Annual Report 2019: State of the Bio Industry
This annual compilation reviews new developments, trends and outlooks in areas such as biotechnology, biosimilars, biopharmaceuticals, biologics, biomarkers and biosimulation.
FDA approval deluge
The Food and Drug Administration set a U.S. regulatory agency record with 59 novel drugs and biologics approved during 2018. During the first five months of 2019, the FDA cleared for marketing potential game-changing therapies for biotechnology and biopharmaceutical companies as regulatory clearance was granted for a variety of first-in-class medicines, promising prescription drugs with blockbuster hype, as well as new indications for established brands.
The most expensive prescription product in the world was cleared for marketing by the U.S. FDA on May 24. Zolgensma represents the first gene therapy for pediatric patients with spinal muscular atrophy (SMA). Zolgensma (onasemnogene abeparvovec-xioi) is approved in the United States for the treatment of pediatric patients younger than 2 years old with SMA including those who are pre-symptomatic at diagnosis. The adeno-associated virus vector-based gene therapy is designed to address the genetic root cause of SMA by replacing the defective or missing SMN1 gene to halt disease progression with a single, one-time infusion. SMA is a muscle-wasting disease and leading genetic cause of infant mortality that affects about one in every 11,000 births.
Zolgensma represents the first approved therapeutic in a proprietary platform to treat rare, monogenic diseases using gene therapy. The Novartis drug is undergoing regulatory review in other major markets and is anticipated to receive regulatory approval in Japan and the European Union in 2019.
Zolgensma came to Novartis via the April 2018 acquisition of the clinical-stage gene therapy company AveXis for $8.7 billion. Novartis management saw AveXis and Zolgensma as an opportunity to transform the care of SMA and expand the Swiss company’s position as a gene therapy and neuroscience leader. The one-time disease-modifying therapy is projected to exceed $2 billion in annual sales during 2023.
The annualized cost of Zolgensma is $425,000 for five years, for a total of $2.125 million. “Zolgensma is a historic advance for the treatment of SMA and a landmark one-time gene therapy,” says Novartis CEO Vas Narasimhan. “Our goal is to ensure broad patient access to this transformational medicine and to share value with the healthcare system.”
May 24 was a big day for Novartis as the company also won U.S. marketing approval for another anticipated blockbuster medicine, Piqray, as the first treatment specifically for patients with a PIK3CA mutation in HR+/HER2- advanced breast cancer. Piqray (alpelisib, formerly BYL719) was approved by U.S. regulators in combination with fulvestrant for treating postmenopausal women – and men – with hormone receptor positive, human epidermal growth factor receptor-2 negative (HR+/HER2-), PIK3CA-mutated, advanced or metastatic breast cancer, as detected by an FDA-approved test following progression on or after an endocrine-based regimen.
PIK3CA represents the most commonly mutated gene in HR+/HER2- breast cancer; 40 percent of patients living with HR+/HER2- breast cancer have this mutation. Such mutations are associated with tumor growth, resistance to endocrine treatment and a poor overall prognosis. The kinase inhibitor Piqray targets the effect of PIK3CA mutations and may help overcome endocrine resistance in HR+ advanced breast cancer.
PI3K inhibitors traditionally have struggled to reach the marketplace, and even some of those successful in doing have been saddled with regulatory conditions such as a non-first-line setting approval and a black-box safety warning that hamper future market potential. For example, during 2018 Roche halted clinical development of taselisib after investigators reported “a slight, 2-month progression-free survival advantage for the drug – along with a sketchy safety profile common to the class – combined with fulvestrant hormone therapy in a Phase III study of metastatic breast cancer.” In another instance, Verastem won accelerated approval from the FDA in September 2018 for the PI3K drug Copiktra (duvelisib) for treating adults with relapsed or refractory chronic lymphocytic leukemia/small lymphocytic lymphoma after at least two prior therapies. In addition to the third-line setting approval, use of Copiktra is associated with a boxed warning.
A brighter future is anticipated for Piqray, which is the first novel drug approved under the FDA Oncology Center of Excellence Real-Time Oncology Review pilot program, which allows for faster evaluation of breakthrough cancer medicines. Novartis launched Piqray with a reported list price of about $15,500 for a 28-day supply.
“Within the next 2 years, Novartis expects 10 or more planned drug launches that could possibly reach blockbuster status,” says Terry Chrisomalis, who runs the Biotech Analysis Central pharma service on Seeking Alpha Marketplace. “I say the company is on the right track based on its two latest approvals. Zolgensma should definitely hit into high gear as it makes its way to the market. Piqray is an approved breast cancer drug that offers a more targeted approach to treating those breast cancer patients with the PIK3CA mutation.”
Pfizer’s Vyndaqel (tafamidis meglumine) and Vyndamax (tafamidis) received a green light from the FDA during May. The drugs were approved for treating cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular mortality and cardiovascular-related hospitalization. The two oral formulations of the first-in-class transthyretin stabilizer tafamidis represent the only medicines approved by the FDA for the treatment of ATTR-CM.
Pfizer has touted tafamidis as a potential blockbuster medicine. Industry trackers have projected annual sales to exceed $1 billion in 2024, with a potential peak amount of $2 billion.
“Once rejected by the FDA, Pfizer’s tafamidis is back and ready to disrupt a rare disease field only recently tapped by Alnylam and Ionis. And the pharma giant’s entrant, now dubbed Vyndaqel, comes with a much lower price tag,” FiercePharma anaylsis says. “The oral drug will bear a list price of $225,000 per year … That’s far lower than the $450,000 list price both Alnylam and Ionis are fielding with their rival meds Onpattro and Tegsedi, respectively. And the Pfizer meds are pills where its competitors are both injections.”
Biopharma company AbbVie received FDA clearance in May for Venclexta (venetoclax) in combination with Gazyva (obinutuzumab) for previously untreated patients with chronic lymphocytic leukemia or small lymphocytic lymphoma. The FDA granted Breakthrough Therapy designation for the combo therapy, and early filing of the clinical data was provided under the Real-Time Oncology Review pilot program, which resulted in approval in just over two months following submission of the complete application.
Venclexta was initially granted accelerated approval by FDA during April 2016 for the treatment of patients with CLL with 17p deletion, as detected by an FDA-approved test, who have received at least one prior therapy. Global sales approaching $3 billion in 2024 have been forecast for Venclexta, which is developed by AbbVie and Roche. The product is jointly commercialized by AbbVie and Genentech – a member of the Roche Group – in the United States and by AbbVie outside of the U.S.
Abbvie also won a significant marketing approval from the FDA in April at a time during which the company’s blockbuster psoriasis medicine Humira – the world’s top-selling prescription drug – is facing patent pressures and biosimilar competition outside the United States. Approved for marketing in Canada and Japan earlier in 2019, Skyrizi (risankizumab-rzaa) received U.S. approval on April 23 as a treatment for plaque psoriasis. The interleukin-23 inhibitor was cleared by U.S. regulators for the treatment of moderate-to-severe plaque psoriasis in adults who are candidates for systemic therapy or phototherapy. Skyrizi is part of a collaboration between Boehringer Ingelheim and
AbbVie, with the latter leading development and commercialization of the new product globally.
Industry analysts believe that Skyrizi should benefit from best-in-category efficacy (i.e. market share gains) and continued rapid market expansion. The product is predicted to generate between $500 million to $1 billion in the second year of launch, which began in May 2019 in the United States. Clarivate Analytics’ Cortellis Competitive Intelligence database projected 2023 sales of $1.74 billion.
Mega-merger: Bristol-Myers Squibb and Celgene
Bristol-Myers Squibb Co. rang in 2019 by entering into a definitive merger agreement to acquire Celgene Corp. in a cash and stock transaction with an equity value of $74 billion. The transaction will create a leading focused specialty biopharmaceutical company well-positioned to address the needs of patients with cancer, inflammatory and immunologic disease and cardiovascular disease via high-value innovative medicines and leading scientific capabilities.
The transaction remains on track to close during third-quarter 2019, subject to the satisfaction of customary closing conditions and regulatory approvals. Bristol-Myers Squibb’s completed purchase of Celgene would represent the second-largest acquisition ever in the pharma industry, trailing only Pfizer’s takeover of Warner-Lambert during 1999.
The Bristol-Myers Squibb-Celgene combination is expected to create the leading oncology company and a top-five immunology franchise with strength in solid tumor and blood cancers. Yet, some Wall Street analysts have questioned if the combination – which the companies said would create $2.5 billion in cost savings and raise earnings – would solve separate challenges that have been facing Bristol-Myers Squibb and Celgene.
“Bristol’s most important cancer immunotherapy and growth driver, Opdivo, has lost much of its luster as Merck & Co.’s (MRK.N) rival drug Keytruda seized dominance in advanced lung cancer, the most lucrative oncology market,” according to a Reuters report when the acquisition was announced. “Meanwhile, Celgene has endured high-profile clinical failures and the U.S. exclusivity on its flagship multiple myeloma drug, Revlimid, starts being phased out in 2022.”
More positive analysis arrived at the end of May when The Motley Fool reported: “Goldman Sachs Picks 4 Big Pharma Stocks to Buy,” with one of those stocks being Bristol-Myers Squibb (NYSE: BMY). According to the analysis from Goldman Sachs, “ … why is Bristol-Myers Squibb stock selling for such a large discount to its peers – less than 15 times trailing earnings? Some investors worry that it’s a “value-trap,” reports StreetInsider, with the company having just anted up $74 billion to acquire Celgene – just as the latter is facing the prospect of its Revlimid patent expiring. Goldman Sachs, however, is looking forward to Bristol-Myers’ Opdivo lung cancer treatment becoming a “front-line” drug in the war against cancer, and predicts positive data from clinical trials will emerge later this summer. Pointing out that Bristol-Myers stock sells at a “significant discount” to other big pharma companies, Goldman argues that it’s too cheap, and its multiple to earnings is bound to expand — transforming from $47 a share to $54 within a year.”
The U.S. FDA on May 28, 2019, approved Revlimid (lenalidomide) in combination with a rituximab product (R²) for treating adults with previously treated follicular lymphoma or marginal zone lymphoma following Priority Review designation. This represents the first FDA-approved combo treatment regimen for patients with these indolent forms of non-Hodgkin’s lymphoma that does not include chemotherapy.
According to a FiercePharma commentary, SVB Leerink analyst Geoffrey Porges was impressed with the 39-month profession-free survival benefit when the data were unveiled during November 2018. R2’s performance “appears to be superior” to what is expected from immunotherapy-chemo combinations based on past results, Porges said in a note to clients at that time. He estimated that the medicine could add another $600 million in yearly revenue from the indication.
“Celgene recently fended off a Dr. Reddy’s bid to invalidate three Revlimid patents at the U.S. Patent and Trademark Office, a key win for the megablockbuster,” according to FiercePharma. “Revlimid pulled in $9.7 billion last year.” Revlimid sales for first-quarter 2019 totaled $2.58 billion, an increase of 15 percent year-over-year. U.S. sales of $1.69 billion and international sales of $891 million rose 13 percent and 19 percent versus Q1 2018, respectively. The product’s sales growth was driven by increases in treatment duration and market share, Celgene reported.
Vantage analysis: Time to restock big pharma’s leaking pipeline
“Biotech develops and big pharma buys; the industry model has long been thus.” But a recent analysis by Vantage suggests that this model is changing. According to the industry trackers, while pharma’s late-stage pipeline is dwindling, there has been a jump in the total number of phase III assets being developed by big biotech, and this surge is even more pronounced among smaller companies.
“The total number of pivotal projects at big pharma groups has shrunk across the board, so it is not as if in-house assets are picking up the slack. If the heavy hitters cannot develop drugs themselves, perhaps it is time for them to get their checkbooks out again to restock their pipelines,” Vantage analysts reported in May.
“However, in the current moribund M&A market this might be easier said than done. Bigger groups continue to bemoan the overblown valuations of their would-be targets, while smaller companies, flush with cash, are happier than ever to go it alone.”
According to Vantage, perhaps larger groups are going for quality over quantity. Another recent analysis by the group reveals that products gained via acquisitions were responsible for a growing proportion of U.S. drug sales, far outstripping the contribution from those developed organically.
“This analysis involved already approved products, so there will be a lag before it becomes clear whether this trend holds for projects currently in phase III. If the current lull in acquisition activity translates into declining sales this could push big pharma back towards the deal-making table.”
Vantage analysis says, “There are still deals to be done, but acquirers are increasingly looking for more reasonably priced, earlier-stage assets. For example, Merck’s takeout of Peloton cost the bigger group a cool $1 billion, but in making its move before Peloton went public, Merck might have saved itself money in the long run.” In May, Merck agreed to acquire Peloton Therapeutics in a move that will bolster the Kenilworth, N.J.-based corporation’s oncology pipeline in a pact that could total $2.2 billion in value.
The analysts say this move towards earlier deals could suggest something else: that the phase III assets still belonging to smaller groups are not worth acquiring. Many biotech firms have struggled with solo launches, though some, such as GW Pharmaceuticals and Insmed, “have made a better fist of the job,” according to Vantage.
“While venture money remains plentiful, and smaller players are not under pressure to sell out, these groups could end up hanging on to even more late-stage projects.”
Biotechs strive to provide patients with more affordable treatment options
Biotech companies are rapidly accelerating their pipelines to develop advanced treatments and therapies to help effectively treat cancer or ultimately even eradicate the disease. Biotechnology firms are heavily investing in clinical trials and studies to develop effective drugs.
The global oncology/cancer drug market is predicted to reach $176.5 billion while registering a CAGR of 7.6 percent from 2018 to 2025, based on data from Allied Market Research.
According to FinancialBuzz.com, the advancement of early screening technology has enabled patients to receive therapy before their cancer worsens. Targeted therapy is additionally becoming a popular treatment because it specifically targets cancerous cells without damaging blood cells. Meanwhile, there is a growing demand for personalized medicine, which is expected to create new opportunities within the marketplace. Personalized medicine tailors treatment towards the patient’s characteristics such as his or her molecular structure and genetic profile.
As pointed out by FinancialBuzz.com, the biggest hindrance is the cost of certain therapies. For an average patient, it is difficult to obtain the monetary funds needed to pay for effective treatments. For example, the U.S. Food and Drug Administration in August 2017 approved Novartis’ Kymriah (tisagenlecleucel) to treat patients with acute lymphoblastic leukemia, a drug that genetically re-engineers the patient’s own T-cells to attack leukemic cells. As the first cell-based gene therapy approved in the United States, Kymriah is priced at $475,000 for a course of treatment.
Meanwhile, at a U.S. annualized cost of $425,000 per year for five calendar terms, Zolgensma becomes the world’s most expensive medicine. “We have used value-based pricing frameworks to price Zolgensma at around 50% less than multiple established benchmarks including the 10-year current cost of chronic SMA therapy,” according to Novartis’ CEO. “In addition, the price of Zolgensma is expected to be within the range of traditional cost-effectiveness thresholds used by ICER when updated for its full labeled indications. We believe by taking this responsible approach, we will help patients benefit from this transformative medical innovation and generate significant cost savings for the system over time.”
Comparatively, Biogen’s SMA medicine Spinraza was approved in December 2016 and has a price tag of $750,000 for the first year of treatment and then costs $375,000 annually for the remainder of a patient’s life. (Editor’s note: For more details about the pricing of Zolgensma and Spinraza, please see the Payer Access cover story).
The industry is undergoing a new era in medicine where groundbreaking therapies can emerge and cure patients in a single treatment – but at high costs.
Biotech companies are tasked with developing effective treatments, but also at an affordable price. “Ultimately, I want to access the best available therapies for the people I treat: the ones most likely to bring meaningful improvements in their quality and length of life, and the ones that reduce the toxicity associated with treatment,” stated Ajay Aggarwal, Oncologist at Guy’s and St Thomas’ NHS Trust in London. “Any new cancer therapy, drug or not, should undergo robust evaluation for outcomes that truly matter to individuals. As it is, limited finances are too often being directed from evidence-based therapies to those that promise false hope.”
FinancialBuzz.com says biopharma/oncology companies making progress on the R&D front include SourcingLink.net (OTC: SNET), Atossa Genetics (NASDAQ: ATOS), Agenus (NASDAQ: AGEN), Sunesis Pharmaceuticals (NASDAQ: SNSS), and OncoMed Pharmaceuticals (NASDAQ: OMED).
Progress on the biosimilars front in Canada
The Government of British Columbia announced in May the Biosimilars Initiative, which will transition patients who use specific reference biologic drugs for some rheumatology, dermatology and diabetes indications to a biosimilar within a six-month period. The program is anticipated to save an estimated C$96.6 million ($71.9 million) during the first three years, and could improve access to prescription medicines for patients in the province.
The Biosimilars Initiative changes PharmaCare coverage for specific biologic products. During the May 27-November 25, 2019, transition period, PharmaCare will cover originator and biosimilar versions of the affected medicines to allow patients time to inform themselves and start the transitioning process with their prescriber. Effective Nov. 26, PharmaCare will only cover the biosimilar versions of the medicines identified for the affected indications.
The new policy targets two arthritis medicines – Janssen’s Remicade and Amgen’s Enbrel – and Sanofi’s long-acting insulin Lantus. The first-of-its-kind in a Canadian public plan could pave the way for similar programs across the country.
The BC will also begin covering two newer biologics: Boehringer Ingelheim and Eli Lilly’s diabetes drug Jardiance and Lilly’s Taltz, which treats psoriatic arthritis. The policy is expected to benefit drugmakers with covered biosimilars: Merck, Sandoz, Pfizer and Lilly, according to a Reuters report.
Public and employer-sponsored benefit plans want to provide patients with access to innovative new treatments. Biosimilar medicines present a significant way to manage costs while additionally supporting positive patient outcomes.
Biologic medicines have revolutionized the treatment and prevention of many disabling and life-threatening diseases during the past 50 years, but they are very expensive and are an important driver of rising prescription drug costs. Only 1.7 percent of Canadian prescriptions were filled with biologic medicines in 2018, yet the cost of these prescriptions represented 29.9 percent of Canada’s total prescription drug costs last year.
Although the use of biosimilar medicines has been well-established in Europe with more than 700 million patient days of use during the past decade, the uptake of these medicines in Canada for chronic treatments has been much slower as public and private payers have not actively implemented policy levers to support their use beyond naïve or new patients. Canadian payers are recognizing that enhanced policies are necessary to fully realize the value of biosimilar drugs.
“Switching or transitioning patients from original biologic treatments to their corresponding biosimilar medicines is the responsible choice for those who manage drug budgets, and will help to ensure patient access to essential treatments for years to come,” says Jim Keon, president of the national association Biosimilars Canada.
“The province’s announcement underscores the value biosimilars can provide to patients and their physicians once a reference biologic medicine has lost market exclusivity, and we urge other provinces across the country to follow BC’s lead,” states Jennifer Chan, a board member of the Canadian Biosimilars Forum and VP, Policy and External Affairs at Merck Canada.
Global cancer biomarkers market projected to reach $32.25 billion by 2026
Analysis from Reports and Data shows that the global cancer biomarkers market was valued at $12.14 billion during 2018 and is predicted to reach $32.25 billion by year 2026, at a CAGR of 12.75 percent.
According to the Centers for Disease Control and Prevention, about 16,000 men and 8,000 women die from liver cancer in the United States per year. Cancer biomarkers are the molecules released from cancerous tumors that are found in blood and other tissues, which can be detected by diagnosing, screening and prediction for the progression of the disease.
Some of the primary growth stimulants for the market between 2018 and 2026 are expected to be the development of advanced genomic analysis technique, introduction of effective guidelines for biomarkers manufacturing, an immense amount of research by Cancer societies and proven effectiveness of transplants. Also, development of products from research institutes has been propelling growth of the cancer biomarkers market. On the other hand, certain manufacturing and pharmacological issues as well as regulatory hurdles are hindering marketplace growth.
Reports and Data analysis shows that North America is expected to account for 39.1 percent of the worldwide cancer biomarkers market through to the presence of high-quality healthcare equipment. Europe represents the second-largest region with a 28.5 percent share because of high cases of cancer in France, Ireland, etc. The cancer biomarkers market is fastest growing at a CAGR of 7 percent in Asia Pacific due to high awareness about biomarkers and an increase in aging population and rising incidences of cancer.
The market for lung cancer disease type segment (i.e. small cell lung cancers, non-small cell lung cancers) is expected to hold the fastest-growing CAGR due to growth in the smoking population and because of very low air purity. Genetics biomarkers is tracking to be second leading segment with a market share of 20.8 percent due to high usage and perfect accuracy rate. The immunoassays segment is projected to have the highest CAGR among other application types. The prognostics segment represents the second-largest share in this market, which is valued at about $3.44 billion, due to maximum population are aware about the new technologies.
Global biosimulation market to grow by $1.42 billion during 2019-2023
Technavio’s May 2019 market research report on the global biosimulation arena predicts that the market will experience a CAGR of more than 14 percent during the 2019-2023 forecast period.
The cost-effective nature of generics and patent expirations are factors contributing to the growth of the worldwide biosimulation market, according to Technavio. As patent expirations create significant opportunities for the entry of generic products, workshops are being conducted for advancing the use of modeling and simulation for modernizing the development of generics. These workshops pertain to the identification of opportunities for modeling and biosimulation in the drug development process, per the market research report. Additionally, pharma companies and CROs are leveraging machine learning tools, quantitative risk modeling, and systems pharmacology to improve the overall drug development process and regulatory decision-making. With an increasing need for stakeholders to use quantitative approaches in the process of generic drug development, the global biosimulation market is anticipated to witness a surge during the next few years, according to Technavio analysis.
Pharma and biotech companies emerged as the largest end-user segment, per the Technavio report. The increasing focus on applications such as high content screening and the use of biosimulation software and services for drug discovery and drug development are driving the segment’s growth. These companies are primarily involved in the discovery and development of drugs for the treatment of complex diseases. These companies are additionally using biosimulation software to simulate the physical reaction of patients towards new drugs to help in evaluating the potential of new drugs as well as enabling better design pivotal studies. This process is expected to further drive the biosimulation market during the 2019-2023 period.
“Manufacturers in the biosimulation industry are witnessing significant demand for biosimulation software and services due to significant investments in R&D,” says a senior research analyst at Technavio. “Biosimulation software and services allow the selection of appropriate drug doses and evaluation of new drug formulations. It also helps in designing optimal clinical trials and predicting drug-to-drug interactions.”
Asia is projected to generated the fastest growth in the global biosimulation market due to significant revenue contributions from emerging economies, including China and Japan. Several factors such as a huge population base, a considerable amount of pharma companies, and CROs working on drug discovery and development outsourced by companies in Western countries will positively impact market growth in the next few years according to Technavio. In addition, the growing amount of conferences aimed at promoting the efficiency of biosimulation techniques will further increase the awareness of biosimulation and its application among different stakeholders, including industry experts, academic institutes and research organizations. As a result, the growing adoption of biosimulation across advancing economies will lead the region to account for the fastest growth during the 2019-2023 time frame.
The importance of specialized research experts in niche biopharma
The ability of a biopharma company to successfully develop experimental drug candidates is mostly dependent on the experience of their research and management teams. As such, the hiring of specialists with decades of experience can make or break future drug candidacy projects, according to Microsmallcap.com. Many big players in the biopharma space, including Pfizer Inc. (NYSE: PFE), Johnson & Johnson (NYSE: JNJ), AbbVie Inc. (NYSE: ABBV), Thermo Fisher Scientific Inc. (NYSE: TMO), and ImmunoPrecise Antibodies Ltd. (OTC: IPATF) (TSX-V: IPA) have been competing for some of the leading minds in the sector.
According to Microsmallcap.com market commentary presented by FN Media Group, “Stock prices of biotech firms are known to jump and crash on the slightest news. Companies invest significant resources in developing specific compounds and drugs as well as undergoing clinical tests and regulatory approvals. These businesses that don’t have sufficient cumulative expertise are much less likely to succeed in the already tricky drug discovery process than those that do. As such, individuals with highly specialized biopharma backgrounds are fought over by major businesses who know full well the importance these experts play in the success of their future drug candidates.”
The market commentary explains that while experienced management teams are essential for every business, this is especially true in certain highly specialized sectors. The amount of technical knowledge necessary in industries such as biopharma means that professionals not frequently hired as executives (ie; doctors, professors, and research experts) often play significant roles in management. According to statistics, roughly 80 percent of all CEOs in the biotechnology industry have direct experience working in the medical or pharma industry, often at first in non-managerial roles.
Sinclair Dunlop, Managing Partner at Epidarex Capital, says “a critical success factor in any life science investment is the management team. The sourcing, development, and retention of experienced talent has a direct impact on technology development and investor returns. A significant challenge, particularly in under-ventured markets, is a shortage of serial CEOs with a track record of fundraising at scale.”
Success rates for biotech companies are already quite low, and in some niche sectors, only one in 200 projects produces a viable drug candidate. Per Microsmallcap.com, this issue is further compounded by the struggle to find specialized experts in many of these fields. “For smaller biotechnology companies, having the right person with specialized expertise can make or break future drug development projects. With the growing trend of large companies outsourcing R&D of specific projects to contract research organizations, there is an ever-growing demand for experts in specific hot fields like gene therapy, biologics, and cancer immunotherapy.”