Company expands pipeline into car‑t and genetic medicine

Original title: “Laverock unveils new in vivo programmes in oncology”

Early findings support phase 2 plans for 2027

Original title: “Atrogi reports human data showing muscle gains with ATR‑258”

Call aims to support research preventing deadly newborn infections

Original title: “Group B Strep mums urge women to join vaccine trial”

The Takeda spinout showed that a high dose of solengepras can shorten the periods during which difficult Parkinson’s disease symptoms like involuntary movements and sleepiness.

Original title: “Cerevance Parkinson’s drug shortens 'off time' in Ph. 3, paving path to FDA”

ArriVent BioPharma’s Phase 3 lung cancer trial was clouded by stronger-than-expected data for patients receiving chemotherapy, prompting analysts to pivot focus to a late-stage test of the tyrosine kinase inhibitor in a different type of lung cancer and to the biotech’s antibody-drug conjugate pipeline.

Original title: “ArriVent sheds nearly half of value on late-stage lung cancer miss”

Qualified sites print doses locally. A U.S. reserve will stock ingredients for about 26 critical drugs; PharmaTher plans a network demo within 12 months. 95% of the roughly 10,000 known rare diseases have no FDA-approved treatment, according to the U.S. National Institutes of Health. Discontinued medicines. Many medicines are discontinued not because they stopped working, but because demand became too small for a production line. Drug shortages. Many medicines are made in central plants, often overseas, far from the patients who need them. Clinical and research supply. Trials often need many strengths in batches of a few hundred units. PharmaTher's pipeline includes personalized 503A products, including peptides and ketamine; FDA rare and ultra-rare disease programs; psychedelic medicines for clinical trials and special access programs; discontinued medicines; medicines in short supply; and medical countermeasures. Focused on unmet medical needs. Personalized doses, rare and ultra-rare diseases, discontinued medicines, drug shortages, clinical and research supply, and emergency preparedness. The network is designed to bring together pharmacies, hospitals, contract manufacturers and government facilities to make personalized, rare disease, discontinued and short-supply medicines and emergency countermeasures, close to the patients who need them.

Original title: “About 95% of roughly 10,000 known rare diseases lack FDA-approved treatment. PharmaTher's new network aims to print medicines locally.”

Guidance supports use when standard therapies fail

Original title: “NICE backs Tryngolza for FCS treatment”

Boehringer reports cardiometabolic improvements in people with t2d

Original title: “Survodutide delivers weight loss and glycemic gains in phase 2 trial”

NIH SBIR grant of $2.98 Million ... treatment for IH · Appointment of new CEO, Katie MacFarlane, PharmD, to lead the company transition into clinical development · The company is launching a Series A financing round · MADISON, Wis., Oct. 6, 2026 /PRNewswire/ -- Arkayli BioPharma Inc. ("Arkayli"), a biopharmaceutical company dedicated to developing innovative therapies for rare dermatologic ... NIH SBIR grant of $2.98 Million acknowledges the high unmet need for a non-systemic treatment for IH · Appointment of new CEO, Katie MacFarlane, PharmD, to lead the company transition into clinical development · The company is launching a Series A financing round · MADISON, Wis., Oct. 6, 2026 /PRNewswire/ -- Arkayli BioPharma Inc. ("Arkayli"), a biopharmaceutical company dedicated to developing innovative therapies for rare dermatologic conditions, today announced that the U.S. The company is preparing to initiate a Phase 1B clinical trial evaluating the safety of ARK001 in infants diagnosed with proliferating infantile hemangioma (IH). In addition, exploratory measures of effect will be assessed as part of the study. Arkayli believes that ARK001 has the potential for improved treatment of IH, through the topical, non-systemic treatment of this disfiguring skin disease. There is a high unmet market need for a safe, effective, topically applied therapy that can be used early in diagnosis and prescribed by pediatricians and dermatologists. ... This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements involve inherent risks and uncertainties, including statements regarding the timing and success of the proposed Phase 1b clinical trial, the ability to secure Series A financing, and regulatory pathways. "ARK001 is designed to address critical unmet needs in this vulnerable patient population, potentially offering a topical approach to treatment of this rare pediatric disease. With the NIH grant, we are focused on preparing for the Phase 1b trial, and we also are looking to accelerate our pipeline development through a Series A financing round."

Original title: “Arkayli Biopharma Inc. Receives Study May Proceed Response to IND from FDA and SBIR Grant from NIH to Advance Clinical Development of ARK001 for a Rare Pediatric Disease - BioSpace”

John Fairbanks, diagnosed with AL Amyloidosis, underwent CAR-T cell therapy as part of a clinical trial, achieving complete remission and improved organ function. Fairbanks became part of a clinical trial testing CAR-T cell therapy as a treatment for amyloidosis. Anderson said the results seen in the clinical trial have been striking, with about 95% of patients achieving complete remission. But when Fairbanks faced a disease that threatened his life, it was another kind of "car" that would make the difference. "Well I started this battle, and I call it a battle, in 2022," Fairbanks said. Fairbanks was diagnosed with AL Amyloidosis, a rare and potentially deadly disease in which abnormal proteins accumulate in the body's organs, damaging vital systems such as the heart, liver and kidneys. "This therapy called NX 201 at the moment and is a form of a CAR-T cell therapy.

Original title: “'Unreal': North Texas man in remission after experimental treatment”

Anderson said the results seen in the clinical trial have been striking, with about 95% of patients achieving complete remission. Anderson said the results seen in the clinical trial have been striking, with about 95% of patients achieving complete remission. To stream KVUE on your phone, you need the KVUE app. ... MANSFIELD, Texas — John Fairbanks knows a thing or two about cars. His pride and joy is a meticulously restored bright red 1967 Ford Mustang Fastback, a classic he rebuilt from the frame up. But when Fairbanks faced a disease that threatened his life, it was another kind of "car" that would make the difference. Fairbanks became part of a clinical trial testing CAR-T cell therapy as a treatment for amyloidosis. Fairbanks was diagnosed with AL Amyloidosis, a rare and potentially deadly disease in which abnormal proteins accumulate in the body's organs, damaging vital systems such as the heart, liver and kidneys. "This therapy called NXC-201 at the moment and is a form of a CAR-T cell therapy.

Original title: “'Unreal': North Texas man in remission after experimental treatment | kvue.com”

Anderson said the results seen in the clinical trial have been striking, with about 95% of patients achieving complete remission. Anderson said the results seen in the clinical trial have been striking, with about 95% of patients achieving complete remission. To stream WFAA on your phone, you need the WFAA app. ... MANSFIELD, Texas — John Fairbanks knows a thing or two about cars. His pride and joy is a meticulously restored bright red 1967 Ford Mustang Fastback, a classic he rebuilt from the frame up. But when Fairbanks faced a disease that threatened his life, it was another kind of "car" that would make the difference. Fairbanks became part of a clinical trial testing CAR-T cell therapy as a treatment for amyloidosis. Fairbanks was diagnosed with AL Amyloidosis, a rare and potentially deadly disease in which abnormal proteins accumulate in the body's organs, damaging vital systems such as the heart, liver and kidneys. "This therapy called NX 201 at the moment and is a form of a CAR-T cell therapy.

Original title: “'Unreal': North Texas man in remission after experimental treatment | wfaa.com”

The better-than-expected results could make Epkinly the new “undisputed” treatment standard in newly diagnosed DLBCL, according to one analyst.

Original title: “Dual-acting drug from AbbVie, Genmab scores in frontline lymphoma trial”

Despite the regulatory delay, Novo is still expecting a 2027 launch for its hemophilia A drug denecimig.

Original title: “FDA extends review of Novo’s hemophilia A drug, citing manufacturing issues”

AbbVie and Genmab’s bispecific antibody blew analyst expectations out of the water with a 51% improvement in progression-free survival when used alongside a standard frontline regimen in diffuse large B cell lymphoma—potentially setting up a battle with Roche's Polivy.

Original title: “AbbVie, Genmab's Epkinly sets ‘new standard of care’ in first-line lymphoma in Phase 3”