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Athersys Amends Securities Purchase Agreement in Connection with Recent Registered Direct Offering

Athersys Amends
Athersys, Inc. a regenerative medicine company developing MultiStem® for critical care indications, announced that it has entered into amended agreements to extend the exercise period of the warrants that were issued as part of a registered direct offering that closed on August 17, 2022, among other changes, and to issue additional warrants to the same warrant holder, a healthcare-focused U.S. institutional investor.

The Purchase Agreement contains certain restrictions that, subject to certain exceptions, prohibit the Company from issuing its common stock in certain variable rate transactions for a period of 180 days following the Closing Date. The Purchase Agreement also gives the Purchasers the right to participate in certain future offerings of the Company in an amount of up to 30 percent in the aggregate of any offered securities during the 12 months following the Closing Date.

On September 22, 2022, the Company entered into an amendment to the Purchase Agreement with the Purchasers to, among other things, (i) extend the exercise period of the Initial Warrants from five years to seven years after the six-month anniversary of the Closing Date (the “Warrant Amendment”), (ii) reduce the Standstill Period from 180 days to 150 days following the Closing Date, (iii) reduce the term and amount of the Participation Right from twelve months to six months following the Closing Date and from 30 percent to 20 percent in the aggregate of any offered securities, respectively, and (iv) require the Purchasers, subject to certain conditions, to participate in the event the Company proposes to offer and sell shares of offered securities during the six months following the Closing Date to investors primarily for capital raising purposes.

In return, Athersys issued to the same warrant holder additional warrants that are exercisable for an aggregate of 2,000,000 shares of common stock and have an exercise price of $6.3850 for a seven-year period after the six-month anniversary of the date of issuance.

A more detailed discussion of the amendments is contained in a Current Report that Athersys filed on September 22, 2022 with the U.S. Securities and Exchange Commission on Form 8-K.

“We are pleased to have entered into this amendment with our new major investor which provides more financial flexibility going forward as well as a commitment for the investor that recently invested $12 million in Athersys to participate in future financings if requested. As Athersys continues to pursue licensing and other business development opportunities for our proprietary technology this amendment can help facilitate future transactions with financial and strategic investors. We are fortunate to have established a strong relationship with a prominent healthcare focused institutional investor that embraces the value of our MultiStem cell therapy and is responsive to our priorities,”

Dan Camardo, Chief Executive Officer of Athersys

“I am also pleased with progress we’ve made in our business transformation and I look forward to updating shareholders during a conference call to be held the week of October 3rd,” he added.

Athersys intends to issue a separate news release that will provide further information on the date and time of the call, as well as instructions regarding how to participate.

About MultiStem®
MultiStem® cell therapy is a patented regenerative medicine product in clinical development that has shown the ability to promote tissue repair and healing in a variety of ways, such as through the production of therapeutic factors in response to signals of inflammation and tissue damage. MultiStem therapy’s potential for multidimensional therapeutic impact distinguishes it from traditional biopharmaceutical therapies focused on a single mechanism of benefit. The therapy represents a unique “off-the-shelf” stem cell product that can be manufactured in a scalable manner, may be stored for years in frozen form, and is administered without tissue matching or the need for immune suppression. Based upon its efficacy profile, its novel mechanisms of action, and a favorable and consistent tolerability demonstrated in clinical studies, we believe that MultiStem therapy could provide a meaningful benefit to patients, including those suffering from serious diseases and conditions with unmet medical need.

About Athersys
Athersys is a biotechnology company engaged in the discovery and development of therapeutic product candidates designed to extend and enhance the quality of human life. The Company is developing its MultiStem® cell therapy product, a patented, adult-derived “off-the-shelf” stem cell product, initially for disease indications in the neurological, inflammatory and immune, and other critical care indications and has several ongoing clinical trials evaluating this potential regenerative medicine product. Athersys has forged strategic partnerships and a broad network of collaborations to further advance MultiStem cell therapy toward commercialization.

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Personalis and ClearNote Health Announce Partnership to Advance Epigenomic Technology

Personalis, Inc. | February 05, 2024

Personalis, Inc. a leader in advanced genomics for cancer, and ClearNote Health, Inc., a pioneer in epigenomic technologies, today unveiled an alliance through which Personalis will expand its pharmaceutical service offerings by distributing ClearNote’s cutting-edge epigenomic 5-hydroxymethylcytosine (5hmC) platform. “We have built a unique pharma channel based on our industry-leading tissue and MRD assays and ClearNote Health’s blood-based epigenomic approach is complementary to our offering as it helps our customers deepen their understanding of a patient’s response to immunotherapies,” said Chris Hall, CEO and President of Personalis. “Commercial partnerships such as this broaden our portfolio and are expected to appeal to a wide range of biopharma customers and accelerate our revenue growth.” Echoing this sentiment, Dave Mullarkey, CEO of ClearNote Health, remarked, “Partnering with Personalis presents an excellent opportunity to bring our 5hmC technology to the forefront of cancer research. This alliance is a testament to the synergy between our two companies, enabling us to expand our reach and significantly impact the biopharmaceutical industry. Together, we can accelerate the development of personalized therapies and make a real difference in the lives of patients.” ClearNote Health’s Epigenomics Platform represents a groundbreaking advance in cancer detection, offering real-time insights into disease-specific pathways. By tracking changes in 5hmC levels coupled with artificial intelligence-based analytical methods, the platform can detect cancer earlier, monitor disease progression, understand mechanisms of resistance, and identify promising drug targets and biomarkers. These insights are invaluable for optimizing drug development programs and delivering more effective treatments to patients. The Epigenomics Platform identifies changes in gene activation and gene regulation by labeling specific changes in the 5hmC landscape from plasma-derived cell-free DNA. This rich biological information, as part of clinical trials, enables the monitoring of cancer therapies in real time and contributes to an understanding of drug resistance mechanisms. The partnership marks a pivotal moment in cancer research, leveraging the strengths of both companies to offer unparalleled solutions in the biopharmaceutical industry. About ClearNote Heath, Inc. ClearNote Health is a cancer detection company focused on enabling people at risk for high-mortality cancers to live longer, healthier lives. Utilizing a standard blood draw, the company applies its proprietary epigenomic platform, combining biology and artificial intelligence, to identify DNA-based changes in biology as cancer develops. With lead programs in non-invasive early detection of pancreatic and ovarian cancers in patients at the highest risk for these diseases, ClearNote Health identifies cancers before they progress and when patients are most likely to benefit from treatment. ClearNote Health’s first commercially available test is the Avantect™ Pancreatic Cancer Test, which detects the presence of pancreatic cancer signals in patients at high risk of the disease, including those recently diagnosed with Type 2 diabetes. ClearNote Health is headquartered in San Diego, with additional presence in the San Francisco Bay area and internationally. The company’s CLIA- and CAP-accredited laboratory is located in San Diego, Calif. About Personalis, Inc. At Personalis, we are transforming the active management of cancer through breakthrough personalized testing. We aim to drive a new paradigm for cancer management, guiding care from biopsy through the life of the patient. Our highly sensitive assays combine tumor-and-normal profiling with proprietary algorithms to deliver advanced insights even as cancer evolves over time. Our products are designed to detect minimal residual disease (MRD) and recurrence at the earliest time points, enable the selection of targeted therapies based on ultra-comprehensive genomic profiling, and enhance biomarker strategy for drug development. Personalis is based in Fremont, California.

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Jnana Therapeutics Announces Positive Clinical Proof of Concept Achieved with JNT-517, a Potential First-in-Class Oral Treatment for PKU

Jnana Therapeutics, Inc. | February 02, 2024

Jnana Therapeutics, a clinical-stage biotechnology company leveraging its next-generation chemoproteomics platform to discover medicines for challenging-to-drug targets, today announced positive, statistically significant interim results from its ongoing clinical study of JNT-517 in individuals with phenylketonuria (PKU). JNT-517, a small molecule inhibitor of the phenylalanine (Phe) transporter SLC6A19, is being evaluated as a potential first-in-class oral treatment for PKU across all ages and genotypes. On the basis of these positive interim results, Jnana has adapted the Phase 1b trial design to support the potential for accelerated progression of JNT-517. “There is an urgent need for an oral, safe, and efficacious therapy for the more than 60% of individuals with PKU not currently on therapy. Across the spectrum of mild to severe disease, our results demonstrate a robust, sustained reduction in blood Phe levels, the registrational endpoint for PKU, giving us high confidence in the path forward for JNT-517,” said George Vratsanos, M.D., Chief Medical Officer and Head of R&D at Jnana Therapeutics. “We are also encouraged by this validation of the power of our RAPID platform to discover small molecules with compelling clinical benefit against challenging-to-drug targets.” JNT-517 is being studied in a randomized, double-blind, placebo-controlled trial in individuals with mild to severe PKU. Following a 28-day screening period focused predominantly on ensuring an average blood Phe level of >600µM, study participants were randomized with no run-in period to 75mg of JNT-517 twice daily (BID) or placebo. The planned interim analysis was based on 13 participants, eight dosed with JNT-517 and five dosed with placebo over 28 days, and demonstrated the following results JNT-517 led to a statistically significant (p=0.0019 vs. placebo) mean blood Phe reduction from baseline of 51%, measured per-protocol at day 28. A high response rate was seen where seven of eight (88%) treated participants achieved >30% reduction in blood Phe from baseline; five of eight (63%) achieved >45% reduction; and two of eight (25%) achieved >65% reduction. A robust response was seen across participants treated with JNT-517 irrespective of baseline blood Phe levels, which ranged from 593µM to 1,526µM with a mean of 1,124µM. A rapid onset of effect was observed with significant blood Phe reduction achieved within seven days after commencing dosing, which was sustained through the full 28 days of dosing. JNT-517 was safe and well tolerated with no serious adverse events and no clinically significant changes in laboratory parameters, consistent with the safety profile demonstrated in the Phase 1a healthy volunteer study. "JNT-517 represents a completely new therapeutic approach that could transform the current treatment paradigm in PKU, in particular for individuals with severe, or classical, PKU where there is the highest unmet medical need,” said Cary O. Harding, M.D., study investigator and Professor of Molecular and Medical Genetics at Oregon Health and Science University School of Medicine. “I am encouraged by the clinical results to date and look forward to working with Jnana and the PKU community to continue to advance this program.” Based on these interim results, Jnana has adapted the protocol of the ongoing trial to include dose exploration. Jnana expects topline data from the second dose cohort in mid-2024 and plans to submit full data from the two dose cohorts for presentation at a scientific meeting in the second half of 2024. Jnana anticipates the company will engage regulators in the second half of 2024 and seek to advance JNT-517 directly into a pivotal Phase 3 study in the first half of 2025. JNT-517 Phase 1b Clinical Trial The ongoing clinical program includes a randomized, double-blind, placebo-controlled trial evaluating the safety, tolerability, pharmacokinetics, and effect on blood and urinary Phe of JNT-517 dosed over a four-week period in individuals diagnosed with PKU. The study dosed its first participant with PKU in August 2023 and is enrolling individuals aged 18 to 65 at clinical sites in the United States and Australia. For more information about the study, please see clinicaltrials.gov (NCT05781399). About JNT-517 JNT-517 is a selective small molecule inhibitor of the Phe transporter SLC6A19 that has the potential to be a first-in-class oral therapy used to treat any person with PKU, regardless of age or genotype. JNT-517 acts at a novel, cryptic allosteric site to block kidney reabsorption of Phe and offers a promising new approach to reduce blood Phe levels. The U.S. Food and Drug Administration granted JNT-517 Rare Pediatric Disease Designation in late 2022. About PKU PKU is a rare inherited metabolic disorder caused by a deficiency of the enzyme phenylalanine hydroxylase (PAH). This enzyme is required for the breakdown of phenylalanine (Phe), an amino acid found in all protein-containing foods. When PAH is deficient or defective, Phe accumulates to abnormally high levels in the blood. If left untreated, toxic levels of Phe in the blood can result in progressive and severe neurological impairment and neuropsychological complications. The SLC transporter SLC6A19 is responsible for kidney reabsorption of Phe back into the bloodstream, and the inhibition of SLC6A19 offers a novel, oral approach for the treatment of PKU by facilitating urinary excretion of excess Phe. About Jnana Therapeutics Jnana Therapeutics is a clinical-stage biotechnology company leveraging its next-generation RAPID chemoproteomics platform to discover medicines for highly validated, challenging-to-drug targets to treat diseases with high unmet needs. Jnana is focused on developing first- and best-in-class therapies to treat a wide range of diseases, including rare diseases and immune-mediated diseases. Jnana’s wholly owned lead program, JNT-517, which targets an allosteric site on the phenylalanine transporter SLC6A19, is a potential first-in-class oral approach for the treatment of PKU, a rare genetic metabolic disease. Located in Boston, Jnana brings together scientific leaders in small molecule drug discovery and development, a highly experienced management team, and the backing of leading life science investors Bain Capital Life Sciences, RA Capital Management, Polaris Partners, Versant Ventures, Avalon Ventures, Pfizer Ventures, and AbbVie Ventures.

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Heron Therapeutics Announces FDA Approval of ZYNRELEF® Indication Expansion to Include Additional Orthopedic and Soft Tissue Procedures

Heron Therapeutics, Inc. | January 29, 2024

Heron Therapeutics, Inc. a commercial-stage biotechnology company announced that the U.S. Food and Drug Administration (the "FDA") has approved its supplemental New Drug Application ("NDA") for ZYNRELEF® extended-release solution to expand the indication for soft tissue and orthopedic surgical procedures including foot and ankle, and other procedures in which direct exposure to articular cartilage is avoided. ZYNRELEF was previously approved for foot and ankle, small-to-medium open abdominal, and lower extremity total joint arthroplasty surgical procedures in adults. This expanded indication for ZYNRELEF will now cover an estimated 13 million procedures annually, an estimated increase of 86% over prior indicated procedures. To obtain this labeling expansion, Heron successfully conducted studies for cesarean section, spinal surgery, augmentation mammoplasty, and total shoulder arthroplasty. No unique safety issues were identified from the new clinical trials, and the bupivacaine and meloxicam blood concentrations were consistent with previous experience following ZYNRELEF administration. "The expanded indication is poised to have a transformative impact on patient care, providing healthcare professionals with a versatile and effective solution for managing postoperative pain across an even wider range of surgical procedures. The new label expansion and recent partnership with CrossLink, combined with the potential approval of the Vial Access Needle ("VAN") later this year, are expected to have a significant positive impact for ZYNRELEF and the Company," said Craig Collard, Chief Executive Officer of Heron. "We're excited for the opportunity to give even more healthcare providers and patients a new, safe and effective option for achieving long-lasting non-opioid pain control after painful surgical procedures," said Bill Forbes, Executive Vice President, Chief Development Officer at Heron. "This new approval further reinforces our commitment to providing meaningful solutions to address unmet medical needs in the acute care and oncology settings." ZYNRELEF is the first and only therapy for postoperative pain management to be rigorously tested in Phase 3 studies and demonstrate superiority to bupivacaine solution, the current standard-of-care. ZYNRELEF demonstrated superiority compared to bupivacaine with lower pain scores, fewer patients experiencing severe pain, and lower opioid consumption. ZYNRELEF was initially approved by the FDA in May 2021 and received approval of the first supplemental NDA for an expanded label in December 2021. "Patients undergoing orthopedic procedures often experience severe pain, slowing down their recovery time and potentially leading to other complications. Reducing patients' pain within the first three days is critical for patient satisfaction, and having a product like ZYNRELEF now available for additional orthopedic procedures is a great benefit to have in my practice," said Alexander Sah, M.D., orthopedic surgeon at Sah Orthopaedic Associates. "ZYNRELEF helps my patients recover fully, be discharged sooner, and have significantly less pain, with little to no opioid use." Important Safety Information for Patients ZYNRELEF contains an NSAID (non-steroidal anti-inflammatory drug), a type of medicine which Can increase the risk of a heart attack or stroke that can lead to death. This risk increases with higher doses and longer use of an NSAID. Cannot be used during heart bypass surgery. Can increase the risk of gastrointestinal bleeding, ulcers, and tears. ZYNRELEF should also not be used if you are allergic to any component of ZYNRELEF, similar local anesthetics, aspirin or other NSAIDs (such as ibuprofen or naproxen), or have had an asthma attack, hives, or other allergic reaction after taking any of these medicines. as a paracervical block, during childbirth. The most common side effects of ZYNRELEF are soft tissue procedures: vomiting and orthopedic procedures: constipation and headache. The medicines in ZYNRELEF (a local anesthetic and an NSAID) may affect the nervous and cardiovascular system; may cause liver or kidney problems; may reduce the effects of some blood pressure medicines; should be avoided if you have severe heart failure; may cause adverse effects on cartilage; may cause a rare blood disorder, or life-threatening skin or allergic reactions; may harm your unborn baby if received at 20 weeks of pregnancy or later; and may cause low red blood cells (anemia). Tell your healthcare provider about all your medical conditions and about all the medicines you take including prescription or over-the-counter medicines, vitamins, or herbal supplements to discuss if ZYNRELEF is right for you. Talk to your healthcare provider for medical advice about side effects. Report side effects to Heron at 1-844-437-6611 The information provided here is not comprehensive. Please see full Prescribing Information, including Boxed Warning. About ZYNRELEF ZYNRELEF is the first and only dual-acting local anesthetic that delivers a fixed-dose combination of the local anesthetic bupivacaine and a low dose of nonsteroidal anti-inflammatory drug meloxicam. ZYNRELEF is the first and only extended-release local anesthetic to demonstrate in Phase 3 studies significantly reduced pain and significantly increased proportion of patients requiring no opioids through the first 72 hours following surgery compared to bupivacaine solution, the current standard-of-care local anesthetic for postoperative pain control. ZYNRELEF was initially approved by the U.S. Food and Drug Administration (the "FDA") in May 2021 for use in adults for soft tissue or periarticular instillation to produce postsurgical analgesia for up to 72 hours after bunionectomy, open inguinal herniorrhaphy and total knee arthroplasty. In December 2021, the FDA approved an expansion of ZYNRELEF's indication to include foot and ankle, small-to-medium open abdominal, and lower extremity total joint arthroplasty surgical procedures. On January 23, 2024, the FDA approved ZYNRELEF for soft tissue and orthopedic surgical procedures including foot and ankle, and other procedures in which direct exposure to articular cartilage is avoided. Safety and efficacy have not been established in highly vascular surgeries, such as intrathoracic, large multilevel spinal, and head and neck procedures. ZYNRELEF was granted a marketing authorization by the European Commission in September 2020 and by the United Kingdom Regulatory Authority in January 2021. In August 2023, we cancelled the ZYNRELEF U.K. marketing authorization and, in October 2023, we cancelled the ZYNRELEF European Union (EU) marketing authorization, as we do not plan to commercially launch ZYNRELEF in the U.K. or the EU. About Heron Therapeutics, Inc. Heron Therapeutics, Inc. is a commercial-stage biotechnology company focused on improving the lives of patients by developing and commercializing therapeutic innovations that improve medical care. Our advanced science, patented technologies, and innovative approach to drug discovery and development have allowed us to create and commercialize a portfolio of products that aim to advance the standard-of-care for acute care and oncology patients.

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Personalis and ClearNote Health Announce Partnership to Advance Epigenomic Technology

Personalis, Inc. | February 05, 2024

Personalis, Inc. a leader in advanced genomics for cancer, and ClearNote Health, Inc., a pioneer in epigenomic technologies, today unveiled an alliance through which Personalis will expand its pharmaceutical service offerings by distributing ClearNote’s cutting-edge epigenomic 5-hydroxymethylcytosine (5hmC) platform. “We have built a unique pharma channel based on our industry-leading tissue and MRD assays and ClearNote Health’s blood-based epigenomic approach is complementary to our offering as it helps our customers deepen their understanding of a patient’s response to immunotherapies,” said Chris Hall, CEO and President of Personalis. “Commercial partnerships such as this broaden our portfolio and are expected to appeal to a wide range of biopharma customers and accelerate our revenue growth.” Echoing this sentiment, Dave Mullarkey, CEO of ClearNote Health, remarked, “Partnering with Personalis presents an excellent opportunity to bring our 5hmC technology to the forefront of cancer research. This alliance is a testament to the synergy between our two companies, enabling us to expand our reach and significantly impact the biopharmaceutical industry. Together, we can accelerate the development of personalized therapies and make a real difference in the lives of patients.” ClearNote Health’s Epigenomics Platform represents a groundbreaking advance in cancer detection, offering real-time insights into disease-specific pathways. By tracking changes in 5hmC levels coupled with artificial intelligence-based analytical methods, the platform can detect cancer earlier, monitor disease progression, understand mechanisms of resistance, and identify promising drug targets and biomarkers. These insights are invaluable for optimizing drug development programs and delivering more effective treatments to patients. The Epigenomics Platform identifies changes in gene activation and gene regulation by labeling specific changes in the 5hmC landscape from plasma-derived cell-free DNA. This rich biological information, as part of clinical trials, enables the monitoring of cancer therapies in real time and contributes to an understanding of drug resistance mechanisms. The partnership marks a pivotal moment in cancer research, leveraging the strengths of both companies to offer unparalleled solutions in the biopharmaceutical industry. About ClearNote Heath, Inc. ClearNote Health is a cancer detection company focused on enabling people at risk for high-mortality cancers to live longer, healthier lives. Utilizing a standard blood draw, the company applies its proprietary epigenomic platform, combining biology and artificial intelligence, to identify DNA-based changes in biology as cancer develops. With lead programs in non-invasive early detection of pancreatic and ovarian cancers in patients at the highest risk for these diseases, ClearNote Health identifies cancers before they progress and when patients are most likely to benefit from treatment. ClearNote Health’s first commercially available test is the Avantect™ Pancreatic Cancer Test, which detects the presence of pancreatic cancer signals in patients at high risk of the disease, including those recently diagnosed with Type 2 diabetes. ClearNote Health is headquartered in San Diego, with additional presence in the San Francisco Bay area and internationally. The company’s CLIA- and CAP-accredited laboratory is located in San Diego, Calif. About Personalis, Inc. At Personalis, we are transforming the active management of cancer through breakthrough personalized testing. We aim to drive a new paradigm for cancer management, guiding care from biopsy through the life of the patient. Our highly sensitive assays combine tumor-and-normal profiling with proprietary algorithms to deliver advanced insights even as cancer evolves over time. Our products are designed to detect minimal residual disease (MRD) and recurrence at the earliest time points, enable the selection of targeted therapies based on ultra-comprehensive genomic profiling, and enhance biomarker strategy for drug development. Personalis is based in Fremont, California.

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Jnana Therapeutics Announces Positive Clinical Proof of Concept Achieved with JNT-517, a Potential First-in-Class Oral Treatment for PKU

Jnana Therapeutics, Inc. | February 02, 2024

Jnana Therapeutics, a clinical-stage biotechnology company leveraging its next-generation chemoproteomics platform to discover medicines for challenging-to-drug targets, today announced positive, statistically significant interim results from its ongoing clinical study of JNT-517 in individuals with phenylketonuria (PKU). JNT-517, a small molecule inhibitor of the phenylalanine (Phe) transporter SLC6A19, is being evaluated as a potential first-in-class oral treatment for PKU across all ages and genotypes. On the basis of these positive interim results, Jnana has adapted the Phase 1b trial design to support the potential for accelerated progression of JNT-517. “There is an urgent need for an oral, safe, and efficacious therapy for the more than 60% of individuals with PKU not currently on therapy. Across the spectrum of mild to severe disease, our results demonstrate a robust, sustained reduction in blood Phe levels, the registrational endpoint for PKU, giving us high confidence in the path forward for JNT-517,” said George Vratsanos, M.D., Chief Medical Officer and Head of R&D at Jnana Therapeutics. “We are also encouraged by this validation of the power of our RAPID platform to discover small molecules with compelling clinical benefit against challenging-to-drug targets.” JNT-517 is being studied in a randomized, double-blind, placebo-controlled trial in individuals with mild to severe PKU. Following a 28-day screening period focused predominantly on ensuring an average blood Phe level of >600µM, study participants were randomized with no run-in period to 75mg of JNT-517 twice daily (BID) or placebo. The planned interim analysis was based on 13 participants, eight dosed with JNT-517 and five dosed with placebo over 28 days, and demonstrated the following results JNT-517 led to a statistically significant (p=0.0019 vs. placebo) mean blood Phe reduction from baseline of 51%, measured per-protocol at day 28. A high response rate was seen where seven of eight (88%) treated participants achieved >30% reduction in blood Phe from baseline; five of eight (63%) achieved >45% reduction; and two of eight (25%) achieved >65% reduction. A robust response was seen across participants treated with JNT-517 irrespective of baseline blood Phe levels, which ranged from 593µM to 1,526µM with a mean of 1,124µM. A rapid onset of effect was observed with significant blood Phe reduction achieved within seven days after commencing dosing, which was sustained through the full 28 days of dosing. JNT-517 was safe and well tolerated with no serious adverse events and no clinically significant changes in laboratory parameters, consistent with the safety profile demonstrated in the Phase 1a healthy volunteer study. "JNT-517 represents a completely new therapeutic approach that could transform the current treatment paradigm in PKU, in particular for individuals with severe, or classical, PKU where there is the highest unmet medical need,” said Cary O. Harding, M.D., study investigator and Professor of Molecular and Medical Genetics at Oregon Health and Science University School of Medicine. “I am encouraged by the clinical results to date and look forward to working with Jnana and the PKU community to continue to advance this program.” Based on these interim results, Jnana has adapted the protocol of the ongoing trial to include dose exploration. Jnana expects topline data from the second dose cohort in mid-2024 and plans to submit full data from the two dose cohorts for presentation at a scientific meeting in the second half of 2024. Jnana anticipates the company will engage regulators in the second half of 2024 and seek to advance JNT-517 directly into a pivotal Phase 3 study in the first half of 2025. JNT-517 Phase 1b Clinical Trial The ongoing clinical program includes a randomized, double-blind, placebo-controlled trial evaluating the safety, tolerability, pharmacokinetics, and effect on blood and urinary Phe of JNT-517 dosed over a four-week period in individuals diagnosed with PKU. The study dosed its first participant with PKU in August 2023 and is enrolling individuals aged 18 to 65 at clinical sites in the United States and Australia. For more information about the study, please see clinicaltrials.gov (NCT05781399). About JNT-517 JNT-517 is a selective small molecule inhibitor of the Phe transporter SLC6A19 that has the potential to be a first-in-class oral therapy used to treat any person with PKU, regardless of age or genotype. JNT-517 acts at a novel, cryptic allosteric site to block kidney reabsorption of Phe and offers a promising new approach to reduce blood Phe levels. The U.S. Food and Drug Administration granted JNT-517 Rare Pediatric Disease Designation in late 2022. About PKU PKU is a rare inherited metabolic disorder caused by a deficiency of the enzyme phenylalanine hydroxylase (PAH). This enzyme is required for the breakdown of phenylalanine (Phe), an amino acid found in all protein-containing foods. When PAH is deficient or defective, Phe accumulates to abnormally high levels in the blood. If left untreated, toxic levels of Phe in the blood can result in progressive and severe neurological impairment and neuropsychological complications. The SLC transporter SLC6A19 is responsible for kidney reabsorption of Phe back into the bloodstream, and the inhibition of SLC6A19 offers a novel, oral approach for the treatment of PKU by facilitating urinary excretion of excess Phe. About Jnana Therapeutics Jnana Therapeutics is a clinical-stage biotechnology company leveraging its next-generation RAPID chemoproteomics platform to discover medicines for highly validated, challenging-to-drug targets to treat diseases with high unmet needs. Jnana is focused on developing first- and best-in-class therapies to treat a wide range of diseases, including rare diseases and immune-mediated diseases. Jnana’s wholly owned lead program, JNT-517, which targets an allosteric site on the phenylalanine transporter SLC6A19, is a potential first-in-class oral approach for the treatment of PKU, a rare genetic metabolic disease. Located in Boston, Jnana brings together scientific leaders in small molecule drug discovery and development, a highly experienced management team, and the backing of leading life science investors Bain Capital Life Sciences, RA Capital Management, Polaris Partners, Versant Ventures, Avalon Ventures, Pfizer Ventures, and AbbVie Ventures.

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Heron Therapeutics Announces FDA Approval of ZYNRELEF® Indication Expansion to Include Additional Orthopedic and Soft Tissue Procedures

Heron Therapeutics, Inc. | January 29, 2024

Heron Therapeutics, Inc. a commercial-stage biotechnology company announced that the U.S. Food and Drug Administration (the "FDA") has approved its supplemental New Drug Application ("NDA") for ZYNRELEF® extended-release solution to expand the indication for soft tissue and orthopedic surgical procedures including foot and ankle, and other procedures in which direct exposure to articular cartilage is avoided. ZYNRELEF was previously approved for foot and ankle, small-to-medium open abdominal, and lower extremity total joint arthroplasty surgical procedures in adults. This expanded indication for ZYNRELEF will now cover an estimated 13 million procedures annually, an estimated increase of 86% over prior indicated procedures. To obtain this labeling expansion, Heron successfully conducted studies for cesarean section, spinal surgery, augmentation mammoplasty, and total shoulder arthroplasty. No unique safety issues were identified from the new clinical trials, and the bupivacaine and meloxicam blood concentrations were consistent with previous experience following ZYNRELEF administration. "The expanded indication is poised to have a transformative impact on patient care, providing healthcare professionals with a versatile and effective solution for managing postoperative pain across an even wider range of surgical procedures. The new label expansion and recent partnership with CrossLink, combined with the potential approval of the Vial Access Needle ("VAN") later this year, are expected to have a significant positive impact for ZYNRELEF and the Company," said Craig Collard, Chief Executive Officer of Heron. "We're excited for the opportunity to give even more healthcare providers and patients a new, safe and effective option for achieving long-lasting non-opioid pain control after painful surgical procedures," said Bill Forbes, Executive Vice President, Chief Development Officer at Heron. "This new approval further reinforces our commitment to providing meaningful solutions to address unmet medical needs in the acute care and oncology settings." ZYNRELEF is the first and only therapy for postoperative pain management to be rigorously tested in Phase 3 studies and demonstrate superiority to bupivacaine solution, the current standard-of-care. ZYNRELEF demonstrated superiority compared to bupivacaine with lower pain scores, fewer patients experiencing severe pain, and lower opioid consumption. ZYNRELEF was initially approved by the FDA in May 2021 and received approval of the first supplemental NDA for an expanded label in December 2021. "Patients undergoing orthopedic procedures often experience severe pain, slowing down their recovery time and potentially leading to other complications. Reducing patients' pain within the first three days is critical for patient satisfaction, and having a product like ZYNRELEF now available for additional orthopedic procedures is a great benefit to have in my practice," said Alexander Sah, M.D., orthopedic surgeon at Sah Orthopaedic Associates. "ZYNRELEF helps my patients recover fully, be discharged sooner, and have significantly less pain, with little to no opioid use." Important Safety Information for Patients ZYNRELEF contains an NSAID (non-steroidal anti-inflammatory drug), a type of medicine which Can increase the risk of a heart attack or stroke that can lead to death. This risk increases with higher doses and longer use of an NSAID. Cannot be used during heart bypass surgery. Can increase the risk of gastrointestinal bleeding, ulcers, and tears. ZYNRELEF should also not be used if you are allergic to any component of ZYNRELEF, similar local anesthetics, aspirin or other NSAIDs (such as ibuprofen or naproxen), or have had an asthma attack, hives, or other allergic reaction after taking any of these medicines. as a paracervical block, during childbirth. The most common side effects of ZYNRELEF are soft tissue procedures: vomiting and orthopedic procedures: constipation and headache. The medicines in ZYNRELEF (a local anesthetic and an NSAID) may affect the nervous and cardiovascular system; may cause liver or kidney problems; may reduce the effects of some blood pressure medicines; should be avoided if you have severe heart failure; may cause adverse effects on cartilage; may cause a rare blood disorder, or life-threatening skin or allergic reactions; may harm your unborn baby if received at 20 weeks of pregnancy or later; and may cause low red blood cells (anemia). Tell your healthcare provider about all your medical conditions and about all the medicines you take including prescription or over-the-counter medicines, vitamins, or herbal supplements to discuss if ZYNRELEF is right for you. Talk to your healthcare provider for medical advice about side effects. Report side effects to Heron at 1-844-437-6611 The information provided here is not comprehensive. Please see full Prescribing Information, including Boxed Warning. About ZYNRELEF ZYNRELEF is the first and only dual-acting local anesthetic that delivers a fixed-dose combination of the local anesthetic bupivacaine and a low dose of nonsteroidal anti-inflammatory drug meloxicam. ZYNRELEF is the first and only extended-release local anesthetic to demonstrate in Phase 3 studies significantly reduced pain and significantly increased proportion of patients requiring no opioids through the first 72 hours following surgery compared to bupivacaine solution, the current standard-of-care local anesthetic for postoperative pain control. ZYNRELEF was initially approved by the U.S. Food and Drug Administration (the "FDA") in May 2021 for use in adults for soft tissue or periarticular instillation to produce postsurgical analgesia for up to 72 hours after bunionectomy, open inguinal herniorrhaphy and total knee arthroplasty. In December 2021, the FDA approved an expansion of ZYNRELEF's indication to include foot and ankle, small-to-medium open abdominal, and lower extremity total joint arthroplasty surgical procedures. On January 23, 2024, the FDA approved ZYNRELEF for soft tissue and orthopedic surgical procedures including foot and ankle, and other procedures in which direct exposure to articular cartilage is avoided. Safety and efficacy have not been established in highly vascular surgeries, such as intrathoracic, large multilevel spinal, and head and neck procedures. ZYNRELEF was granted a marketing authorization by the European Commission in September 2020 and by the United Kingdom Regulatory Authority in January 2021. In August 2023, we cancelled the ZYNRELEF U.K. marketing authorization and, in October 2023, we cancelled the ZYNRELEF European Union (EU) marketing authorization, as we do not plan to commercially launch ZYNRELEF in the U.K. or the EU. About Heron Therapeutics, Inc. Heron Therapeutics, Inc. is a commercial-stage biotechnology company focused on improving the lives of patients by developing and commercializing therapeutic innovations that improve medical care. Our advanced science, patented technologies, and innovative approach to drug discovery and development have allowed us to create and commercialize a portfolio of products that aim to advance the standard-of-care for acute care and oncology patients.

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