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Flash Updates
Thiogenesis Flash Update
“A prodrug for mito dysfunction?”
2024 progress - Leigh syndrome is a new indication
Thiogenesis’ key driver, TTI-0102, is a “pipeline in a product” for diseases characterized by mitochondrial dysfunction. 2024 highlights included the allowance of a core patent protecting TTI-0102 in Europe until 2037, a collaboration with UCSD to start a POC trial in pediatric MASH, a new development program for TTI-0102 in Leigh syndrome, and acceptance of the CTA-Part I for MELAS in the EU. TTI-0102 will now target four pediatric indications, with global peak sales amounting to CAD 9.8 bn (USD 7 bn) and ample patent life until at least 2037. In 2025, POC trials are expected to start in MELAS, Leigh syndrome, and pediatric MASH. Substantial equity upside should be unlocked once the POC trials start and positive results are reported.
Key catalysts include:
1. Start TTI-0102 POC trial in MELAS (Q1 2025)
2. Start TTI-0102 POC trial in Leigh syndrome (H1 2025)
3. Start TTI-0102 POC trial in p
“A pivotal year”
KOLs highlight evenamide's potential at Investor Day
On June 25th, Newron hosted an upbeat Investor Day in New York City, highlighting the blockbuster potential of its late-stage schizophrenia drug evenamide. Three key opinion leaders explained the underlying biology of poorly responding and treatment-resistant schizophrenia (TRS) and how evenamide’s unique mechanism of action targets the core abnormalities in schizophrenia. This led to unprecedented positive efficacy results and superior safety and tolerability of evenamide in this difficult-to-treat and unresponsive patient population, as seen in the pivotal phase III “Study 008A” trial and the open-label “Study 014/015” trial. Newron expects to sign a lucrative global or regional partnering agreement in the next few months and start the second pivotal “Study 017” trial of evenamide in TRS in Q4 2024.
Key catalysts include:
1. Partnering evenamide with a major CNS player (before starting “Study
"Back to the roots"
2023 guidance reiterated - strong Winlevi H2 ahead
Cosmo reported H1 2023 results driven by strong GI Genius, Lialda, and Contract manufacturing growth. Winlevi’e royalties were up sharply, offset by a drop in manufacturing income, expected to rebound in H2 2023. Cosmo will repay the EUR 175 mn convertible bonds in November 2023 with cash at hand. FY 2023 guidance was maintained despite the USD currency headwind signaling a strong H2 boosted by strong upfront and sales milestones for Winlevi with several new commercialization agreements underway.
Key catalysts include:
1. CB-01-33 (colesevelam) start of phase II POC trial in bile acid diarrhea (H2 2023)
2. Winlevi licensing agreements (during 2023)
3. Winlevi filing for EU approval (H2 2023)
“Against all odds”
A much-needed Catalyst - substantial dilution avoided
The Catalyst licensing agreement for vamorolone rights in North America, valued at up to USD 231 mn plus royalties, significantly improves the risk/reward profile for Santhera. Based on our forecasts, the stock continues to provide significant equity upside - a multiple from the current depressed share price - but without substantial share dilution to raise cash for a US and EU specialist sales force with the risk of running out of cash in the crucial launch stage if unsuccessful. Santhera is now a much de-risked company with an experienced partner in North America with the potential to accelerate vamorolone development in new indications outside DMD and a cash runway into Q1 2025 (from Q4 2023).
Key catalysts include:
1. Vamorolone US approval in DMD - PDUFA date (26 October 2023)
2. Vamorolone EU approval in DMD (Q4 2023)
3. Vamorolone UK approval in DMD (Q4 2023)
“Setting a new course”
Bright future under the stewardship of a new CEO
Cosmo is one of the few SIX-listed biopharma companies with sufficient cash resources to fund its clinical development plans and pay dividends thanks to a strong balance sheet and sustainable revenues and profits from 8 marketed products. Growth products GI Genius (AI-enhanced colonoscopy) and Winlevi (acne) should provide substantial growth and margin expansion in the near and long-term, with the potential of sustained annual dividends. Pipeline products such as Breezula (hair loss), colesevelam MMX (bile acid diarrhea), and rifamycin enema (distal ulcerative colitis) should add to future growth.
Key catalysts include:
1. Winlevi EU approval (H2 2024)
2. Breezula complete patient enrolment phase III AGA trial (H2 2024)
3. Colesevelam MMX start POC tria in bile acid diarrhea (Q4 2024)
“A stable and exciting growth story”
Profitable distribution business and emerging pipeline
Curatis is a unique risk-balanced company, including a profitable and growing Specialty Distribution business and a Search & Development business seeking drugs with existing safety and clinical data to develop new indications with high unmet medical need with a focus on rare and ultra-rare diseases. It has three advanced/late-stage clinical pipeline projects with peak sales ranging from USD 250 mn to over USD 500 mn and one project ready for clinical development. Successful development and commercialization of any of its key compounds would be transformational, providing substantial revenues on top of its profitable Specialty Distribution business with the potential of future dividends.
Key catalysts include:
1. C-PTBE-01 - Apply for orphan and rare pediatric disease designation (H2 2024)
2. C-PTBE-01 - Scientific advice meeting FDA & EMA (H2 2024)
3. C-PTBE-01 - Partnering agreement bef
“On steroids - but better!”
AGAMREE launch signals a new era for Santhera
Key driver AGAMREE is off to a flying start in the US and EU, with initial sales surpassing expectations backed by strong patient demand. AGAMREE is set to replace mainstay steroids in DMD and become a foundational treatment that can be given to all patients irrespective of genetic mutation and in combination with other DMD treatments. We forecast global peak sales of CHF 750+ mn (incl. partner sales), which would be transformational. Santhera guides for a cash reach into 2025 (excluding maturing convertible bonds in August 2024) and expects to reach breakeven on a cash basis by H1 2026.
Key catalysts include:
1. AGAMREE UK launch in DMD (summer 2024)
2. AGAMREE partnerships (during 2024)
3. AGAMREE China approval; launch in France, Italy, Spain, Benelux (Q1 2025)
“A prodrug for mito dysfunction?”
TTI-0102: three POCs to prove its blockbuster potential
Thiogenesis’ key driver, TTI-0102, is a “pipeline in a product” for diseases characterized by mitochondrial dysfunction. TTI-0102 will initially target three pediatric indications, MELAS, Rett syndrome and pediatric NASH with estimated total peak sales amounting to CAD 9.6 bn and ample patent life until at least 2037. TTI-0102, being a prodrug of cysteamine, is eligible to use the US expedited 505(b)(2) regulatory pathway and the EU hybrid MAA pathway, substantially cutting development timelines and costs. In the next 9 months, POC trials should to start in MELAS, Rett syndrome, and pediatric NASH. Substantial equity upside should be unlocked once the POC trials start and positive results are reported.
Key catalysts include:
1. Start TTI-0102 POC trial in MELAS (Q1 2024)
2. Start TTI-0102 POC trial in Rett syndrome (mid-2024)
3. Start TTI-0102 POC trial in pediatric NASH (mid-2024)
FY 2023 revenue surprised positively, largely due to a one-off Xadago payment. Newron has a cash runway well into 2025 and beyond major value inflection points, thanks to cash of EUR 12.6 mn, Italian R&D tax credits, funding of up to EUR 15 mn from an institutional healthcare investor, and the deferral of the repayment of the first three tranches of the EUR 40 mn EIB loan. Substantial equity upside should be unlocked upon positive results of the pivotal “Study 008A” trial results of evenamide in non-treatment-resistant schizophrenia (non-TRS), a partnering deal with a major CNS player, and the start of the pivotal phase III “Study 017” trial of evenamide in TRS.
Key catalysts include:
1. Results “Study 008A” of evenamide in non-TRS (April 2024)
2. Partnering evenamide with a major CNS player (before starting “Study 017”)
3. Start pivotal “Study 017” trial of evenamide in TRS (Q3 2024)
"Return to anti-infectives and profits"
2023 net profit to at least triple, slight US Zevtera delay
Basilea guides 2023 net profit to at least triple on already strong results in 2022. The slight delay of Zevtera’s US filing in three indications (SAB, ABSSSI, and CABP) to Q3 2023 (from March/April 2023) has a negligible effect as QIDP status provides 10-year market exclusivity from the day of launch. Zevtera should get a considerable boost on contracting a US commercialization partner and the launch in the lucrative US market. Basilea is one of the few Swiss biotechs with sustainable profits.
Key catalysts include:
1. US NDA filing of Zevtera for severe bacterial infections (Q3 2023)
2. US commercialization partner for Zevtera (before Q2 2024)
3. US approval of Zevtera for severe bacterial infections (Q2 2024)
“Play ACE!”
Olpruva US launch early July - reverse share split
Upon shareholder approval (EGM 28 April 2023), Relief plans a reverse share split of 400 current for 1 new share, complying with the minimum share price required for a secondary listing on the US NASDAQ stock exchange. The US launch of PKU GOLIKE and the upcoming US launch of Olpruva (ACE-001) in UCDs boost near-term revenue. Successful development of Relief’s diversified pipeline, including RLF-100 in rare respiratory diseases, RLF-OD032 in PKU, and RLF-TD011 in EB, are future revenue generators. Relief has a cash runway through Q3 2023. An estimated CHF 30 mn additional funding, needed to reach breakeven guided for late 2024, should primarily be raised through non-dilutive regional product out-licensing agreements.
Key catalysts include:
1. US launch of Olpruva (ACER-001) in UCDs (early July 2023)
2. RLF-100 INHALED start of phase IIb sarcoidosis trial (Q2/Q3 2023)
3. EU filing of ACER-001 in UCDs (H2 2023)
“Catching breath in lung disease”
ColiFin US phase III CF trial to start in H1 2023
Spexis provides a unique, low clinical risk, high reward investment, yet, dependent on securing sufficient funding. Lead drug ColiFin (branded inhaled colistin), approved in the EU for cystic fibrosis (CF), needs only a single positive phase III trial to gain US approval. In the US, generic versions of inhaled colistin are prescribed “off label” by physicians at their own risk to treat CF patients, given its favorable profile. Following US approval, the roughly 36% of CF patients on generic colistin could be rapidly switched to branded ColiFin, while gaining traction in new patients and enjoying 12 years of US market exclusivity from FDA approval. We forecast CHF 300+ mn US peak sales for ColiFin.
Key catalysts include:
1. Strategic decision on balixafortide (H2 2022)
2. Start of ColiFin “COPILOT” dosing trial in CF (H1 2023)
3. Start of ColiFin single pivotal US phase III “COPA” trial
“Back to the future - fast forward”
USD 339 mn Indivior deal highlights pipeline potential
The USD 339 mn strategic agreement with Indivior to accelerate development of ADX71441 in addiction highlights the underlying value of Addex’ allosteric modulator pipeline and extends it cash runway into 2021. The company plans to raise sufficient funds in 2018 to finance pivotal development of dipraglurant in PD-LID and dystonia, both with blockbuster peak sales potential. Successful funding is the last step in Addex' development strategy to unlock the value of its key pipeline projects.
Key catalysts include:
1) Start phase IIa POC trial of dipraglurant ER in dystonia (H2 2018)
2) Start phase I trial of ADX71441 in addiction (H2 2018)
3) Start 1st pivotal phase III trial of dipraglurant IR in PD-LID (H2 2018)
"Fit for Growth"
KUR-023, the first product to reach the market in 2017
Kuros’ key projects target sizable market opportunities in surgical sealants with KUR-023 (dural membrane sealant); and orthobiologics with KUR-111 (bone graft substitute) and KUR-113 (bone fracture healing), with combined peak sales of more than CHF 1.5 bn. With cash of CHF 18 mn (June 30, 2016), the company has sufficient funds to launch KUR-023 in the EU in 2017, and prepare KUR-111 and KUR-113 for phase III development. Additional financing will be needed to bring KUR-111 and KUR-113 successfully to market. Legacy Cytos collaborations (not in our forecasts) provide significant upside potential without any further funding requirements.
Key catalysts include:
1. Filing CE marking dural membrane sealant KUR-023 (~end 2016)
2. Start US PMA clinical trial KUR-023 in spine patients (2017)
3. EU approval, partnering agreement, and launch of KUR-023 (2017)