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STOCK RECAP #PipelineProspector

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Pipeline Prospector Feb 2025: Bain buys Mitsubishi Tanabe for US$ 3.4 bn; Japan’s Ono gets FDA nod for rare joint tumor drug
February was a mixed bag for biopharma indices, underscoring the volatility and uncertainty in the sector. The Nasdaq Biotechnology Index (NBI) managed a modest gain of 1.16 percent, climbing from 4,466.41 to 4,518.06. However, the SPDR S&P Biotech ETF (XBI) fell 2.06 percent from 90.58 to 88.71, and the S&P Biotechnology Select Industry Index (SPSIBI) experienced a more substantial drop of 4.26 percent, declining from 7,222.3 to 6,914.8.The month saw several notable developments among Asian pharmaceutical companies, signaling their growing influence. Meanwhile, the confirmation of Robert F. Kennedy Jr. as the US Secretary of Health and Human Services (HHS) cast a veil of uncertainty over the sector, prompting investors to brace for potential shifts in vaccine policy and regulatory oversight. Access the Pipeline Prospector Dashboard for February 2025 Newsmakers (Free Excel)Bain buys Mitsubishi Tanabe in US$ 3.4 bn deal; Japan’s Ono secures FDA nod for joint tumor drugUS private equity firm Bain Capital acquired Mitsubishi Tanabe Pharma for approximately JPY 510 billion (US$ 3.4 billion). This strategic acquisition positions Bain to leverage the Osaka-based pharmaceutical company’s expertise in central nervous system disorders, immuno-inflammation, and oncology.Bain’s investment reflects growing confidence in Japan’s life sciences sector, particularly in light of the recent initiatives by the Japanese government to accelerate the development and approval of innovative medicines. Japan’s Ono Pharmaceutical (stock up 2 percent) received FDA approval for Romvimza (vimseltinib), a treatment for tenosynovial giant cell tumor (TGCT), a rare condition affecting joints. The approval follows Ono’s US$ 2.4 billion acquisition of Deciphera Pharmaceuticals, which included Romvimza and other cancer drugs. Romvimza will compete with Daiichi Sankyo’s Turalio (pexidartinib), the only other FDA-approved systemic treatment for TGCT.Meanwhile, Radiance Biopharma has acquired an antibody-drug conjugate (ADC) asset from China’s CSPC Megalith Biopharmaceutical. The ADC targets ROR1 (receptor tyrosine kinase-like orphan receptor 1), a protein implicated in embryonic development and cancer pathways. The transaction structure includes an upfront payment of US$ 15 million, with potential milestone payments exceeding US$ 1 billion.Continuing with its aggressive dealmaking, Novartis agreed to acquire Anthos Therapeutics, an anticoagulant drug development company majority-owned by investment firm Blackstone, for up to US$ 3.1 billion, with US$ 925 million paid upfront. Anthos emerged from a partnership between Blackstone and Novartis to develop abelacimab, a novel blood-thinning drug aimed at preventing strokes and dangerous blood clots in patients with atrial fibrillation. Access the Pipeline Prospector Dashboard for February 2025 Newsmakers (Free Excel) FDA okays SpringWorks’ med for rare genetic condition, AbbVie-Pfizer’s antibiotic for drug-resistant infectionsFDA approved SpringWorks Therapeutics’ Gomekli (mirdametinib) for the treatment of neurofibromatosis type 1 (NF1), a rare genetic condition that causes tumors to grow along nerve tissues throughout the body, often resulting in severe pain and physical deformity. Gomekli is the first and only medicine approved for addressing this condition in both pediatric and adult populations with inoperable tumors. SpringWorks’ shares skyrocketed 57 percent in February. Meanwhile, reports suggest that Germany’s Merck KGaA is currently in advanced talks to acquire Connecticut-based SpringWorks.AbbVie and Pfizer received FDA approval for Emblaveo (aztreonam and avibactam), a novel intravenous antibiotic developed jointly for the treatment of complicated intra-abdominal infections (cIAIs) in adults with limited or no alternative treatment options. To be used in combination with metronidazole, Emblaveo targets gram-negative bacteria, including drug-resistant strains.AbbVie (stock up 9 percent) further strengthened its oncology portfolio with a collaboration with Xilio Therapeutics to develop novel tumor-activated immunotherapies, including masked T-cell engagers. This partnership could potentially be worth over US$ 2 billion.Mirum Pharma’s Ctexli (chenodiol) was approved as the first targeted treatment for cerebrotendinous xanthomatosis (CTX), a rare lipid storage disease.In drug-device combinations, Supernus Pharmaceuticals received FDA approval for Onapgo, a treatment of motor fluctuations in adults with advanced Parkinson’s disease. Onapgo is a wearable device that continuously delivers apomorphine hydrochloride subcutaneously throughout waking hours.Meanwhile, Roche’s Susvimo (ranibizumab injection) eye implant received a label expansion to include the treatment of diabetic macular edema (DME). Additionally, Roche (stock up 8 percent) reported promising late-stage clinical data for its blood cancer drug Gazyva (obinutuzumab) in lupus nephritis. Access the Pipeline Prospector Dashboard for February 2025 Newsmakers (Free Excel) RFK Jr’s confirmation as HHS Secy hits vaccine makers; GSK gains on buyback plan; FDA okays 5-strain meningitis vaccineWith RFK Jr taking charge of HHS, stocks of vaccine makers took a hit. For instance, Bavarian Nordic’s stock fell by 11 percent, even though its chikungunya vaccine Vimkunya got approved by the FDA on February 14.Meanwhile, FDA paused a late-stage trial of Moderna’s experimental norovirus vaccine, mRNA-1403. The pause came after a single reported case of Guillain–Barré syndrome — a rare neurological side effect. Moderna’s stock fell 21 percent.In contrast, GSK’s stock rose 9 percent after it launched a £ 2 billion (US$ 2.5 billion) share buyback and raised its long-term sales target to nearly US$ 50 billion by 2031, largely driven by robust growth in specialty medicines, such as its HIV and cancer treatments. The British pharma giant also received FDA approval for its meningitis combination vaccine, Penmenvy. This five-in-one vaccine protects against five strains of Neisseria meningitidis, combining protective components of two existing vaccines into a single shot. Sanofi (stock up 2 percent) announced plans to repurchase € 5 billion (US$ 5.21 billion) worth of its shares in 2025 following the anticipated sale of its Opella consumer healthcare unit to private equity firm Clayton, Dubilier & Rice for € 15.5 billion (US$ 16.11 billion). The French giant also indicated it could be “a bit more” active with mergers and acquisitions in the near future. As part of Sanofi’s share buyback program, L’Oréal has agreed to sell a € 3 billion (US$ 3.11 billion) stake in Sanofi back to the drugmaker.The month saw some dramatic exits in the gene therapy space. Bluebird Bio, which was once valued at about US$ 10 billion, agreed to be acquired by private equity firms Carlyle and SK Capital Partners for less than US$ 30 million. Similarly, Pfizer announced it would discontinue the global development and commercialization of its hemophilia B gene therapy Beqvez (fidanacogene elaparvovec-dzkt), which is priced at US$ 3.5 million per treatment. Access the Pipeline Prospector Dashboard for February 2025 Newsmakers (Free Excel) Our view The biopharma sector is doing the right things by taking strategic business decisions, focusing on innovation and a strong drug pipeline. However, the geopolitical environment is uncertain. With a trade war ensuing between the US, China, Mexico and Canada, and the Trump administration suspending all military aid to Ukraine, we are entering uncertain times when it's difficult to say what the next few months would look like for the biopharma sector.

Impressions: 3279

https://www.pharmacompass.com/pipeline-prospector-blog/pipeline-prospector-feb-2025-bain-buys-mitsubishi-tanabe-for-us-3-4-bn-japan-s-ono-gets-fda-nod-for-rare-joint-tumor-drug

#PharmaFlow by PHARMACOMPASS
06 Mar 2025

WEEKLY NEWS RECAP #Phispers

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Lilly to invest US$ 27 bn in US as Trump threatens big pharma with tariffs; FDA scrambles to rehire after mass layoffs
In the US, President Donald Trump has been putting pressure on Big Pharma to reshore or face tariffs. Eli Lilly has been the first to comply as it unveiled an ambitious plan to invest at least US$ 27 billion in four new plants across the US over the next five years.Though layoffs have been continuing unabated across agencies in America, the US Food and Drug Administration (FDA) reversed its controversial mass layoffs and reportedly moved to rehire hundreds of employees to stabilize critical operations.In drug approvals, Mirum Pharma’s Ctexli became the first FDA-approved treatment for a rare lipid storage disease. In trials, AstraZeneca’s experimental drug camizestrant demonstrated significant improvements in progression-free survival in a late-stage breast cancer trial.In the field of gene therapy, Pfizer announced it will discontinue Beqvez for hemophilia B citing limited market interest, while Bluebird Bio said it is selling itself to private equity firms.In regulatory news, FDA has shot off warning letters to two Indian API manufacturers for critical cGMP violations, and issued a Form 483 to Piramal Pharma post an inspection held at its Navi Mumbai plant this month.Lilly to invest US$ 27 bn in US, as Trump threatens Big Pharma with tariffsPharmaceutical giant Eli Lilly has unveiled an ambitious plan to invest at least US$ 27 billion in constructing four new manufacturing plants across the US over the next five years. The announcement comes amid increasing pressure from President Donald Trump, who met privately with top executives from Big Pharma — including Pfizer, Merck, Lilly and lobby group PhRMA.During the meeting, Trump warned that failing to reshore manufacturing operations could result in tariffs as high as 25 percent. Lilly’s new facilities are expected to generate over 3,000 skilled jobs in engineering and science, alongside approximately 10,000 construction roles. This boosts the Indiana drugmaker’s total investment in the US to over US$ 50 billion since 2020.Compounders sue FDA over GLP-1 drug shortages: Pharmaceutical compounders are suing FDA following the agency’s recent declarations that shortages of key weight loss drugs have ended. Compounders are contesting that the “reckless and arbitrary decision” to remove Novo Nordisk’s semaglutide (Ozempic and Wegovy) from the shortage list is premature. In a parallel development, Lilly has cut the price for vials of its weight-loss drug Zepbound (tirzepatide) by US$ 50 or more in order to stave off competition from compounding pharmacies and its Danish rival.FDA reverses course, moves to rehire hundreds after mass layoffsIn a swift policy reversal, FDA is working to rehire hundreds of employees who were terminated just days earlier as part of a mass layoff initiative. The initial firings, ordered by the Trump administration and led by Elon Musk's Department of Government Efficiency, affected over 1,000 FDA staffers responsible for critical areas such as drug and medical device review, food safety, and tobacco regulation. The layoffs drew significant backlash from industry stakeholders. Reports suggest that approximately 300 staff members are being asked to return.Mirum’s Ctexli bags FDA approval for rare lipid storage diseaseFDA has approved Mirum Pharma’s Ctexli (chenodiol) as the first targeted treatment for cerebrotendinous xanthomatosis (CTX), a rare lipid storage disease. Patients with CTX are unable to effectively break down cholesterol, leading to a toxic build-up of cholestanol in tissues, and suffer from neurological abnormalities.AstraZeneca’s camizestrant shows promise in late-stage breast cancer trialAstraZeneca has announced that its experimental drug, camizestrant, has demonstrated  significant improvement in progression-free survival for patients with advanced breast cancer. The trial specifically targeted patients with hormone receptor-positive, HER2-negative advanced breast cancer possessing a particular (emergent ESR1) genetic mutation. The British-Swedish drugmaker has set a peak sales target of US$ 5 billion for this drug.Emalex’s ecopipam cuts Tourette syndrome relapse: Emalex Biosciences has reported a major win in its phase 3 trial on ecopipam, a med for Tourette syndrome (a neurological disorder that causes involuntary tics, repetitive movements or sounds). In the study, pediatric patients who continued treatment with ecopipam experienced a relapse rate of 41.9 percent compared to 68.1 percent in those who switched to placebo. Similar benefits were seen in the combined pediatric and adult groups. Pfizer shifts focus to Hympavzi after discontinuing US$ 3.5 mn hemophilia gene therapyThe field of gene therapy has been facing a tough period for the last few years. Ergo, Pfizer has announced the discontinuation of global development and commercialization of Beqvez, a gene therapy designed to treat adults with moderate to severe hemophilia B.Despite receiving approvals in Canada, the US, and Europe throughout 2024, the company cited limited patient and physician interest in hemophilia gene therapies as the primary reason for this decision. Beqvez (fidanacogene elaparvovec-dzkt) is priced at US$ 3.5 million per treatment.This move effectively leaves Pfizer without any active gene therapy programs in its portfolio. The company plans to redirect its resources toward Hympavzi (marstacimab-hncq), its other therapy for hemophilia A and B that received FDA approval in October 2024.Bluebird Bio gets sold for ~US$ 30 million: Cash-strapped Bluebird Bio that develops gene therapies for severe genetic disorders has entered into a definitive agreement to be acquired by private equity firms Carlyle and SK Capital Partners for less than US$ 30 million.FDA slaps two Indian API firms with warning letters; Piramal Pharma gets Form 483FDA has issued warning letters and import alerts to two Indian API manufacturers — Jagsonpal Pharmaceuticals and Tyche Industries — following inspections that revealed significant cGMP violations. The agency discovered that Jagsonpal had engaged a third-party contract manufacturer to produce APIs, which it then claimed as its own on import records. The company, based in Gurugram near New Delhi, downright refused entry to FDA inspectors on March 15, 2024, FDA said. Inspectors were eventually allowed entry on March 20, 2024, but with limited access to the requested documents. In the case of Tyche Industries, FDA inspectors reported that during an inspection, a member of management stated that two operators admitted to falsifying temperature data for a drying oven that was not turned on during the manufacture of a batch.Form 483 for Piramal Pharma: FDA has issued a Form 483, with six observations, to Piramal Pharma after conducting a Good Manufacturing Practice (GMP) inspection at its Turbhe, Navi Mumbai facility from February 11-17, 2025.

Impressions: 1271

https://www.pharmacompass.com/radio-compass-phisper/lilly-to-invest-us-27-bn-in-us-as-trump-threatens-big-pharma-with-tariffs-fda-scrambles-to-rehire-after-mass-layoffs

#Phispers by PHARMACOMPASS
27 Feb 2025

NEWS #PharmaBuzz

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https://www.reuters.com/business/healthcare-pharmaceuticals/us-fda-approves-mirum-pharmas-genetic-disorder-drug-2025-02-21/

REUTERS
22 Feb 2025

https://www.businesswire.com/news/home/20231002522656/en

BUSINESSWIRE
02 Oct 2023

https://www.biospace.com/article/fda-approves-retrophin-s-new-formula-thiola-for-cystine-kidney-stones/

Mark Terry BIOSPACE
01 Jul 2019
Dutch doctors resist pharma firms 500-fold price hike
Dutch doctors resist pharma firms 500-fold price hike

04 Sep 2018

// Louis Goss PHARMA FILE

http://www.pharmafile.com/news/518520/dutch-doctors-resist-pharma-firms-500-fold-price-hike

Louis Goss PHARMA FILE
04 Sep 2018

https://endpts.com/a-shkreli-like-drug-price-controversy-leadiant-finds-itself-in-the-spotlight-after-500-fold-price-hike-ft/

John Carroll ENDPTS
04 Sep 2018

https://www.forbes.com/sites/nicolefisher/2018/06/26/sticker-shock-the-real-cost-of-americas-10-most-expensive-drugs/?ss=pharma-healthcare#1781663714d1

Nicole Fisher FORBES
26 Jun 2018