PureTech Health – Half-Year Report

PureTech Health – Half-Year Report

Strong capital base with PureTech Level Cash and Cash Equivalents of $341.4 million1 and Consolidated Cash and Cash Equivalents of $365.9 million2 as of June 30, 2022, excluding up to $115.4 million added post-period3; Operational runway extended into Q1 2026

Significant advancement of PureTech’s Wholly Owned Programs, with three clinical trials underway, four completed, and human proof-of-principle achieved for a key PureTech platform

Excellent progress across the Founded Entities, including Karuna’s positive topline Phase 3 results for KarXT in schizophrenia, Akili’s Nasdaq listing and Gelesis’ commercial progress with Plenity®4 in the post-period, and four clinical data publications across Founded Entities

Company to host a webcast and conference call today at 9:00am EDT / 2:00pm BST

PureTech Health plc (Nasdaq: PRTC, LSE: PRTC) (“PureTech” or the “Company”) today announces its half-yearly results for the six months ended June 30, 2022. The following information will be filed on Form 6-K with the United States Securities and Exchange Commission (the “SEC”) and is also available at

https://investors.puretechhealth.com/financials-filings/reports.

Webcast and conference call details

Members of the PureTech management team will host a conference call at 9:00am EDT / 2:00pm BST today, August 25, 2022, to discuss these results. A live webcast and presentation slides will be available on the investors section of PureTech’s website under the Events and Presentations tab. To join by phone, please dial:

United Kingdom Toll-Free: +44 800 640 6441

United Kingdom Toll/International: +44 20 3936 2999

United States: +1 646 664 1960

United States Toll-Free: +1 855 979 6654 / +1 800 249 2588

Access Code: 563263

For those unable to listen to the call live, a replay will be available on the PureTech website.

Commenting on PureTech’s half-yearly results, Daphne Zohar, Founder and Chief Executive Officer of PureTech, said:

“The first half of 2022 has been an exceedingly strong period for PureTech. Our mission is to change the treatment paradigm for patients with devastating diseases, and we have made great progress towards that goal, particularly on the heels of stellar topline results from Karuna’s Phase 3 trial evaluating KarXT in adults with schizophrenia. Schizophrenia is a severe and debilitating disorder affecting approximately 21 million people worldwide. KarXT, which was invented at PureTech, demonstrated notable improvements across symptom domains, and was not associated with the debilitating side effects of weight gain, sedation and movement disorders seen with existing treatments. It is now poised to potentially be the first new class of medicine in over 50 years for patients living with schizophrenia. As a co-inventor of KarXT, we have the right to receive royalties, sublicense income and milestone payments in addition to the value of our equity.

“Across our Wholly Owned Pipeline are examples of other programs that we have developed in a similar way to Karuna’s KarXT, where we start with an approach or candidate that has proof of human efficacy, but key limitations have hindered the class from reaching its full potential. Through the expertise of our experienced R&D team and our network of industry-leading collaborators, we strive to overcome barriers to unlock potential new classes of therapeutics for the benefit of patients.

“Another example of our clinically de-risked development approach is LYT-300, which is an oral form of natural allopregnanolone. LYT-300 could make a difference for patients with a range of mental health conditions, such as depression, where there is a growing need but standard of care treatments like selective serotonin reuptake inhibitors (SSRIs) can have mixed efficacy, delayed onset of action and poor tolerability. In June, we announced that we can orally administer LYT-300 and achieve therapeutic levels of allopregnanolone in systemic circulation. This is exciting because allopregnanolone has proven efficacy but is only available for the treatment of postpartum depression as a 60-hour IV infusion. Demonstration of human oral bioavailability of allopregnanolone is therefore a key milestone for LYT-300 and for our Glyph platform, which enabled this innovation. Similarly, we are advancing LYT-100, a deuterated form of pirfenidone, to make a meaningful difference in the lives of patients with lung fibrosis and other inflammatory and fibrotic conditions by potentially offering better therapeutic effect without the poor tolerability associated with current standard of care drugs. In the first half of 2022, we initiated a late-stage clinical trial of LYT-100 for the potential treatment of idiopathic pulmonary fibrosis (IPF), a terminal condition that affects about three million people worldwide. These milestones are just a few of the many accomplishments from our Wholly Owned Pipeline that demonstrate our commitment to improving the lives of millions of patients.

“In addition to Karuna, several of our other Founded Entities also made notable progress. Most recently, Akili began trading on Nasdaq, becoming the fourth of our Founded Entities to be publicly traded, and – together with Karuna, Gelesis and Vor Bio – bringing the value of publicly traded Founded Entities created by PureTech to over $9 billion. Gelesis also continued to grow its revenue from Plenity4 sales, generating $16.5 million in net product revenue in the first half of 2022, resulting in an increase of 212% year-over-year.

“I’m particularly proud of our track record of clinical success, which is approximately six times better than the biopharma industry average.5 This clinical success has led us to financial success as one of a few cash generating biotech companies in the world. We have generated over $680 million in non-dilutive cash in less than three years3 and have not had to raise money from the capital markets in over four years. Based on a strong balance sheet, our Board approved a share buyback program in May. We are delighted to have received positive feedback from shareholders thus far, and we are confident that we can maintain sufficient cash on hand to support the advancement of our Wholly Owned Pipeline, including the completion of all currently initiated clinical trials and certain strategic investments in our Founded Entities. Additionally, we have updated our guidance to extend our operational runway to the first quarter of 2026.

“We look forward to carrying this success forward into another catalyst rich period as we unlock the potential of validated efficacy to deliver new classes of medicine for patients with devastating diseases.”

Operational Highlights

• Strong progress across our Wholly Owned Programs6, including the progression of four clinical trials
• Initiated a registration-enabling trial of LYT-100 (deupirfenidone) for the potential treatment of IPF
• Completed four clinical trials with LYT-100 to validate the thesis of the anti-fibrotic and anti-inflammatory activity of pirfenidone with a differentiated pharmacokinetic (PK) profile, affirming both the strong safety and tolerability profile of LYT-100
• Completed the bi-monthly, monotherapy dose escalation portion of the Phase 1 program assessing the safety and tolerability of escalating doses of LYT-200 (anti-galectin-9 mAb) as a potential treatment for metastatic solid tumors
• Achieved oral bioavailability of LYT-300 (oral allopregnanolone) in a multi-part Phase 1 program, representing the first mechanistic proof-of-principle in the clinic for our Glyph™ platform

Momentum across Founded Entities7, which we initiated and co-invented, demonstrates success of our R&D model

• Karuna Therapeutics, Inc. (Nasdaq: KRTX) (Karuna) announced positive topline Phase 3 data evaluating the efficacy, safety and tolerability of KarXT in adults with schizophrenia, meeting its primary endpoint and key secondary endpoints in the August 2022 post-period.
• Akili, Inc. (Nasdaq: AKLI) (Akili) recently began trading on the Nasdaq Stock Market and announced its partner, Shionogi, had started a pivotal Phase 3 randomized, controlled study of SDT-001 in children with attention-deficit hyperactivity disorder (ADHD), both in the August post-period, and Akili partnered with Roblox (NYSE: RBLX) in May.
• Gelesis Holdings, Inc. (NYSE: GLS) (Gelesis) began trading on the New York Stock Exchange in January 2022, and generated net product revenue of $16.5 million in the first half of 2022 for Plenity4, an increase of 212% year-over-year.
• Gelesis presented results from a clinical trial demonstrating that approximately 6 out of 10 adults in the trial who were treated with GS200 lost on average 11% of their body weight; Akili announced the publication of data in adults with systemic lupus erythematosus (SLE), adults with major depressive disorder (MDD) and children with ADHD in major scientific journals; and Vedanta Biosciences, Inc. (Vedanta) published data in a major journal for its lead program, VE303.

Financial Highlights:

• PureTech Level Cash and Cash Equivalents as of June 30, 2022, were $341.4 million1 (December 31, 2021: $418.9 million) and Consolidated Cash and Cash Equivalents as of June 30, 2022, were $365.9 million2 (December 31, 2021: $465.7 million).
• Founded Entities have strengthened their collective balance sheets by attracting gross proceeds of $113.5 million8 in equity investments during the six months ended June 30, 2022. In the post-period, Founded Entities attracted additional gross proceeds of more than $1 billion.9 Since July 2018 through the date of this report, our Founded Entities have raised funding of $3.1 billion,8 of which $2.9 billion (95.3%) was from third parties.
• Operating Expenses for the period were $108.2 million (June 30, 2021: 73.9 million).
• PureTech initiated a share buyback program up to a maximum consideration of $50 million.
• PureTech will receive aggregate proceeds of up to approximately $115.4 million, net of transaction fees, through the sale of Karuna shares in the August 2022 post-period.3

Key Upcoming Milestones (next 12 to 24 months)

• Multiple important milestones are anticipated, including those announced by our Founded Entities:

Wholly Owned Pipeline

• We expect topline results from the registration-enabling trial of LYT-100 in IPF by the end of 2023 as part of a streamlined development program that capitalizes on efficiencies of the 505(b)(2) pathway. Pending positive clinical and regulatory feedback, we believe the results of the Phase 2 clinical trial, together with a Phase 3 clinical trial, could serve as the basis for registration in the U.S.
• We expect results from the Phase 1/2 clinical trial evaluating LYT-200 in single agent cohorts by the end of 2022 and will soon begin to enroll patients in cohorts designed to evaluate LYT-200 in combination with chemotherapy. Results from the combination cohorts are expected in 2023.
• We plan to initiate a clinical trial to evaluate LYT-200 as a single agent for the treatment of acute myeloid leukemia (AML) by the end of 2022.
• We expect to complete the multi-part Phase 1 program of LYT-300 by the end of 2022, and - based on the data - a Phase 1b/2a clinical trial is planned to initiate in 2023.
• We expect additional preclinical validation of our key technology platforms.

Founded Entities

• Karuna plans to submit a New Drug Application (NDA) for KarXT in schizophrenia with the U.S. Food and Drug Administration (FDA) in mid-2023 and Karuna’s Phase 3 ADEPT program, evaluating KarXT for the treatment of psychosis in elderly patients with Alzheimer’s disease (AD) is expected to initiate in the third quarter of 2022.
• Akili expects to bring its digital therapeutic to more families and healthcare providers with the broader commercial launch of EndeavorRx®10 in the second half of 2022.
• Vor Biopharma Inc. (Nasdaq: VOR) (Vor) expects to report initial clinical data from VBP101, a Phase 1/2a clinical trial for VOR33 for patients with AML, in the fourth quarter of 2022, and data from the ongoing Phase 1/2 National Marrow Donor Program (NMDP)-sponsored clinical trial evaluating VCAR33AUTO in young adult and pediatric patients with relapsed/refractory AML in a bridge-to-transplant study are expected in 2022, depending on investigator’s timing of data release.11
• Vedanta plans to initiate a Phase 3 clinical trial of VE303 in patients at high risk for recurrent Clostridioides difficile infection (CDI) in the first half of 2023.
• Four additional Founded Entities also expect multiple near-term milestones.