Therapeutic solutions for rare diseases

Home » Areas of activity

Our aim is to find therapeutic solutions for neglected orphan diseases: pathologies so rare that others have unfortunately deemed it difficult to implement a medicine development and registration plan.

Today in the EU, a rare disease is defined as a disease affecting less than one in two thousand people.

About 80% of recognized rare disease are pathologies of genetic origin, while the remaining 20% are typically multifactorial diseases derived not only from individual characteristics, but also from other factors (for example: comorbidities, certain environmental factors, food) or from the interaction between genetic and environmental causes.

(source: https://www.iss.it/malattie-rare )

Our commitment today is geared towards two serious rare diseases: SBS (short bowel syndrome) and CDD (congenital diarrheal disorders). The first is a disorder caused by the reduction of the intestinal surface necessary for the absorption of liquids and nutrients. CDD, on the other hand, refers to a group of rare hereditary enteropathies characterized by severe chronic diarrhea in the first months of children’s life. Both SBS and CDD frequently cause chronic diarrhea, massive dehydration, metabolic acidosis or alkalosis, and malnutrition: symptoms that can occur quickly and be life-threatening for patients.

It’s estimated that SBS affects approximately between 1 and 9 people in every 100,000.
(Source: orpha.net | sigenp.org)

Focus: How rare are rare diseases?

300 million people around the world.

It is estimated that more than 300 million people worldwide suffer from a rare disease, including over 30 million in Europe. Of the around two million rare disease patients in Italy, approximately 1.4 million are pediatric patients. (osservatoriomalattierare.it).

EARLY ACCESS PROGRAMS (EAPs)

Early Access Programs (EAPs) are programs offering access to experimental medicines – or to medicines not yet authorized in a particular country – for patients with rare or very serious diseases for which no other treatments exist. Access to these programs is regulated by ethical, compliant and controlled mechanisms. Only a doctor, under her/his responsibility, has the right to indicate whether a person suffering from a serious illness will be able to access these programs.

EAPs may be considered in the early stages of the development of medicines showing promising results for patients who cannot participate in clinical trials because, for example, the patients do not meet inclusion criteria. Sometimes, these programs are the only option to provide life-saving medicines to severely ill patients, considering that even the approval processes of these medicines can take years.

Massimo Radaelli

Pharmaceutical manager and entrepreneur with over 30 years of experience in the biopharmaceutical sector and innovation in therapies dedicated to rare diseases.
He founded Noventia Pharma, Pint Pharma Group, Ferrer Italia and he is co-founder of Dupont-Merck JV Italian Subsidiary.
He was president and CEO of Dompé International, as well as member of the boards of directors of Ariad Pharmaceuticals, Novabay, Philogen and IDRI. Today he is member of the boards of Pint Pharma Group, Bioakos Pharma Labs and Cytovia Threapeutics.

Michela Pantaleoni

Michela brings with her almost 20 years of experience in market access of innovative new therapies and orphan drugs from different perspectives, both local and international.

She began her career in consulting, where she spent about 7 years working in different settings and for both public and private healthcare clients. During this experience she completed successful projects involving both regional and national stakeholders and where she also worked on the development of international access strategies working with HQs of multinational companies.

She then moved to AstraZeneca Italy in 2010, where she had the opportunity to work in the access team, in roles of increasing responsibility, gaining proven experience in the development of pricing and access strategies for innovative drugs. He worked closely with the territory and had the opportunity to develop important partnership projects with payers and institutions.

Prior to joining Napo Therapeutics, Michela served as Head of Market Access Rare Diseases Europe & Emerging Markets at Chiesi Global Rare Diseases, where she was responsible for developing access and reimbursement strategies for the rare disease drug portfolio, working closely with local market access teams or partners to ensure implementation.

Michela holds a Bachelor’s degree in Management Engineering from the University of Bologna and a Master’s degree in International Health Care Management, Economics and Policy from SDA Bocconi in Milan.

Massimiliano Di Cicco

Over the last 15+ years he worked for top-tier pharmaceutical companies developing extensive local and international commercial experience in different therapeutic areas with a particular focus in rare haematologic neoplasms, oncology, and neuroscience. Prior to joining Napo Therapeutics, between 2019 and 2022 Massimiliano served as Franchise Head Multiple Myeloma Europe & Canada at Takeda Pharmaceutical International leading the regional team based in Zurich for the development and commercialization of the multiple myeloma franchise. In his previous role at Takeda, between 2016 and 2019 he successfully led the cross-functional efforts to bring an oral orphan drug for multiple myeloma to the Italian market. Between 2007 and 2016 Massimiliano worked for Eli Lilly where he held roles of increasing responsibility in various functional areas such as financial planning, business development and alliance management, commercial strategy, marketing, and sales. Massimiliano holds a master’s degree in Economics of Institutions and Financial Markets and an Executive MBA with focus on Entrepreneurship from LUISS Business School. Most recently he complemented his executive education completing the Leading Strategic Growth program at Columbia University.

Stefano Cesare Ronconi

Before joining Napo Therapeutics, Stefano served from 2015 to mid-September 2022 as Chief Financial Officer and M&A Director at Consulcesi Group, a leading international healthcare service group, based in Lugano (Switzerland) and oversighting EU subsidiaries.
He started his professional career in 1998 as an auditor for KPMG, based in Milan (Italy) and secondment in Southampton (UK), focusing on assurance, transaction services, process engineering and IFRS conversion projects.
In 2006 he spent one year as a Finance Manager for Bioxell SpA, a biotech company listed on the main segment of the Zurich stock exchange, and from 2007 to 2012 served as Director of Finance and Chief Operating Officer for Savills Investment Management, a real estate investment manager headquartered in London (UK) and offices in Luxembourg and Milan (Italy), dealing with investments, finance, operating activities and transactions involving EU regulated entities and cross-border institutional funds.
From 2013 to 2014 Stefano also worked as Chief Financial Officer for an Italian pharmaceutical retail company and successfully led the company’s listing process (IPO) on both the Alternative Investment Market of the Milan stock exchange and the Marché Libre (Euronext) segment of the Paris stock exchange.
Stefano holds a bachelor’s degree in Business Economics and a master’s degree in Corporate Finance. He also was a member of the Board of Directors of several European companies.

People Manager

Responsibilities:

Able to operate independently and responsible for developing initiatives in areas such as Organization Design, Staffing, Recruitment, Comp Ben, Training and Development, Change Management and Employee Engagement
Act as a business partner and ensure all People processes and systems operate effectively
Manage the recruitment process in a timely and cost-effective manner (pre-selection, interviewing, reference checks, advising managers) and ensure hiring of high potentials consistent with Napo Way
Ensure compliance with state and national employment laws and turn them, into consistency with the company culture and objectives, in its policies and procedures
Ensure payroll and related data accuracy
Manage the performance review processes in line with company culture
Manage the organization and talent planning process to ensure effective organization design, identify key performers, and build their succession plans
Identify training and development priorities within the organization, provide appropriate programs and support managers in their efforts to track the initiative’s success
Participate in business and functional planning meetings and reviews
Represent the company in front of attorneys, various institutions, tax auditors, labor courts etc.
Initiate, support and/or drive the implementation of organizational fine tunings that improves Napo’s performance
Ensure consistency within policies and procedures for Compensation and Benefits in compliance with local markets and internal equity

Head of Marketing

Key responsibilities:

maximize growth and profitability by proposing an adequate promotional approach and strategy, translating the vision into clear strategies and focusing the organization to deliver results
work closely with the CEO in the development of priorities and strategies for new products launches, promotional plans and budgets
creation of European marketing plans and monitoring the execution, managing new product launches and registrations, multichannel roll-out campaigns, training and educational activities for the field force, and guaranteeing the delivery of its element
supervise the portfolio penetration in the key markets conducting analysis of market segments, identifying key drivers for growth, and providing insights and solutions
development of strong relationships with key customers and opinion leaders, assuring strong partnership programs with Key Scientific Societies
support the creation of a customer-centric organization through tailored customer programs and KOL interactions
management and inspiration of direct reports in the strategic development, integration and implementation of the Marketing Plans and development of their professional growth
establish the budgeting, forecasting and medium-long term planning P&L
oversee the marketing spending and allocation of resources driving profitable growth
coordination of multifunctional local and global teams (production, logistics, R&D, Sales) for the implementation and commercialization of product launches
organization of training activities, congresses, and workshop
responsible for two resources at European HQ located in Milan and supervising the Marketing organization across European Affiliates

Head of Market Access, Pricing and HEOR

The company is seeking an experienced Head of Market Access, Pricing and HEOR, owner of pre-market access strategic plan in key selected markets for Europe, commercial pricing strategy and accountable for international market access policies.

He/she will assess the markets’ existing health care environment and create stakeholder value propositions.

Key responsibilities:

Understand, describe and prioritize most relevant market access stakeholders (payers, governmental bodies, industry associations, professional associations and other stakeholders at European level)
Build trusted and strategic relationships with payers and influencing stakeholders to anticipate payers´ agendas, plans and objectives
Understand the specific needs of Pricing and reimbursement strategies, Negotiation strategies, commercial launch sequencing across European markets
Development of the global value dossiers and health economic models
Articulation of value messages to payers, creation of objection handlers
Responsible for the development of payer communication materials and the generation of health economic evidence
Supervise the writing, publishing and presentation of the health economic studies sustaining the value of Napo Thera’s products in key international congresses
Supervise and assist in the risk assessments for European price referencing
Assist countries in the adaptation of P&R dossiers and HE models and support them throughout the negotiation process with HTA bodies
Provide continuous ad hoc market access guide to affiliates
Lead cross-functional Market Access Product teams
Ensure best practice sharing and cross-fertilization among markets
Ensure consistency in messaging throughout Europe; verify that the national health economic communications are in line with the European health economic strategy (e.g. abstract reviewing, poster reviews)
Develop and implement a market access and health economic training for the Napo EU market access network

Clinical Trial Lead

The role reports to the Chief Medical Officer and works closely with CRO, clinical development functions and medical affairs. The overall scope of the role is to plan, execute, maintain oversight and report on clinical trials, from planning, vendor selection and site feasibility, through study close out. Ensure all trial outputs from protocol development to final clinical study report (including archiving of the Trial Master File) are delivered on time, within budget, and with high quality, in adherence to internal SOPs, GCP and applicable regulatory guidelines.

The responsibilities for this role include the following:

Provide clinical operations related input to the Clinical Development Plan
Ensure that the design of clinical studies under area of responsibility is in line with the objectives defined.
Develop or coordinate development and approval of study documentation such as the study protocol, Informed Consent Form (ICF), study-specific guidelines, regulatory documents, monitoring, data management and statistical analysis plans and clinical study report, in accordance with internal SOPs and GCP requirements.
Define and drive study timelines and milestones.
Define the needs for External Service Providers (ESPs), identify and select ESPs, including negotiation of scope of work and budget and ensure that related contracts comply with internal SOPs and GCP requirements.
Form and lead a cross functional teams including representatives of appropriate internal or external functions such as Regulatory Affairs, Clinical Science and any expertise required to implement and oversee the study according to GCP, the Protocol and the requirements of the Management Team.
Coordinate the development of specifications for ESPs and ensure appropriate quality control of deliverables
Contribute to the development of regulatory documents, responses to Health Authority, Ethic Committees (EC) and Institutional Review Board (RB) and oversee study approval processes for Health Authority, EC/IRB approvals for all study document as required
Oversee forecasting of Investigational New DrugI (MP) and study supplies
Review and approve feasibilitity
Plan, organize and lead Investigator meeting and CRA training & review related material
Perform ongoing ESP management, performance management and issue resolution
Oversee monitoring activities and conduct co-monitoring visits to ensure data quality
Ensure adequate trial resources in personnel and material are available cross-functionally (internally and at ESP) and escalate issues to respective Function Head if needed
Ensure study team members are timely informed, trained and updated on their role and responsibilities through-out the duration of the study
Provide study specific direction to study team members and ensure that they are regularly updated on the study progress, challenges and risks through-out the duration of the study
Address enrolment and retention issues, identify and implement actions to keep study on track
Ensure proper study documentation is maintained and archived in the TMF
Chair SMT meetings and ensure timely follow up of agreed upon actions
Resolve issues in a proactive and timely fashion and escalate unresolved issues and identified risks to the CMO and to the Managing Director
Create, manage and ensure tracking of study budget including revisions and perform final reconciliation at trial close out
Check and approve ESP invoices and ensure appropriate tracking and reconciliation
Implement best practices and lessons learned and share outcome with the teams
Oversee database lock activities to ensure timely data availability and coordinate study close out with ESPs as needed
Coordinate pre-audit and CAPA resolution activities ensuring satisfactory outcome
Adhere to personal development plan and maintain training records to ensure appropriate level of competence in compliance with this job description including GCP etc

Annabella Amatulli

A well-recognized expert in global regulatory affairs, Ms. Amatulli previously served as a Global Regulatory Affairs Director at Dompé, an Italian bio-pharmaceutical company that markets 50 million medication packages in roughly 40 countries around the world. Ms. Amatulli also spent more than six years in regulatory affairs with the Janssen Pharmaceutical Companies of Johnson & Johnson. Her recent track record includes approval of the first biotech drug (eye drops) for the treatment of a rare disease of the eye in the US, China, EU and four other countries; discussion with FDA, EMA and ANVISA about the development plan of 2 investigational products for COVID-19 treatment; the preparation and submission of three Investigational New Drug Applications (INDs) for different therapeutic areas (diabetes, oncology and ophtha); the development of a Global Early Access Program (Managed Access Program and Named-Patient Program); and the set-up of local affiliates in EU-5 countries (France, Germany, Italy, Spain and the United Kingdom), the US, and China from a regulatory standpoint. Ms. Amatulli holds a BS degree in Pharmaceutical Biotechnology from the Università Degli Studi Di Milano, and a master’s degree in Molecular Biology from the Università Degli Studi Milano-Bicocca.

Martire Particco

Aphysician with 30+ years of experience in Europe’s pharmaceutical industry and in clinical practice, Dr. Particco possesses in-depth experience in the field of rare diseases, having been involved in the clinical development and launch of Pfizer’s pulmonary hypertension indication for sildenafil and the clinical development of Kedrion Biopharma’s ligneous conjunctivitis indication for plasminogen, with direct experience with patients and experts treating these rare pathologies.

From 2016 to 2021, Dr. Particco served as Vice President, Head of Medical Affairs for Europe for Shionogi Europe, the subsidiary of a leading Japanese pharmaceutical company with global sales of €2.5 billion. Prior to this role, Dr. Particco was Shionogi Europe’s Country Medical Director for Italy, and in 2013 he served as the Country Medical Director for Italy for UCB Italy, a leading European biopharmaceutical company with global sales of €4.6 billion. He also previously served as Director of Global Pharmacovigilance, Medical & Clinical Affairs at Italian firm Kedrion Biopharma, and as the Country Medical Director for Italy for Warner Chilcott, the Italian affiliate of a US pharmaceutical company that is now part of Abbvie. Dr. Particco was with Pfizer/Wyeth from 1999-2010, in the role of Senior Medical Advisor for Biotechnology from 2007-2010, and previously as both a Medical Advisor and a Training Specialist at Pfizer. From 1990-1999, Dr. Particco worked as an obstetrician-gynecologist in Rome, both in private practice and in public health. He received his degree in Medicine and Surgery and his degree in the Specialization in Gynecology and Obstetrics from the Sapienza University of Rome.