoma, Phase III

Most-anticipated products

AbbVie/Boehringer Ingelheim, Phase III

Indication: Plaque psoriasis

What the clinical trials found: Risankizumab, which has orphan designation in Crohn’s disease, is in P3 trials in a variety of indications, including Psoriasis, Ankylosing spondylitis, Crohn’s disease, and Asthma. According to P3 results in psoriasis, the drug bested AbbVie’s approved psoriasis drug, Humira, as well as Johnson & Johnson’s Stelara. Across three clinical trials, the drug cleared 90 percent of plaque psoriasis in about three quarters of patients after 16 weeks of treatment. In addition, the drug was found to be relatively safe, with only 2% of patients taking risankizumab encountering serious adverse events.

Credit Suisse success probability and inThought comment: 50%, with expected launch in 2019. “It’s going to be a blockbuster. It turns out when you inhibit IL23 without inhibiting IL12, [outcomes are] so much better. In addition, risankizumab’s dosing is such that it could be given quarterly.” — Ben Weintraub, president, inThought Research

Credit Suisse revenue forecast: $283 million global annual sales by the end of 2019 and $680 million by the end of 2020

What physicians are saying: “Although risankizumab has offered compelling efficacy data to date and a comprehensive data package including head-to-head Phase II trials outperforming Stelara with a greater reduction in Psoriasis Area and Severity Index (PASI) score, it will remain a late IL-23 inhibitor entry, after Janssen’s Tremfya [guselkumab] and Sun Pharma’s tildrakizumab. It is likely to be prescribed in second line or later, eclipsing its potential for blockbuster status. Additional data showing long-term efficacy, safety, and adherence, and addressing patient unmet needs and impact on quality of life, could improve its position in the current market, particularly if it is able to differentiate itself from key competitors.” — Anita Agier, head, real world evidence, GfK Health

Celgene, Phase III

Indication: Multiple sclerosis

What the clinical trials found: Ozanimod performed well in two Phase III trials in MS, meeting primary endpoints in both. There have been no severe adverse events reported to date. Overall, adverse-event profile was similar to placebo in a Phase II trial.

Credit Suisse success probability and inThought comment: 50%, with expected launch in 2018. “Celgene’s drug is thought to be safer than its rival, Novartis’ Gilenya [fingolimod], because it may be approved without the need for heart rate monitoring. In addition, Celgene is going after IBD, so it might be first-in-class there.”— Weintraub

Credit Suisse revenue forecast: $81 million global annual sales by the end of 2018 and $740 million by the end of 2019

What physicians are saying: “Ozanimod will be a welcome additional treatment option into the complex MS market, where physicians will likely prescribe it among RRMS patients with active disease at launch. It will continue to gain share from Novartis’ Gilenya as physicians increase their experience with the drug.”— Agier

Biogen, Phase II

Indication: Lupus

What the clinical trials found: In a Phase Ib study in 12 patients, a single dose of BIIB059 inhibited IFN-responsive genes in peripheral blood, consistent with the drug’s MOA. BIIB059 was well tolerated, with no discontinuations due to AEs.

inThought comment: “Although [BIIB059] trails behind AstraZeneca’s Anifrolumab, people are really excited about it in lupus. We now believe that type I interferons are crucial to lupus pathology, and the Biogen drug appears to target those cells directly.” — Weintraub

What physicians are saying: “If Phase II results show superior efficacy and safety data, as well as a quicker onset of action over GSK’s Benlysta [belimumab], then it could have blockbuster potential and increase overall penetration of targeted treatments among systemic lupus erythematosus patients.” — Agier

Other key products in the pipeline:

RA, Phase III

MS, Phase III

lupus, Phase III

lupus, Phase III

Eli Lilly and Incyte
RA, companies plan to refile in January 2018

Galapagos NV Gilead Sciences
RA, Phase III

Crohn’s, Phase III

GSK 165
RA, Phase IIb

lupus, Phase III

Johnson & Johnson
RA, company plans to reassess in early 2018

Biotin capsules (MD 1003)
MedDay Pharmaceuticals
MS, Phase III

MS, Phase III

Rigel Pharmaceuticals
chronic and persistent immune thrombocytopenia, NDA submitted April 2017

TG Therapeutics
MS, Phase III

Most-anticipated products

NeuroDerm/Mitsubishi, Phase III

Indication: Parkinson’s disease

What the clinical trials found: ND0612, a liquid formulation of levodopa/carbidopa administered subcutaneously via a mini-pump, has been tested at two doses in two Phase II trials. At the lower dose, ND0612 maintained drug concentrations and reduced “off time” associated with oral levodopa in Parkinson’s patients. At the higher dose, the Phase II trial indicated ND0612 might be a viable alternative to deep brain stimulation or intestinal gel. In 2016, the FDA gave NeuroDerm a green light to replace two Phase III clinical trials with smaller PK trials.

BioPharm Insight and inThought comment: BioPharm Insight notes analysts are predicting global sales starting in 2019. “This is the first successful attempt to make a convenient liquid formulation that would allow for continuous subcutaneous administration, therefore avoiding ‘off times’ in medication. The safety has already been demonstrated for this drug.” — Lisa Kennedy, senior principal, inThought Research

BioPharm Insight revenue forecast: $1.28 billion in U.S. and EU annual sales by 2026

What physicians are saying:“Improvement in levodopa therapies are always welcome and necessary, but it is only one factor in the management of Parkinson’s disease. Still, ND0612 should significantly improve motor and non-motor complications, including dyskinesia and ‘off’ periods, and is the first non-surgical continuous levodopa therapy.” — Edwin Bas, industry lead, health, GfK Netherlands and Els Bilman, senior research manager, brand and customer experience (health), GfK Netherlands

vTv Therapeutics, Phase III

Indication: Alzheimer’s disease

What the clinical trials found: In a Phase IIb trial, patients who received a lower dose of azeliragon saw 26% benefit in cognitive decline relative to placebo. However, at a higher dose the drug resulted in neurological worsening, prompting vTv’s partner, Pfizer, to pull out of the joint-development arrangement. Still, a Phase III trial is being conducted based on a protocol agreed upon with the FDA.

BioPharm Insight and inThought comment: BioPharm Insight notes analysts are predicting global sales starting in 2019. “This is a high-risk, high-reward program. Azeliragon targets three main pathways. [Several drugs] have looked at inhibiting amyloid beta and none worked, so it makes sense to put hope into a compound that’s separate from that. — Kennedy

BioPharm Insight revenue forecast: $1.1 billion in global annual sales by 2023

What physicians are saying: “Azeliragon works by inhibiting the receptor for RAGE [receptor for advanced glycation endproducts] and, with that, reduces downstream effects as vascular dysfunction and metabolic dysregulation. RAGE has been correlated with disease severity and progression. Current licensed treatments are directed at symptoms, while Azeliragon is a disease-modifying candidate. There is a clear need for this type of medication.” — Bas and Bilman

Other key products in the pipeline:

Alzheimer’s disease, Phase III

Axovant Sciences
Alzheimer’s disease, Phase III

Bayer Healthcare
Alzheimer’s disease, Phase III

Biogen and Neurimmune
Alzheimer’s disease, Phase III

Biorex and Orphazyme
Niemann-Pick diseases, Phase III

Concert Pharmaceuticals
Agitation, Phase III

Corplex Donepezil transdermal patch
Corium International
Alzheimer’s disease, Phase I

Alzheimer’s disease, Phase III

Alzheimer’s disease, Phase III

Alzheimer’s disease, Phase III

pantothenate kinase-associated neurodegeneration, Phase III

Alzheimer’s disease, Phase III


Most-anticipated products

Juno Therapeutics/Celgene, Phase I/II

Indication: Non-Hodgkin’s lymphoma (NHL)

What the clinical trials found: JCAR017 was granted breakthrough designation by the FDA. Updated data from the Phase I TRANSCEND study found that almost three quarters of patients with relapsed or refractory aggressive B-cell NHL had a complete response after three months when treated with the higher dose of 100 million cells. JCAR017 is also being tested in relapsed or refractory diffuse large B-cell lymphoma (DLBCL). Also, in the Phase I TRANSCEND study, the drug demonstrated 59% CR and 86% ORR among those with DLBCL.

BioPharm Insight and inThought comment: Global sales start in 2019. According to AdisInsight, Juno anticipates FDA approval by 2018. “JCAR017 is Juno’s second attempt at a CAR-T breakthrough after its first candidate was associated with five deaths in an earlier trial. Although Juno is behind Kite in the same indication, this compound might have an improved toxicity profile, with lower rates of neurotoxicity and cytokine-related syndrome.” — Lisa Kennedy, senior principal, inThought Research

BioPharm Insight revenue forecast: $789 million in global annual sales by 2023

What physicians are saying: “For many practicing hematologists, CAR T-cell therapies are still far away from being on their radar screen, probably because use will likely focus on specialized treatment centers. It will take time until hematologists and oncologists get used to coping with this new era of genetically engineered drugs — as it took time for stem cell transplants to get widely used.” — Petra Maertens, team lead, brand and customer experience (health) — GfK Switzerland

BlueBird/Celgene, Phase III

Indication: Multiple myeloma

What the clinical trials found: The CAR-T therapy BB2121 demonstrated strong Phase I interim results, with all patients in the first dosing cohort showing a response. The ORR rate was 100%, with 25% achieving a complete response. Across all dosing levels, ORR was 89%.

BioPharm Insight and inThought comment: Global sales start in 2020. “BB2121 showed excellent data in Phase I and BlueBird quickly moved on to a pivotal study. It seems to have high response rates and good durability. This candidate generated a lot of excitement at ASCO and ASH.” — Kennedy

BioPharm Insight revenue forecast: $937 million in global annual sales by 2027

What physicians are saying: “BB2121 is a genetically engineered immune T-cell therapy to create a chimeric antigen receptor, or CAR, that targets the BCMA protein often found in myeloma cells. The success of CAR T-cells against leukemia and lymphoma has encouraged development of CAR T-cell therapies also for multiple myeloma. Toxicities, including cytokine-release syndrome, have been similar to toxicities observed in CAR T-cell trials for leukemia including unique acute toxicities that are uncommon with other cancer therapies.” — Maertens

Other key products in the pipeline:

sarcoma, Phase III

sarcoma, Phase II

Durvalumab +/- tremelimumab
lung cancer, Phase III

Boehringer Ingelheim
AML, Phase III

Bristol-Myers Squibb
small cell lung cancer, Phase III

Daiichi Sankyo

Daiichi Sankyo
AML, Phase III

Eli Lilly
advanced breast cancer, approved

breast cancer, prostate cancer, Phase III

breast cancer, NSCL, Phase III

Gilead Sciences
gastric cancer, solid tumors, Phase III

Johnson & Johnson
prostate cancer, Phase III

BRCA-mutated breast cancer, Phase III

Syndax Pharmaceuticals
HR+ and HER2- breast cancer, Phase III

Most-anticipated products

Eli Lilly/Pfizer, Phase III

Indication: Chronic pain

What the clinical trials found: Tanezumab, a nerve growth factor (NGF) inhibitor granted fast track status by the FDA in June 2017, has demonstrated clinically meaningful efficacy versus placebo in multiple clinical trials of more than 11,000 patients.

Credit Suisse success probability and inThought comment: 50%, with an expected launch date of 2019. “Tanezumab is definitely promising because of its mechanism of action as a potential alternative to opioids for pain relief.” — Julie Hoggatt, senior principal, inThought Research Group

Credit Suisse success probability and inThought comment: $3 billion global sales by 2019

What physicians are saying: “There are safety concerns with the NGF class due to an elevated risk of arthopathy (the rapid destruction of a joint). This caused the FDA to put the entire NGF class under a clinical hold in 2011, although this was subsequently relaxed in 2015, allowing some NGF inhibitors to restart clinical testing. Tanezumab offers an improvement upon the standard of care by providing targeted pain control and subcutaneous administration every eight weeks. This may improve adherence and compliance, and therefore provide more effective overall pain control without the associated risk of dependency or the co-administration of agents to protect against GI damage.” — Louise Hogg, senior consultant, pricing and market access, GfK

Regeneron/Teva, Phase III

Indication: Chronic pain

What the clinical trials found: Fasinumab has been studied in chronic lower back pain and osteoarthritis knee or hip pain. A Phase IIb trial in 800 patients with back pain showed significant improvement over placebo, as did a Phase II/III trial in 421 patients with pain due to osteoarthritis of the knee or hip, as measured by the WOMAC pain score.

inThought comment: “Anti-NGFs for chronic pain are very interesting. Although fasinumab is expected to get to market a year after Pfizer/Lilly’s tanezumab, fasinumab’s lower dose may be safer.” — Hoggatt

Credit Suisse revenue forecast: $1.05 billion in global annual sales by 2019

What physicians are saying: “Fasinumab continues to be investigated in a Phase II/III osteoarthritis study, although the companies are planning to advance only lower doses. Putting safety aside, fasinumab has shown promising efficacy for the treatment of patients with chronic lower back pain or pain due to osteoarthritis of the knee or hip. It has the potential to be an alternative to prescription opioids that are effective for pain relief but are associated with high rates of addiction and overdose.” — Hogg

Medivir, Phase II  

Indication: Pain from osteoarthritis

What the clinical trials found: Phase IIa data released in September demonstrated 65% reduction in femoral joint bone area progression at both 100mg and 200mg doses, while the placebo arm demonstrated only a 1% increase in disease progression. In addition, the 200mg group actually showed a slight overall increase in cartilage thickness.

inThought comment: “The success of this Phase IIa trial is likely to make MIV-711 an intriguing commodity in the OA field. The demonstration of a slight increase in knee cartilage growth suggests that cathepsin K inhibition is indeed a key mechanism in OA. However, it will be important to see if this drug causes any other long-term safety issues. Merck discontinued development of a cathepsin K inhibitor for OA in September 2016 due to a risk of stroke. We still do not know if this was a result of cathepsin K inhibition or an off-target side-effect of the drug.” — Hoggatt

What physicians are saying: “MIV-711 is generating excitement with potential to be the first disease modifying drug for osteoarthritis. Medivir has retained strategic advisers to seek a partner for the future development of MIV-711, a highly selective cathepsin K inhibitor that has the potential to be a future disease modifying osteoarthritis drug. Cathepsin K is a protease that breaks down collagen, a protein that plays an important role in the structural integrity of both bone and cartilage. Medivir’s research has shown that inhibition of cathepsin K can reduce the rate of joint destruction in preclinical models of osteoarthritis.” — Hogg

Other key products in the pipeline:

pain due to osteoarthritis of the knee, Phase III

Bristol-Myers Squibb
migraine, Phase III

Daiichi Sankyo
pain, Phase III

chronic pain, Phase III

Eli Lilly
cluster headache/migraine, Phase III

Eli Lilly
migraine, Phase III

acute pain, Phase III

Ferring Pharmaceuticals
postoperative pain/pruritus, Phase III

acute pain, prereg.

pain due to osteoarthritis of the knee, Phase III

Takeda and Myovant Sciences
pain, Phase III

migraine (prevention), preregistration

postoperative pain,Phase III

Most-anticipated products


Alnylam/Sanofi, preregistration

Indication: TTR amyloidosis

What the clinical trials found: Patisiran met its primary endpoint in a Phase III trial, which evaluated change in baseline in a modified neuropathy score at 18 months in patients with ATTR.

Credit Suisse success probability and inThought comment: 95%, with expected launch in 2019. “This RNAi therapy is competing against Ioni’s inotersen, yet appears to have an edge as far as net benefit is concerned. Not only is inotersen associated with excess thrombocytopenia and renal AEs, but also its dynamics (as they relate to TTR knockdown magnitude) appear preferable.” — Dr. Leon Henderson-MacLennan, medical adviser, inThought Research

Credit Suisse revenue forecast: $3.4 billion in global annual sales by 2020

What physicians are saying: “Current therapies for hereditary ATTR amyloidosis treatment are insufficient. The primary treatment option is liver transplantation, and the disease is often misdiagnosed due to its heterogeneity. Now, Patisiran has the potential to address unmet medical needs. Phase III data suggest Patisiran could help improve the lives of people living with hATTR amyloidosis with polyneuropathy, a patient population in urgent need of additional treatment options.” — Edwin Bas, industry lead, health, GfK Netherlands, and Els Bilman, senior research manager, brand and customer experience (health), GfK Netherlands


Novo Nordisk, preregistration

Indication: Type 2 diabetes

What the clinical trials found: In a Phase III trial, Novo’s drug cut HbA1c by 1.5% at a lower dose and 1.8% at a higher dose, compared with Trulicity’s 1.1% at the lower dose and 1.4% at the higher dose.

Credit Suisse success probability and inThought comment: 75%, with expected launch in 2017. “Novo’s semaglutide — from the GLP-1 class, another class in which we are seeing CV risk-reduction capacity — is also compelling in these regards. The more classes we have against not only glycemic control but also downstream effects, the better. Plus, the promise of once-weekly dosing is quite attractive. The caveat is an excess retinopathy signal that awaits more mature data.” — Henderson-MacLennan

Credit Suisse revenue forecast: $330 million in global annual sales by 2018

What physicians are saying: “Semaglutide could transform diabetes treatment. Besides the blood glucose beat and weight-loss advantage, semaglutide joins another differentiating factor: proven cardiovascular benefits. Analysts predict that GLP-1 drugs will grow, volume-wise, to 7% of the overall diabetes market from 4% now. When the oral semaglutide (currently in Phase III) is registered, it will be world’s first biologic drug in an oral formulation.” — Bas and Bilman

Merck/Pfizer, preregistration

Indication: Type 2 diabetes

What the clinical trials found: The primary endpoint was met in a placebo-controlled, Phase III monotherapy trial designed to test ertugliflozin in patients who are uncontrolled by diet and exercise.

Credit Suisse success probability and inThought comment: 70%, with expected launch in December 2017. “The market will welcome another SGLT2 inhibitor. There’s real momentum in this class in terms of glycemic control, and macrovascular morbidity. A real inflection point will also occur around the second half of 2019, when its long-term CV outcomes study reads out.” — Henderson-MacLennan

Credit Suisse revenue forecast: $320 million global annual sales by 2018

Other key products in the pipeline:

Macimorelin Acetate

Aeterna Zentaris
adult growth deficiency, preregistration


Bristol-Myers Squibb (originator)/ViiV Healthcare (owner)
HIV-1, Phase III

cytomegalovirus, Phase III

malaria, preregistration

HIV-1, Phase III

Doravirine/lamivudine/tenofovir disoproxil fumarate
HIV-1, Phase III

Motif Bio
skin and soft tissue infections, Phase III

Cabotegravir plus rilpivirine
ViiV Healthcare
HIV-1, Phase III


Bayer Healthcare Pharmaceuticals
diabetic nephropathies, Phase III

GZ 402666
Genzyme Pharmaceuticals
glycogen storage disease type II, Phase III

Gilead Sciences

anemia associated with CKD, Phase III

Lexicon Pharmaceuticals and Sanofi
T1D and T2D, Phase III

Takeda and Tobira Therapeutics


Alnylam Pharmaceuticals
hemophilia A and hemophilia B, Phase III

Gilles de la Tourette’s syndrome, Phase II

sickle cell anemia, Phase III


Bayer Healthcare Pharmaceuticals
uterine leiomyoma, Phase III

preterm labor, Phase III

Neurocrine Biosciences and AbbVie
endometriosis, prereg.

breast cancer, Phase III