The past year has been one to remember for biotech investors. The FDA smashed through its previous record for new drug approvals in a single season, and a long list of experimental drugs getting close to the finish line promise to make 2019 just as interesting.
These experimental treatments aren't approved to treat anything yet, but their continued success could send shares of the drugmakers developing them soaring in the new year.
Data source: Yahoo! Finance, and EvaluatePharma.
Every year, EvaluatePharma ranks experimental treatments in late-stage studies by their consensus net present values. This measurement takes into account sales expectations as well as their odds of earning approvals from the FDA and other regulators.
Here's what you need to know about potential new blockbusters and the stocks that could soar if they exceed expectations.
Vertex Pharmaceuticals launched the first cystic fibrosis (CF) treatment in 2012, and it's still the only company marketing drugs to treat the root cause of this progressive, life-shortening disorder. The company's new triple combination treatment produced such convincing late-stage data that analysts are nearly certain the FDA will approve an application that the company expects to file in the middle of 2019.
Vertex thinks total revenue from CF products could reach $3.0 billion this year, which is 38% more than a year earlier. Although an approval and successful launch for VX-659 seems likely, investors need to realize a great deal of growth is already baked into Vertex shares, which have been trading at 15.6 times trailing sales.
Around 2,000 genetic mutations are known to cause CF for around 75,000 people in North America, Europe, and Australia. Many of those with the most common mutations are already on a Vertex therapy, which makes it hard to predict just how much new growth the company's new triplet can generate.
Celgene's late-stage star is a chimeric antigen receptor T-cell (CAR-T) therapy, which is really an IV bag full of a patient's own immune cells after they've been modified off-site to recognize and attack cancer. It's a complicated process that appears worth the effort for non-Hodgkin's lymphoma patients who no longer respond to standard care. Six months after treatment with JCAR017, a whopping 55% of patients were in complete remission.
While JCAR017's results are nothing short of spectacular, they're comparable to another CD19-directed CAR-T that aims for the same cancer cell target, Yescarta. Gilead Sciences (NASDAQ: GILD) launched this treatment for the same limited group of non-Hodgkin's patients over a year ago, and sales have been a huge disappointment so far.
It looks like Celgene may be repositioning JCAR017 to aim for a leukemia indication down the road instead of attempting to compete with Gilead for non-Hodgkin's patients. With plenty of potential pitfalls ahead, forward-looking estimates for this program should be taken with a big grain of salt.
Weekly GLP-1 injections are a popular new way to treat type-2 diabetes, and Novo Nordisk is working on a once-daily GLP-1 tablet that could become the most popular of all.
Novo's own GLP-1 injections generated around $2.8 billion in sales during the first nine months of 2018, and the company's depending on it to maintain its share of an increasingly crowded market.
Oral semaglutide probably has what it takes to become Novo's next blockbuster. Adding it to standard care reduced patients' risk of death from cardiovascular disease by 51%, plus investigators measured a 49% all-cause mortality risk reduction during a long-term outcome study with thousands of patients.
Intensifying competition for diabetes care has pressured the price of Novo Nordisk shares down to just 19.1 times trailing earnings at recent prices. That's well below the S&P 500 average, which gives this stock a chance to outperform if oral semaglutide can surpass some fairly reasonable expectations.
In case you hadn't noticed, the candidate that topped the list is also the only one that will be running unopposed in 2020. That said, Novo's oral semaglutide has a convenience advantage over injections, plus it's backed up with impressive outcome data that could set it apart. If I had to pick a candidate most likely to exceed expectations, it's the one in Novo's pipeline.
每年， EvaluatePharma 都会根据其共识净现值对后期研究中的实验治疗进行排名。该评估考虑了销售预期以及他们从 FDA 和其他监管机构获得批准的可能性。
Vertex Pharmaceuticals 于2012年启动了第一个囊性纤维化（ CF ）治疗，目前仍是公司唯一一种治疗这种进行性、缩短生命障碍的根源性药物。公司新的三联疗法产生了如此令人信服的后期数据，分析师几乎肯定 FDA 会批准该公司预计在2019年年中提交的申请。
Vertex 认为，今年 CF 产品的总收入可能达到30亿美元，较上年同期增长38%。尽管 VX-659获得批准和成功上市的可能性似乎很大，但投资者需要意识到， Vertex 的股票已经获得了很大的增长。 Vertex 股票的市盈率为15.6倍。
在北美、欧洲和澳大利亚，大约有2,000个基因突变导致了约75,000人的 CF 。许多具有最常见突变的人已经接受了 Vertex 疗法，这使得很难预测公司新的三重基因能产生多少新的增长。
Celgene 晚期的明星是嵌合抗原受体 T 细胞（ CAR-T ）疗法，它实际上是一个充满患者自身免疫细胞的 IV 袋，在经过非现场修饰后可以识别和攻击癌症。这是一个复杂的过程，似乎值得努力的非霍奇金淋巴瘤患者谁不再响应标准护理。在 JCAR017治疗6个月后，55%的患者完全缓解。
尽管 JCAR017的结果并不引人注目，但它们与另一个 CD19导向的 CAR-T 相比，后者的目标是相同的癌细胞靶标 Yescarta 。吉利德科学公司（ NASDAQ ： GILD ）在一年多前推出了这一疗法，用于同样数量有限的非霍奇金患者，到目前为止销售一直是一个巨大的失望。
看起来 Celgene 可能正在重新定位 JCAR017，以在路上寻找白血病迹象，而不是试图与 Gilead 争夺非霍奇金的患者。在未来有很多潜在的隐患的情况下，对这个项目的前瞻性估计应该用大量的盐。
每周注射 GLP-1是治疗2型糖尿病的一种流行的新方法，诺和诺德公司正在研制一种每天一次的 GLP-1片剂，它可能成为最受欢迎的。
Novo 公司自己的 GLP-1注射液在2018年前9个月产生了约28亿美元的销售额，公司依靠其保持在日益拥挤的市场中的份额。
口服精索可能会成为 Novo 的下一部大片。加上标准护理，使患者死于心血管疾病的风险降低了51%，此外，调查人员在对数千名患者的长期结果研究中测量了49%的全因死亡率降低风险。
如果你没有注意到，排在榜首的候选人也是唯一一个在2020年没有得到支持的候选人。尽管如此， Novo 公司的口服精液疗法比注射疗法有一个方便的优势，而且它还得到了令人印象深刻的结果数据的支持，这可能使它与众不同。如果我不得不选择一位最有可能超出预期的候选人，那就是 Novo 的人选。