UBX-0101

It has not been an easy road for the anti-aging startup Unity Biotechnology. From staff cuts to a lead program loss, the company has run into several obstacles on its journey, but their latest news points to both the perils and potential ahead. In the biotech’s Q2 results on Friday, the company revealed it has only enough cash to fund its operations through the first quarter of next year, with posted results showing that Unity had $64.5 million in cash and market securities at the end of the quarter. This is compared to the $79.2 million the biotech had at the end of March. The company also had an operating loss of $12.3 million for the quarter as well. Endpoints News reached out to Unity about the situation but has not received a response by press time and will update accordingly. The company price $UBX dropped into the penny stock range earlier this year and has been gyrating in and out since. However, data released this morning are bringing better news for investors, pushing the stock price up by more than 100% Friday morning. And that may help pave the way to bringing in more cash, if they can follow the road to a follow-on. The company posted 12- and 18-week data from its Phase II BEHOLD study of UBX1325, a senolytic Bcl-xL inhibitor, in patients with diabetic macular edema (DME). UBX1325 was one of the major focuses of the company after its lead candidate flopped in 2020 . At 18 weeks, after a single injection of UBX1325, the change in patients’ best corrected visual acuity (BCVA) was an increase of 6.1 ETDRS letters, representing an improvement of +5.0 ETDRS letters compared to sham-treated subjects and a value of P= 0.0368. The data also showed that patients treated with UBX1325 maintained a central subfield thickness (CST) compared to sham-treated patients who demonstrated progressive worsening of CST. Patients treated with a single injection of UBX1325 for 12 weeks had a mean improvement in BCVA of +4.7 ETDRS letters from baseline compared to +1.3 ETDRS letters in sham-treated patients, leading to a value of p=0.1148. “The vision gains observed are greater than what has been previously reported with the standard of care in similar patient populations, and the durability of effect suggests that UBX1325 could address the large unmet need for longer-lasting, disease-modifying treatments for patients with DME. These data represent an important and exciting step in validating the senolytic therapeutic concept that is core to UNITY’s platform,” said Unity CEO Anirvan Ghosh. The company is bullish on the findings and is forging ahead with more studies for UBX1325 for now. This positive news is a welcome reprieve after several recent setbacks for Unity. In 2020, after the company saw massive investments from names such as Jeff Bezos, Peter Thiel and Bob Nelsen, its Phase II study testing whether their lead drug UBX0101 could alleviate pain in people with osteoarthritis of the knee showed virtually no difference in patients on the placebo arm. This resulted in a 30% cut in the biotech’s staff and Nelsen’s eventual departure, and the company pivoted to ophthalmology and neurology. At the start of this year, the company sliced away another 50% of its workforce, primarily in its discovery research unit, to stretch its cash into 2023.

UBX1325 demonstrates favorable tolerability in Phase 1 safety study and improvement in visual acuity and central subfield thickness in majority of patients with diabetic macular edema (DME) and wet age-related macular degeneration (AMD) Results from Phase 1 study of UBX1325 in additional patients with wet AMD expected by end of year UBX1325 Phase 2a proof-of-concept study enrolling patients with DME; data expected first half of 2022 SOUTH SAN FRANCISCO, Calif., Aug. 10, 2021 (GLOBE NEWSWIRE) -- UNITY Biotechnology, Inc. (UNITY) [NASDAQ:UBX], a biotechnology company developing therapeutics to slow, halt, or reverse diseases of aging, today reported financial results for the second quarter ended June 30, 2021. “This quarter has been exciting and productive, marked by the compelling data from the Phase 1 study of UBX1325. In addition to the reassuring safety profile, we observed initial signs of efficacy in both vision and corresponding ocular structures in patients suffering from advanced DME and wet AMD for whom anti-VEGF therapy was no longer considered beneficial – and who in fact hadn’t received any anti-VEGF therapy for at least three months. This initial data has generated strong enthusiasm and support from the physician community, and we have both completed the enrollment of additional wet AMD patients in our Phase 1 study and are rapidly recruiting in our international Phase 2a study in DME,” said Anirvan Ghosh, Ph.D., chief executive officer of UNITY. “There’s a large unmet need for treatment options beyond anti-VEGF therapy, and UBX1325 may fill that void as a senolytic option working through an entirely novel and potentially disease-modifying mechanism. We look forward to sharing additional data from the Phase 1 study in the coming months, and the results of the Phase 2a study in the first half of 2022.” Key Business Highlights Ophthalmology – UBX1325 Phase 1 Results In July, UNITY announced positive data from its Phase 1 safety study of UBX1325 in patients with advanced disease from DME or wet AMD for whom anti-VEGF therapy was no longer considered effective. In addition to a favorable safety and tolerability profile, UBX1325, a small molecule inhibitor of Bcl-xL, showed rapid improvements in these advanced patients. Specifically, the majority of patients from this study showed a positive gain in key clinical measures of disease progression, including in best-corrected visual acuity (BCVA), central subfield thickness (CST), and sub- and intra-retinal fluid (SRF, IRF). The following improvements in vision and retinal structure were observed as summarized below: Treatment of patients with UBX1325 resulted in the following clinical changes as of June 30, 2021: Gain in ETDRS Letters from Baseline in Best-Corrected Visual Acuity (BCVA) Decrease in Central Subfield Thickness (CST) Reduction in Subretinal / Intraretinal Fluid Based on these data, the Company has, as of August 5, 2021, completed the enrollment of additional patients with advanced wet AMD in the Phase 1 study to inform a potential Phase 2a study in wet AMD to start this fall. A parallel Phase 2a proof-of-concept study in DME is actively recruiting patients, with information about that trial available here. Second Quarter Financial Results Cash, cash equivalents, and marketable securities totaled $97.5 million as of June 30, 2021 compared with $115.6 million as of December 31, 2020. UNITY believes that current cash, cash equivalents, and marketable securities are sufficient to fund operations through the third quarter of 2022. Operating loss for the three months ended June 30, 2021 was $17.0 million compared to $23.3 million for the three months ended June 30, 2020. Cash used in operations during the first and second quarters of 2021 was $28.5 million compared to $44.4 million for the first and second quarters of 2020. Research and development expenses decreased by $5.1 million, to $11.0 million for the three months ended June 30, 2021 from $16.1 million for the three months ended June 30, 2020. The decrease was primarily due to decreases of $3.2 million in personnel costs due to reduction in force, $0.6 million in laboratory supplies, $0.8 million in facilities-related costs, $0.1 million in consultant expenses, and $0.4 million in net direct research and development expenses mainly due to termination of UBX0101 offset by increase of $2.0 million in license expense from Ascentage International reaching a milestone, which was a non-cash expense. General and administrative expenses decreased by $0.3 million, to $6.0 million for the three months ended June 30, 2021 from $6.3 million for the three months ended June 30, 2020. The decrease was primarily due to a decrease of $0.6 million in personnel costs due to reduction in force, offset by increases of $0.1 million in facilities-related costs, $0.1 million in professional fees and $0.1 million in insurance-related expense. About UBX1325 UBX1325 is an investigational compound being studied for age-related diseases of the eye, including diabetic macular edema (DME), age-related macular degeneration (AMD), and diabetic retinopathy (DR). UBX1325 is a potent small molecule inhibitor of Bcl-xL, a member of the Bcl-2 family of apoptosis-regulatory proteins. UBX1325 is designed to inhibit the function of proteins senescent cells rely on for survival. In preclinical studies, UNITY has demonstrated that targeting Bcl-xL with UBX1325 preferentially eliminates senescent cells from diseased tissue while sparing healthy cells. In its Phase 1 safety trial in patients with advanced DME or wet AMD who were no longer expected to benefit from anti-VEGF therapies, UBX1325 showed a favorable safety and tolerability profile as well as initial evidence of relevant biological efficacy. UNITY’s goal with UBX1325 is to transformationally improve real-world outcomes for patients with DME, AMD, and DR. About UNITY UNITY is developing a new class of therapeutics to slow, halt, or reverse diseases of aging. UNITY’s current focus is on creating medicines to selectively eliminate or modulate senescent cells and thereby provide transformative benefit in age-related ophthalmologic and neurologic diseases. More information is available at  or follow us on Twitter and LinkedIn. Forward-Looking Statements This press release contains forward-looking statements including statements related to UNITY’s understanding of cellular senescence and the role it plays in diseases of aging, the potential for UNITY to develop therapeutics to slow, halt, or reverse diseases of aging, including for ophthalmologic and neurologic diseases, our expectations regarding potential benefits, activity, effectiveness, and safety of UBX1325, the potential for UNITY to successfully commence and complete clinical studies of UBX1325 for DME, AMD, and other ophthalmologic diseases, the expected timing of results of our studies of UBX1325, the timing of the expected commencement, progression, and conclusion of our studies including those of UBX1325, and UNITY’s expectations regarding the sufficiency of its cash runway. These statements involve substantial known and unknown risks, uncertainties, and other factors that may cause our actual results, levels of activity, performance, or achievements to be materially different from the information expressed or implied by these forward-looking statements, including the risk that the COVID-19 worldwide pandemic may continue to negatively impact the development of preclinical and clinical drug candidates, including delaying or disrupting the enrollment of patients in clinical trials, risks relating to the uncertainties inherent in the drug development process, and risks relating to UNITY’s understanding of senescence biology. We may not actually achieve the plans, intentions, or expectations disclosed in our forward-looking statements, and you should not place undue reliance on our forward-looking statements. Actual results or events could differ materially from the plans, intentions, and expectations disclosed in the forward-looking statements we make. The forward-looking statements in this press release represent our views as of the date of this release. We anticipate that subsequent events and developments will cause our views to change. However, while we may elect to update these forward-looking statements at some point in the future, we have no current intention of doing so except to the extent required by applicable law. You should, therefore, not rely on these forward-looking statements as representing our views as of any date subsequent to the date of this release. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to the business of UNITY in general, see UNITY’s most recent Quarterly Report on Form 10-Q for the quarter ended June 30, 2021, filed with the Securities and Exchange Commission on August 10, 2021, as well as other documents that may be filed by UNITY from time to time with the Securities and Exchange Commission. Unity Biotechnology, Inc. Statements of Operations and Comprehensive Loss (In thousands, except share and per share amounts) (Unaudited) Unity Biotechnology, Inc. Condensed Balance Sheets (In thousands)