Saniona : 08.26.21 Q2 2021 Report

INTERIM REPORT FOR SANIONA AB (PUBL) 556962-5345 January - June 2021

Published August 26, 2021

Saniona Achieves Clinical Progress in Q2 2021 and Subsequent Period, with Orphan Status for Tesomet in HO and Start of Phase 1 for SAN711

Three Months Ended June 30, 2021 (2020)	Six Months Ended June 30, 2021 (2020)
Revenue was SEK 1.9 M (2.0 M)	Revenue was SEK 5.3 M (4.4 M)
Operating loss was SEK -103.6 M (-26.0 M)	Operating loss was SEK -197.7 M (-53.5 M)
Net loss was SEK -103.9 M (-23.9 M)	Net profit (loss) was SEK -187.3 M (19.3 M)
Basic loss per share was SEK -1.67(-0.81)	Basic earnings (loss) per share was SEK -3.00 (0.65)
Diluted loss per share were SEK -1.67(-0.81)	Diluted earnings (loss) per share were SEK -3.00 (0.65)

Business highlights in Q2 2021

• Saniona achieved a key milestone with the initiation of a Phase 1 clinical trial of SAN711, an ion channel modulator that may be applicable in the treatment of rare neuropathic disorders. This is the first wholly-owned asset from Saniona's proprietary ion channel drug discovery engine to advance into a clinical trial. Data from the trial are expected in the first half of 2022.
• Saniona provided multiple updates on its progress toward initiating Phase 2b clinical trials of Tesomet in hypothalamic obesity (HO) and Prader-Willisyndrome (PWS), announcing a partnership with the Foundation for Prader- Willi Research to increase clinical trial awareness and the receipt of manufacturing feedback from the U.S. Food and Drug Administration (FDA) on the transition from tablets to capsules. Both Phase 2b clinical trials are expected to start in the second half of 2021, with top-linedata expected from the PWS clinical trial in the first half of 2023 and from the HO clinical trial in the second half of 2023.
• Saniona presented preclinical data from its ion channel program SAN903 in a model of idiopathic pulmonary fibrosis at the American Society of Pharmacology and Experimental Therapeutics (ASPET) Annual Meeting. SAN903 is expected to enter Phase 1 in the second half of 2022. Saniona also presented SAN711 preclinical data in a model of facial neuropathic pain at the prestigious European Academy of Neurology (EAN) Congress.
• Saniona hosted a Research and Development (R&D) day featuring presentations highlighting its ion channel drug discovery engine, including its IONBASE™ database now consisting of more than 20,000 proprietary molecules targeting various ion channels.
• Saniona provided an update from its partner Medix that additional information requested by a Mexican regulatory committee may delay the anticipated final decision in Mexico regarding tesofensine for obesity into 2022.
• Saniona successfully monetized its position in the 2017 spin-out Scandion Oncology, completing the sale of its remaining shares on the open market.
• Saniona received a minority ownership stake in Cephagenix, as per the terms of the previously announced February 2020 collaboration agreement through which the company was formed to explore ion channel modulators for the treatment of migraine.

Significant events after the reporting period

• Saniona achieved orphan drug designation from the FDA for Tesomet for the treatment of HO. Tesomet is the first and only investigational treatment for HO to receive orphan drug designation.
• Saniona entered into a non-dilutiveterm loan agreement for SEK 87 million ($10 million) with Formue Nord Fokus A/S to support new activities aimed at accelerating clinical development programs.

INTERIM REPORT FOR SANIONA AB (PUBL)

January - June 2021

Comments from the CEO

"In the first half of 2021 and into the subsequent period, Saniona has made significant progress on the clinical and regulatory fronts, achieving orphan drug designations from the U.S. FDA for Tesomet in both hypothalamic obesity and Prader-Willi syndrome. These designations put us in a strong position as we prepare to initiate our Phase 2b trials of Tesomet in HO and PWS before the end of this year," said Rami Levin, President & Chief Executive Officer of Saniona. "Additionally, in Q2 we initiated a Phase 1 clinical trial of SAN711, our second wholly-owned proprietary pipeline asset to advance into clinical trials. SAN711 is our lead molecule from our ion channel drug discovery engine, which has already generated a number of additional discovery-stage and preclinical assets to fuel our pipeline well into the future."

For more information, please contact

Trista Morrison, Chief Communications Officer, Saniona. Office: + 1 [phone]. Email: [e-mail]

INTERIM REPORT FOR SANIONA AB (PUBL)

January - June 2021

Letter from the CEO

In the first half of 2021 and the following months, Saniona has made important progress executing on our strategy to discover, develop and commercialize innovative medicines for rare disease patients. Notable achievements so far this year include:

• Tesomet in the treatment of hypothalamic obesity (HO) and Prader-Willi syndrome (PWS): We are now seeing the benefits of investments we made in building an experienced U.S. clinical and regulatory team. This year, we achieved orphan drug designations from the U.S. FDA for Tesomet in both HO and PWS - and the HO designation was notably the first ever granted by the FDA in this indication. I am proud of this truly pioneering work by our team. Importantly, these two orphan drug designations qualify Saniona for certain development benefits, including tax credits, elimination of certain FDA license application fees, and seven years of market exclusivity in the U.S. following approval. They put us in a strong position as we prepare to initiate a Phase 2b clinical trial of Tesomet in each of these two indications in the second half of this year. We expect top-linedata from the PWS study in the first half of 2023 and from the HO study, which is a longer trial, in the second half of 2023.
• Ion channel pipeline and drug discovery engine: This year Saniona has had the opportunity to give multiple presentations about the encouraging data we are seeing from our ion channel programs, including the ability of SAN711 to reduce pain in in-vivo models of neuropathic pain and the ability of SAN903 to reduce inflammation and fibrosis in in-vivo models of idiopathic pulmonary fibrosis. In Q2, we advanced SAN711 into a Phase 1 clinical trial, making it the first wholly-owned asset from our proprietary ion channel drug discovery engine to advance into clinical trials. We anticipate reporting top-line data on SAN711 in the first half of 2022 and initiating a Phase 1 clinical trial with SAN903 in the second half of 2022.
• Partnerships: Saniona's strategy is to retain and develop our innovative molecules for rare diseases. We will continue to evaluate opportunities to leverage our expertise and portfolio in non-rare indications through business development activities that may provide non-dilutive funding. We also continue to monitor our existing programs that are advancing with partners. In the second quarter, our partner Medix reported that additional information requested by a Mexican regulatory committee may delay the anticipated final decision in Mexico regarding tesofensine for obesity into 2022. Medix is entirely responsible for this program including communications with Mexican regulators, and we will continue to update the market as we receive information.
• Ensuring Saniona is well-funded: We view Tesomet, SAN711 and SAN903 - our wholly-owned, proprietary pipeline assets - as the primary value-drivers of our business. Thus, ensuring we have the funding and expertise to continue to advance these programs is a high priority. After the close of the second quarter, we entered into a non- dilutive term loan agreement for SEK 87 million ($10 million) with Formue Nord Fokus A/S to support new activities aimed at accelerating clinical development programs. We also continue to evaluate listing our shares on the U.S. Nasdaq exchange, and will provide updates as appropriate.

Overall, I am proud of the clinical and regulatory milestones our team has achieved in the first half of 2021, and we look forward to achieving the very significant milestones of initiating our Phase 2b clinical trials with Tesomet in HO and PWS in the second half of this year. We appreciate the continued support of our shareholders on our journey to transform Saniona into a fully-integrated biopharmaceutical company with the ability to discover, develop and ultimately commercialize our own innovative treatments for rare disease patients around the world.

Rami Levin

President & CEO

INTERIM REPORT FOR SANIONA AB (PUBL)

January - June 2021

About Saniona

Saniona is a clinical-stage biopharmaceutical company focused on discovering, developing, and commercializing innovative therapies for patients suffering from rare diseases for which there are a lack of available treatment options. The company's lead product candidate, Tesomet, is in mid-stage clinical trials for hypothalamic obesity and Prader-Willi syndrome, serious rare disorders characterized by severe weight gain, disturbances of metabolic function and uncontrollable hunger. Saniona has developed a proprietary ion channel drug discovery engine anchored by IONBASE, Saniona's database of more than 130,000 ion channel modulators, of which more than 20,000 are Saniona's proprietary compounds. Through its ion channel expertise, Saniona is advancing two wholly-owned ion channel modulators, SAN711 and SAN903. SAN711 is in a Phase 1 clinical trial and may be applicable in the treatment of rare neuropathic disorders, and SAN903 is in preclinical development for rare inflammatory, fibrotic and hematological disorders. Led by an experienced scientific and operational team, Saniona has an established research organization in the Copenhagen area, Denmark and a corporate office in the Boston, Massachusetts area, U.S. The company's shares are listed on Nasdaq Stockholm Small Cap (OMX: SANION). Read more at www.saniona.com.

Our vision

Improve the lives of rare disease patients around the world through scientific innovation.

Our mission

We leverage our ion channel targeting expertise to discover, develop and deliver innovative rare disease treatments.

Our values

• Put People First
  Treat all people with kindness, respect and equity. Support people on their journey and enable a sense of belonging.
• Innovation With Impact
  Push boundaries with courage. Embrace empowerment. And deliver excellence.
• Integrity, Always
  Maintain the highest ethical standards in all that we do as we deliver with urgency for patients in need.

Our Strategy

Our strategy is to discover, develop and commercialize innovative treatments for patients suffering from rare diseases around the world. We intend to achieve this initially by advancing our lead asset, Tesomet, for HO and PWS, and our ion channel modulators, SAN711 and SAN903, for rare neuropathic and rare inflammatory, fibrotic and hematological disorders, respectively. We also intend to utilize our ion channel drug discovery engine to identify additional novel assets for new indications, with a focus on rare diseases for which there are currently no FDA-approved treatment options or those for which there remains significant unmet medical need.

Investment rationale:

INTERIM REPORT FOR SANIONA AB (PUBL)

January - June 2021

Strategic priorities:

• Completing the clinical development of, and seeking FDA approval for, Tesomet for the treatment of HO and PWS. We have completed initial Phase 2 clinical trials of Tesomet for HO and PWS. We are planning to initiate Phase 2b clinical trials for each of these indications in the second half of 2021. The FDA has confirmed that Tesomet may be advanced via the 505(b)(2) pathway for both indications and granted orphan drug designation status for Tesomet for the treatment of HO and PWS, respectively.
• Advancing our earlier stage programs, SAN711 and SAN903, through clinical development in rare neuropathic disorders and rare inflammatory, fibrotic and hematological disorders, respectively. We have identified two ion channel modulator product candidates, SAN711 and SAN903, from our proprietary ion channel discovery engine. We dosed the first participant in our Phase 1 clinical trial of SAN711 in healthy volunteers in June 2021 in the United Kingdom and anticipate reporting top-linedata in the first half of 2022. Additionally, we are continuing to progress SAN903 through preclinical studies and anticipate initiating a Phase 1 clinical trial in the second half of 2022.
• Continuing to expand our pipeline and develop innovative therapies targeting underserved patient populations by leveraging our rare disease expertise and ion channel drug discovery engine. We have been pioneers in the field of ion channel modulation since our founding and believe that the market for this recognized drug class has significant, untapped potential across many disease areas. We believe that our proprietary drug discovery engine overcomes many of the significant limitations of historical ion channel drug development, as does our IONBASE database of more than 130,000 ion channel modulators targeting various subtypes of ion channels, of which more than 20,000 are our proprietary compounds. We expect to continue to leverage our drug discovery engine and IONBASE to expand our wholly-ownedpipeline in rare diseases. Additionally, we are focused on expanding the breadth of our identified clinical programs through life cycle management beyond our initial target indications.
• Commercializing Tesomet independently in the key major markets, if approved, and exploring other markets through strategic collaborations. We have worldwide development and commercialization rights for Tesomet. Due to the rare incidence and prevalence of HO and PWS, the market for these indications is concentrated around key opinion leaders (KOLs) and key centers of excellence. Since our founding, we have built and continue to expand our established relationships with leading KOLs, clinicians and patient advocacy groups to help inform our product development. We plan to build a targeted sales force, initially in North America. We may explore expanding into other select markets, notably the European Union, either alone or in collaboration with one or more global or regional partners in order to provide patients around the world with access to our therapies.
• Maximizing the value of our ion channel engine and IONBASE by collaborating with global pharmaceutical and biotechnology companies as appropriate. Our expertise in the field of ion channel drug discovery has led to several out-licensingarrangements, spin-outsand collaborations with pharmaceutical companies globally, including Novartis and Boehringer Ingelheim, particularly for assets outside of our focus area of rare diseases. These transactions serve as a source of non-dilutivecapital, in the form of upfront payments, milestone payments, royalties and/or equity stakes that we intend to reinvest in both our discovery engine and core development efforts to treat rare diseases. We intend to continue to strategically evaluate additional opportunities to license out or collaborate with leading industry partners in disease areas addressing larger patient populations.

Our Pipeline

Consistent with our goal of developing innovative therapies for underserved patient populations, our wholly-owned pipeline consists of four programs in clinical and preclinical development across multiple rare diseases. The following table summarizes our wholly-owned programs: