In the clinic, much of the work is carried out by vendors on a clock: the CRO is enrolling patients, the CDMO is making the next batches, the agency expects safety reports and answers on schedule, and investors are waiting on the readout. The decisions stay with you, and the team that got the program into the clinic is now making them while running it.
We work alongside your team where it needs added leadership or capacity: leading the clinical function, managing the study and its vendors on your side, and keeping clinical, regulatory and CMC plans moving together.
We also give you an independent read of your own data. Optimistic interpretation of early results is a familiar route to a failed later-stage trial, and a clinical lead who is not invested in a particular answer can raise a concern while the plan can still change.
Since 2009, we have worked with clinical-stage companies from private biotechs with a first program in Phase 1 to listed companies preparing for approval. Earlier in development? See our support for early-stage and virtual biotechs.
Facing a leadership gap, a new study or an agency meeting? Talk to us about it.
Why Alacrita
People who have held the role
Alacrita associate consultants average 20+ years of experience and have been chief medical officers, clinical operations leads and regulatory leads at biotechs, so they can take on work on your program without a long handover.
Work that follows the program
Clinical-stage clients often bring us back as the program moves on. One oncology company engaged us for interim medical leadership and later for advice on manufacturing its clinical batches.
Clinical, regulatory and CMC in one plan
When a study raises a question outside the clinical team's lane, such as an agency query or a delay at the manufacturer, the specialist joins the same engagement and works from the same plan.
Clinical operations support for an oncology company's first clinical study
Clinical-stage biotech · Oncology
Client situation
An oncology company preparing to take its lead program into the clinic wanted experienced clinical operations support for its first study.
What Alacrita did
Following an earlier regulatory strategy project, Alacrita provided senior clinical operations consultants who have worked with the company's team through study start-up and into the conduct of its Phase 1 trial, over more than two years.
Where We Typically Support
Clinical-stage companies usually bring us in at one of a few points. An engagement can start with one of these and continue as the program moves forward.
A gap in medical leadership
Your chief medical officer has left, a search is running longer than planned, or the program needs CMO-level judgment before a full-time hire makes sense.
Your first trial with a CRO, or a larger one
The study needs someone on your side managing the CRO, the sites and the budget, and your team is already stretched.
Moving into the next phase
Phase 1 data are coming in and the questions turn to dose, indication and the design of the next study. This is the point to set the criteria the next result has to meet.
Before an agency interaction
A pre-IND, end-of-phase or scientific advice meeting, or a filing in a new region. We can prepare the briefing package, questions and company positions, and attend the meeting with you.
Manufacturing for later-stage trials
Larger trials need more material and sometimes a new process or manufacturer, on a timeline set by the clinic. We can plan the CMC work against the trial and oversee the CDMOs doing it.
How We Support Clinical-Stage Companies
Our work with clinical-stage companies centers on four areas, each described in full on its own page. Many engagements combine two or more.
Medical Leadership
When a chief medical officer leaves mid-trial or a search runs long, an interim or part-time CMO can lead the clinical team, oversee safety, and represent the company with the board, investors and regulators. Some engagements start as a defined project and continue as interim leadership.
Clinical Operations
We can lead clinical operations on your side of the CRO relationship, from choosing the CRO and negotiating the contract to managing the study through database lock. The CRO conducts the study; our lead manages it for you and brings decisions to you when they are needed.
Clinical Strategy
Before the next study, we can build or review the development plan, design the protocol, and agree the go/no-go criteria the data will be judged against before the data arrive.
Regulatory and CMC
Agency meetings and manufacturing run on the clinic's timeline. We can prepare meetings and filings, manage CMC work and CDMOs, and plan both against the trial so neither holds up the next study.
Selected Engagements
Examples of our work with companies running clinical programs.
Clinical-stage biotech
A biotech with two programs in Phase 1 needed medical leadership for both studies during a change in its chief medical officer.
An Alacrita physician served as medical director and sponsor medical lead for both programs, reporting to the CEO. He was the medical contact for investigators and the project teams, reviewed protocol amendments and regulatory filings, and worked with the company's clinical operations, regulatory and nonclinical teams, its CROs and outside clinical experts.
Clinical-stage biotech · Small molecules · Oncology
After earlier work together, an oncology biotech engaged Alacrita to provide an interim chief medical officer for its clinical program.
The company later brought Alacrita back to advise on manufacturing GMP batches for its clinical trials at its CDMO.
Clinical-stage biotech · Infectious disease
A biotech moving its lead program from early clinical studies into a randomized, placebo-controlled Phase 2 trial needed medical monitoring and statistical support.
Alacrita provided a medical monitor and biostatistics support. The statistics work covered the Phase 2 design and sample size justification, review of the protocol's statistical sections and the statistical analysis plan, and support in the company's meetings with regulators on the trial design.
Biotech · Antibody · Inflammation and fibrosis
A biotech had completed a Phase 1 single ascending dose study of its lead antibody in healthy volunteers and planned a Phase 2a proof-of-concept study. It wanted the regulator's view on whether the protocol and supporting data would allow the trial to proceed.
Alacrita managed the scientific advice process: strategy, the meeting request, draft questions and company positions, the briefing package and its submission, meeting attendance and minutes. The agency found the protocol and supporting data acceptable.
Biotech · Oral protein · Endocrine and metabolic
A company with an oral form of an approved biologic had a Phase 2 trial in an orphan indication underway in Europe and, after a pre-IND meeting with FDA, planned trials in the US.
Alacrita prepared the CMC, nonclinical and clinical sections of the IND from the existing clinical trial application, assembled the administrative information and source documents, managed publishing and submission, and prepared responses to FDA's questions.
If your program is at a similar point, we can talk through how we'd approach it. Get in touch.
Related Reading
Alacrita's writing on the decisions clinical-stage companies face.
Related Case Studies
ISO13485 Audit by medical device consultant
Gene therapy preclinical, CMC, & regulatory strategy
Assessing a new inhalation device and assisting with partnering
Clinical and regulatory support for respiratory delivery specialist
Frequently Asked Questions
Timing, scope and how an engagement works.
Alacrita is often brought in at a transition: a gap in medical leadership, a first or larger trial with a CRO, the move into a new phase, an upcoming agency meeting, or a manufacturing scale-up. Companies also engage Alacrita for a single question, such as a protocol review.
Around the decisions and work products you need. Alacrita's proposal sets out the scope, the work products and who will do the work. A protocol review is a bounded piece of work, while interim leadership or a clinical operations lead run longer and can be revisited as the program changes. There is no fixed package.
Yes. Alacrita's consultants work with your team and your vendors, and can take on a defined part of the program or support the people already doing it. You keep final authority over the program's decisions.
Yes. Alacrita engagements with clinical-stage companies often extend or change shape: a project can lead to interim leadership, and a clinical engagement can add regulatory or CMC specialists when the program needs them.
Have Something Coming Up in the Clinic?
Whether it's leadership through a transition, a study about to start or an agency meeting on the calendar, a short first conversation can tell you whether we're the right people for it.
The below list of recent projects can help illustrate the type of support we typically provide to biotechs:
Product Development
- Virtual Stewardship of a Targeted Cancer Therapy to an IND Submission: An early-stage biotech company developing a targeted cancer therapy engaged Alacrita to provide expertise to help them navigate an upcoming IND submission. An Alacrita partner and medical oncologist with over 25 years of industry experience in drug development was integrated virtually into their team as interim CMO.
-
Medical oversight support for oncology clinical trials: A listed biotech company conducting Phase III clinical trials in an oncology indication needed Chief Medical Officer support for multiple clinical activities in Europe and the United States.
-
Indication strategy for reformulated chemotherapy: A biotech company developing improved versions of available chemotherapy drugs approached Alacrita for indication strategy support, seeking to sanity check and refine its current clinical development strategy and products' differentiation and clinical placement, as well as brainstorm other avenues to pursue.
Click here for more information on our Product Development services
Strategy & Commercialization
- Commercialization options and roadmap for orphan drug: A publicly-listed biotech company preparing for commercialization of its lead asset had retained Alacrita to sketch out the relevant options for its European go to market strategy and to define the critical activities that needed to be undertaken in the ensuing pre-launch 18-24 month period.
- Indication opportunity assessment for a gene therapy: An early-stage, venture-backed, private biotechnology company developing a novel multi-gene therapy approach to addressing chronic, age-related diseases requested an opportunity mapping assessment and target indication prioritization to identify the optimal indication strategy for development and commercialization of the therapy.
- R&D pipeline planning for gene therapy company: A biotechnology company developing proprietary synthetic biology technology in gene therapy expression for several clients was looking to evolve its business model. The company engaged Alacrita to find a way to build their own internal cell and gene therapy (including gene editing) pipeline and to put together a plan in order to establish medical indications the technology could be applied to.
- Opportunity mapping for hemojuvelin antibody: A US-based biotech company developing a novel biologic which impacts iron metabolism, wanted help deciding which disease and patient groups would make the best lead indication for development and commercialization.
Click here for more information on our Strategy & Commercialization services
Business Development
- Due diligence on a novel T cell therapy and discovery platform: A VC investor wished to make an investment in a California-based preclinical biotechnology company with a discovery platform and novel T cell therapies for cancer and other diseases. Alacrita was commissioned to use its experience in cell therapeutics to perform a technical due diligence on the company to highlight any key risks and to propose mitigation strategies.
- Valuation of a re-purposed combination drug for Alzheimer's disease: Valuation of a re-purposed combination drug for Alzheimer's disease.
-
Valuation of a cancer therapy for licensing deal: A biotech company developing small molecule drugs for oncology was in the process of licensing negotiations with a pharma company for its lead asset. The asset, set to enter the clinic in the next year, was being developed for four CNS cancer indications. The company asked Alacrita to develop a current and future valuation of the lead asset and model various deal term scenarios to guide ongoing licensing discussions.
-
Screening for rare disease licensing opportunities: Our client, a commercial stage biotech company focused on rare and ultra-rare diseases was seeking to build its pipeline of development products. Whilst it had an established business development function in North America, it had limited business development capacity in Europe and therefore a more limited awareness of and reach into the academic, disease advocacy and early stage biotech networks in Europe.
Alacrita was engaged to assist in identifying promising drug candidates, technologies and research capabilities which offered transformational potential in a selected group of rare and ultra-rare diseases.
Click here for more information on our Business Development services
Case studies
I. Valuation of a re-purposed combination drug for Alzheimer's disease
Challenge: A clinical stage biotech company developing treatments for Alzheimer's disease was looking for a valuation of its Phase II-ready therapeutic drug. The drug was a re-purposed combination of two known drugs with a well-documented safety profile, however unlike most other Alzheimer's drugs in development, it was part of a small pool of pipeline drugs that claimed to be disease-modifying, and in particular its primary aim was to stop disease progression at the prodromal/mild disease stage.
The company was looking for Series A financing of £20m in venture investment in order to progress the drug through a Phase II clinical trial. To prepare for discussions with investors, Alacrita was asked to develop an independent valuation of the drug.
Solution: Given the historical failures of Alzheimer's drug development and therefore lack of benchmarks for probability of technical success, developing a risk-adjusted valuation for this product was particularly challenging. Alacrita's Monte Carlo approach to pharma valuations proved to be especially useful in this case, as we were able to develop a valuation range that truly reflected the risk associated with Alzheimer's drug development whilst at the same time appreciating the potential value in a differentiated product with a known safety profile that could stop the disease at an early stage.
II. Providing regulatory strategy for T-cell therapy
Challenge: Our client is a leading T Cell Receptor (TCR) biotechnology company, focused on delivering first-in-class biological therapies for serious diseases. The client wanted to understand whether it had the capabilities to launch its lead product if it were to receive accelerated approval without partnering with larger companies and asked Alacrita for commercialization assistance. The overall objective of the project was to ensure the client was adequately prepared for the eventuality of an early commercialization event for the product. This included an assessment of the regulatory pathway for the product and any obstacles that had to be considered.
Solution:
Our assessment comprised three umbrella categories:
- Marketing approval pathway and data requirements: As a prerequisite for commercialization, the marketing approval pathway and data requirements were evaluated based on the sponsor’s interactions with relevant regulatory authorities and development plans. Having a preferred marketing approval pathway agreed with the agency(ies) and well-defined data requirements and desired labelling for the application reduces risk to achieving a timely approval and, in turn, market launch, for a commercially viable product.
- Regulatory strategy plans and execution: The second group of criteria evaluated organizational capabilities to analyze and mitigate development risks and to adequately resource, plan and execute a regulatory strategy leading to marketing approval.
- Processes for post approval compliance: Lastly, the regulatory assessment examined the organization’s preparedness to support post-approval compliance requirements to maintain a product license.
A written report was provided to the client summarizing the findings from each analyses and highlighting areas for focus as they work towards
III. Commercialization options and roadmap for Orphan Drug
Challenge: A publicly-listed biotech company preparing for commercialization of its lead asset had already decided to launch the product directly in the USA but was not yet certain how to approach the European markets. The product, a therapeutic for a rare disease, was on track for pivotal trial readout in two years so commercial strategy for Europe had become a critical path issue. The client retained Alacrita to sketch out the relevant options for its European go to market strategy and to define the critical activities that needed to be undertaken in the ensuing pre-launch 18-24 month period.
Solution: Alacrita's commercialization team determined, unsurprisingly, that there were four options for European commercialization: Go-alone, Partial infrastructure, Outsource, Partner. Each had its own unique set of advantages and drawbacks and through a structured workshop with the company BoD the Alacrita team helped the client select its preferred option. At the same time, we set out the essential commercial & project management activities that would be required to underpin a viable launch, highlighting the market access and supply chain activities that urgently needed attention.
IV. IND-enabling studies plan for a small molecule in NASH
Challenge: A biotech developing a novel drug for NASH asked Alacrita to create a project plan outlining costs and timelines for preclinical IND-enabling studies.
Solution: We recommended the best animal models, such as DIAMOND and STAM, of NASH for evaluating the activity of the novel drug. We also identified the most suitable IND-enabling studies for this indication, specifically focusing on preclinical studies in module 2 of the IND which includes pharmacology (in vitro and in vivo pharmacodynamic test), ADME, toxicology, and pharmacokinetics. We also estimated the time and the cost for these preclinical studies.
V. Indication opportunity assessment for a gene therapy
Challenge: An early-stage, venture-backed, private biotechnology company developing a novel multi-gene therapy approach to addressing chronic, age-related diseases requested an opportunity mapping assessment and target indication prioritization to identify the optimal indication strategy for development and commercialization of the therapy.
Solution: Alacrita first assessed ~50 potential target indications that could benefit from the gene therapy based upon biological rationale and US patient prevalence as scoring metrics. Indications were sorted into biological families depending on the key drivers of disease (cardiovascular, metabolic, obesity, renal/liver). Due to the innovative and potentially disruptive nature of the client’s approach, it was recognized that the indication strategy should involve a development journey from an initial “gateway” indication (high scientific rationale, low disease prevalence) through to larger biologically-related larger patient populations once POC had been demonstrated.
Thus, we proposed a shortlist of potential ‘indication journeys’ for client consideration, in each case profiling the severity and progression of disease, the optimal stage of disease for treatment with the “preventative” gene therapy, preclinical models and likely costs to IND, current standards of care, clinical benchmarks, the estimated cost and requirements for clinical studies to POC and regulatory submission. Finally, we provided a recommendation on the best initial indication to prioritize and its associated downstream journey indications.
Contact Us
For more information on our biotech consulting services, please reach out.