After growing rapidly in recent years, China’s pharma market will begin to experience some slowdown, but market watchers said it is not necessarily all bad news. According to data from Frost & Sullivan, China’s pharma market is expected to reach ¥2.13 trillion (US$300 billion) by 2023, up 6.8% from ¥1.53 trillion in 2018. In comparison, the market grew 8.1% between 2014 and 2018. The market intelligence provider predicts that the Chinese market for small-molecule drugs will grow 18.4% to reach ¥27.4 billion, while for monoclonal antibodies, it will soar 57.8% to ¥156.5 billion by 2023.

Biopharma stocks continue their upward swing in May

Blue chip public biopharmaceutical companies continued their positive trajectory in May, with the BioWorld Biopharmaceutical index recording an 8% jump in valuation and contributing to its year-to-date performance of approximately 17%. Expectations that the sector will provide a therapeutic solution, or solutions, to the COVID-19 pandemic has certainly been the catalyst for growth during the past two months.

ASCO impresses investors

While the halls remained silent at McCormick Place in Chicago – the traditional meeting spot for thousands of delegates attending the American Society of Clinical Oncology (ASCO) meeting – the online version still managed to attract about 40,000 registrants, like years past. The abbreviated meeting concentrated on scientific presentations, with 250 oral presentations along with 2,500 posters on tap. Overall, data presented at the meeting appeared to have garnered investor approval, with the BioWorld Cancer index advancing almost 5% last week.

Product approvals

The FDA has approved Recarbrio (imipenem-cilastatin and relebactam), from Merck & Co. Inc., to treat adults with hospital-acquired and ventilator-associated bacterial pneumonia (HABP/VABP). The approval is for patients whose HABP/VABP is caused by these susceptible gram-negative microorganisms: Acinetobacter calcoaceticus-baumannii complex, Enterobacter cloacae, Escherichia coli, Haemophilus influenzae, Klebsiella aerogenes, Klebsiella oxytoca, Klebsiella pneumoniae, Pseudomonas aeruginosa and Serratia marcescens.

Clinical trials

Two phase III stumbles of Iterum Therapeutics plc’s sulopenem have cast the company’s future in doubt. The latest problem is the failure of the penem anti-infective compound, with oral and I.V. formulations failing to achieve statistical noninferiority relative to ertapenem in treating complicated urinary tract infection (cUTI). The primary endpoint for the SUlopenem for Resistant Enterobacteriaceae, or SURE 2, trial was overall clinical and microbiologic response on day 21 in the micro-modified intent-to-treat population as evaluated using a 10% noninferiority margin. The company stock (NASDAQ:ITRM) suffered on the news, with shares plummeting 59.6% on June 1 to close at $1.70 each.

COVID-19 developments

By the numbers: At the end of May, 318 therapeutics and 115 vaccines were in development for COVID-19, according to BioWorld. Tracked diagnostics featured in BioWorld MedTech have reached 275, including antibody, antigen and molecular-based tests, of which 117 have emergency use authorization (EUA) from the FDA. WHO reported 6.06 million confirmed COVID-19 cases worldwide, with a total of 371,166 deaths. The U.S. accounts for about 28% of the cases and deaths.

Abbvie Inc., Harbour Biomed Therapeutics Ltd., Utrecht University (UU) and Erasmus Medical Center (EMC) said they entered a collaboration to develop a novel antibody therapeutic to prevent and treat COVID-19. The focus of the collaboration is on advancing the fully human, neutralizing antibody 47D-11 discovered by UU, EMC and Harbour and recently reported in Nature Communications. Codiak Biosciences Inc. signed collaborations with the Ragon Institute of Massachusetts General Hospital, Massachusetts Institute of Technology and Harvard to investigate the potential of its Exovacc vaccine platform in SARS-CoV-2, the virus that causes COVID-19, and in HIV. CSL Ltd., the Coalition for Epidemic Preparedness Innovations and the University of Queensland (UQ) said they entered a new partnering agreement to accelerate the development, manufacture and distribution of a COVID-19 vaccine candidate pioneered by researchers at UQ. Dimerix Ltd. saw its shares gain 66% on the news that its lead candidate, DMX-200, has been chosen to enter the global REMAP-CAP platform trial as a potential treatment for COVID-19-related acute respiratory distress syndrome. Intravacc and Epivax Inc. will collaborate to advance a COVID-19 vaccine. Intravacc will combine its outer membrane vesicle delivery platform with synthetically produced COVID-19 epitopes (protein allergens), to generate a T-cell response against SARS-CoV-2 and related coronaviruses. Modular Bioscience Inc. said it has a definitive agreement with the University of Illinois for an exclusive global patent license to the school’s artificial restriction enzyme platform. Modular's initial focus will be on synthetic biology, diagnosis of inherited genetic diseases, liquid biopsy and detecting infectious disease agents such as COVID-19. Trevena Inc. is collaborating with Imperial College London to evaluate the potential of TRV-027, an AT1 receptor selective agonist, to treat acute lung injury contributing to acute respiratory distress syndrome in COVID-19 patients. Imperial College will be sponsoring and funding the study, with additional support through the British Heart Foundation Center for Research Excellence Award. Twist Bioscience Corp. and Proteona Pte. Ltd. said they'll work together as part of an international alliance to develop broadly neutralizing antibodies against coronaviruses, including SARS-CoV-2. Twist will employ its DNA synthesis platform to support the manufacture of millions of oligos of different lengths, enabling its Twist Biopharma division to identify numerous neutralizing antibody sequences and use in-house capability to generate and optimize IgG antibodies ready for analysis, it said. Vault Pharma Inc. said it's working with the University of California, Los Angeles, and other universities to advance a vaccine with the potential to treat SARS-CoV-2 in addition to protecting against it. Candidate vaccines will be tested at Northern Arizona University. Vitro Diagnostics Inc. has entered an exclusive memorandum of understanding with Global Institute of Stem Cell Therapy and Research Inc. to collaborate on an IND using Vitro Biopharma’s umbilical cord mesenchymal stem cell product, Allorx Stem Cells, in a clinical trial to treat COVID-19 patients

Deals of the week

Abbvie Inc. and Jacobio Pharmaceuticals Co. Ltd. disclosed a global collaboration to develop and commercialize SHP2 inhibitors, which target a key node in cancer and immune cells. SHP2 is an important protein mediator of cellular signaling through the RAS/MAP kinase pathway. Aduro Biotech Inc. and privately held Chinook Therapeutics Inc. will merge to focus mainly on kidney diseases. Two complementary drugs are involved in the deal. Chinook brings atrasentan, licensed from Abbvie Inc., an endothelin receptor antagonist, which is entering a phase III trial for treating primary glomerular disease in the second half of the year and a phase II glomerular basket trial to begin in 2021. Aduro brings BION-1301, a humanized IgG4 monoclonal antibody that blocks APRIL binding to the BCMA and TACI receptors, which is in nonclinical phase I testing in healthy volunteers for treating IgA nephropathy. Accent Therapeutics Inc. will receive $55 million up front and as much as $1.1 billion in milestones as part of a substantial new three-molecule deal with Astrazeneca plc, which committed to exploring the field of targeting RNA-modifying proteins (RMPs) for the treatment of cancer. Alligator Bioscience AB and Scandion Oncology A/S signed a collaboration agreement to explore the antitumor efficacy of the CD40 antibody mitazalimab from Alligator in combination with SCO-101 from Scandion as an addition to chemotherapy in resistant preclinical tumor models. CSL Behring and the Seattle Children's Research Institute said they established a strategic alliance to develop stem cell gene therapies for primary immunodeficiency diseases. Initially, the alliance will focus on the development of treatment options for patients with Wiskott-Aldrich syndrome and X-linked agammaglobulinemia. Jounce Therapeutics Inc. has regained worldwide rights to JTX-8064 from Bristol Myers Squibb Co. JTX-8064, a potential first-in-class antibody that targets the leukocyte immunoglobulin-like receptor B2 on macrophages, was licensed in July 2019 to Celgene Corp., which was later acquired by BMS. As part of its Celgene integration process, BMS said it is streamlining its pipeline and notified Jounce that the JTX-8064 license agreement is being terminated. Juvenescence Ltd. and G3 Therapeutics Inc. formed Juvenomics Ltd., a joint venture. Juvenomics will focus on developing validated nutraceuticals and medicines to combat aging and aging-related diseases such as those of the musculoskeletal system. Regeneron Pharmaceuticals Inc. has agreed to pay Intellia Therapeutics Inc. $70 million up front, alongside making a $30 million equity investment in the company, in an expansion of an ongoing partnership, first inked in 2016. The revised collaboration provides Regeneron with rights to develop products for additional in vivo CRISPR/Cas9-based therapeutic targets and for the companies to jointly develop potential products for the treatment of hemophilia A and B. Shanghai Junshi Biosciences Co. Ltd. and Merck KGaA said they are collaborating on a clinical trial program designed to investigate the efficacy and safety of anti-PD-1 monoclonal antibody Tuoyi (toripalimab) in combination with EGFR-targeting antibody Erbitux (cetuximab) as a treatment for recurrent and/or metastatic squamous cell carcinomas of the head and neck in China. UCB SA has acquired Engage Therapeutics Inc., a clinical-stage company developing Staccato Alprazolam for the rapid termination of an active epileptic seizure, for $125 million in cash (subject to certain adjustments) and up to $145 million in further potential milestone payments related to clinical development, submission and launch of Staccato Alprazolam.

Financial transactions

Public offerings: Adaptimmune Therapeutics plc priced its public offering for gross proceeds of $225.5 million. Allogene Therapeutics Inc. priced its public offering for gross proceeds of about $550 million. Argenx NV closed its global offering for gross proceeds of about $862.5 million. Biocryst Pharmaceuticals Inc. generated gross proceeds of about $115 million from a public offering. Greenwich Lifesciences Inc. has filed with the SEC to raise up to $24 million in an IPO. Intellia Therapeutics Inc. priced its public offering, grossing $100 million. Iovance Biotherapeutics Inc. closed its public offering generating $603.7 million. Kaleido Biosciences Inc. priced its public offering, grossing the company $35.6 million, and Marinus Pharmaceuticals Inc. raised about $46 million from its offering. CAR T-focused Legend Biotech Corp. raised $423.8 million in its IPO to fund its Janssen Biotech Inc.-partnered BCMA-targeting CAR T candidate, LCAR-B38M. Menlo Therapeutics Inc. priced an underwritten public offering, resulting in gross proceeds of $50.04 million. Mersana Therapeutics Inc. closed its public offering, grossing the company approximately $174.8 million. Pliant Therapeutics Inc. raised $144 million from its IPO.

Other public offerings: Appili Therapeutics Inc. priced an overnight marketed offering of equity securities for gross proceeds of CA$13.5 million (US$9.99 million). G1 Therapeutics Inc. entered a debt financing agreement with Hercules Capital Inc. for up to $100 million. Humanigen Inc. priced a private placement of about $72 million of common stock. Mereo Biopharma Group plc said it completed a $70 million (£56 million) private placement with new and existing U.S.-based institutional and accredited investors.

Venture funding: Athira Pharma Inc. closed on an $85 million series B financing designed to advance its small molecule, NDX-1017, into a phase II/III trial for treating Alzheimer’s disease later this year. Cullinan Oncology LLC closed a $98.5 million series B financing. Everest Medicines Ltd. closed one of the biggest financing rounds in China’s health care market this year, adding $310 million to its war chest. The firm is aiming to advance its late-stage assets in-licensed from global partners to the China market soon. The series C round included a $260 million series C-2 led by Janchor Partners and a $50 million series C-1 joined by Jiashan SDIC. Nodthera Ltd. has raised $55 million in a series B round, providing funding for phase II proof-of-concept studies and to advance a second, brain-penetrant, compound to the clinic. Prilenia Therapeutics BV that is developing an experimental therapy for Huntington's disease and amyotrophic lateral sclerosis, has raised $62.5 million in a series A financing round.

Atlas Venture said it closed its 12th fund, raising $400 million from an oversubscribed fundraise. The new fund will be deployed to create breakthrough biotech startups focused on cutting-edge innovation, sourced from leading global institutions and the team’s entrepreneurial networks.

Regulatory news

Responding to the challenges clinical trial investigators are facing with getting informed consent during the COVID-19 pandemic, the FDA is making its Mystudies app available as a free platform. Offered under the name COVID Mystudies, the app allows investigators to obtain informed consent securely from patients when face-to-face contact isn’t possible or practical because of COVID-19 control measures. Before using the app, investigators will provide their informed consent documents to the COVID Mystudies team so they can be added to the app. Investigators will be able to review the informed consent within the app before using it in their trials. Investigators still must have institutional review board review and approval of the informed consent document and process, the FDA said.

Logistics and supplies are not the sexy part of drug and device development, but they’re what makes using a diagnostic, therapy or vaccine possible. During a massive disaster or a pandemic, securing the necessary manufacturing capacity, needles, syringes, vials, properly labeled caps, reagents and other supplies is as critical as the development of the product itself. Some experts have been warning about these needs since COVID-19 first began spreading outside of China. Now members of Congress are sounding the alarm. Echoing concerns raised in a recent whistleblower complaint filed by former Biomedical Advanced Research and Development Authority Director Rick Bright, 25 U.S. lawmakers, from both parties, wrote to House leaders last week urging them to address the shortage of syringes, needles and other materials that will be needed to effectively administer a coronavirus vaccine while maintaining access to other necessary vaccines and drugs.

Word on the Street: Industry responses to the issues of the week

“By 2023, the growth of China’s pharma market will be pretty much the same as the country’s GDP. We used to say the pharma market grew rapidly and was lucrative, but this growth is expected to slow because a lot of generics will be squeezed out of the market to make room for innovative drugs.”
Fred Mao, partner at Frost & Sullivan Greater China, at the Enmore Bio Conference

“The U.S. drug supply chain is safe, and the supply chain is secure.”
Judith McMeekin, associate commissioner for regulatory affairs at the FDA, testifying before the Senate Finance Committee

“There is a lot of growing evidence that there are high concentrations of MCP-1 in the lungs of patients with ARDS, and the resulting movement of immune cells into the lung may be one of the factors of the cytokine storm in the lung. For this reason, there is strong scientific evidence that DMX-200 on its own or in combination with an angiotensin receptor blocker may have the unique potential to reduce the COVID-related lung damage.”
Nina Webster, CEO, Dimerix Ltd., whose DMX-200 has been chosen to enter the global REMAP-CAP platform trial as a potential treatment for COVID-19-related acute respiratory distress syndrome

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