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UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
WASHINGTON, D.C. 20549
FORM
CURRENT REPORT
Pursuant to Section 13 or 15(d) of the Securities Exchange Act of 1934
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Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions:
Securities registered pursuant to Section 12(b) of the Act:
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Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 (§ 230.405 of this chapter) or Rule 12b-2 of the Securities Exchange Act of 1934 (§ 240.12b-2 of this chapter).
Emerging growth company
If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act.
Item 2.02 Results of Operations and Financial Condition.
On August 15, 2022, Biora Therapeutics, Inc. issued a press release announcing its financial results for the second quarter ended June 30, 2022 and an updated corporate presentation. The press release and corporate presentation are furnished as Exhibit 99.1 and Exhibit 99.2, respectively, to this Current Report on Form 8-K.
As provided in General Instruction B.2 of Form 8-K, the information in this Item 2.02 and Exhibit 99.1 and 99.2 incorporated herein shall not be deemed to be “filed” for purposes of Section 18 of the Securities Exchange Act of 1934, as amended (the “Exchange Act”) or otherwise subject to the liabilities of that section, nor shall such information or Exhibit 99.1 and 99.2 be deemed to be incorporated by reference in any filing under the Securities Act of 1933, as amended, or the Exchange Act, except as shall be expressly set forth by specific reference in such a filing.
Item 9.01 Financial Statements and Exhibits.
(d) Exhibits.
99.1 |
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99.2 |
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104 |
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Cover Page Interactive Data File (embedded with the Inline XBRL document) |
SIGNATURES
Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned thereunto duly authorized.
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Biora Therapeutics, Inc. |
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Date: |
August 15, 2022 |
By: |
/s/ Aditya P. Mohanty |
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Aditya P. Mohanty |
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Exhibit 99.1 |
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Biora Therapeutics Provides Corporate Update and Reports
Second Quarter 2022 Financial Results
Successfully completed PM-602 human study for its targeted therapeutics platform demonstrating promising device performance in active ulcerative colitis patients
Successfully completed PM-611 human study for its targeted therapeutics platform in healthy volunteers showing promising performance regardless of feeding schedule
Confirmed planned phase 1 IND filing in Q1 based on FDA feedback on PGN-600 program in Ulcerative Colitis
Strengthened patent position for both targeted and systemic therapeutics platforms
Management will host conference call and webcast today at 4:30 PM Eastern / 1:30 PM Pacific
SAN DIEGO, August 15, 2022 – Biora Therapeutics, Inc. (Nasdaq: BIOR), the biotech company that is reimagining therapeutics, today provided a corporate update and reported financial results for the second quarter ended June 30, 2022.
During the second quarter, the company completed its PM-611 device function study which assessed whether the autonomous location functionality of the ingestible devices designed for targeted drug delivery was impacted by a fed state as compared to a fasted state. The study demonstrated that all analyzed capsules indicated entry to the colon, activation, and deployment, regardless of fasted or fed schedule, with no failure modes observed in the analyzed devices. These study results suggest that the Drug Delivery System (DDS) capsule could be the first ingestible therapeutic delivery device that does not require fasting or other food restriction for use, which may be an important consideration for patients who need frequent dosing in chronic diseases like ulcerative colitis.
The company also recently announced topline results from its PM-602 device function study that assessed the safety and performance of the targeted delivery device in active ulcerative colitis patients. The study demonstrated that the device was well tolerated, and that the device performed as intended in active ulcerative colitis (UC) patients. In all seven patients, the device accurately identified entry into the colon, triggered release of a non-drug liquid payload, and achieved distribution across the entire colon. The company is not aware of any other oral drug delivery technology that accurately detects colon entry, especially in the environment of inflammation, blood and highly variable motility seen in active ulcerative colitis.
In the second quarter the company submitted a Type C filing to the United States Food and Drug Administration (FDA) asking for feedback on its proposed PGN-600 clinical development plan related to its targeted therapeutics platform. In a constructive response received recently, the FDA generally agreed with the proposed clinical trial design and provided helpful feedback on protocols. The FDA also reviewed the proposed supporting data package and confirmed the anticipated need for additional toxicology data. The company believes it will be in a position to file an IND in Q1 2023 to support the initiation of its Phase 1 clinical study.
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Exhibit 99.1 |
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Finally, Biora strengthened its intellectual property (IP) portfolio during the second quarter with a newly issued US patent and the allowance of two additional US patent applications. The issued US patent (No. 11,363,964) is directed to the company’s targeted delivery localization technology used in the DDS device which enables detection of device entry into the colon. This builds on the company’s proprietary localization technology portfolio, which includes 29 issued patents worldwide, including multiple patents issued in both the US and Europe. Related to the company’s systemic therapeutics platform, the company was pleased to receive a Notice of Allowance for a US patent application covering liquid jet delivery of GLP-1 receptor agonists to the small intestine for the treatment of any disease, including Type 2 diabetes.
“We’ve continued to generate important human safety and performance data in support of our targeted therapeutics platform, where so far we have demonstrated that our DDS capsule functions as intended in humans, and especially in UC patients. After receiving constructive feedback from the FDA, we are now preparing to initiate our clinical program for PGN-600,” said Adi Mohanty, Chief Executive Officer of Biora Therapeutics. “This is an important step for this program, and we are very excited about the opportunity to improve treatment for patients with ulcerative colitis. We also continue to strengthen our IP portfolio, illustrating the innovative and proprietary aspects of our technology and platforms for both systemic and targeted drug delivery.”
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Exhibit 99.1 |
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Second Quarter 2022 and Recent Corporate Highlights
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Exhibit 99.1 |
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Second Quarter 2022 Financial Results
Comparison of Three Months Ended June 30, 2022 and March 31, 2022
The company generated $0.9 million in revenues during the second quarter, out of which $0.8 million came from discontinued operations. The company generated $1.4 million in revenues during the first quarter, out of which $1.3 million came from discontinued operations. Operating expenses were $14.3 million for the three months ended June 30, 2022, compared to $20.0 million for the three months ended March 31, 2022.
Net loss was $5.5 million and net loss per share was $0.03 for the three months ended June 30, 2022, compared to net loss of $13.8 million and net loss per share of $0.08 for the three months ended March 31, 2022.
Net gain from discontinued operations was $0.5 million for the three months ended June 30, 2022 with no impact to loss per share, compared to net gain from discontinued operations of $0.7 million with no impact to loss per share for the three months ended March 31, 2022.
Comparison of Three Months Ended June 30, 2022 and 2021
Operating expenses were $14.3 million for the three months ended June 30, 2022, compared to $36.1 million for the three months ended June 30, 2021.
Net loss was $5.5 million and net loss per share was $0.03 for the three months ended June 30, 2022, compared to net loss of $78.5 million and net loss per share of $1.23 for the three months ended June 30, 2021.
Net gain from discontinued operations was $0.5 million for the three months ended June 30, 2022, with no impact to loss per share, compared to net loss from discontinued operations of $37.1 million and net loss per share for discontinued operations of $0.58 for the three months ended June 30, 2021.
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Exhibit 99.1 |
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Webcast and Conference Call Information
Biora Therapeutics will host a webcast and conference call to discuss the second quarter financial results and answer investment community questions today, Monday, August 15, 2022 at 4:30 p.m. Eastern / 1:30 p.m. Pacific.
The live call may be accessed by dialing 1-877-423-9813 (domestic) or 1-201-689-8573 (international) and entering the conference code: 13731511. A live webcast will be available via the Investors section of the company website, with a replay available online for 60 days following the call.
About Biora Therapeutics
Biora Therapeutics is the biotech company that is reimagining therapeutics. By creating innovative smart pills designed for targeted drug delivery to the GI tract, and systemic, needle-free delivery of biotherapeutics, the company is developing therapies to improve patients’ lives. Biora envisions a world where patients have access to needle-free drug delivery and better therapeutic outcomes.
For more information, visit bioratherapeutics.com or follow the company on LinkedIn or Twitter.
Safe Harbor Statement or Forward-Looking Statements
This press release contains “forward-looking statements” within the meaning of the “safe harbor” provisions of the Private Securities Litigation Reform Act of 1995, which statements are subject to substantial risks and uncertainties and are based on estimates and assumptions. All statements, other than statements of historical facts included in this press release, including statements concerning future expectations of our research and development efforts and clinical trials and programs, are forward-looking statements. In some cases, you can identify forward-looking statements by terms such as “may,” “might,” “will,” “objective,” “intend,” “should,” “could,” “can,” “would,” “expect,” “believe,” “design,” “estimate,” “predict,” “potential,” “plan” or the negative of these terms, and similar expressions intended to identify forward-looking statements. These statements reflect our plans, estimates, and expectations, as of the date of this press release. These statements involve known and unknown risks, uncertainties and other factors that could cause our actual results to differ materially from the forward-looking statements expressed or implied in this press release. Such risks, uncertainties, and other factors include, among others, our ability to innovate in the field of precision medicine, our ability to obtain and maintain regulatory approval or clearance of our products on expected timelines or at all, our plans to research, develop, and commercialize new products, the unpredictable relationship between preclinical study results and clinical study results, our expectations regarding future revenue generating opportunities with current or future pharmaceutical collaborators, our ability to raise sufficient capital to achieve our business objectives, the ongoing COVID-19 pandemic, and those risks described in “Risk Factors” and “Management’s Discussion and Analysis of Financial Condition and Results of Operations” in our Annual Report on Form 10-K for the year ended December 31, 2021 filed with the SEC and other subsequent documents, including Quarterly Reports, that we file with the SEC.
Biora Therapeutics expressly disclaims any obligation to update any forward-looking statements whether as a result of new information, future events or otherwise, except as required by law.
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Exhibit 99.1 |
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Investor Contact
Chuck Padala
Managing Director, LifeSci Advisors
IR@bioratherapeutics.com
(646) 627-8390
Media Contact
media@bioratherapeutics.com
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Exhibit 99.1 |
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Biora Therapeutics, Inc.
Condensed Consolidated Statements of Operations
(Unaudited)
(In thousands, except share and per share amounts)
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Three Months Ended |
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June 30, |
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March 31, |
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Revenues |
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$ |
104 |
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$ |
107 |
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Operating expenses: |
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Research and development |
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5,904 |
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6,558 |
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Selling, general and administrative |
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8,410 |
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13,457 |
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Total operating expenses |
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14,314 |
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20,015 |
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Loss from operations |
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(14,210 |
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(19,908 |
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Interest expense, net |
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(2,772 |
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(2,760 |
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Gain (loss) on warrant liability |
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4,413 |
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8,989 |
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Other income (expense), net |
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5,735 |
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(811 |
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Loss before income taxes |
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(6,834 |
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(14,490 |
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Income tax benefit |
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(837 |
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— |
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Loss from continuing operations |
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(5,997 |
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(14,490 |
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Gain (loss) from discontinued operations |
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484 |
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682 |
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Net loss |
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$ |
(5,513 |
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$ |
(13,808 |
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Net loss per share from continuing operations, basic and diluted |
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$ |
(0.03 |
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$ |
(0.08 |
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Net loss per share from discontinued operations, basic and diluted |
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$ |
— |
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$ |
— |
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Net loss per share, basic and diluted |
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$ |
(0.03 |
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$ |
(0.08 |
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Weighted average shares outstanding, basic and diluted |
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184,371,626 |
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183,201,663 |
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Exhibit 99.1 |
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Biora Therapeutics, Inc.
Condensed Consolidated Statements of Operations
(Unaudited)
(In thousands, except share and per share amounts)
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Three Months Ended |
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2022 |
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2021 |
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Revenues |
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$ |
104 |
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$ |
463 |
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Operating expenses: |
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Research and development |
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5,904 |
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13,401 |
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Selling, general and administrative |
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8,410 |
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22,715 |
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Total operating expenses |
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14,314 |
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36,116 |
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Loss from operations |
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(14,210 |
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(35,653 |
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Interest expense, net |
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(2,772 |
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(3,502 |
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Gain (loss) on warrant liability |
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4,413 |
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(5,146 |
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Other income, net |
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5,735 |
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2,901 |
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Loss before income taxes |
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(6,834 |
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(41,400 |
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Income tax benefit |
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(837 |
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— |
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Loss from continuing operations |
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(5,997 |
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(41,400 |
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Gain (loss) from discontinued operations |
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484 |
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(37,131 |
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Net loss |
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$ |
(5,513 |
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$ |
(78,531 |
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Net loss per share from continuing operations, basic and diluted |
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$ |
(0.03 |
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$ |
(0.65 |
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Net loss per share from discontinued operations, basic and diluted |
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$ |
— |
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$ |
(0.58 |
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Net loss per share, basic and diluted |
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$ |
(0.03 |
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$ |
(1.23 |
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Weighted average shares outstanding, basic and diluted |
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184,371,626 |
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63,942,298 |
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Exhibit 99.1 |
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Biora Therapeutics, Inc.
Condensed Consolidated Balance Sheets
(Unaudited)
(In thousands)
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June 30, |
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December 31, |
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(1) |
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Assets |
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Current assets: |
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Cash and cash equivalents |
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$ |
48,506 |
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$ |
88,397 |
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Accounts receivable, net |
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— |
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653 |
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Income tax receivable |
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817 |
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— |
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Prepaid expenses and other current assets |
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8,675 |
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7,232 |
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Current assets of disposal group held for sale |
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2,147 |
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2,147 |
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Total current assets |
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60,145 |
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98,429 |
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Property and equipment, net |
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2,454 |
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4,012 |
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Right-of-use assets |
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2,203 |
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— |
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Other assets |
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6,227 |
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326 |
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Goodwill |
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6,072 |
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6,072 |
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Total assets |
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$ |
77,101 |
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$ |
108,839 |
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Liabilities and Stockholders' Deficit |
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Current liabilities: |
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Accounts payable |
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$ |
4,315 |
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$ |
8,709 |
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Accrued expenses and other current liabilities |
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30,940 |
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34,157 |
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Warrant liability |
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5,329 |
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18,731 |
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Current portion of capital lease obligations |
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— |
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12 |
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Total current liabilities |
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40,584 |
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61,609 |
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Convertible notes, net |
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127,086 |
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126,392 |
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Other long-term liabilities |
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5,537 |
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5,814 |
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Total liabilities |
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$ |
173,207 |
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$ |
193,815 |
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Stockholders' deficit: |
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Common stock |
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150 |
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146 |
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Additional paid-in capital |
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730,833 |
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722,646 |
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Accumulated deficit |
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(808,007 |
) |
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(788,686 |
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Treasury stock |
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(19,082 |
) |
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(19,082 |
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Total stockholders' deficit |
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(96,106 |
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(84,976 |
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Total liabilities and stockholders' deficit |
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$ |
77,101 |
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$ |
108,839 |
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(1) The condensed consolidated balance sheet data as of December 31, 2021 has been derived from the audited consolidated financial statements
CORPORATE PRESENTATION August 2022 Exhibit 99.2
FORWARD-LOOKING STATEMENTS This presentation contains “forward-looking statements” within the meaning of the federal securities laws, which statements are subject to substantial risks and uncertainties and are based on estimates and assumptions. All statements, other than statements of historical fact included in this presentation, including statements concerning our plans, objectives, goals, strategies, future events, plans or intentions relating to product candidates, estimates of market size, the anticipated timing, design and conduct of our planned pre-clinical and clinical trials, the anticipated timing for pre-clinical and clinical data, the development of our product candidates, the potential clinical benefits of our product candidates, including efficacy and safety benefits, the potential benefits of strategic partnerships and licensing arrangements and our intent to enter into any strategic partnerships or licensing arrangements, the timing and likelihood of success, plans and objectives of management for future operations and future results of anticipated product development efforts, are forward-looking statements. In some cases, you can identify forward-looking statements by terms such as “may,” “might,” “will,” “objective,” “intend,” “should,” “could,” “can,” “would,” “expect,” “believe,” “design,” “estimate,” “predict,” “potential,” “plan” or the negative of these terms, and similar expressions intended to identify forward-looking statements. These statements involve known and unknown risks, uncertainties and other factors that could cause our actual results to differ materially from the forward-looking statements expressed or implied in this presentation, including difficulties in managing changes to our organization due to our strategic transformation; competition from third parties with respect to our product candidates; risks related to the supply and manufacturing of and complexity of components in our devices; whether we will be able to develop our precision medicine products, and, if developed, that such product candidates will be authorized for marketing by regulatory authorities, or will be commercially successful; and those described in “Risk Factors” and “Management’s Discussion and Analysis of Financial Condition and Results of Operations” in our Annual Report on Form 10-K for the fiscal year ended December 31, 2021, and elsewhere in such filing and in other subsequent disclosure documents, including our Quarterly Reports on Form 10-Q, filed with the U.S. Securities and Exchange Commission. We cannot assure you that we will realize the results, benefits or developments that we expect or anticipate or, even if substantially realized, that they will result in the consequences or affect us or our business in the way expected. Forward-looking statements are not historical facts and reflect our current views with respect to future events. Given the significant uncertainties, you should evaluate all forward-looking statements made in this presentation in the context of these risks and uncertainties and not place undue reliance on these forward-looking statements as predictions of future events. All forward-looking statements in this presentation apply only as of the date made and are expressly qualified in their entirety by the cautionary statements included in this presentation. We disclaim any intent to publicly update or revise any forward-looking statements to reflect subsequent events or circumstances, except as required by law. Industry and Market Data: We obtained the industry, market, and competitive position data used throughout this presentation from our own internal estimates and research, as well as from industry and general publications, and research, surveys, and studies conducted by third parties. Internal estimates are derived from publicly available information released by industry analysts and third-party sources, our internal research and our industry experience, and are based on assumptions made by us based on such data and our knowledge of the industry and market, which we believe to be reasonable. In addition, while we believe the industry, market, and competitive position data included in this prospectus is reliable and based on reasonable assumptions, we have not independently verified any third-party information, and all such data involve risks and uncertainties and are subject to change based on various factors. These and other factors could cause results to differ materially from those expressed in the estimates made by the independent parties and by us.
ABOUT BIORA THERAPEUTICS Our mission is to reimagine therapeutics and their delivery TARGETED ORAL DELIVERY OF BIOTHERAPEUTICS Treatment at the site of disease in the GI tract could improve outcomes for patients with inflammatory bowel disease SYSTEMIC ORAL DELIVERY OF BIOTHERAPEUTICS Ingestible technology designed to enable needle-free, systemic delivery of large molecules for improved management of chronic diseases Innovating smart capsule technologies to deliver the right dose to the right place, safely
THERAPEUTIC PIPELINE PROGRAM PROGRAM INDICATION DESIGN/FEASIBILITY PRECLINICAL CLINICAL TARGETED THERAPEUTICS DDS Device -- PGN-600 Tofacitinib + Device UC PGN-001 Adalimumab + Device UC SYSTEMIC THERAPEUTICS OBDS Device -- PGN-OB2 GLP-1 agonist + Device Diabetes PGN-OB1 Adalimumab variant + Device Autoimmune Ionis Collaboration Antisense therapy + Device Undisclosed Large Pharma 1 Collaboration Undisclosed drug + Device Undisclosed Large Pharma 2 Collaboration Undisclosed drug + Device Undisclosed
TARGETED THERAPEUTICS
ULCERATIVE COLITIS: THE TREATMENT GAP Despite therapeutics targeting different pathways, few patients achieve long-term remission Alsoud D, Verstockt B, Fiocchi C, Vermeire S. Breaking the therapeutic ceiling in drug development in ulcerative colitis. Lancet Gastroenterol Hepatol. 2021;6(7):589-595. Hirten RP, Sands BE. New Therapeutics for Ulcerative Colitis. Annu Rev Med. 2021;72:199-213. Shivashankar R, Tremaine WJ, Harmsen WS, Loftus EV Jr. Incidence and Prevalence of Crohn's Disease and Ulcerative Colitis in Olmsted County, Minnesota From 1970 Through 2010. Clin Gastroenterol Hepatol. 2017;15(6):857-863. Inflammatory bowel disease (IBD) includes Crohn’s disease and ulcerative colitis (UC) UC causes inflammation and damage to the large intestine About 1 million people in the U.S. are affected with UC, and ~40,000 cases are diagnosed each year3 ABOUT ULCERATIVE COLITIS Treatment begins with 2-3 months of drug therapy to reach therapeutic levels 15-30% of patients achieve remission of symptoms after induction with any drug therapy1,2 UC TREATMENT CYCLE Patients undergo induction (2-3 months) with different drug Efficacy rate decreases with successive rounds of therapy1 60% of patients in remission relapse within 12 months2 INDUCTION TRIAL INDUCTION TRIAL RELAPSE NO RESPONSE MAINTENANCE Patients in remission continue drug therapy to prevent relapse
UNMET NEED IN ULCERATIVE COLITIS Targeted delivery could enable rapid induction and improve patient response THERAPEUTIC CHALLENGES POTENTIAL SOLUTION Systemic toxicity issues may limit daily dosage of UC drugs Reduced systemic uptake is designed to reduce toxicity and adverse events Difficulty of achieving sufficient drug levels at site of disease 1 Targeted delivery is designed to increase drug levels at the site of disease, which is correlated with improved outcomes1 2 Combination therapy is limited by toxicity 3 Reduced toxicity could enable combination therapy2 TARGETED THERAPEUTICS Research in partnership with: Verstockt B, Alsoud D, van Oostrom J, et al. Tofacitinib tissue exposure correlates with endoscopic outcome. Poster presented at: 17th Congress of the European Crohn’s and Colitis Organisation (ECCO), February 18, 2022, virtual. van Oostrom J, Verstockt B, Hanzel J, et al. Pharmacokinetic stratification of cytokine profiles during anti-TNF induction treatment in moderate-to-severe ulcerative colitis. Poster presented at: 17th Congress of the European Crohn’s and Colitis Organisation (ECCO), February 18, 2022, virtual.
RESEARCH DATA SUPPORTS TARGETED APPROACH Tissue drug concentration correlates with endoscopic outcomes in UC Verstockt B, Alsoud D, van Oostrom J, et al. Tofacitinib tissue exposure correlates with endoscopic outcome. Poster presented at: 17th Congress of the European Crohn’s and Colitis Organisation (ECCO), February 18, 2022, virtual. RESULTS 30 consecutive UC patients with active endoscopic disease initiated treatment with tofacitinib and prospectively monitored median tissue exposure Responders Non-responders IC90 P= 0.04 TOFACITINIB TISSUE EXPOSURE EXCEEDED IC90 IN RESPONDERS Research presented at ECCO 2022 and DDW 2022 in collaboration with: Tofacitinib tissue exposure at the end of induction was associated with endoscopic improvement by week 16 (p=0.04) In responders (n=14), median tofacitinib tissue exposure exceeded IC90
TARGETED THERAPEUTIC DELIVERY FOR IBD Needle-free, oral drug delivery to the colon ORAL ADMINISTRATION Convenient oral capsule the size of a fish oil pill TARGETED THERAPEUTICS 1. Biora Therapeutics internal data AUTONOMOUS LOCATION Proprietary autolocation in the GI tract for accurate drug delivery regardless of fasted or fed state1 TARGETED DRUG DELIVERY Method designed to coat the length of the colon with liquid formulation, minimizing systemic uptake
DEVICE FUNCTION STUDIES Demonstrated accurate localization and delivery to colon Biora Therapeutics internal data ACCURATE LOCALIZATION AND DELIVERY TO HUMAN COLON Clinical device validation for localization and delivery function using scintigraphic imaging in normal, healthy volunteers Achieved distribution across the entire colon TARGETED THERAPEUTICS ACCURATE DELIVERY TO COLON IN CANINES Pharmacokinetic data from two marker drugs administered in canine model Successful delivery to colon via DDS No early release of drug No drug absorption in upper GI tract
Biora Therapeutics internal data DEVICE FUNCTION STUDIES Three successful human studies show device functions with and without food, and in active UC patients TARGETED THERAPEUTICS PM-601 Device Function Study in Healthy Volunteers – Fasted State PM-611 Device Function Study in Healthy Volunteers – Fasted and Fed PM-602 Device Function Study in Patients with Active UC 83% of devices accurately identified entry into the colon (10/12) Achieved distribution of payload across the entire colon No early deployment before colon detection Presenting data at upcoming conference 100% of analyzed devices indicated entry to the colon, activation, and deployment, and were unaffected by food (39/39) No failure modes observed in the analyzed devices No serious adverse events reported 100% of devices accurately identified entry into the colon, triggered release of a liquid payload, and achieved distribution across the entire colon (7/7) Device was well tolerated and performed as intended in active ulcerative colitis patients Presenting data at upcoming conference
PGN-600 PRECLINICAL TOX STUDY RESULTS Reduced systemic uptake, better PK effect and coverage Biora Therapeutics internal data RESULTS Non-GLP tox study; 7 days/QD in canine model compared PGN-600 (tofacitinib 10mg liquid formulation delivered via DDS capsule) vs. standard oral tablet (tofacitinib 10mg) Reduced drug levels in blood vs. standard oral tablet Tissue drug levels at average ~100X higher along the length of the colon vs. standard oral tablet Data suggest that a dose lower than the standard 10mg tofacitinib may provide increased tissue levels while reducing systemic exposure TARGETED THERAPEUTICS PLASMA LEVEL CMAX 5X LOWER COLON TISSUE COVERAGE ~100X HIGHER
PGN-600 (TOFACITINIB + DDS DEVICE) Clinical Development Plan Q4 2022 Q1 2023 Q2 2023 Q3 2023 Q4 2023 14-DAY GLP TOX STUDY 56-DAY GLP TOX STUDY TARGETED THERAPEUTICS INTERIM DATA Preclinical / IND enabling Clinical FINAL DATA 2 weeks/QD 8 weeks/QD Design: SAD/MAD Primary endpoint Safety & tolerability Key secondary endpoints PK/PD Design: randomized vs. placebo Primary endpoint Remission after 8 weeks in moderate to severe UC patients Key secondary endpoints Safety & tolerability PK/PD PHASE 2 IN UC PATIENTS PHASE 1 IN HEALTHY VOLUNTEERS
PHASE 1: SINGLE AND MULTIPLE ASCENDING DOSE STUDIES Evaluate safety, tolerability, pharmacokinetics and pharmacodynamics of PGN-600 in healthy volunteers SAD 24 subjects MAD 24 subjects D7 Blood Stool D1 Blood Stool D2 Blood Stool D3 Blood Stool PGN-600 Baseline D1 Blood Stool D2 Blood Stool D3 Blood Stool D7 Blood & Tissue Stool Placebo or PGN-600 7 days/QD Baseline Blood Stool PATIENT POPULATION Normal healthy volunteers Total of 48 subjects (24 SAD and 24 MAD subjects) STUDY DESIGN Randomized, double-blind (participant and site), placebo-controlled study to evaluate the safety, tolerability, and PK/PD of SAD and MAD doses of PGN-600 in healthy subjects OBJECTIVES Demonstrate safety and tolerability of PGN-600, assess PK and PD effects of tofacitinib released from PGN-600 over 8 days in NHV in blood and in tissue. TARGETED THERAPEUTICS N=9 N=3 5mg Placebo N=9 N=3 10mg Placebo N=9 N=3 10mg 7x QD Placebo N=9 N=3 5mg 7x QD Placebo
Source: Evaluate Pharma; GlobalData Global annual sales forecast for ulcerative colitis therapeutics: $7 billion in 20221 >10 FDA-approved drugs for UC ULCERATIVE COLITIS: TREATMENT LANDSCAPE Potential for market-leading efficacy in tofacitinib creates sizeable opportunity Vedolizumab Adalimumab Ustekinumab Infliximab Tofacitinib / Ozanimod* PRE-BIOLOGICS FIRST-LINE BIOLOGIC SECOND-LINE BIOLOGIC THIRD-LINE BIOLOGIC Adalimumab Infliximab Vedolizumab TARGETED THERAPEUTICS *Non-biologic drug therapies
SYSTEMIC THERAPEUTICS
UNMET NEED Needles are associated with poor disease management Frost & Sullivan research commissioned by Rani Therapeutics Holdings, Inc. https://ir.ranitherapeutics.com/static-files/b1f080bf-a860-4136-87cb-d6f7c49c1502 Spain CV, Wright JJ, Hahn RM, Wivel A, Martin AA. Self-reported Barriers to Adherence and Persistence to Treatment With Injectable Medications for Type 2 Diabetes. Clin Ther. 2016;38(7):1653-1664.e1. doi:10.1016/j.clinthera.2016.05.009 of diabetics miss 4+ injections per week1 38 % of patients fail to maintain diabetes treatment due to injection concerns when using an injectable GLP-1 agonist2 42 % higher discontinuation rate for diabetes patients initiating treatment with an injectable GLP-1 agonist vs. those starting oral therapy2 71 %
ORAL BIOTHERAPEUTICS DELIVERY SYSTEM Needle-free, oral delivery to small intestine ORAL CAPSULE Convenient oral capsule the size of a multivitamin for ease of swallowing PRECISE DELIVERY Enteric trigger for precise timing of drug delivery to the small intestine NEEDLE-FREE ADMINISTRATION Liquid jet injection to the small intestine to maximize systemic uptake RESEARCH COLLABORATIONS Ionis Large Pharma 1 Large Pharma 2 SYSTEMIC THERAPEUTICS
Biora Therapeutics internal data See also Lee SN, Stork C, Smith J, et al. Assessing the performance of an oral biotherapeutic delivery system (OBDS) using intra-duodenal endoscopy delivery in Yucatan minipigs. Poster presented at: Controlled Release Society Annual Meeting, July 13-14, 2022, Montreal, Canada. Lee SN, Stork C, Smith J, et al. Development of ex-vivo and in-vivo models to assess the performance of an oral biotherapeutic delivery system (OBDS) capsule. Poster presented at: Controlled Release Society Annual Meeting, July 13-14, 2022, Montreal, Canada. PGN-OB1 PRECLINICAL PERFORMANCE Excellent systemic uptake for orally delivered large molecules demonstrated in animal models RESULTS Multiple studies in swine model with endoscopically placed, autonomous device compared to IV administration In multiple studies in swine model, an average of ~22% bioavailability was observed in animals where drug was detected in blood1 SYSTEMIC THERAPEUTICS BIOAVAILABILITY COMPARABLE TO IV Achieved up to 67% bioavailability for a variant of adalimumab1 For comparison, commercially available oral large molecules achieve 1% or less bioavailability Precise and reliable release of payload in small intestine demonstrated in canine studies No issues observed with safety or tolerability of the device
DEVELOPMENT TIMELINE
DEVELOPMENT TIMELINE PROGRAM Q3 2022 Q4 2022 Q1 2023 Q2 2023 Q3 2023 Q4 2023 MILESTONES/CATALYSTS TARGETED THERAPEUTICS Establish device performance in UC patients Preclinical toxicology to support phase 1 and phase 2 study Evaluate the safety & tolerability, PK/PD of PGN-600 subject to FDA review SYSTEMIC THERAPEUTICS Bioavailability of oral delivery of biologics using OBDS; enables human studies Establish device performance in healthy volunteers PM-602 14-Day Tox Clinical Studies Phase 1 Clinical Phase 2 Clinical Preclinical PK Studies 56-Day Tox INTERIM DATA
APPENDIX
TARGETED THERAPEUTICS PUBLICATIONS bioratherapeutics.com/publications Development of targeted therapeutic antibodies for the treatment of inflammatory bowel disease: A proof of concept. Presented at DDW 2019. A comparison of systemic versus targeted anti-TNFα antibody in treatment of colitis induced by adoptive transfer of CD44-/CD62L+ T-cells into RAG2-/- mice recipients. Presented at DDW 2019. Targeted delivery of soluble tofacitinib citrate to the site of inflammation to improve efficacy and safety. Presented at DDW 2021. Development of a novel drug delivery system for treatment of Ulcerative Colitis. Presented at DDW 2021. Development of a Novel Drug Delivery System to Deliver Drugs Directly to the Colonic Mucosa, Resulting in Improved Efficacy and Reduced Systemic Exposure for the Treatment of Ulcerative Colitis. Crohn's & Colitis 360. 2021, 3, 1–5. Tofacitinib tissue exposure correlates with endoscopic outcome. Presented at ECCO 2022 and DDW 2022. Pharmacokinetic stratification of cytokine profiles during anti-TNF induction treatment in moderate-to-severe UC. Presented at ECCO 2022 and DDW 2022. TARGETED THERAPEUTICS
SYSTEMIC THERAPEUTICS PUBLICATIONS bioratherapeutics.com/publications SYSTEMIC THERAPEUTICS Development of ex-vivo and in-vivo models to assess the performance of an oral biotherapeutic delivery system (OBDS) capsule. Poster presented at: Controlled Release Society Annual Meeting, July 13-14, 2022, Montreal, Canada. Assessing the performance of an oral biotherapeutic delivery system (OBDS) using intra-duodenal endoscopy delivery in Yucatan minipigs. Poster presented at: Controlled Release Society Annual Meeting, July 13-14, 2022, Montreal, Canada.
SAMPLING & DIAGNOSTICS 17 patent families covering: GI sample preservation GI analyte detection & quantification systems Complementary diagnostic markers Protein and nucleic acid markers & assays THERAPEUTICS 28 patent families covering: Treatment via ingestible device GI delivery PK/PD profiles GI delivery dosing regimens GI delivery drug combinations Liquid drug formulations DEVICES 37 patent families covering: Device designs, materials, components & manufacturing GI localization Devices for targeted delivery to GI tract Devices for targeted GI sampling systems Devices for jet delivery into GI tissue INTELLECTUAL PROPERTY PORTFOLIO Diverse patent portfolio with 82 distinct patent families1 Approximately 170 issued patents and 170 pending applications in major countries and regions around the world
