On May 13, 2024 Think Bioscience ("Think"), a Boulder-based synthetic biology company focused on developing small-molecule therapeutics that target "undruggable" proteins, reported a $6M Seed Expansion (Press release, Think Bioscience, MAY 13, 2024, View Source;utm_medium=rss&utm_campaign=think-bioscience-extends-seed-round-to-accelerate-pocket-finding-platform [SID1234643158]). Existing investors are joined by YK Bioventures, bringing total funding to $26M. The additional raise will accelerate the company’s pipeline and expand their unique approach for finding new functional pockets on challenging targets.

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Ultimately, the biochemical properties of any small-molecule drug are constrained by its binding site, a pocket on its protein target. Despite advances in computational chemistry and structural biology, new functional pockets remain challenging to find. Think’s pocket-finding process programs microbes to build small molecules that bind to functional pockets, and they use these pockets to find hits in drug-like space. They have extended their platform technology to a striking variety of targets such as protein tyrosine phosphatases (PTPs), kinases (PTKs), proteases, and GTPases.

"Nature has endowed living systems with an extraordinary ability to build potent bioactive compounds, usually to fulfill important ecological functions, such as defense," said Dr. Jerome Fox, co-founder and CEO of Think Bioscience. "We are using a similar selection process to discover new functional pockets on challenging drug targets. New pockets are gold."

Gary Yeung, a managing partner at YK Bioventures, added, "We invested in Think Bioscience because of its transformational platform for new discoveries. Think Bio has already identified multiple novel pockets and generated strong pre-clinical data for its lead programs. We are eager to support Think Bio’s science-driven leadership team to advance these programs and to fund its growth."

Dr. Wendy Young will join the Board of Directors, which includes fellow veteran drug developer Nick Saccomano. Dr. Young has held leadership positions in biopharma for over 30 years and most recently spent 15 years at Genentech where she served as Senior Vice President of Small Molecule Drug Discovery and co-led research. She currently serves as a SAB member to Think, as well as several other biotech companies, and is an advisor at Google Ventures. "We look forward to working even more closely with Wendy," said Dr. Jerome Fox, co-founder and CEO at Think Bioscience. "Wendy and Nick bring a wealth of drug development expertise. Their guidance will be invaluable as we expand our platform to new classes of important targets and push pipeline programs toward the clinic."

On May 13, 2024 Terns Pharmaceuticals, Inc. ("Terns" or the "Company") (Nasdaq: TERN), a clinical stage biopharmaceutical company developing a portfolio of small molecule product candidates to address serious diseases, including oncology and obesity, reported financial results for the first quarter ended March 31, 2024 and provided corporate updates (Press release, Terns Pharmaceuticals, MAY 13, 2024, View Source [SID1234643157]).

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"We continue to execute our strategy and make important progress on our pipeline of potential best-in-class small molecule therapies as we prepare for key data readouts from our two lead programs," said Amy Burroughs, chief executive officer of Terns, "We look forward to reporting both interim dose escalation data from the ongoing Phase 1 CARDINAL trial of TERN-701 in CML and top-line data from the Phase 1 trial of TERN-601 in obesity in the second half of this year."

"We are particularly pleased with the recent findings from our Phase 1 study of TERN-701 in healthy volunteers, which showed lack of food effect and supports once-daily dosing. This represents a key potential differentiator as the only approved allosteric BCR-ABL inhibitor requires three hours of fasting with each dose and twice-daily dosing in multiple clinical settings," added Ms. Burroughs.

Recent Pipeline Developments and Anticipated Milestones

TERN-701: Oral, allosteric BCR-ABL inhibitor for chronic myeloid leukemia (CML)

•
Terns’ Phase 1 CARDINAL trial of TERN-701 in CML is ongoing and interim data from initial CARDINAL dose escalation cohorts are expected in the second half of 2024
o
CARDINAL is a global, multicenter, open-label, two-part Phase 1 clinical trial to evaluate the safety, pharmacokinetics (PK), and efficacy of TERN-701 in patients with previously treated CML
•
In April, Terns announced findings from a concurrent Phase 1 PK study of TERN-701 in U.S. healthy volunteers, which indicated TERN-701 can be administered once-daily (QD) with or without food at doses that achieve clinically efficacious exposures
•
In March 2024, the United States Food and Drug Administration (FDA) granted Orphan Drug Designation for TERN-701 for the treatment of CML
•
Terns plans to host a TERN-701-focused virtual key opinion leader (KOL) event in mid-2024

TERN-601: Oral, small molecule glucagon-like peptide-1 (GLP-1) receptor agonist for obesity

•
Phase 1 first-in-human clinical trial of Terns’ lead oral GLP-1 receptor agonist in obese and overweight participants is progressing
•
The multiple ascending dose (MAD) portion of the study is underway, testing once-daily administration of TERN-601, and is on track to report top-line 28-day weight loss data in the second half of 2024
•
Preliminary safety findings from the ongoing, blinded Phase 1 SAD/MAD study have been unremarkable to date with no observations of liver enzyme elevations, drug induced liver injury or discontinuations due to treatment-related adverse events

TERN-501: Oral, thyroid hormone receptor-beta (THR-β) agonist

•
Terns continues to evaluate opportunities for TERN-501 in metabolic diseases
o
Based on non-clinical studies, THR-β is an orthogonal mechanism to GLP-1, potentially providing broader metabolic and liver benefits in addition to increased weight loss
o
Non-clinical data suggests that TERN-501 may augment the weight loss effects of a GLP-1 receptor agonist, as demonstrated in a diet-induced obese mouse model

TERN-800 Series: Oral, small molecule glucose-dependent insulinotropic polypeptide receptor (GIPR) modulators

•
Discovery efforts are ongoing for small molecule GIPR modulators for obesity, which have the potential for combination with GLP-1 receptor agonists, such as TERN-601
•
Terns is prioritizing its discovery efforts on nominating a GIPR antagonist development candidate based on in-house discoveries and growing scientific rationale supporting the potential of GLP-1 agonist/GIPR antagonist combinations for obesity

Corporate Updates

•
In April 2024, Terns announced the appointment of Melita Sun Jung as chief business officer of Terns
•
In May 2024, Terns announced the appointment of Scott Harris as chief development officer and the upcoming departure of Erin Quirk, M.D., president, head of research and development

First Quarter 2024 Financial Results

Cash Position: As of March 31, 2024, cash, cash equivalents and marketable securities were $240.7 million, as compared with $263.4 million as of December 31, 2023. Based on its current operating plan, Terns expects these funds will be sufficient to support its planned operating expenses into 2026.

Research and Development (R&D) Expenses: R&D expenses were $18.6 million for the quarter ended March 31, 2024, as compared with $17.1 million for the quarter ended March 31, 2023.

General and Administrative (G&A) Expenses: G&A expenses were $6.9 million for the quarter ended March 31, 2024, as compared with $7.1 million for the quarter ended March 31, 2023.

Net Loss: Net loss was $22.4 million for the quarter ended March 31, 2024, as compared with $21.5 million for the quarter ended March 31, 2023.

Financial Tables

Terns Pharmaceuticals, Inc.

Condensed Consolidated Statements of Operations

(Unaudited; in thousands except share and per share amounts)

Three Months Ended March 31,

2024

2023

Operating expenses:

Research and development

$

18,587

$

17,056

General and administrative

6,859

7,101

Total operating expenses

25,446

24,157

Loss from operations

(25,446

)

(24,157

)

Interest income

3,182

2,693

Other expense, net

(12

)

(4

)

Loss before income taxes

(22,276

)

(21,468

)

Income tax expense

(97

)

(60

)

Net loss

$

(22,373

)

$

(21,528

)

Net loss per share, basic and diluted

$

(0.30

)

$

(0.31

)

Weighted average common stock outstanding, basic and diluted

74,399,378

69,778,420

Terns Pharmaceuticals, Inc.

Selected Balance Sheet Data

(Unaudited; in thousands)

March 31, 2024

December 31, 2023

Cash, cash equivalents and marketable securities

$

240,654

$

263,440

Total assets

246,766

268,517

Total liabilities

10,046

13,150

Total stockholders’ equity

236,720

255,367

On May 13, 2024 Sutro Biopharma, Inc. (Sutro or the Company) (NASDAQ: STRO), a clinical-stage oncology company pioneering site-specific and novel-format antibody drug conjugates (ADCs), reported its financial results for the first quarter of 2024, its recent business highlights, and a preview of select anticipated milestones (Press release, Sutro Biopharma, MAY 13, 2024, View Source [SID1234643156]).

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"The Sutro team executed on multiple fronts in the first quarter of 2024, advancing luvelta through the clinic in multiple indications of high unmet need, continuing to progress our pipeline and collaboration programs, and establishing a new licensing relationship with Ipsen. The upfront funding from the Ipsen deal and our recent financing also augmented our strong cash position," said Bill Newell, Sutro’s Chief Executive Officer. "We plan to deliver on important catalysts throughout 2024, reporting on expanded patient data with luvelta in combination with bevacizumab, the initiation of a registrational trial for pediatric patients with a rare form of acute myeloid leukemia (AML), and a Phase 2 trial in non-small cell lung cancer (NSCLC). We continue to build upon our momentum and are well positioned on our goal to rapidly deliver precisely designed ADCs to patients in need."

Recent Business Highlights and Select Anticipated Milestones

Luveltamab Tazevibulin (luvelta), FolRα-Targeting ADC Franchise:

•
Part 1 (dose-optimization) of the registration-directed trial, REFRαME-O1, for treatment of platinum-resistant ovarian cancer (PROC), has completed enrollment. Part 2 (randomized portion) is now enrolling, with an anticipated ~140 sites in ~20 countries planned to be opened by the end of 2024.
•
Enrollment of REFRαME-P1, a registration-enabling trial for pediatric patients with CBFA2T3::GLIS2 (CBF/GLIS; RAM phenotype) AML, is expected to be initiated in the second half of 2024.
•
An Investigational New Drug (IND) application for the treatment of NSCLC has been cleared by U.S. Food and Drug Administration (FDA). The Phase 2 trial is expected to begin enrolling patients in the second half of 2024. Initial data is expected in the first half of 2025.
•
A Phase 2 expansion study in combination with bevacizumab is well underway. Enrollment is expected to be complete in the first half of 2024.

Additional Pipeline Development and Collaboration Updates:

•
In April 2024, Sutro announced a global licensing agreement for STRO-003, a ROR1-targeting ADC, with Ipsen. Sutro is eligible to receive up to $899 million in upfront and potential milestone payments, including up to $92 million in near-term payments, of which $75 million, including an equity investment, have been received in April. Sutro is also eligible to receive tiered royalties ranging from low double-digit to mid-teen digit percentages on annual global sales of STRO-003.
•
Sutro plans to submit an IND for STRO-004, a tissue factor-targeting ADC, in 2025.
•
Sutro continues to seek to maximize the value of its proprietary cell-free platform by working with partners on programs in multiple disease spaces and geographies and has generated from collaborators an aggregate of approximately $864 million in payments through March 31, 2024, including equity investments.
Corporate Updates:

•
Additionally, Sutro strengthened its cash position with an underwritten offering of 14,478,764 shares of its common stock at a price of $5.18 per share, resulting in gross proceeds of $75.0 million. The offering was led by a high-quality group of new and existing healthcare-focused institutional investors.
Upcoming Events: Sutro will participate in two upcoming investor conferences. Webcasts of the presentations will be accessible through the News & Events page of the Investor Relations section of the Company’s website at www.sutrobio.com. Archived replays will be available for at least 30 days after the events.

•
The Citizens JMP Life Sciences Conference in New York, May 13-14, 2024
•
Jefferies Healthcare Conference in New York, June 5-6, 2024
First Quarter 2024 Financial Highlights

Cash, Cash Equivalents and Marketable Securities

As of March 31, 2024, Sutro had cash, cash equivalents and marketable securities of $267.6 million, and approximately 0.7 million shares of Vaxcyte common stock with a fair value of $45.6 million.

Unrealized Gain from Increase in Value of Vaxcyte Common Stock

The non-operating, unrealized gain of $3.7 million for the quarter ended March 31, 2024 was due to the increase since December 31, 2023 in the estimated fair value of Sutro’s holdings of Vaxcyte common stock. Vaxcyte common stock held by Sutro will be remeasured at fair value based on the closing price of Vaxcyte’s common stock on the last trading day of each reporting period, with any non-operating, unrealized gains and losses recorded in Sutro’s statements of operations.

Revenue

Revenue was $13.0 million for the quarter ended March 31, 2024, as compared to $12.7 million for the same period in 2023, with the 2024 amount related principally to the Astellas collaboration, and the Tasly and Vaxcyte agreements. Future collaboration and license revenue under existing agreements, and from any additional collaboration and license partners, will fluctuate as a result of the amount and timing of revenue recognition of upfront, milestones, and other agreement payments.

Operating Expenses

Total operating expenses for the quarter ended March 31, 2024 were $69.6 million, as compared to $54.9 million for the same period in 2023. The 2024 quarter includes non-cash expenses for stock-based compensation of $6.1 million and depreciation and amortization of $1.8 million, as compared to $6.0 million and $1.6 million, respectively, in the comparable 2023 period. Total operating expenses for the quarter ended March 31, 2024 were comprised of research and development expenses of $56.9 million and general and administrative expenses of $12.7 million.

On May 13, 2024 Cartesian Therapeutics, Inc. (NASDAQ:RNAC) (the "Company), a clinical-stage biotechnology company pioneering mRNA cell therapy for autoimmune diseases, reported that management will participate in a fireside chat at the H.C. Wainwright 2nd Annual BioConnect Investor Conference on Monday, May 20, 2024, at 3:00 p.m. ET (Press release, Cartesian Therapeutics, MAY 13, 2024, View Source [SID1234643155]).

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A live webcast of the fireside chat is expected to be accessible in the Events section of the Company’s website at www.cartesiantherapeutics.com, where an archived replay of the event will be accessible for a limited time.

On May 13, 2024 Quince Therapeutics, Inc. (Nasdaq: QNCX), a late-stage biotechnology company developing an innovative drug delivery technology designed to leverage a patient’s own biology to deliver rare disease therapeutics, reported an update on the company’s development pipeline and announced financial results for the first quarter ended March 31, 2024 (Press release, Quince Therapeutics, MAY 13, 2024, View Source [SID1234643154]).

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Dirk Thye, M.D., Quince’s Chief Executive Officer and Chief Medical Officer, said, "Our primary corporate objective is the advancement of our lead asset, EryDex, for the treatment of patients with ataxia-telangiectasia. We remain on track to begin enrollment of our pivotal Phase 3 study in the second quarter of 2024 and will diligently pursue enrollment at U.S. and European study sites to provide an opportunity for patients living with this rare, devastating disease to participate in research to identify a beneficial therapeutic solution.

"We are pleased to report the selection of Duchenne muscular dystrophy (DMD) as Quince’s second development program for EryDex. We consider DMD a promising indication for EryDex as corticosteroids are the standard of care for this rare disease, but its utility is limited by significant chronic toxicity due to adrenal suppression. We believe EryDex has the potential to provide the therapeutic benefit of corticosteroids without this chronic toxicity. Physicians caring for patients with DMD, along with DMD advocacy groups, also have encouraged the development of EryDex for DMD as a potentially safer alternative to conventional corticosteroids for their patients," concluded Dr. Thye.

Pivotal Phase 3 NEAT Clinical Trial

Secured regulatory approvals in the U.S. and European Union related to the company’s pivotal Phase 3 NEAT (Neurologic Effects of EryDex on Subjects with A-T; IEDAT-04-2022/NCT06193200) clinical trial.
Commenced NEAT study site initiation and activation activities throughout the U.S., U.K., and European Union as NEAT enrollment remains on track to begin in the second quarter of 2024.
NEAT is an international, multi-center, randomized, double-blind, placebo-controlled study to evaluate the neurological effects of the company’s lead asset, EryDex (dexamethasone sodium phosphate [DSP] encapsulated in autologous red blood cells), in patients with A-T.

Plan to enroll approximately 86 patients with A-T ages six to nine years old (primary analysis population) and approximately 20 patients with A-T ages 10 years or older.

Pivotal Phase 3 NEAT clinical trial is being conducted under a Special Protocol Assessment (SPA) agreement with the U.S. Food & Drug Administration (FDA).

Participants will be randomized (1:1) between EryDex or placebo and treatment will consist of six infusions scheduled once every 21 to 30 days. The primary efficacy endpoint will be measured by the change from baseline to last visit completion in rescored modified International Cooperative Ataxia Rating Scale (RmICARS) compared to placebo.
Participants who complete the full treatment period, complete study assessments, and provide informed consent will be eligible to transition to an open label extension study.
Expect to report Phase 3 NEAT topline results in the second half of 2025 with a potential NDA submission in 2026, assuming positive study results.
Pipeline and Corporate Updates

Completed initial patient sizing project with third-party analysis from IQVIA Medical Claims (Dx), IQVIA Analytics confirming approximately 3,400 diagnosed patients with A-T in the U.S., which aligns with an estimated U.S. prevalence of approximately 5,000 patients with A-T in the U.S. There are currently no approved therapeutic treatments for A-T, and the market represents a $1+ billion peak commercial opportunity globally, based on the company’s internal estimates and assumptions.
Advanced evaluation of other potential indications for EryDex with the selection of Duchenne muscular dystrophy (DMD) as the company’s second development program. DMD is an ideal indication for EryDex as corticosteroids are the standard of care for this rare disease, but its utility is limited by significant chronic toxicity due to adrenal suppression. Corticosteroid treatment in patients with DMD is commonly interrupted during adolescence due to interference with sexual maturation and delayed puberty.
Targeting EryDex for the potential treatment of patients with DMD would leverage the company’s AIDE technology designed to encapsulate the corticosteroid DSP in a patient’s own red blood cells, which have several characteristics that make them an ideal vehicle for drug delivery. EryDex is designed to alter the biodistribution, pharmacokinetics, and pharmacodynamics of the DSP, allowing for potentially safe and effective treatment for patients with DMD.
Focused on generating proof-of-concept clinical trial study designs to evaluate EryDex for the potential treatment of patients with DMD, including corticosteroid intolerant populations, in addition to evaluating optimal capital efficient study approaches such as investigator initiated trials and Phase 2/3 options.
Investigating other potential indications for EryDex where chronic corticosteroid treatment is – or has the potential to become – a standard of care, if there were not corticosteroid-related safety concerns. This evaluation process is expected to span across ataxias, neuromuscular indications, hematology, cancer, and autoimmune diseases, with a focus on rare diseases.
Plan to evaluate additional potential applications of Quince’s proprietary AIDE technology platform using drugs and biologics targeted at rare and debilitating diseases to further expand the company’s drug development pipeline.
Evaluate potential strategic partnerships to out-license ex-U.S. rights to extend operational runway to support potential NDA approval of EryDex in the U.S., as well as further advance other potential indications and programs using the AIDE platform.
Participation at The Citizens JMP Life Sciences Conference on Monday, May 13, 2024 beginning at 1:30 p.m. Eastern Time. A live webcast and archive of the presentation will be accessible here.
First Quarter 2024 Financial Results

Reported cash, cash equivalents, and short-term investments of $67.8 million for the first quarter ended March 31, 2024. Quince expects its existing cash runway to be sufficient to fund the company’s capital efficient development plan into 2026.

Expect to fully fund lead asset, EryDex, through Phase 3 NEAT topline results and prepare for a potential NDA submission in 2026, assuming positive study results. This includes approximately $20 million for the NEAT clinical trial and approximately $15 million in direct trial costs for the open label extension study.
Reported research and development (R&D) expenses of $3.7 million for the quarter ended March 31, 2024. R&D expenses for the quarter primarily reflected costs related to the advancement of lead asset EryDex, the startup of related Phase 3 NEAT clinical trial activities, and stock-based compensation expense.
Reported general and administrative (G&A) expenses of $5.0 million for the quarter ended March 31, 2024. G&A expenses for the quarter primarily included personnel-related expenses, insurance, professional and legal fees, and stock-based compensation.

Reported a net loss of $11.1 million, or a loss of $0.26 per basic and diluted share, for the quarter ended March 31, 2024. Weighted average shares outstanding for the quarter were 43.0 million.
Reported net cash used in operating activities of $8.4 million for the quarter ended March 31, 2024, which included adjustments for $3.6 million of non-cash items: a $2.5 million change in the fair value of contingent consideration liabilities due to passage of time, $1.3 million in stock-based compensation, and $0.4 million change in the fair value of long-term debt due to passage of time, offset by a $0.6 million amortization of discount on the company’s investments.