Precigen Reports Second Quarter and First Half 2023 Financial Results and Provides Update on Portfolio Prioritization and Capital Allocation Strategies to Extend Projected Cash Runway into 2025
– The FDA confirmed that the ongoing Phase 1/2 single arm study of PRGN-2012 in RRP will serve as the pivotal study to support accelerated approval and no additional randomized, placebo-controlled trial will be required to support submission of a BLA –
– Company to prioritize portfolio activities to accelerate PRGN-2012 and continue advancement of other key programs, by implementing strategies to reduce clinical costs (e.g., reducing CRO costs without internal R&D headcount reduction) and reduce SG&A costs –
– Completely retired the outstanding balance of convertible notes –
– Cash, cash equivalents, short-term and long-term investments totaled
"Today we announced that the FDA confirmed the ongoing Phase 1/2 study of PRGN-2012 will serve as the pivotal study to support accelerated approval, an important milestone that brings
"As a result of our capital raise in January, our portfolio prioritization and other cost-saving measures, including completely retiring our convertible notes, the Company's balance sheet is well positioned for the future," said
Program Highlights
PRGN-2012 AdenoVerse™ Immunotherapy in RRP
- The Company announced that the
US Food and Drug Administration (FDA) has agreed that the ongoing Phase 1/2 single arm study of the first-in-class investigational PRGN-2012 AdenoVerse™ immunotherapy for the treatment of recurrent respiratory papillomatosis (RRP) will serve as pivotal for the purpose of filing an accelerated approval request for licensure. The FDA also confirmed no additional randomized, placebo-controlled trial will be required to support submission of a biologics license application (BLA). Based on the FDA guidance, the Company also plans to initiate a confirmatory study prior to submission of the BLA. - PRGN-2012 is an investigational off-the-shelf AdenoVerse immunotherapy designed to elicit immune responses directed against cells infected with human papillomavirus (HPV) 6 or HPV 11 for the treatment of RRP.
- The Company completed enrollment and dosing in the Phase 2 portion of the study (N=23) bringing the total number of enrolled patients to 35 at the recommended Phase 2 dose. Patient follow up is currently ongoing and data collection is anticipated to be completed by the second quarter of 2024.
- The Company announced that the FDA granted Breakthrough Therapy Designation for PRGN-2012 for the treatment of RRP, adding to the existing Orphan Drug Designation. The Breakthrough designation is based on positive Phase 1 clinical data that showed that 50% of adult RRP patients (who had ≥3 surgeries to treat the disease in the year prior treatment) were "surgery-free" (Complete Response) after PRGN-2012 treatment during the 12 month follow-up. All complete responders continue to be surgery-free with a minimum follow-up of 18 months post-treatment.
PRGN 2009 AdenoVerse™ Immunotherapy in HPV-associated Cancers
- PRGN-2009 is an investigational off-the-shelf AdenoVerse immunotherapy designed to activate the immune system to recognize and target HPV-positive solid tumors.
- The Company completed the Phase 1 study and presented positive Phase 1 clinical data from the monotherapy (N=6) and combination therapy (N=11) arms in patients with recurrent or metastatic HPV-associated cancers at the 2023
American Society of Clinical Oncology (ASCO) Annual Meeting. PRGN-2009 was safe and well-tolerated with only Grade 1 or 2 treatment related adverse events and resulted in a 30% objective response rate (ORR) in the combination arm in patients with heavily pre-treated HPV-associated cancers that were naïve or resistant to checkpoint blockade with prolonged duration of responses. - Enrollment was completed in the Phase 2 monotherapy arm with 20 evaluable patients in newly diagnosed oropharyngeal squamous cell carcinoma (OPSCC) patients.
- The Company announced that the FDA has cleared the Investigational New Drug (IND) application to initiate a Phase 2 study of PRGN-2009 in combination with pembrolizumab in patients with recurrent or metastatic cervical cancer. The Phase 2 randomized, open-label, two-arm study will evaluate the efficacy and safety of PRGN-2009 in combination with pembrolizumab versus pembrolizumab monotherapy in patients with recurrent or metastatic cervical cancer who are pembrolizumab resistant.
PRGN-3006 UltraCAR-T® in AML
- PRGN-3006 is an investigational multigenic, autologous chimeric antigen receptor T (CAR-T) cell therapy engineered to express a CAR specifically targeting CD33, membrane bound IL-15 (mbIL15), and a kill switch. The FDA granted Orphan Drug Designation and Fast Track Designation for PRGN-3006 UltraCAR-T for patients with relapsed or refractory acute myeloid leukemia (AML).
- The Company completed the Phase 1 dose escalation study and announced positive data at the 64th
American Society of Hematology (ASH) Annual Meeting and Exposition. PRGN-3006 was well-tolerated with no dose-limiting toxicities. A single infusion of autologous PRGN-3006 cells resulted in a 27% ORR in heavily pre-treated relapsed or refractory AML patients infused following lymphodepletion. A single infusion of UltraCAR-T cells with or without lymphodepletion demonstrated robust expansion and persistence in blood and bone marrow and PRGN-3006 infusion with lymphodepletion resulted in a decrease in bone marrow blasts in 60% of heavily pre-treated patients. - The Phase 1b dose expansion study of PRGN-3006 is ongoing and an interim clinical data presentation is expected in 2024.
PRGN-3005 UltraCAR-T® in Ovarian Cancer
- PRGN-3005 UltraCAR-T is an investigational multigenic, autologous CAR-T cell therapy engineered to express a CAR specifically targeting the unshed portion of MUC16, mbIL15, and a kill switch.
- The Company completed the Phase 1 dose escalation cohorts of the intraperitoneal (IP) and intravenous (IV) arms without lymphodepletion as well as in the lymphodepletion cohort in the IV arm and presented positive Phase 1 clinical data in patients with advanced platinum resistant ovarian cancer at the 2023 ASCO Annual Meeting. PRGN-3005 was well-tolerated with no dose-limiting toxicities, no cytokine release syndrome (CRS) greater than Grade 2, and no neurotoxicity. PRGN-3005 cells demonstrated expansion and persistence when delivered via either IP or IV infusion without lymphodepletion or via IV infusion after lymphodepletion. A single IV infusion following lymphodepletion decreased tumor burden in 67% of the heavily pretreated patients (median of 8 or more prior therapies).
- The Phase 1b dose expansion study of PRGN-3005 is ongoing.
PRGN-3007 UltraCAR-T® in Advanced ROR1+ Hematological and Solid Tumors
- PRGN-3007, based on the next generation of the UltraCAR-T platform, is an investigational multigenic, autologous CAR-T cell therapy engineered to express a CAR targeting receptor tyrosine kinase-like orphan receptor 1 (ROR1), mbIL15, a kill switch, and a novel mechanism for the intrinsic blockade of PD-1 gene expression.
- The Phase 1 dose escalation part of the Phase 1/1b study is ongoing. The target patient population for the Phase 1/1b study includes hematological cancers (chronic lymphocytic leukemia (CLL), mantle cell lymphoma (MCL), acute lymphoblastic leukemia (ALL), and diffuse large B-cell lymphoma (DLBCL)) and solid tumors (triple negative breast cancer (TNBC)).
Financial Highlights
- Completely retired the outstanding balance of convertible notes in the second quarter.
- Cash, cash equivalents, short-term and long-term investments totaled
$95.6 million as ofJune 30, 2023 . - Selling, general, and administrative (SG&A) costs decreased versus the prior year, by 27% and 21% for the three and six months ended
June 30, 2023 , respectively.
Second Quarter 2023 Financial Results Compared to Prior Year Period
Research and development expenses decreased
SG&A expenses decreased
Revenues decreased
Total other income, net, increased
Loss from continuing operations was
First Half 2023 Financial Results Compared to Prior Year Period
Research and development expenses increased
SG&A expenses decreased
Revenues decreased
Total other income, net, increased
Loss from continuing operations was
UltraCAR-T®
UltraCAR-T is a multigenic autologous CAR-T platform that utilizes
UltraCAR-T® Clinical Program
The UltraCAR-T platform has shifted the autologous CAR-T manufacturing paradigm using an advanced non-viral multigene delivery system and an overnight, decentralized manufacturing process for administration of autologous CAR-T cells one day after gene transfer to reduce vein-to-vein time.
UltraCAR-T® Library Approach
UltraPorator®
The UltraPorator system is an exclusive device and proprietary software solution for the scale-up of rapid and cost-effective manufacturing of UltraCAR-T therapies and potentially represents a major advancement over current electroporation devices by significantly reducing the processing time and contamination risk. The UltraPorator device is a high-throughput, semi-closed electroporation system for modifying T cells using
AdenoVerse™ Immunotherapy
AdenoVerse™ Immunotherapy Clinical Program
For patients interested in enrolling in NCI-led clinical studies, please call NCI's toll-free number 1-800-4-Cancer (1-800-422-6237) (TTY: 1-800-332-8615), email NCIMO_Referrals@mail.nih.gov, and/or visit the website: https://trials.cancer.gov.
Trademarks
Cautionary Statement Regarding Forward-Looking Statements
Some of the statements made in this press release are forward-looking statements. These forward-looking statements are based upon the Company's current expectations and projections about future events and generally relate to plans, objectives, and expectations for the development of the Company's business, including the timing and progress of preclinical studies, clinical trials, discovery programs and related milestones, the promise of the Company's portfolio of therapies, and in particular its CAR-T and AdenoVerse therapies. Although management believes that the plans and objectives reflected in or suggested by these forward-looking statements are reasonable, all forward-looking statements involve risks and uncertainties and actual future results may be materially different from the plans, objectives and expectations expressed in this press release. The Company has no obligation to provide any updates to these forward-looking statements even if its expectations change. All forward-looking statements are expressly qualified in their entirety by this cautionary statement. For further information on potential risks and uncertainties, and other important factors, any of which could cause the Company's actual results to differ from those contained in the forward-looking statements, see the section entitled "Risk Factors" in the Company's most recent Annual Report on Form 10-K and subsequent reports filed with the
Investor Contact:
Vice President, Investor Relations
Tel: +1 (301) 556-9850
investors@precigen.com
Media Contacts:
press@precigen.com
glenn.silver@finnpartners.com
|
||
Consolidated Balance Sheets |
||
(Unaudited) |
||
(Amounts in thousands) |
|
|
Assets |
||
Current assets |
||
Cash and cash equivalents |
$ 16,546 |
$ 4,858 |
Restricted cash |
- |
43,339 |
Short-term investments |
71,888 |
51,092 |
Receivables |
||
Trade, net |
1,354 |
978 |
Other |
13,052 |
12,826 |
Prepaid expenses and other |
2,792 |
5,066 |
Total current assets |
105,632 |
118,159 |
Long-term in investments |
7,127 |
- |
Property, plant and equipment, net |
6,574 |
7,329 |
Intangible assets, net |
42,656 |
44,455 |
|
36,966 |
36,923 |
Right-of-use assets |
7,623 |
8,086 |
Other assets |
949 |
1,025 |
Total assets |
$ 207,527 |
$ 215,977 |
Liabilities and Shareholders' Equity |
||
Current liabilities |
||
Accounts payable |
$ 2,510 |
$ 4,068 |
Accrued compensation and benefits |
4,820 |
6,377 |
Other accrued liabilities |
3,257 |
4,997 |
Settlement and Indemnification Accrual |
18,750 |
18,750 |
Deferred revenue |
15 |
25 |
Current portion of long-term debt |
- |
43,219 |
Current portion of lease liabilities |
1,421 |
1,209 |
Total current liabilities |
30,773 |
78,645 |
Deferred revenue, net of current portion |
1,818 |
1,818 |
Lease liabilities, net of current portion |
6,545 |
6,992 |
Deferred tax liabilities |
2,181 |
2,263 |
Total liabilities |
41,317 |
89,718 |
Shareholders' equity |
||
Common stock |
- |
- |
Additional paid-in capital |
2,080,348 |
1,998,314 |
Accumulated deficit |
(1,911,620) |
(1,868,567) |
Accumulated other comprehensive loss |
(2,518) |
(3,488) |
Total shareholders' equity |
166,210 |
126,259 |
Total liabilities and shareholders' equity |
$ 207,527 |
$ 215,977 |
Consolidated Statement of Operations |
||||
(Unaudited) |
||||
Three months ended |
Six months ended |
|||
(Amounts in thousands, except share and per share data) |
|
|
|
|
Revenues |
||||
Product revenues |
$ 324 |
$ 621 |
$ 648 |
$ 1,113 |
Service revenues |
1,438 |
2,213 |
2,965 |
7,146 |
Other revenues |
5 |
77 |
5 |
165 |
Total revenues |
1,767 |
2,911 |
3,618 |
8,424 |
Operating Expenses |
||||
Cost of products and services |
1,697 |
1,811 |
3,224 |
3,505 |
Research and development |
11,874 |
11,954 |
24,037 |
23,755 |
Selling, general and administrative |
9,316 |
12,670 |
20,954 |
26,359 |
Impairment of goodwill |
- |
- |
- |
482 |
Impairment of other noncurrent assets |
- |
638 |
- |
638 |
Total operating expenses |
22,887 |
27,073 |
48,215 |
54,739 |
Operating loss |
(21,120) |
(24,162) |
(44,597) |
(46,315) |
Other Expense, Net |
||||
Interest expense |
(136) |
(2,063) |
(460) |
(4,101) |
Interest income |
828 |
37 |
1,460 |
75 |
Other income, net |
44 |
40 |
424 |
238 |
Total other income (expense), net |
736 |
(1,986) |
1,424 |
(3,788) |
Equity in net loss of affiliates |
- |
- |
- |
(1) |
Loss from continuing operations before income taxes |
(20,384) |
(26,148) |
(43,173) |
(50,104) |
Income tax benefit |
65 |
89 |
120 |
147 |
Loss from continuing operations |
$ (20,319) |
$ (26,059) |
$ (43,053) |
$ (49,957) |
Income from discontinued operations, net of income taxes |
- |
8,424 |
- |
13,071 |
Net loss |
$ (20,319) |
$ (17,635) |
$ (43,053) |
$ (36,886) |
Net Loss per share |
||||
Net loss from continuing operations per share, basic and diluted |
$ (0.08) |
$ (0.13) |
$ (0.18) |
$ (0.25) |
Net income from discontinued operations per share, basic and diluted |
- |
0.04 |
- |
0.07 |
Net loss per share, basic and diluted |
$ (0.08) |
$ (0.09) |
$ (0.18) |
$ (0.18) |
Weighted average shares outstanding, basic and diluted |
248,003,322 |
200,461,441 |
240,307,403 |
200,047,629 |
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