Biotech Start-ups Research Articles

Characteristics of Drugs from Non-Global Companies for Hematologic Malignancies and Impact on Global Regulatory Approval.

The number of drugs developed by non-global companies, including biotech start-ups, has increased; however, their characteristics and impact on global regulatory approval are not well understood. Using a public database, we identified new molecular entities (NMEs) approved for hematologic malignancies in the US from January 2011 to December 2022. They were divided into those submitted by non-global companies (non-global group) and those by global companies (global group). We identified 48 NMEs, of which 19 (40%) were classified as non-global. Of these, 13 (68%) were from US-based companies. In the non-global group, 63% (12/19) of the NMEs had received accelerated approval in the US, of which only 50% (6/12) had a post-approval confirmatory trial by September 2023. Regarding the impact on the approval in the European Union (EU) and Japan, the unapproval rate of 2 years after US approval was higher in the non-global group than in the global group in the EU (56% vs. 21%) and Japan (94% vs. 64%). In conclusion, many NMEs from non-global companies had received accelerated approval in the US based on phase I/II trials. NMEs from non-global companies had a higher unapproval rate at 2 years in both the EU and Japan.

First Patient Dosed in First-in-human trial of AGX101, a Novel ADC targeting TM4SF1

A first patient has been dosed in a Phase 1 clinical study (NCT06440005) with AGX101, a novel TM4SF1-directed Nuclear-Delivered Antibody-Drug Conjugate™ (ND-ADC) therapy for the treatment of solid tumors. AGX101, which is being developed by privately held biotech startup Angiex, is based on a new, groundbreaking ADC technology platform with a unique mechanisms of action.

Bio-entrepreneurs' bugbear: Regulatory rigmarole.

Indian biotech startup sector - a rapidly growing business segment focused on the development of innovative products has the potential to make significant contributions to the country's economy. Indian bio-entrepreneurs' optimistic expectation of rapidly moving product development from bench to bedside faces tremendous challenges of the complex Indian regulatory system, which is shaped by a diversity of regulatory authorities, rules, guidelines, and processes. This brief review discusses specific regulatory issues faced by bio-entrepreneurs investing in a variety of innovative products - new drugs, vaccines, medical devices, cell, and gene therapy and suggests approaches which can ease Indian entrepreneur's endeavors.

Creating a capital-efficient organization: Accele BioPharma

Research methodology Case information was mainly acquired through interviews with Richard Gammans, chief operating officer. Dr Gammans was a visiting professor at Fayetteville State University for a year, and two of the case authors developed personal friendships with Richard. Interviews were conducted over a two-year period as the accelerator got started. In addition, one author conducted a team-building session with the management team and one of the bio-startup researchers. An interview was also conducted with Clayton Duncan, chief executive officer, to gain his agreement with developing the case. The Accele website included a write-up on each of the pharmaceutical startup companies. The write-up included a company summary, description of the science (disease and cure), the size of the market, results from testing, regulatory considerations and intellectual property. A literature review was conducted as the basis for the information on the pharmaceutical industry. Case overview/synopsis This case is about a biopharmaceutical accelerator founded in 2011 by two senior executives with experience in both large pharmaceutical companies and running biotech startup companies. The founders were successful in raising capital to start their first venture capital fund which they used to invest in four biotech startups. All four startups were working in very different disease areas. For example, one developed a drug to help with hearing loss that the department of defense was funding. Another of the startups discovered drug candidates that attack antibiotic-resistant bacteria. Biopharmaceutical accelerators were relatively new. They differed from business incubators because they invest in the startups and provide operational support, but the degree of support provided varies across accelerators. The Accele BioPharma accelerator operated in virtual, network type of organization, and Accele BioPharma provided primary strategic and operational management for the startups. The challenge in this case is to identify how the leaders managed the virtual network, and what additional resources were needed so that the management team could expand their ability to assist startups to get drugs approved by the food and drug administration. Complexity academic level This case is suitable recommended for undergraduate/graduate strategy, undergraduate/graduate organizational behavior, entrepreneurship and health-care management courses.

PHARMACEUTICAL INDUSTRY ANALYSIS: A COMPREHENSIVE REVIEW OF MARKET TRENDS, COMPETITIVE LANDSCAPE, AND FUTURE OUTLOOK

The pharmaceutical industry, a cornerstone of modern healthcare, undergoes constant evolution to meet the dynamic demands of the healthcare landscape. This comprehensive review delves into the multifaceted dimensions of the industry, encompassing market trends, competitive dynamics, and future projections. Market trends reveal a shift towards personalized medicine, leveraging advancements in genomics and biotechnology, alongside a growing focus on specialty drugs catering to niche patient populations. The prevalence of chronic diseases and an aging populace accentuate the necessity for innovative therapies, while the digital revolution introduces new paradigms in patient care and drug development, propelled by telemedicine, digital therapeutics, and artificial intelligence. Simultaneously, regulatory pressures and pricing constraints exert significant influence, necessitating adaptability among industry players. In the competitive landscape, established pharmaceutical giants vie for market dominance amidst patent expirations and generic competition, while biotech startups and niche players disrupt traditional models. Mergers and acquisitions serve as strategic imperatives for pipeline fortification and market expansion. Looking forward, the industry must navigate a terrain characterized by rapid technological advancements, evolving patient needs, and regulatory complexities, all while striving for innovation, accessibility, and sustainability in the delivery of healthcare solutions. Keywords: Pharmaceutical industry, healthcare landscape, market trends, personalized medicine, specialty drugs, chronic diseases.

Brazilian Northern biotech startups: an examination of supporting factors for web portal modeling

The purpose of this research project is to determine the elements that would facilitate the establishment of a biotechnology startup help site in the northern part of Brazil. As part of the research, data on startups was gathered, obstacles to growth were identified, and issues faced by the companies were noted. Based on the responses to the questionnaire, conclusions were made on the major difficulties these businesses encountered. The main barrier is found to be the difficulty in acquiring finance; other challenging elements include revenue generation and equipment availability. It is acknowledged that regional concerns, such input availability, and logistics, have a significant impact on the difficulties that local businesses encounter. Recommendations are made based on the challenges found to improve the startup growth environment, hence promoting sustainable innovation and the bioeconomy's development.

Rethinking new venture growth: A time series cluster analysis of biotech startups’ heterogeneous growth trajectories

Startups are crucial job creators and drivers of economic growth. Research on startups has predominantly targeted high-growth startups, while a comprehensive understanding of alternative growth journeys remains limited. Addressing this gap, we employ the theory of early firm growth and the time-calibrated theory of entrepreneurial action to examine 416 biotech startups. We use time series cluster analysis to unveil four heterogeneous new venture growth trajectories. These are characterized by unique timings, paces, and sequences of financial, human, and innovative resource-related activities. This study contributes to new venture growth research, particularly in science-based high-tech startups, with its nuanced understanding of diverse growth pathways, including intriguing notions of early failure, growth reversal, and high and moderate steady growth.

Attitudes Towards Patent Law Among Biotech Startups

The rapid pace of innovation in biotechnology necessitates effective patent protection to ensure competitive advantage and investment attraction for startups. However, the impact of patent law on the strategic operations and innovation within biotech startups is complex and multifaceted. This study aims to explore the attitudes of biotech startup leaders towards patent law, identifying the perceived strategic values, challenges, and the overall impact on innovation within their companies. This qualitative study employed semi-structured interviews with 25 leaders (founders, CEOs, and senior managers) from biotech startups across various global regions. The data was collected until theoretical saturation was reached and analyzed using NVivo software to facilitate thematic analysis. This approach allowed for an in-depth exploration of the nuanced perspectives on patent law in the biotech sector. Three main themes were identified: Strategic Value of Patents, Challenges with Patent Law, and Perceptions and Attitudes Towards Patent Law. Under these themes, key categories such as Competitive Advantage, Innovation Incentives, and Regulatory Navigation highlighted patents' strategic benefits. Conversely, significant challenges included Cost Implications, Complexity of Processes, and Intellectual Property Risks. Diverse perceptions were also noted, ranging from viewing patents as crucial to innovation to concerns over their potential to stifle technological progress and ethical considerations. The study concludes that while patents are critical for protecting innovations and securing economic benefits for biotech startups, they also present substantial challenges that can impede these companies' operational and technological agility. Balancing these benefits and drawbacks is essential for fostering an environment that supports sustainable innovation and ethical practices within the biotechnology industry.

Leveraging biotechnology design principles to advance brain understanding and improve biotechnological device efficiency and scalability

The field of Biodesign holds immense potential in transforming our understanding of the brain and enhancing the capabilities of biotech devices. Over the past decade, this interdisciplinary area has experienced rapid growth, driven by pioneering research, the rise of design-focused biotech startups, and the expansion of educational programs globally.

Approval Delay in Drugs from Non-Global Company in Europe and Japan: Analysis of Novel Drugs for Hematologic Malignancies

Background: Since most therapeutic agents for hematologic malignancies are developed and first approved in the US, it is important for each country to review and approve these agents without delay. Recently, the number of drugs developed by non-global companies including biotech start-ups has increased. Most of these non-global companies are located in the US, and typically do not have offices outside of the US. Thus, it is possible that drugs developed by non-global companies are likely to be approved later in countries other than the US, including the EU. However, their characteristics have not been fully evaluated. Study Design and Methods: Using the public database of the Food and Drug Administration (FDA) from January 2011 to December 2020, new molecular entity (NME) drugs for hematological malignancies that were approved in the US during the relevant period were identified. Cellular products, including chimeric antigen receptor (CAR)-T cells, were excluded from this analysis. NMEs for hematological malignancies were divided into two categories according to the applicant company: NMEs from non-global companies (non-Global group) and NMEs from global companies (Global group). Global companies were defined as those with branches or headquarters in all following regions: the US, the EU, and Japan. The others were classified as non-global companies. Based on the public databases of Japanese regulatory agency (PMDA) and European regulatory agency (EMA), we estimated the rate at which these drugs remained unapproved (unapproved rate) using the Kaplan-Meier method. The unapproved rate of NMEs was compared between the non-Global and Global groups. Results: A total of 39 NMEs for hematologic malignancies approved in the US during the study period were identified. Of these, 13 (33%) NMEs were from non-global companies (non-Global group) and 26 (67%) NMEs were from global companies (Global group). The proportion of orphan drug (13/13 vs. 26/26, p=1.000) and priority review designation (12/13 [92%] vs. 22/26 [85%], p=0.648) were not significantly different between the non-Global and Global groups. In the non-Global group, 12 (92%) NMEs were from the companies headquartered in the US, while 10 (39%) NMEs were from the US companies in the Global group. The proportion of pivotal phase Ⅰ/Ⅱ trial used for marketing authorization in the US was significantly higher in the non-Global group (11/13 [85%] vs. 13/26 [50%], p=0.045). The proportion of pivotal trials enrolling Japanese participants was lower in the non-Global group (0/13) than in the Global group (6/26 [23%]), while the proportion was similar in the EU (9/13 [69%] in the non-Global group and 16/26 [62%] in the Global group). In the EU, the unapproved rate 2 years after the US approval was higher in the non-Global group (54% vs. 22%), but the 5-year unapproved rate was rather lower than that in the Global group (15% vs. 22%) (Figure 1a). Similarly, in Japan, the unapproved rate of NMEs in the non-Global group was higher at 2 years (92% vs. 65%), but rather slightly lower than that in the Global group at 5 years (20% vs. 35%) (Figure 1b). After the US approval, 9 (69%) of the 13 NMEs filed by non-global companies had subsequently partnered with or been acquired by the global companies. Discussion and Conclusion: In both the EU and Japan, drugs in the non-Global group had a higher unapproved rate at 2 years. Notably, 54% of the drugs in the non-Global group were not approved in the EU, even though the participation rate in the pivotal trial used for US approval was similar between the non-Global and Global groups in the EU. However, at 5 years, the unapproved rate had decreased to approximately 20% in both the EU and Japan, presumably due to the fact that lots of the non-global companies were subsequently partnered with or acquired by the global companies.

Financing repurposed drugs for rare diseases: a case study of Unravel Biosciences

BackgroundWe consider two key challenges that early-stage biotechnology firms face in developing a sustainable financing strategy and a sustainable business model: developing a valuation model for drug compounds, and choosing an appropriate operating model and corporate structure. We use the specific example of Unravel Biosciences—a therapeutics platform company that identifies novel drug targets through off-target mechanisms of existing drugs and then develops optimized new molecules—throughout the paper and explore a specific scenario of drug repurposing for rare genetic diseases.ResultsThe first challenge consists of producing a realistic financial valuation of a potential rare disease repurposed drug compound, in this case targeting Rett syndrome. More generally, we develop a framework to value a portfolio of pairwise correlated rare disease compounds in early-stage development and quantify its risk profile. We estimate the probability of a negative return to be 80.8% for a single compound and 56.1% for a portfolio of 8 drugs. The probability of selling the project at a loss decreases from 79.2% (phase 3) for a single compound to 55.4% (phase 3) for the 8-drug portfolio. For the second challenge, we find that the choice of operating model and corporate structure is crucial for early-stage biotech startups and illustrate this point with three concrete examples.ConclusionsRepurposing existing compounds offers important advantages that could help early-stage biotech startups better align their business and financing issues with their scientific and medical objectives, enter a space that is not occupied by large pharmaceutical companies, and accelerate the validation of their drug development platform.

Success factors in drug discovery and development

The article aimed to give a personal perspective on drug discovery and development. The author has worked both in Big Pharma as a scientist and manager and more recently also in start-up biotech companies. Drug companies have played a major important role in improving population health and will continue to do so. The hurdles and costs for drug development have continuously risen without a parallel enhancement of productivity. There is no single explanation for this and the article outlines success factors and hurdles for effective drug development. Aspects of the external and internal environments that influence Big Pharma productivity is outlined and discussed.

Accelerating drug discovery process with genome editing technology from Tokushima

In a series of drug development processes from basic research to non-clinical and clinical trials, genome editing technologies have been making innovative breakthroughs. Genome editing using CRISPR/Cas9 system, which was awarded the Novel Prize in Chemistry in 2020, has greatly streamlined the production of genetically modified mice and cells, which have been used in a variety of drug discovery research and non-clinical trials. Setsuro Tech Inc. (Setsurotech) established in 2017 is a biotech startup originated in Tokushima University. In this paper, we will briefly review genome editing technology using CRISPR/Cas9 system, and then introduce our company, our fundamental technologies; GEEP method (Genome Editing by Electroporation of Cas9 Protein) developed by Takemoto et al., and VIKING method (Versatile NHEJ-based Knock-in using Genome Editing) established by Sawatsubashi et al. Also, we will introduce our contribution to the field of drug discovery research and industrial application of genome editing technology.

HPR83 Coming to America: How Ex-US Drug Companies May Best Prepare for the Complexities of a US Market Product Launch

To determine the unique US policy and market access nuances which must be addressed for ex-US biotech companies or startups to enter the US market. The aim of this study is to determine what ex-US biotech startups must understand and focus on within the US market access space to best position new drug launches, given the differences among landscapes. Additionally, this work provides a framework of required issues to explore during early decision-making process in a pre-launch setting.

Medical institutions in the geography of innovation: evidence from public tertiary hospitals and biotech start-ups in Chinese cities

ABSTRACT Concerning China’s growth in biotechnology, the research on biotech start-ups in China yields significant implications. However, studies have not given enough attention to hospitals, which are critical players in the biotech industry. Because of the hospital-centric healthcare system, public tertiary hospitals in China are important partners for biotech start-ups. This study investigates the geographical relationship between public tertiary hospitals and biotech start-ups in Chinese cities. The findings suggest evidence of collocation, which reflects their alliances. This research thereby contributes to the existing knowledge of the biotech industry by integrating the healthcare system into the location of biotech firms.

Complement-targeted therapeutics: An emerging field enabled by academic drug discovery.

Within a short few years, the number of complement inhibitors that are either approved for therapeutic application or evaluated in late-stage clinical trials has expanded remarkably. The sudden emergence of this target area in the pipelines of many biotech start-ups and even large pharmaceutical companies appears even more surprising when considering that the involvement of the complement system in various clinical conditions had long been recognized. In many aspects, however, the complement system is far from being a traditional drug target, which may explain the delayed breakthrough of this therapeutic strategy. While complement modulation is now considered an attractive "platform technology" with applications in a wide spectrum of disorders, the broad yet heterogeneous disease involvement of the complement system has long restricted its placement in traditional drug discovery programs. Concerns about the safety of complement-targeted interventions, the large number and high plasma concentrations of target proteins, and the complexity of the complement system's engagement in biological processes are among other factors that kept complement off the drug discovery radar for decades. Alongside technical advances and financial incentives, the innovation and persistence of academic and clinical researchers have been the critical driving force to navigate complement therapeutics out of the shadow into the spotlight. In this commentary, we document this remarkable development using select examples and aim to venture some predictions where this promising field may be headed to.

Global or local: The future of biotech

Biotech start-ups often begin as domestic companies relying on local resources and talent, but this approach might not be effective in achieving rapid growth and long-term success, particularly for developing new therapeutics that require significant resources and extensive commitment. Here, we argue that born-global biotechs are better equipped to tackle major industry challenges, such as innovation, resource constraints, and limited talent diversity, especially in current challenging times. We also highlight the importance of capital efficiency in maximizing the benefits of being a born-global biotech, and provide an operational framework, based on the FlyWheel concept, for becoming a successful born-global biotech.

Studying the governmental, industrial, and academic partnerships between incubation centers for furthering innovation: Evidence from biotechnology firms in Singapore

In Singapore, the number of incubation centers is rapidly increasing to fulfill the demands of the associated audience. These centers are vital in providing important facilities and capitals for new start-ups. This study aimed at identifying the governmental, industrial, and academic partnerships between incubation centers for furthering innovation in biotechnology firms in Singapore. For this purpose, semi-structured interviews were conducted with five employers from top management in biotechnology firms in Singapore. After the collection data, qualitative analysis was conducted in two steps (step 1: thematic analysis and step 2: word cloud analysis). The results obtained from this study showed that incubation centers play an important role in establishing early-stage biotech start-ups, promoting bio-entrepreneurship. It has also been observed that different challenges are faced during incubation process which include legal issues, infrastructure issues, strategic alliances issues and others. Moreover, the governmental, industrial, and academic partnerships between incubation centers have proven to be effective in promoting innovation processes in biotechnology firms.

How to Develop a Rare Disease Treatment

How to Develop a Rare Disease Treatment

Patient Advocacy Leads to Synthetic Antibodies for Mycobacteria

Patient Advocacy Leads to Synthetic Antibodies for Mycobacteria