We see continued strong underlying market growth of 6% for drugs based on small molecules1. The continuing role of small molecules is apparent in recent approval trends with 83% of 2019 approved FDA drugs being small molecules2. In 2019, early phase drug candidates based on chemical technologies have continued to grow driven by increased investment in new therapeutic treatments (e.g. oncology indications); streamlined regulatory approval processes; and demand for increasingly specialized medicines inclusive of orphan disease applications.
Specialty medications are usually defined as high-cost, high-complexity medicines. These are often associated with biologics although small molecules today still represent 60% of all innovative drugs3. The demand for these specialty medications continues to increase especially in highly developed areas such as USA, Europe and Japan. As a result, sales of specialty medications are growing at roughly twice the rate of traditional drug products whose growth rate is primarily driven by increased access to medicines in heavily populated countries such as China and India4. More than 25% of drug products in development are highly potent active pharmaceutical ingredients (HPAPIs)5. The highly potent nature of these active pharmaceutical ingredients (APIs) means that they are highly toxic and require specialized manufacturing and handling capabilities.
In addition, dosage form and delivery of complex small molecule drugs pose challenges including the prevalence of poor solubility in highly potent compounds. An estimated 70% of new compounds require an enabling technology to reach sufficient bioavailability6.
1 Source: Evaluate Pharma (2019)
2 Source: DCAT (2019)
3 Source: Evaluate Pharma (2019) (no generics or biosimilars considered)
4 Source: IQVIA/Midas (2019)
5 Sources: Internal Analysis; FDA (2019)
6 Sources: Internal Analysis (2019); PharmaCircle (2019)
We remain a global leader in the contract development and manufacture of drug substances or API, leveraging our expertise in more complex molecules, economies of scale, our many years of experience in manufacture, and the dedication and expertise of our employees.
We offer world-leading expertise and capabilities for the safe and efficient development and manufacture of HPAPI. With more than 20 years of experience in HPAPI, we have progressed more than 30 drug products to commercialization. Our specialized capabilities include handling HPAPI to exposure levels to 100ng/m3 across all manufacturing scales, as well as specific expertise in rapidly scaling up drug substances for commercialization.
Our HPAPI handling capabilities include contained processing for particle engineering as well as specialized drug product for highly potent/low dose applications, inclusive of liquid and semi-solid oral drug product as well as sterile fill and finish. This integrated offering adds substantial value to our customers as it simplifies interfaces, reduces costs and accelerates timelines. Lonza is an established partner in early development programs (preclinical through to Phase II) for drug products. The majority of early development is associated with addressing bioavailability and drug delivery challenges as well as accelerated development for clinical trials.
Our industry-leading bioavailability enhancement services portfolio includes all primary technologies for handling solubility, dissolution rate and/or drug delivery challenges. It also includes proprietary capabilities developed over 25 years. We have predictive modelling tools for rapid technology selection, specialized processing techniques and phase-appropriate equipment, which all complement our product development teams’ experience in meeting required bioavailability targets for new compounds or improving existing drug product performance.
Our Global Footprint
With a global network of eight sites in Europe, USA and China covering drug substance and drug product development and manufacturing, we are geographically aligned with the major growth drivers of the biopharmaceutical industry, e.g. small/emerging biopharma companies that hold the majority of the early phase pipeline for new molecules.
At our Visp (CH) facility, we have a well-established base for current good manufacturing practices (cGMP) chemical synthesis with more than 40 years’ track record in the production of highly potent active pharmaceutical ingredients HP(API) and their intermediates. The seven ISO-certified plants within the Visp complex provide more than 600m3 of reactor volume and a full range of capabilities across an array of chemical technologies to service customer requirements.
The Nansha-based (CN) production of drug substance complements our Visp site in ensuring high quality, secure supply of active ingredients to global biopharma customers.
In the USA, we provide particle engineering, bioavailability enhancement and drug delivery services from our sites in Quakertown, PA (USA), Bend, OR (USA) and Tampa, FL (USA). Additionally, in 2019, we completed the expansion of our solid oral dose development and manufacturing capabilities and capacity at our Tampa site. The expansion enables the team to provide more integrated services for customers across early-stage product development, clinical trial material manufacture and commercialization of innovative drug products. It will also further strengthen our speed-to-market capabilities.
Additional capacity for design, development and manufacturing services across drug products is available from our sites in Monteggio (CH), Ploërmel (FR) and Edinburgh (UK).
For further information about our offerings in small molecules, including our expertise in HPAPI and other active pharmaceutical ingredients, visit our website. Click here for a 360° Virtual Tour of our facilities. Visit our Capsugel and Micro-Macinazione websites.
Highlights and Initiatives 2019
Throughout the year our small molecule business continued to benefit from innovative business models (including tailored capacity optimized to customer needs), formulation and encapsulation capabilities. Lonza’s HPAPI offerings have made a positive contribution, with a number of new long-term contracts signed.
Drug Substance Development and Manufacturing
Our API/HPAPI offerings have positively contributed to our 2019 performance with a number of new long-term contracts signed. Examples include:
- AstraZeneca signing a long-term manufacturing agreement with us for the delivery of a number of HPAPI based products from our Visp (CH) site. To meet this increased demand, we will expand our Visp-based HPAPI development and manufacturing capacities, with planned operations to start from July 2020. This investment will add two 4m3-scale, multi-purpose production lines for HPAPI manufacturing to complement our existing range of production capacities from lab to large commercial scale.
- A previously-announced long-term agreement to produce drug substance payloads for antibody drug conjugates, was expanded in 2019 to accommodate higher production commitments by a major biopharmaceutical partner. The inherent expansion, which includes two new suites designed specifically for the production of ADC payloads, is based on a tailored agreement that ensures flexible and secure supply at reduced cost.
- Several substantial new contracts for manufacturing were also secured for our Nansha (CN) drug substance development and manufacturing site.
Leveraging our leading expertise in addressing bioavailability challenges, we launched SimpliFiHTM Solutions. This service package is designed for innovator companies, especially small/emerging companies that require development and manufacturing partnerships for early-stage development and first-in-human studies. This new offering consists of streamlined service agreement packages to further reduce time, cost and complexity for early clinical studies, demonstrating our continued commitment to constant innovation and adding value for our customers.
Drug Product Development and Manufacturing
The first long-term commercial supply agreement for a spray dried dispersion-based drug product was signed with a biotech company partner. Our Bend, OR (USA) site utilizes proprietary spray-dried dispersion technology to provide sufficient drug bioavailability to a highly insoluble kinase inhibitor drug substance. This agreement provides supply security to the client whose new drug product is expected to be granted regulatory approval in Q1 2020.
We signed an agreement with Chiasma for the commercial supply of their late clinical phase product Mycapssa®. Combining Chiasma’s technology with our liquid-filled hard capsule delivery systems provides the potential to improve the treatment options for adult patients with acromegaly. We have provided early stage development and specialized encapsulation services for this new orphan application.
We are providing commercial supply of a new drug product using our proprietary Xcelodose® Precision Powder Micro-Dosing Technology. This agreement is the first instance in which this specialized equipment, designed for supporting rapid clinical trial studies, will be used for the commercial supply of a specialized encapsulated drug product. Lonza has developed micro-dosing best practices through more than fifteen years’ experience across hundreds of compounds.
In addition, our team in Tampa, FL (USA) supported Takeda’s brigatinib program, a new oral, once-daily monotherapy for treating non-small cell lung cancer. We provided the product, method and process development support to meet accelerated timelines to bring this breakthrough treatment to patients.